First‐line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy

Abstract

We retrospectively compared the outcomes of children with severe aplastic anemia (SAA) who received immunosuppressive therapy (IST) or who underwent hematopoietic stem cell transplantation (HSCT) from a haploidentical donor (HID), between 2007 and 2016. A total of 52 children with SAA under the age of 17 years were initially treated with IST (n = 24) or haploidentical HSCT (n = 28) as first-line treatment. The estimated 10-year overall survival was 73.4 ± 12.6% and 89.3 ± 5.8% in patients treated with IST or HID-HSCT (P = .806). The failure-free survival was significantly inferior in patients receiving IST than in those undergoing transplantation from an HID (52.6 ± 10.5% vs 89.3 ± 5.8, P = .008). In univariate and multivariate analysis, the choice of first-line immunosuppressive therapy was the only adverse predictor for failure-free survival. At the last follow-up, completely normal blood count was observed in 11 of 20 (55.0%) and 24 of 25 (96.0%) live cases in IST and HID-HSCT cohort (P = .003). These suggest that HSCT from a haploidentical donor could be considered as first-line treatment in children who lack a matched related donor, especially in experienced transplantation centers.

Effect of Stem Cell Source and Dose on Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Idiopathic Aplastic Anemia: Data from the Korean Aplastic Anemia Trials

“…Our prior study suggested the noninferiority of MUDs to MSDs when the pretransplant conditions were well matched in this high-resolution human leukocyte antigen (HLA) typing era, and several studies have supported our analysis [1, 4, 5]. Therefore, alloHSCT from MUDs and haplo-identical family donors is a plausible strategy for severe AA patients without an MSD [6, 7]. AlloHSCT cases using PBSCs surpass those treated using BM.…”

Allo-HSCT compared with immunosuppressive therapy for acquired aplastic anemia: Is superiority a one-sided understanding?

“…This is consistent with the present controversial treatment situation. In China, HID-HSCT has been advocated as a rst-line treatment for children with AA [19], but in the United States and Europe, HID-HSCT is regarded as an experimental treatment for the relatively limited number of cases reported with unknown long-term effects of complicated regimens and a mismatched immune system. In addition, we observed a great disparity in FFS for patients treated with allo-HSCT versus IST.…”

“…All studies reported the outcomes of AA patients treated with rst-line allo-HSCT or IST [11][12][13][14][15][16][17][18][19][20][21][22][23][24][25]. The allo-HSCT group was divided into two subgroups: BMT-MRD and BMT-MUD.…”

Allo-HSCT compared with immunosuppressive therapy for acquired aplastic anemia: a system review and meta-analysis

“…All studies reported the outcomes of AA patients treated with first-line allo-HSCT or IST [11][12][13][14][15][16][17][18][19][20][21][22][23][24][25]. The allo-HSCT group was divided into two subgroups: BMT-MRD and BMT-MUD.…”

“…This is consistent with the present controversial treatment situation. In China, HID-HSCT has been advocated as a first-line treatment for children with AA [24], but in the United States and Europe, HID-HSCT is regarded as an experimental treatment for the relatively limited number of cases reported with unknown long-term effects of complicated regimens and a mismatched immune system. In addition, we observed a great disparity in FFS for patients treated with allo-HSCT versus IST.…”