| Primary | Percentage of Participants With Overall Survival (OS) at 2 Years After Biologic Assignment | Due to incomplete accrual, there was not adequate statistical power to analyze the primary endpoint as specified. Instead, point estimates for the observed proportion of patients surviving at two years post-biologic assignment in each arm were generated descriptively, with 95% CI, using the Kaplan Meier methodology. The event of interest was death from any cause. | Using the intent-to-treat principle, participants remained in their assigned treatment arm regardless of treatment received. | Posted | | Number | 95% Confidence Interval | percentage of participants | | 2 Years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
| | | Title | Denominators | Categories |
|---|
| | | Title | Measurements |
|---|
| - OG00089(78 to 100)
- OG00194(87 to 99)
|
|
| |
| Secondary | Frequencies of Sickle Cell Disease (SCD) Events of Special Interest | Examination of the occurrence of the SCD-related events was performed. Participants can have multiple events. The following sickle cell disease related events of special interest (SCD-EOSI) are expected events for all participants, regardless of biologic assignment:
- pulmonary hypertension
- significant cerebrovascular events, including: stroke; transient ischemic attack; seizure
- renal function compromise, including: proteinuria; increased creatinine grades ≥2 per CTCAE version 4.0
- avascular necrosis of the hip or shoulder
- leg ulcers
- acute chest syndrome (ACS) requiring hospitalization
- vaso-occlusive pain crisis (VOC) requiring hospitalization or administration of parenteral opioid drugs in an outpatient setting. Self-reported events without clinical documentation should not be included.
| Using the intent-to-treat principle, participants remained in their assigned treatment arm regardless of treatment received. | Posted | | Number | | event | | 2 Years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | |
|
| Secondary | Change in 6-minute Walk Distance (6MWD) Assessment From Baseline | The 6-minute walk distance (6MWD) test is a test to measure how far you can walk in 6 minutes. It is used in this study to assess exercise capacity. The number of meters that a participant could walk in 6 minutes was selected as a direct measurement of physical function. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | meter | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
| |
| Secondary | Change in Transcuspid Regurgitant Jet Velocity (TRJV) Assessment From Baseline | Transcuspid regurgitant jet velocity (TRJV) is a measure of how fast blood is pumped through one of the heart valves on the right side of the heart, measured using an ultrasound of the heart (echocardiogram). Higher values can indicate pulmonary hypertension and more severe sickle cell disease. Increasing TRJV over time indicates worsening disease. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | meters per second (m/s) | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Albuminuria Assessment From Baseline | Albuminuria is the ratio of albumin (a protein) in the urine to creatinine in the urine. This may be assessed based on a 24-hour urine sample or a spot urine sample. Albumin in the urine may indicate kidney disease. Increases in albuminuria over time indicate increasing renal (kidney) disease. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | mg of albumin per gram of creatine | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component Physical Function Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. Physical Function asks about degree of difficulty in performing activities of daily living such as housework, errands, and going up and down stairs. 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 better physical function. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component Anxiety Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. Anxiety asks about the frequency of feelings of fear, worry, and anxiety. 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 more anxiety. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component Depression Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. Depression asks about the frequency of feelings of worthlessness, failure, unhappiness and depression. 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 more depression. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component Fatigue Score Changes in From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. Fatigue asks about the extent to which fatigue interferes with physical functioning and completing tasks. 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 more fatigue. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component Sleep Disturbance Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. Sleep Disturbance asks about the quality of sleep and the frequency with which sleep was restless or difficult to achieve. 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 more sleep disturbance. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component Participation in Social Roles Score Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. Participation in Social Roles asks about the frequency of limitations of social activities with friends and family. 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 more satisfaction with participation in social roles. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component Pain Interference Score Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. Pain Interference asks about the frequency with which pain interferes with household chores, social activities and enjoyment of life. 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 more pain interference in daily life. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component Pain Intensity Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. Pain Intensity is scored from 0 to 10 reflecting the level of pain over the previous 7 days. A score of 0 reflects no pain; a score of 10 reflects the maximum intensity of pain. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
| |
| Secondary | Health-Related Quality of Life (HRQoL) Component 28-Day Pain Diary Average Pain Intensity Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. The 28-Day Pain Diary Average Pain Intensity assesses pain on a score of 0 to 10 twice each day for 28 days. A score of 0 reflects no pain, and a score of 10 reflects maximum intensity of pain. The scores on a given day are averaged (if only one score is obtained on a given day, that score is taken as the measure), and then daily scores are averaged over the number of days on which a score is available. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | units on a scale | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | |
|
