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HLA-haploidentical bone marrow benefited patients with sickle cell disease
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Ten of 14 patients with sickle cell disease who received bone marrow from HLA-haploidentical donors were asymptomatic at follow-up, according to study results.
The current report discusses the development of a non-myeloablative bone marrow transplantation platform that employs related donors for patients with sickle cell disease. The study population included HLA-haploidentical donors, who often are difficult to locate.
The treatment regimen employed by the study investigators included anti-thymocyte globulin, fludarabine, cyclophosphamide and total body irradiation, as well as graft-versus-host disease prophylaxis with post-transplantation high-dose cyclophosphamide, mycophenolate mofetil and tacrolimus or sirolimus.
The final analysis included 17 patients, 14 of whom received marrow from HLA-haploidentical donors and three of whom received marrow from HLA-matched donors.
Durable grafts were reported in 11 patients. The median follow-up duration was 711 days, with the minimum 224 days. At follow-up, 10 patients were asymptomatic and six were no longer on immunosuppression.
One patient developed skin-only acute graft-versus-host disease. This case resolved with no treatment, and there was no mortality reported.
Graft failure, which occurs in 43% of haploidentical pairs, continues to be a challenge for clinicians to overcome, according to the researchers. However, this complication may be acceptable in a small ratio of patients if the majority may be cured without toxicities, the investigators said.
“Non-myeloablative conditioning with post-transplantation high-dose cyclophosphamide expands the donor pool, making marrow transplantation feasible for most patients with sickle cell disease,” they concluded. “[It also] is associated with a low risk of complications, even with haploidentical-related donors.”
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