| Primary | Event-Free Survival Probability During the 5 Years After Transplant | Event-free survival (EFS) is survival without death or disease activity from any one of the following criteria: 1) loss of neurological function, defined as a change in pretransplant Extended Disability Status Scale (EDSS) of > 0.5. 2) Relapse, defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit/disability, and lasting over 48 hours. 3) New lesions on magnetic resonance imaging (MRI), defined as presence of 2 or more independent multiple sclerosis brain lesions detected on MRI 1 year or more after stem cell transplant. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT). | Posted | | Number | 90% Confidence Interval | Probability | | 5 years | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| | | Title | Measurements |
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| - OG0000.692(0.502 to 0.821)
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| Secondary | Event-Free Survival Probability During the 3 Years After Transplant | Event-free survival (EFS) is survival without death or disease activity from any one of the following criteria: 1) loss of neurological function, defined as a change in pretransplant Extended Disability Status Scale (EDSS) of > 0.5. 2) Relapse, defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit/disability, and lasting over 48 hours. 3) New lesions on magnetic resonance imaging (MRI), defined as presence of 2 or more independent multiple sclerosis brain lesions detected on MRI 1 year or more after stem cell transplant. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT). | Posted | | Number | 90% Confidence Interval | Probability | | 3 years | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants received Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to > 500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Survival From Treatment-Related Mortality | The probability that a participant did not experienced a treatment-related death estimated at 1, 2, 3, 4, and 5 years following transplant via the Kaplan-Meier Method. Greenwood's formula for standard error was used to calculate 90% confidence intervals. Participants that did not experience a treatment-related death were censored at the time of last follow-up. A treatment-related death was defined as death that occurred at any time after study entry and that was possibly, probably, or definitely related to the cellular product or possibly, probably, or definitely related to mobilization of autologous peripheral blood hematopoietic progenitor cells with G-CSF and prednisone or to the high-dose immunosuppressive therapy. There were no treatment-related mortality events in the study. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT). | Posted | | Number | 90% Confidence Interval | Probability | | From study entry to death, loss to follow-up, or the end of the study, whichever came first, up to 6 years | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥ 2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Overall Survival | The probability that a participant did not experienced a death estimated at 1, 2, 3, 4, and 5 years following transplant via the Kaplan-Meier Method. Greenwood's formula for standard error was used to calculate 90% confidence intervals. Participants that did not die were censored at the time of last follow-up. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Number | 90% Confidence Interval | Probability | | From study entry to death, loss to follow-up, or the end of the study, whichever came first, up to 6 years | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Survival From MS-Related Mortality | The probability that a participant did not experienced a MS-related death estimated at 1, 2, 3, 4, and 5 years following transplant via the Kaplan-Meier Method. Greenwood's formula for standard error was used to calculate 90% confidence intervals. Participants that did not experience a MS-related death were censored at the time of last follow-up. A MS-related death was defined as death that occurred at any time after study entry and that was possibly, probably, or definitely related to disease progression. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Number | 90% Confidence Interval | Probability | | From study entry to death, loss to follow-up, or the end of the study, whichever came first, up to 6 years | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Percent of Participants Who Experienced All-Cause Morbidity | Morbidity is the occurrence of NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0 adverse event grade 3 or higher. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Number | | percentage of participants | | From the time of enrollment until completion of the 5-year follow-up, an average of 6 years. | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Percent of Participants Who Experienced All-Cause Morbidity Within 12 Months of Post-HCT | Morbidity is the occurrence of NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0 adverse event grade 3 or higher. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT). | Posted | | Number | | Percentage of Participants | | From the time of Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) to 1 year after HCT. | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Time to Neutrophil Engraftment | Neutrophil engraftment, or neutrophil count recovery, is defined as an Absolute Neutrophil Count (ANC) > 500/ μL for 2 consecutive measurements on different days. Normal range is 1500 to 8000/μL. Reference: http://www.medicinenet.com | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Mean | Standard Deviation | Days | | From time of graft infusion to time of engraftment, up to 6 years | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Time to Platelet Engraftment | | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Mean | Standard Deviation | Days | | From time of graft infusion to time of engraftment, up to 6 years | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Event-Free Survival Probability After Transplant | Event-free survival (EFS) is survival without death or disease activity from any one of the following criteria: 1) loss of neurological function, defined as a change in pretransplant Extended Disability Status Scale (EDSS) of > 0.5. 2) Relapse, defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit/disability, and lasting over 48 hours. 3) New lesions on magnetic resonance imaging (MRI), defined as presence of 2 or more independent multiple sclerosis brain lesions detected on MRI 1 year or more after stem cell transplant. Kaplan-Meier estimates of survival probability, with 90% confidence intervals based on Greenwood's formula for standard error. