| Primary | Percentage of Participants With Two-Year Post-Transplant Event Free Survival (EFS) | EFS is defined as survival without a qualifying event from transplant. Primary graft failure (PGF), secondary graft failure (SGF), second infusion of hematopoietic cells, or death from any cause will count as events for this endpoint. This endpoint was adjudicated by an Endpoint Review Committee. PGF is lack of engraftment on or before Day 42 post-transplant. Engraftment is defined as having greater than or equal to 5% donor cells post-transplant, from any molecular chimerism assessment (e.g., unsorted, myeloid, or T-cell) on a peripheral blood or bone marrow aspirate sample. SGF is defined as < 5% donor whole blood or myeloid chimerism in peripheral blood or bone marrow beyond day +42 post-transplant in patients with prior documentation of hematopoietic recovery with >5% donor cells by day +42 post-transplant. Infusion of a second stem cell product will be considered graft rejection, and counted toward primary or, depending on timing of the second infusion, secondary graft rejection | All participants who consented and received transplantation for each stratum. | Posted | | Number | 95% Confidence Interval | Percentage of Participants | | 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
| | | Title | Denominators | Categories |
|---|
| | | Title | Measurements |
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| - OG00088.0(73.5 to 94.8)
- OG00179.3(62.8 to 89.1)
|
|
| |
| Secondary | Percentage of Participants With Two-Year Post-Transplant Overall Survival (OS) | Death from any cause will be the event and patients will be censored at the date of last contact or two years post-transplant, whichever comes first. | All participants who consented and received transplantation for each stratum. | Posted | | Number | 95% Confidence Interval | Percentage of Participants | | 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Number of Participants With Graft Failure | Primary Graft Failure (PGF) is lack of engraftment on or before Day 42 post-transplant. Engraftment is defined as having greater than or equal to 5% donor cells post-transplant, from any molecular chimerism assessment (e.g., unsorted, myeloid, or T-cell) on a peripheral blood or bone marrow aspirate sample. Secondary Graft Failure (SGF) is defined as < 5% donor whole blood or myeloid chimerism in peripheral blood or bone marrow beyond day +42 post-transplant in patients with prior documentation of hematopoietic recovery with >5% donor cells by day +42 post-transplant. Infusion of a second stem cell product will be considered graft rejection, and counted toward primary or, depending on timing of the second infusion, secondary graft rejection. | All participants who consented and received transplantation for each stratum. | Posted | | Count of Participants | | Participants | | 2 Years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes |
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| Secondary | Percentage of Participants With Disease Recurrence Post-Transplant | Disease Recurrence is defined as primary graft failure, secondary graft failure, or sickle hemoglobin (HbS) > 70% within 2 years post-transplant. Cumulative Incidence of Disease Recurrence was estimated with a 95% confidence interval using the Aalen-Johansen estimator with death prior to disease recurrence treated as a competing risk. This endpoint was adjudicated by an Endpoint Review Committee. | All participants who consented and received transplantation for each stratum. | Posted | | Number | 95% Confidence Interval | Percentage of Participants | | 1 year and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Percentage of Participants With Neutrophil Recovery Post-Transplant | Neutrophil recovery is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of ≥500/μL after conditioning. The incidence of neutrophil recovery from transplant will be estimated using the cumulative incidence function with a 95% confidence interval using the Aalen-Johansen estimator with death or second transplant without neutrophil recovery as a competing risk. | All participants who consented and received transplantation for each stratum. | Posted | | Number | 95% Confidence Interval | Percentage of Participants | | 42 Days post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Percentage of Participants With Platelet Recovery Post-Transplant | Platelet recovery is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count > 50,000/μL AND did not receive a platelet transfusion in the previous 7 days. The incidence of platelet recovery from transplant will be estimated using the cumulative incidence function with a 95% confidence interval using the Aalen-Johansen estimator with death or second transplant without platelet recovery as a competing risk. | All participants who consented and received transplantation for each stratum. One pediatric participant did not have post-transplant platelet data available. | Posted | | Number | 95% Confidence Interval | Percentage of Participants | | 60 Days, 100 Days post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum |
