ArticleBlood advances2026
Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years.
Article in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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Who cites it
1 citing paper in PubMed.
- Haploidentical HSCT vs immunosuppressive therapy for severe aplastic anemia in patients aged 40-65 years: a retrospective comparative study.Stem cells translational medicine · 2026Article
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25 authors.
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Abstract
abstractWe investigated the outcomes of 179 pediatric patients with severe aplastic anemia (SAA) who received up-front hematopoietic stem cell transplantation (HSCT; n = 87) or received HSCT after failure of immunosuppressive therapy (IST; n = 92). With a median follow-up of 4.0 years (range, 0.2-10.7) after HSCT, 161 patients were alive at last follow-up. The cumulative incidence of graft failure, grade 2 to 4 acute and chronic graft-versus-host disease (GVHD) was 10%, 18%, and 8%, respectively. Five-year overall survival (OS) and GVHD-free, relapse/rejection-free survival (GRFS) were 89% and 79%, respectively. Patients who received up-front HSCT from a matched sibling donor (MSD) or ≥9/10 HLA-matched unrelated donor (MUD) both achieved an OS of 95%. OS was superior in patients who received up-front HSCT than those who received HSCT after failure of IST (up-front, 95% [95% CI, 89-100] vs after IST failure, 83% [95% CI,75-91]; P < .01). However, OS in the HSCT group after IST failure improved over time, resulting in comparable OS between up-front HSCT and HSCT after IST failure from 2015 (97% vs 89%; P = .09). Moreover, there was no difference in GRFS of MUD-HSCT comparing patients who received a transplant up front or after IST failure (up-front, 77% vs after IST failure, 76%; P value not significant). Our data highlight the good transplant outcomes of a consecutive pediatric SAA cohort. Although up-front HSCT from a readily available MUD is a viable option if an MSD is not available, HSCT after IST failure from a well-matched unrelated donor also yields favorable outcomes.
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