Excellent Outcome of Haploidentical Hematopoietic Stem Cell Transplantation in Children and Adolescents with Acquired Severe Aplastic Anemia

Abstract

We evaluated the outcome of children and adolescents with acquired severe aplastic anemia (SAA) who received haploidentical hematopoietic stem cell transplantation (HHCT) with in vitro T cell-depleted peripheral blood stem cells. Twelve patients with acquired SAA received a total of 15 HHCTs with in vitro CD3-depleted grafts between July 2009 and July 2012. Among the 12 patients, 11 achieved neutrophil engraftment at a median of 10 days (range, 9 to 13 days) after HHCT. One patient failed to achieve primary engraftment, and two experienced graft rejection soon after engraftment. All three patients who experienced early graft failure received a second HHCT and achieved sustained engraftment. Thus, the final engraftment rate was 100%. Acute graft-versus-host disease was assessed in 9 patients, excluding the 3 patients with early graft failure. Three of these patients developed acute graft-versus-host disease (two ≥ grade II and one with grade III). All 12 patients survived and were transfusion-independent at a median follow-up of 14.3 months (range, 4.1 to 40.7 months). Hematopoietic stem cell transplantation from haploidentical family donors with in vitro CD3 T cell depletion is a reasonable therapeutic option for children and adolescents with acquired SAA. Our future trial with a uniform protocol will help to solve the problems associated with HHCT and provide a valuable platform for the further development of HHCT as a therapy for SAA.

Haploidentical hematopoietic stem cell transplantation for nonresponders to immunosuppressive therapy against acquired severe aplastic anemia

“…3 When the partially matched family donor transplant option was initially used, it was unsuccessful owing to the high rate of graft failure (GF) and refractory GvHD, despite its ability to render donors available to almost all patients. 2,4 Delayed immune recovery and the high incidence of infection were also major clinical obstacles. 5,6 These drawbacks affect the universal use of haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) for SAA.…”

Refinement of treatment strategies in ex vivo T-cell-depleted haploidentical SCT for pediatric patients

“…We reported our experience with CD3-depleted HHCT for patients with acquired SAA and patients with graft failure (GF) after allogeneic HCT. 5,6 These patients constitute part of the current patient cohort: of 28 patients in the present cohort, 17 were included in the previous two reports. The purpose of the present study was to assess the efficacy and feasibility of CD3-depleted HHCT in the children and adolescents of our cohort with an additional 11 patients and with an extended follow-up of previously reported cases.…”

Acquired aplastic anemia in Korean children: treatment guidelines from the Bone Marrow Failure Committee of the Korean Society of Pediatric Hematology Oncology

“…Most strikingly, Im et al [46] reported excellent survival of 100 % for 12 AA patients after CD3-depleted HSCT in Korean children. The cumulative incidence of grade III-IV acute GVHD and extensive chronic GVHD were 14 and 11 %, respectively.…”