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| Secondary | Mean Percentage of Donor Chimerism Post-Transplant | A point estimate and confidence interval will be provided for the mean percent donor chimerism at the time points specified including Day 28, Day 100, Day 180, 1 year, and 2 years post-transplant. For each participant's visit with multiple chimerism sources recorded, donor percentage is determined by taking the first non-missing chimerism percentage in the following order of sources: marrow, blood, myeloid, and t-cell. | All participants who consented and received transplantation for each stratum. At later timepoints, participants are excluded as they are lost to follow-up, withdraw consent, and experience death. | Posted | | Mean | 95% Confidence Interval | Percentage of Donor Chimerism | | Days 28, 100, and 180 and at 1 and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum |
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| Secondary | Percentage of Participants by Donor Chimerism Category Post-Transplant | At Day 28, Day 100, Day 180, 1 year and 2 years post-transplant, the proportions with low chimerism (<5%), mixed chimerism (5-95%), or full chimerism (>95%) will be tabulated and described. For each participant's visit with multiple chimerism sources recorded, donor percentage is determined by taking the first non-missing chimerism percentage in the following order of sources: marrow, blood, myeloid, and t-cell. | All participants who consented and received transplantation for each stratum. At later timepoints, participants are excluded as they are lost to follow-up, withdraw consent, and experience death. | Posted | | Number | | Percentage of Participants | | Days 28, 100, and 180 and at 1 and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum |
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| Secondary | Number of Participants by Maximum Acute GVHD Grade Post-Transplant | Acute GVHD was graded according to Consensus Criteria with higher grade indicating worse outcomes. Grade I aGVHD is defined as stage 1-2 skin rash and no liver or GI involvement. Grade II is stage 3 skin rash, or stage 1 liver involvement, or stage 1 GI involvement. Grade III is stage 0-3 skin rash, with stage 2-3 liver involvement, or stage 2-3 GI involvement. Grade IV is stage 4 skin rash, liver, or GI involvement. Maximum grade is defined as the maximum grade of acute GVHD (0-IV) that a participant experiences by Day 100 post-transplant. | All participants who consented and received transplantation for each stratum. | Posted | | Count of Participants | | Participants | | 100 Days post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | |
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| Secondary | Percentage of Participants With Acute GVHD Post-Transplant | Acute GVHD was graded according to Consensus Criteria with higher grade indicating worse outcomes. Grade I aGVHD is defined as stage 1-2 skin rash and no liver or GI involvement. Grade II is stage 3 skin rash, or stage 1 liver involvement, or stage 1 GI involvement. Grade III is stage 0-3 skin rash, with stage 2-3 liver involvement, or stage 2-3 GI involvement. Grade IV is stage 4 skin rash, liver, or GI involvement. The cumulative incidence of acute GVHD grade II-IV and III-IV at Day 100 was estimated using the Aalen-Johansen estimator with 95% confidence intervals, treating death prior to aGVHD as a competing event. Time to aGVHD is defined as time from transplant until onset of grades II-IV and III-IV aGVHD, respectively. | All participants who consented and received transplantation for each stratum. | Posted | | Number | 95% Confidence Interval | Percentage of Participants | | 100 Days post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Number of Participants by Maximum Chronic GVHD Severity Post-Transplant | Chronic GVHD is based on NIH Consensus Criteria (2014 NIH Consensus Criteria) and includes mild, moderate and severe chronic GVHD. Skin/hair, ocular, oral, pulmonary, gastrointestinal, hepatic, genitourinary, musculoskeletal, and hematologic systems were scored on a 0-3 scale to reflect degree of cGVHD involvement. Maximum grade is defined as the maximum grade of chronic GVHD (mild, moderate, or severe) that a participant experiences by 2 years post-transplant. | All participants who consented and received transplantation for each stratum. | Posted | | Count of Participants | | Participants | | 2 Years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Percentage of Participants With Chronic GVHD Post-Transplant | Percentage of participants with chronic GVHD (cGVHD) at will be estimated with 95% confidence intervals for each treatment group using the cumulative incidence estimate with the complementary log-log transformation, treating death prior to cGVHD as a competing event. Chronic GVHD is based on NIH Consensus Criteria (2014 NIH Consensus Criteria) and includes mild, moderate and severe chronic GVHD. Skin/hair, ocular, oral, pulmonary, gastrointestinal, hepatic, genitourinary, musculoskeletal, and hematologic systems were scored on a 0-3 scale to reflect degree of cGVHD involvement. This endpoint considers any cGVHD onset. | All participants who consented and received transplantation for each stratum. | Posted | | Mean | 95% Confidence Interval | Percentage of Participants | | 6 months, 1 year, 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 |
