Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

Study Overview
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Brief Summary
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Patients with severe, refractory aplastic anemia have a severe, life threatening disease in their bone marrow. Refractory disease means that disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed by bone marrow transplantation (BMT) has been used to treat blood diseases like aplastic anemia but complications from Graft vs Host disease (GVHD) and graft failure have limited the survival for those patients. Another study done here at Johns Hopkins has shown that in patients with other diseases (blood cancers) some immunosuppressive drugs given after the BMT has decreased how often patients had complications of GVHD and engraftment failure. This research is being done to find if this approach will help patients with aplastic anemia who have failed other treatments will have better outcomes.

Official Title
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A Phase II Trial of Myeloablative Conditioning and Transplantation of HLA-matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated (MUD) Bone Marrow for Patients With Refractory Severe Aplastic Anemia

Conditions
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Severe Aplastic Anemia

Intervention / Treatment
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* Procedure: Bone Marrow Transplant


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients who have refractory or relapsed SAA following treatment with one or more immunosuppressive regimens. Age 6 months - 70 years Patients must meet medical criteria for myeloablative BMT Patients or their parents/guardians and donors must be able to sign consent forms. Patients must be geographically accessible and willing to participate in all stages of treatment. Exclusion Criteria: Poor cardiac function: left ventricular ejection fraction <45% as determined by MUGA or ECHO. Poor pulmonary function: FEV1 and FVC <50% predicted. Poor renal function Positive leukocytotoxic crossmatch Women of childbearing potential who currently are pregnant (Β-HCG+) or who are not practicing adequate contraception Uncontrolled viral, bacterial, or fungal infections

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Procedure: Bone Marrow Transplant|Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To estimate the overall survival (OS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia |  | 1 year | 
| To estimate the event-free survival (EFS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia |  | 1 year |