Severe Aplastic Anemia
Conditions
Brief summary
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.
Interventions
PROCEDUREBone Marrow Transplant
Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy
Sponsors
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 70 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | 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 |
Countries
United States
Outcome results
None listed