Experts at The Johns Hopkins Kimmel Cancer are nationally and internationally recognized as leaders in the research and treatment of aplastic anemia and other blood disorders. Our experienced and dedicated team provides our patients with innovative treatments and the best quality of care possible.

Research Papers

Durable treatment-free remission after high-dose cyclophosphamide therapy for previously untreated severe aplastic anemia. Ann Intern Med. 2001 Oct 2;135(7):477-83. doi: 10.7326/0003-4819-135-7-200110020-00006. PMID 11578150.

Summary:
Johns Hopkins researchers led by Robert Brodsky show in a clinical trial that high-dose cyclophosphamide treatment can cause durable, treatment-free remission of disease in some aplastic anemia patients. Cyclophosphamide is a potent immunosuppressive drug that is used to pre-treat patients who receive bone marrow transplants. In the study, 19 patients with severe untreated aplastic anemia received four days of high-dose cyclophosphamide treatment. At 24 months after treatment, the probability of survival for the patients was 84% and the probability of achieving treatment-free remission was 73%, and at 50 months after treatment the probability of complete remission was 65%. None of the patients who responded to the treatment had a relapse or secondary disorders such as a proliferation of blood stem cell mutations.

Haploidentical Donor Bone Marrow Transplantation for Severe Aplastic Anemia. Hematol Oncol Clin North Am. 2018 Aug;32(4):629-642. doi: 10.1016/j.hoc.2018.04.001. Epub 2018 May 28. PMID 30047416.

Summary:
This study by Amy DeZern and Robert Brodsky is an overview of how bone marrow transplantation from “half-matching” or haploidentical donors is used to treat patients with severe aplastic anemia. Bone marrow donors are matched with patients based on protein markers on blood cells. Haploidentical donors share half of these markers with patients. In aplastic anemic patients with hard-to-treat disease, haploidentical bone marrow treatment is increasingly recommended. The procedure used to lead to high rates of the transplant being rejected by the patient, but post-transplant high doses of the immune-suppressing drug cyclophosphamide has lessened the risk of rejection-related disease, they note.

Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide. Blood Adv. 2020 Apr 28;4(8):1770-1779. doi: 10.1182/bloodadvances.2020001729. PMID 32343796.

Summary:
In a study led by John Hopkins physicians, bone marrow transplantation from “half-matching” or haploidentical donors, along with post-transplant doses of the drug cyclophosphamide, was used to treat 37 patients with severe aplastic anemia. The researchers compared the success of the transplants in different types of patients, including those who received radiation treatments as well. All 20 of the patients in the study who had resistant or relapsing disease were alive and disease free with no signs of transplant rejection one to two years later. Patients who had never been treated for aplastic anemia before had higher rates of transplant failure, but radiation treatment may increase the rate of durable transplants.