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| Secondary | Number of Participants With Complications and Events Post-Transplant | The number of participants experiencing the following complications and events will be tabulated at baseline, 1 year and 2 years post-transplant: Idiopathic pneumonia syndrome (IPS), Veno-occlusive disease (VOD), Various Central Nervous System (CNS) toxicities, Stroke, participants on immunosuppression for GVHD, and significant infections reported (any bacterial/fungal sepsis, Cytomegalovirus (CMV) reactivation with/without clinical disease, adenovirus, Epstein-Barr Virus (EBV)). | All participants who consented and received transplantation for each stratum. As participants are followed, the number evaluable at 1 and 2 years decreases due to various reasons (lost to follow-up, death, withdrawal of consent, assessment not performed, etc.). | Posted | | Count of Participants | | Participants | | Baseline, 1 year, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | |
|
| Secondary | Number of Participants With Sickle Cell Disease Events of Special Interest Post-Transplant | Participants will be followed for the entire 2 year duration of their time on study for the recurrence of Sickle Cell Disease (SCD) related complications. These SCD related complications are referred to as SCD events of special interest (SCD-EOSI) and will be summarized with frequency. These SCD-EOSI include: pulmonary hypertension, new onset of significant cerebrovascular event, renal function compromise, new onset of avascular necrosis of hip or shoulder, new onset of leg ulceration, new onset of acute chest syndrome, and new onset of painful vaso-occlusive crisis requiring hospitalization or parenteral opioid drug in the outpatient setting. | All participants who consented and received transplantation for each stratum. As participants are followed, the number evaluable at later post-transplant timepoints decreases due to various reasons (lost to follow-up, death, withdrawal of consent, assessment not performed, etc.). | Posted | | Count of Participants | | Participants | | Baseline, 6 months, 1 year, 18 months, and 2 Years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Summary of Hematological Outcomes in Percentages | Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Depending on the measurement, "baseline" could be pre-hydroxyurea conditioning (day -70 pre-transplant) and/or pre-thymoglobulin (day -7 pre-transplant). Measures include: hemoglobin (Hgb) %, reticulocyte count %, and hemoglobin S level %. | All participants who consented and received transplantation in the each stratum. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | percentage of lab measure | | Baseline, Day 28, Day 100, 6 months, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Summary of Glomerular Filtration Rate (GFR) | Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-thymoglobulin (day -7 pre-transplant). Measures include: GFR (mL/min/1.73m2) and Estimated GFR. Estimated GFR is computed using the CKD-EPI Creatinine Equation 2021 for Adults and the Bedside Schwartz 2009 equation for Pediatrics. | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | mL/min/1.73m2 | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
|
| Secondary | Summary of Lung Function in Liters | Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1 (L), FVC (L), and TLC (L). All values in this table were reported by study site. | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | liters (L) | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
|
| Secondary | Summary of Cardiac Function | Cardiac Function is measured through tricuspid regurgitant jet velocity (TRJV). It will be summarized with mean and standard deviation at Baseline, 1, and 2 years post-transplant. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | m/sec | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
|
| Secondary | Summary of Six Minute Walk Distance | Six Minute Walk Distance is measured in meters to assess how far a participant can walk in 6 minutes. It will be summarized with mean and standard deviation at Baseline, 1, and 2 years post-transplant. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Change from baseline will also be provided at the 1 and 2 years post-transplant visit. | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | meters (m) | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
|