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Number | 90% Confidence Interval | Probability | | 1, 2, and 4 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | MS Progression-Free Survival Probability After Transplant | MS progression is measured as number of days from transplant to first Kurtzke's Expanded Disability Status Scale (EDSS) increase of more than 0.5 relative to the baseline measurement. EDSS assesses disability in Multiple Sclerosis patients. Eight functional systems are evaluated: visual, brain stem, pyramidal, cerebellar, sensory, bowel and bladder, cerebral, and ambulation. The overall score ranges from 0 (normal neurological exam) to 10 (death due to MS). Kaplan-Meier estimates of survival probability, with 90% confidence intervals based on Greenwood's formula for standard error. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Number | 90% Confidence Interval | Probability | | 1 to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | MRI Activity-Free Survival Probability After Transplant | MS disease activity is measured as days from transplant to first occurrence of >= 2 new MS lesions on Magnetic resonance imaging (MRI) relative to baseline. Kaplan-Meier estimates of survival probability, with 90% confidence intervals based on Greenwood's formula for standard error. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Number | 90% Confidence Interval | Probability | | 1 to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | MS Relapse-Free Survival Probability After Transplant | MS clinical relapse is defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit or disability, and lasting over 48 hours. Clinical relapse was determined by the participant's neurologist and was measured as days from transplant to new or worsening neurological symptom relative to baseline. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Number | 90% Confidence Interval | Probability | | 1 to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Disease-Modifying Therapy Survival Probability After Transplant | Treatment with disease-modifying therapy was measured by the number of days from transplant to the first treatment with an additional disease-modifying therapy. Examples of therapy include interferon beta-1a, glatiramer acetate, natalizumab, alemtuzumab, other immunosuppressive medications, or experimental therapies directed against MS activity. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Number | 90% Confidence Interval | Probability | | 1 to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Change From Baseline in Extended Disability Status Scale (EDSS) | Kurtzke's Expanded Disability Status Scale (EDSS) assesses disability in Multiple Sclerosis patients. Eight functional systems are evaluated: visual, brain stem, pyramidal, cerebellar, sensory, bowel and bladder, cerebral, and ambulation. The overall score ranges from 0 (normal neurological exam) to 10 (death due to MS). Change from baseline was computed as the value at the time point minus the baseline value. A negative value in change from baseline indicates an improvement and a positive value indicates worsening. A change of > 0.5 in EDSS was a treatment-failure criterion. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Mean | Standard Deviation | units on a scale | | 6 months to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Change From Baseline in Number of Gadolinium-Enhanced Lesions | Multiple sclerosis disease-related lesions were assessed by gadolinium-enhanced magnetic resonance imaging (MRI). Change from baseline was computed as the value at the time point minus the baseline value. A negative value in change from baseline indicates an improvement and a positive value indicates worsening. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Mean | Standard Deviation | Lesions per scan | | 8 weeks to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Number of New T2-Weighted Lesions From Baseline | A T2-weighted magnetic resonance imaging (MRI) scan was used to determine the number of new T2 lesions in the brain relative to Baseline. A value of 0 means that the participant didn't worsen. Values greater than 0 indicate an increase in disease activity from baseline. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Mean | Standard Deviation | Lesions per scan | | 6 Months to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Change From Baseline in T2-Weighted Lesion Volume | A T2-weighted magnetic resonance imaging (MRI) scan was used to assess the volume of T2 lesions in the brain. Change from baseline was computed as the value at the time point minus the baseline value. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Mean | Standard Deviation | milliliters | | 8 weeks to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Change From Baseline in T1-Weighted Lesion Volume | A T1-weighted magnetic resonance imaging (MRI) scan was used to assess the volume of T1 lesions in the brain. Change from baseline was computed as the value at the time point minus the baseline value. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Mean | Standard Deviation | milliliter | | 8 weeks to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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| Secondary | Percent Change From Screening in Brain Volume | Magnetic resonance imaging (MRI) scan techniques measured ventricular volumes and grey and white matter brain volumes. Change from screening was computed as the value at the time point minus the screening value. | Participants who received High-Dose Immunosuppressive Therapy (HDIT) and Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) | Posted | | Mean | Standard Deviation | Percent Change | | 8 weeks to 5 years after HCT | | | | ID | Title | Description |
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| OG000 | HDIT and HCT | Participants recv'd Granulocyte Colony Stimulating Factor (G-CSF) by injection for 4-5 days to increase the number of blood stem cells in the blood stream by mobilizing them from the bone marrow. Leukapheresis was performed until ≥2.0 x 10^6 CD34+ hematopoietic progenitor cells (HPCs) per kg were collected and frozen for future use. After ≥7 days following the last G-CSF dose, patients were hospitalized and received high-dose immunosuppression (HDIT) and immunosuppressive agent thymoglobulin 2.5 mg/kg over the course of 6 days. HDIT consisted of carmustine 300mg/m^2, etoposide 200 mg/m^2 cytarabine 200 mg/m^2, and melphalan 140 mg/m^2. CD34+ HPCs were thawed and infused according to institutional best practice for blood component transfusion over approximately 30 minutes. Patients were hospitalized until recovery of ANC to >500/uL for at least 2 days. Prednisone was administered (0.5 mg/kg/day) from Days 7 to 21 after HCT and tapered over 2 weeks to prevent engraftment syndrome. |
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