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New Transplant Platform Successful in Sickle Cell

Last Updated: September 27, 2012.

Non-myeloablative conditioning with post-transplantation high-dose cyclophosphamide expands the donor pool for bone marrow transplantation for patients with sickle cell disease, according to a preliminary study published online Sept. 6 in Blood.

THURSDAY, Sept. 27 (HealthDay News) -- Non-myeloablative conditioning with post-transplantation high-dose cyclophosphamide expands the donor pool for bone marrow transplantation for patients with sickle cell disease, according to a preliminary study published online Sept. 6 in Blood.

Javier Bolaños-Meade, M.D., from Johns Hopkins University in Baltimore, and colleagues developed a non-myeloablative bone marrow transplantation platform using related, including HLA-haploidentical, donors for patients with sickle cell disease.

The researchers performed a transplant in 17 sickle cell patients: 14 from HLA-haploidentical and three from HLA-matched related donors. Eleven patients engrafted durably. At a median follow-up of 711 days (minimal follow-up, 224 days), 10 patients were asymptomatic and six patients were off immunosupression. Skin-only acute graft-versus-host disease occurred in one patient and resolved without any therapy. No mortality was observed.

"Non-myeloablative conditioning with post-transplantation high-dose cyclophosphamide expands the donor pool making marrow transplantation feasible for most patients with sickle cell disease and is associated with a low risk of complications, even with haploidentical related donors," the authors write.

Several authors are listed as inventors on a patent application related to the study subject matter; one author disclosed financial ties to HemaQuest Pharmaceuticals.

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