| Secondary | Mean Patient Reported Quality of Life (QoL) Score | Health-Related Quality of Life (QoL) assessed using the NIH's PROMIS short forms administered to English- and Spanish-speaking patients in the adult stratum. Questions about fatigue (short form 8a), pain interference (short form 8a), and pain intensity (short form 3a) are asked regarding the degree of difficulty in performing activities of daily living such as housework, social activities, and day to day activities. It is scored from 0 to 10 and converted to a standardized T-score with mean 50 and standard deviation 10. A higher T-score represents worse fatigue, pain interfering with more daily activities, and worse pain intensity. | Participants who consented and received transplantation in the adult stratum. Some participants who were not transplanted were assessed at baseline. Less participants have QoL data collected as time goes on due to participant censoring and other reasons for QoL data not being collected. | Posted | | Mean | Standard Error | T-Score | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Mean Patient Reported Pain Intensity Score From Pain Diary | Subjects who speak English and are 15 years or older will be asked to use a web-based diary to report pain intensity on a scale from 0 (no pain) to 10 (worst pain). Subject pain intensity score is reported as an aggregate of several AM and PM scores at Baseline, one year, and two years post-transplant timepoints. | Participants who enrolled in the adult stratum and submitted an electronic pain diary. Some participants who were not transplanted were assessed at baseline. Less participants have data collected as time goes on due to participant withdrawal, lost to follow-up, death, and other reasons for QoL data not being collected. | Posted | | Mean | Standard Error | T-Score | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
| |
| Secondary | Number of Participants by Cause of Death | Cause of Death is tabulated by age strata. Deaths among participants who were not transplanted are included. | All participants who enrolled in either age stratum. | Posted | | Count of Participants | | Participants | | From Enrollment to 2 Years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Number of Participants by Maximum Grade of Infection | Number of participants who reported the Maximum Infection Severity of Grade 2 and Grade 3. Only grade 2 and grade 3 infections occurring post transplantation were reported on the study. Grade 2 and grade 3 infections are defined by the BMT CTN Technical MOP. Higher infection grade indicates worse infection severity. The infection grading criteria are published online (https://bmtctn.net/administrative-manual-procedures-moppolicy-guidelines). Severity of grade 1, 2 and 3 are described for bacterial, fungal, viral, parasitic, and nonmicrobiological infections. For example, grade 2 fungal infections are defined as candida esophagitis, or proven or probably fungal sinusistis confirmed radiologically without orbital, brain or bone involvement. Grade 3 fungal infections are defined as Fungemia including candidemia, Proven or probably invasive fungal infections, Disseminated infections with histoplasmosis, blastomycosis, coccidiomycosis, or Cryptococcus, or Pneumocystis jiroveci pneumonia. | All participants who consented and received transplantation for each stratum. | Posted | | Count of Participants | | Participants | | From Transplant to 2 Years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Frequencies of Infections Categorized by Infection Type | The number of systemic infections is reported. Infections are categorized by infection type. A participant can report multiple types of infections, so the categories are not mutually exclusive for participants. All grade 2 and grade 3 infections, as defined by the BMT CTN Technical MOP, occurring post transplantation were reported on the study. | All participants who consented and received transplantation for each stratum. | Posted | | Number | | infections | | From Transplant to 2 Years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Frequency of Hospital Readmissions by Cause | Participants could be readmitted to a hospital for many reasons during follow-up. The causes of each readmission are tabulated by age strata. The protocol-specified scheduled transplant hospitalization is not included in this table. | All participants who consented and received transplantation for each stratum. | Posted | | Number | | hospitalizations | | From Transplant to 2 Years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Other Pre-specified | Mean Follow-up Days Since Transplant | Average number of post-transplant follow-up days by age strata. | Only surviving and transplanted participants are included, so 2 pediatrics and 3 adults are excluded from this table. | Posted | | Mean | Standard Deviation | days | | From transplant up to 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Summary of Lactate Dehydrogenase (LDH) | Hematological Outcomes will be summarized with mean and standard deviation at various post-transplant timepoints. Measures in this table include: LDH (U/L) | All participants who consented and received transplantation in the each stratum. Laboratory measurements are not mandatory at all time points. Laboratory measurements at various timepoints may be missing for some participants. | Posted | | Mean | Standard Deviation | U/L | | Day 28, Day 100, 6 months, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Summary of Bilirubin | Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Baseline timepoint is pre-thymoglobulin (day -7 pre-transplant). Measures in this table include: Bilirubin (mg/dL) | All participants who consented and received transplantation in the each stratum. Laboratory measurements are not mandatory at all time points. Laboratory measurements may be unavailable for some participants at various timepoints. | Posted | | Mean | Standard Deviation | mg/dL | | Baseline, Day 28, Day 100, 6 months, 1, and 2 years post-transplant | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