| Secondary | Health-Related Quality of Life (HRQoL) Component ASCQ-Me Stiffness Changes From Baseline | Health-Related Quality of Life (HRQoL) assessed using the NIH's PROMIS 57 instrument. The ASCQ-Me Stiffness Scale asks about the frequency and severity of joint stiffness. 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 more stiffness. | Participants that are biologically assigned to different arms and that completed both the baseline and 2-year assessment; this follows the intent-to-treat principle. | Posted | | Median | Full Range | T-score | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Forced Expiratory Volume 1 Second (FEV1) From Baseline | Pulmonary function is assessed by the change from baseline in Forced Expiratory Volume 1 second (FEV1). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of FEV1 | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Forced Vital Capacity (FVC) From Baseline | Pulmonary function is assessed by the change from baseline in forced vital capacity (FVC). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of FVC | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Ratio of FEV1 to FVC (FEV1/FVC) From Baseline | Pulmonary function is assessed by the change from baseline in ratio of FEV1 to FVC (FEV1/FVC). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of FEV1/FVC | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Vital Capacity (VC) From Baseline | Pulmonary function is assessed by the change from baseline in vital capacity (VC). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of VC | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Total Lung Capacity (TLC) From Baseline | Pulmonary function is assessed by the change from baseline in total lung capacity (TLC). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of TLC | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Residual Volume (RV) From Baseline | Pulmonary function is assessed by the change from baseline in residual volume (RV). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of RV | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Expiratory Reserve Volume (ERV) From Baseline | Pulmonary function is assessed by the change from baseline in expiratory reserve volume (ERV). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of ERV | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Inspiratory Capacity (IC) From Baseline | Pulmonary function is assessed by the change from baseline in inspiratory capacity (IC). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of IC | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Functional Residual Capacity (FRC) From Baseline | Pulmonary function is assessed by the change from baseline in functional residual capacity (FRC). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of FRC | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Diffusing Capacity for Carbon Monoxide (DLCO) From Baseline | Pulmonary function is assessed by the change from baseline in diffusing capacity for carbon monoxide (DLCO). The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of DLCO | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Change in Pulmonary Function of Oxygen Saturation From Baseline | Pulmonary function is assessed by the change from baseline in oxygen saturation. The complete pulmonary function test will include forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), ratio of FEV1 to FVC (FEV1/FVC), vital capacity (VC), total lung capacity (TLC), residual volume (RV), expiratory reserve volume (ERV), inspiratory capacity (IC), functional residual capacity (FRC), diffusing capacity for carbon monoxide (DLCO), and oxygen saturation. | Analysis population includes participants that are biologically assigned to different arms and completed both baseline and 2-year assessment. | Posted | | Median | Full Range | percentage of oxygen saturation | | Baseline, 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
|
| Secondary | Frequencies of Infections | All Grade 2 and 3 infections were reported according to the BMT CTN Technical Manual of Procedures (MOP) after biological assignment. The frequency of Grade 2-3 infections are tabulated by biological assignment at the event level. | Using the intent-to-treat principle, participants remained in their assigned treatment arm regardless of treatment received. | Posted | | Number | | infection | | 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft | | OG001 | No-Donor Arm | No-donor arm patients will continue with standard of care per their SCD physician. Standard of Care: Continue to receive standard of care treatment per patient's SCD physician. |
| |
| Secondary | Percentage of Participants With Grades II-IV Acute GVHD at Day 180 After Biological Assignment | The time interval from day 0 of transplant until grade II-IV aGVHD will be described for each treatment arm using the the cumulative incidence estimator developed by Gray, with death prior to aGVHD treated as a competing risk. Estimates and 95% CIs of the cumulative incidence of grade II-IV aGVHD will be provided at Day 180 after day 0 of transplant and compared between matched related and matched unrelated donors using the Gray test. Acute GVHD was graded according to the BMT CTN Technical Manual of Operating Procedures (MOP). Higher aGVHD grade indicate worse outcomes. Grade I is defined as Skin stage 1-2 and stage 0 for both GI and liver. Grade II is stage 3 skin, stage 1 GI, or stage 1 liver. Grade III is stage 2-3 GI or stage 2-3 liver. Grade IV is stage 4 skin or stage 4 liver. | Patients who enrolled on the donor arm and received transplant | Posted | | Number | 95% Confidence Interval | percentage of participants | | 180 days after biological assignment | | | | ID | Title | Description |
|---|
| OG000 | HLA Matched Sibling Donor | For patients who enrolled on the donor arm, 10 patients received transplant from sibling donors. | | OG001 | Unrelated Donor | For patients who enrolled on the donor arm, 14 patients received transplant from unrelated donors. |
| |
| Secondary | Percentage of Participants With Chronic GVHD at Day 600 After Biological Assignment | Chronic GVHD was collected according to the recommendations of the NIH Consensus Conference. Eight organs will be scored on a 0-3 scale to reflect degree of chronic GVHD involvement. Liver and pulmonary function test results and use of systemic therapy for treatment of chronic GVHD was also recorded. Percentage of Participants with chronic GVHD (cGVHD) at Day 600 was estimated with 95% confidence intervals for each donor type group using the cumulative incidence estimate, treating death prior to cGVHD as a competing event. | Patients who enrolled on the donor arm and received transplant. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 600 after biological assignment | | | | ID | Title | Description |
|---|
| OG000 | HLA-matched Sibling Donor | For patients who enrolled on the donor arm, 10 patients received transplant from sibling donors. | | OG001 | Unrelated Donor | For patients who enrolled on the donor arm, 14 patients received transplant from unrelated donors. |
| |
| Secondary | Number of Participants With Primary and Secondary Graft Failure | Primary graft failure defined as never achieving ANC ≥ 500/µL or never achieving ≥ 5% donor whole blood or myeloid chimerism (myeloid is preferable) assessed by bone marrow or peripheral blood chimerism assays by day +42 post-transplant. Second infusion of hematopoietic cells is also considered indicative of primary graft failure by day +42 post-transplant. Secondary graft failure is defined as < 5% donor whole blood or myeloid chimerism (myeloid is preferable) 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. Second infusion of hematopoietic cells is also considered indicative of secondary graft failure. | Analysis population includes transplanted participants who were biologically assigned to donor arm. | Posted | | Number | | participants | | 2 years | | | | ID | Title | Description |
|---|
| OG000 | Donor Arm | Donor Arm patients will undergo hematopoietic cell transplant. Patients with a matched unrelated donor will receive a bone marrow transplant (unless PBSC graft is pre-approved per section 2.5.1 using a preparative regimen with Busulfan, Fludarabine and rabbit ATG. Patients with an HLA-identical sibling donor can receive a transplant using one of three regimens: A. Busulfan, Fludarabine, and rabbit ATG using a bone marrow graft (preferred regimen) B. Alemtuzumab/TBI 300 cGy using a peripheral blood graft C. Alemtuzumab, fludarabine, melphalan using a bone marrow graft |
| |