A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes
Johns Hopkins Kimmel Cancer Center in Baltimore
This research is being done to find out if bone marrow transplantation (BMT) followed by chemotherapy will help people with aplastic anemia who have failed other treatments.
Inclusion criteria:-Patients with relapsed or refractory SAA or very SAA -Age 0-73-Adequate end-organ function Exclusion criteria:-Prior transfusions from selected donor (as this could have cause recipient alloimmunization against the donor)-Women of childbearing potential who currently are pregnant (HCG+) or who are not practicing adequate contraception.-Patients who have any debilitating medical or psychiatric illness that would preclude their giving informed consent or their receiving optimal treatment and follow up.-Uncontrolled viral, bacterial, or fungal infections (HIV infection permitted if viral load undetectable)
You have a severe, life threatening disease (severe aplastic anemia) in your bone marrow. Your 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 yours but complications from Graft vs. Host disease (GVHD) and graft failure have limited the survival for those people.A small study done at Johns Hopkins has shown that in subjects with other diseases (blood cancers) some immunosuppressive drugs given after the BMT have decreased how often subjects had complications of GVHD and engraftment failure.In this study, chemotherapy will be administered before and after bone marrow transplantation. Immunosuppressive agents will be given after BMT for GVHD prophylaxis.
03/05/2019 05:03 AM