Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

Johns Hopkins Medicine

Status and phase

Terminated

Phase 2

Conditions

Severe Aplastic Anemia

Treatments

Procedure: Bone Marrow Transplant

Study type

Interventional

Funder types

Other

Identifiers

NCT01383434

NA_00042991 (Other Identifier)

J10117

Details and patient eligibility

About

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.

Enrollment

4 patients

Sex

All

Ages

6 months to 70 years old

Volunteers

No Healthy Volunteers

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