First-line haploidentical stem cell transplantation in children and adolescents with severe aplastic anemia using mobilized peripheral blood as source of CD34+: Single-institutional experience in a transplant center from northeast Mexico.

Abstract

The only curative treatment for severe aplastic anemia in children is an allogeneic stem cell transplant; however, few patients have a matched related or unrelated donor. Haploidentical stem cell transplantation (haplo-SCT) using bone marrow (BM) and peripheral blood stem cells (PBSC) has been recently described as effective and safe. In this study, we retrospectively report the outcome of twelve pediatric patients who underwent haplo-SCT using only PBSC. The conditioning regimen consisted on rabbit anti-thymocyte globulin (r-ATG) 2.5mg/kg/d on days -7, -6,-5, and -4, and cyclophosphamide (Cy) 50mg/kg/d on days -3 and -2. We used Cy 50mg/kg/d on days +3 and +4, tacrolimus and mycophenolic acid as graft versus host disease (GVHD) prophylaxis. The median follow-up was 1,099days (45-1258days). The overall survival rate up-to-date is 83.3%. In 10 of the 12 patients, a sustained graft was achieved. None of the patients had acute or chronic GVHD. Haplo-SCT could be established as a first-line treatment when there is no matched related or unrelated donor. According to this short sample and previous reports, PBSC are a feasible option effectively used as the sole source of stem cells. Additionally, post-transplant cyclophosphamide remains a good strategy for GVHD prevention.