Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years

We 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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Publication Details

Journal
Blood Advances
Published
2026-01-20
DOI
https://doi.org/10.1182/bloodadvances.2025018285
Primary Topic
Hematopoietic Stem Cell Transplantation
Type
article
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article

Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years

Krisztián Kállay, Felicia J. Andresen, Miriam Erlacher, Ingo Müller et al.
Blood Advances
Hematopoietic Stem Cell Transplantation
article

Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years

Krisztián Kállay, Felicia J. Andresen, Miriam Erlacher, Ingo Müller, M. Schmugge, Jochen Buechner, Katarzyna Pawelec, Veerle Labarque, J. Starý, A. Yoshimi, M. Dworzak, B. Strahm, Marc Bierings, Federica Locatelli, Petr Sedláček, OP Smith, Peter Nöllke, Andrea Malone, Peter Bader, Birgit Burkhardt, Victoria Bordon
article en

Abstract

We 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.

Blood AdvancesVol. 10(6)
Università Cattolica del Sacro Cuore (IT), Goethe University Frankfurt (DE), Oslo University Hospital (NO), Universität Hamburg (DE), Universität Ulm (DE), Charles University (CZ), Medical University of Warsaw (PL), Ghent University Hospital (BE), University Medical Center Freiburg (DE), Copenhagen University Hospital (DK), Children's Health Ireland at Crumlin (IE), National Institute of Cardiovascular Diseases (SK), University Hospital in Motol (CZ), University Hospital Münster (DE), University Medical Center Hamburg-Eppendorf (DE), Hospital Infantil Universitario Niño Jesús (ES), National Institute of Oncology (HU), Cancer Research Institute of the Slovak Academy of Sciences (SK), Bambino Gesù Children's Hospital (IT), St Anna Children's Hospital (AT), Princess Máxima Center (NL), University Children's Hospital Zurich (CH), University Hospital Frankfurt (DE), Istituti di Ricovero e Cura a Carattere Scientifico (IT), University Hospital Ulm (DE), KU Leuven (BE)
Bundesministerium für Bildung und Forschung
Good health and well-being
Openalex Percentile: Top 84%
Hematopoietic Stem Cell Transplantation
DOIOpen Access PDFOpenAlex
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