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Expanding the Donor Pool for Severe Aplastic Anemia
Biol Blood Marrow Transplant; ePub 2016 Dec 22; DeZern, et al
Non-myeloablative allo-bone marrow transplantation (alloBMT) using post-transplant cyclophosphamide (PTCy) allowed inclusion of HLA-haploidentical donors for recipients with severe aplastic anemia (SAA) in a study involving 32 individuals.
Participants included patients with refractory SAA and no matched sibling donors (n=16), as well as haploidentical (n=13) and unrelated (n=3) donors. Patients underwent alloBMT, and received PTCy 50mg/kg/day IV on days +3, +4. They also received mycophenolate mofetil beginning day+5 through 35 and tacrolimus day +5 through 1 year. Median time to:
- Neutrophil recovery over 1000 × 103/mm3 for 3 consecutive days: 19 days.
- Red cell engraftment: 25 days.
- Last platelet transfusion to keep platelets counts over 50 × 103/mm3: 28 days.
Additionally, by the study’s conclusion:
- No patients experienced graft failure.
- All patients were alive, transfusion independent, and without evidence of clonality.
- 2 patients had grade 1-2 skin-only acute GVHD; both were able to come off all immunosuppression at ~16 months.
- All other patients stopped immunosuppression at 1 year.
DeZern A, Zahurak M, Symons H, Cooke K, Jones R, Brodsky R. Alternative donor transplantation with high-dose post-transplant cyclophosphamide for refractory severe aplastic anemia. [Published online ahead of print December 22, 2016]. Biol Blood Marrow Transplant. doi:10.1016/j.bbmt.2016.12.628.