What is a possible rescue therapy for aplastic anemia?

Updated: Jan 29, 2021
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Nonmyeloablative peripheral blood stem cell transplantation from HLA-haploidentical family donors has been proposed as rescue therapy in patients with refractory SAA or VSAA. [85] A systematic review and meta-analysis concluded that haploidentical HCT is a promising approach in idiopathic aplastic anemia, with high engraftment rates and reduced complication rates. In 15 studies that included 577 patients, successful engraftment occurred in 97.3% of patients (95% confidence interval [CI], 95.9-98.7). Grades II-IV acute and chronic GVHD were reported in 26.6% and 25.0% of patients, respectively. The pooled incidence of transplant-related mortality was 6.7% per year (95% CI, 4.0-9.4%). [86]

Rates of successful engraftment were higher with reduced-intensity conditioning compared with nonmyeloablative conditioning (97.7% vs 91.7%, P = 0.03) and rates of acute GVHD were lower (29.5% vs 18.7%, P = 0.008). No differences in the incidence rates of chronic GVHD or mortality were found. Posttransplantation regimens containing cyclophosphamide were associated with reduced rates of acute GVHD, cytomegalovirus (CMV) viremia, and CMV disease in initially viremic patients, compared with methotrexate-containing regimens and other regimens. The authors of this study recommend prospective trials to identify the preferred conditioning regimen, GVHD prophylaxis, and graft source for haploidentical HCT for aplastic anemia. [86]


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