|
| Secondary | Summary of Serum Ferritin | Hematological Outcomes will be summarized with mean and standard deviation at various timepoints. Baseline refers to pre-thymoglobulin (day -7 pre-transplant). Measures in this table include: Serum Ferritin Level (ng/dL) | All participants who consented and received transplantation in the each stratum. Laboratory measurements may be unavailable for some participants. | Posted | | Mean | Standard Deviation | ng/dL | | Baseline pre-thymoglobulin visit (day -7 pre-transplant) | | | | ID | Title | Description |
|---|
| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Summary of Creatinine Clearance | Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-thymoglobulin (day -7 pre-transplant). Measures include: Creatinine Clearance (mL/min). | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | mL/min | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
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| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Summary of Urine Albumin Creatine Ratio | Renal Function Outcomes will be summarized with mean and standard deviation at various timepoints. Measures include: Urine Albumin Creatine Ratio (mg/g). | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | mg/g | | 1 and 2 years post-transplant | | | | ID | Title | Description |
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| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Summary of Lung Function Percent Predicted | Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1 (percent predicted), FVC (percent predicted), FEV1/FVC (percent predicted), and TLC (percent predicted). All values in this table, including percent predicted, were reported by study site. | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | percent predicted | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
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| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | |
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| Secondary | Summary of Lung Function Ratio of Liters | Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1/FVC (ratio of Liters). All values in this table were reported by study site. | All participants who consented and received transplantation in each stratum were expected to provide data. However, the lung function FEV1 and FVC assessments were not collected at baseline in the beginning of the study, and several participants did not contribute lung function data at one and two years due to a COVID-19 pandemic era requirement at some sites for a negative COVID test before collecting lung function data. | Posted | | Mean | Standard Deviation | ratio of liters (L) | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
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| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum |
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| Secondary | Summary of Lung Function Percentages of Lab Measure | Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: DLCO (%) and Oxygen Saturation (%). All values in this table were reported by study site. | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | percent of lab measure | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
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| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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| Secondary | Summary of Lung Function Ratio of Percent Predicted | Lung Function Outcomes will be summarized with mean and standard deviation at various timepoints. "Baseline" refers to pre-hydroxyurea (day -70 pre-transplant). Measures include: FEV1/FVC (ratio of percent predicted). All values in this table were reported by study site. | All participants who consented and received transplantation in the each stratum. Some participants who were not transplanted were assessed at baseline. Laboratory measurements are not mandatory at all time points. | Posted | | Mean | Standard Deviation | ratio of percent predicted | | Baseline, 1, and 2 years post-transplant | | | | ID | Title | Description |
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| OG000 | Adult Stratum | Ages 15.00 - 45.99 years at enrollment with severe SCD Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. | | OG001 | Pediatric Stratum | Ages 5.00 - 14.99 years at enrollment with SCD with strokes. After the adult stratum closed, two participants were enrolled to the pediatric stratum at ages 15.1 and 16.3 with protocol officer and DSMB approval. Participants in both strata receives a BMT preparative regimen will start on Day -70 with hydroxyurea 30mg/kg daily through Day -10. The conditioning regimen will also include Thymoglobulin (rATG) (0.5mg/kg on Day -9, 2mg/kg on Day -8, Day -7), Thiotepa 10mg/kg on Day -7, Fludarabine (30mg/m2 on Day -6 to Day -2), Cyclophosphamide 14.5mg/kg on Day -6 and Day -5, and TBI 200cGy on Day -1. Day 0 is the day of infusion with non T-cell depleted bone marrow. Cyclophosphamide 50 mg/kg and Mesna 40mg/kg is given on Days +3 and +4, and GVHD prophylaxis (sirolimus and mycophenolate mofetil) begin on Day +5. |
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