Hematopoietic Cell Transplantation in Children with Sickle Cell Disease: A Single-Center Experience and Exploration of an Age Threshold for Post-Transplant Complications
6 affiliations
- 1Department of Pediatric Onco-Hematology, Hôpitaux Universitaires de Strasbourg, 67200 Strasbourg, France
- 2Referral Center for SCD, Pediatrics Department, Intercommunal Hospital Center, 94000 Créteil, France
- 3Department of Pediatric Onco-Hematology, CHU Reims, 51092 Reims, France
- 4Department of Pediatric Hematology and Oncology, Rouen University Hospital, 76000 Rouen, France
- 5Hematology Clinical Laboratory, Hôpitaux Universitaires de Strasbourg, 67200 Strasbourg, France
- 6Laboratoire d’ImmunoRhumatologie Moléculaire, INSERM UMR_S 1109, LabEx Transplantex, Fédération de Médecine Translationnelle de Strasbourg, 67085 Strasbourg Cedex, France
Abstract
Background/Objectives: Hematopoietic cell transplantation (HCT) is the only established curative option for sickle cell disease (SCD), yet the optimal timing in pediatric cohorts remains incompletely defined. Methods: We retrospectively analyzed all patients under 20 years with SCD who underwent HCT at the University Hospital of Strasbourg between 2006 and 2024 and explored whether an age threshold exists for post-HCT complications. Results: Regarding the MSD-HCT (n = 30), median age at HCT was 8.4 years (3.7–16.7). All received myeloablative conditioning with thymoglobulin. Median follow-up was 4.1 years. Five-year overall and event-free survival were 93%. The single death was accidental; therefore, there was no transplant-related mortality. Grade II-IV acute GVHD occurred in two patients (7%) and chronic GVHD in one (3%). GVHD-free rejection-free survival was 87%. No graft failure occurred, and 70% achieved complete donor chimerism. No statistically significant age threshold for post-transplant complications was identified, although PRES tended to be more frequent from 8 years. Cerebral vasculopathy was stable or improved in all evaluated patients. In the haploidentical group, one primary graft failure occurred despite thiotepa and post-transplant cyclophosphamide. Conclusions: This cohort demonstrates excellent survival and low morbidity following MSD-HCT in children with SCD and justifies a larger registry-based analysis of age and complications. The growing role of haploidentical HCT warrants evaluation in larger prospective cohorts, with particular attention to conditioning strategies.
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BibTeX
@article{nazon2026hematopoietic,
title = {{Hematopoietic Cell Transplantation in Children with Sickle Cell Disease: A Single-Center Experience and Exploration of an Age Threshold for Post-Transplant Complications}},
author = {Charlotte Nazon and Chloé Gerber and Alexandra Salmon and Annie Kamdem and Claire Pluchart and Bruno Filhon and Anne-Cécile Galoisy and Françoise Bernaudin and Catherine Paillard},
journal = {Journal of Clinical Medicine},
year = {2026},
month = oct,
doi = {10.3390/jcm15208155},
url = {https://www.mdpi.com/2077-0383/15/20/8155},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Hematopoietic Cell Transplantation in Children with Sickle Cell Disease: A Single-Center Experience and Exploration of an Age Threshold for Post-Transplant Complications
AU - Charlotte Nazon
AU - Chloé Gerber
AU - Alexandra Salmon
AU - Annie Kamdem
AU - Claire Pluchart
AU - Bruno Filhon
AU - Anne-Cécile Galoisy
AU - Françoise Bernaudin
AU - Catherine Paillard
PY - 2026
DA - 2026/10/01
JO - Journal of Clinical Medicine
DO - 10.3390/jcm15208155
UR - https://www.mdpi.com/2077-0383/15/20/8155
AB - Background/Objectives: Hematopoietic cell transplantation (HCT) is the only established curative option for sickle cell disease (SCD), yet the optimal timing in pediatric cohorts remains incompletely defined. Methods: We retrospectively analyzed all patients under 20 years with SCD who underwent HCT at the University Hospital of Strasbourg between 2006 and 2024 and explored whether an age threshold exists for post-HCT complications. Results: Regarding the MSD-HCT (n = 30), median age at HCT was 8.4 years (3.7–16.7). All received myeloablative conditioning with thymoglobulin. Median follow-up was 4.1 years. Five-year overall and event-free survival were 93%. The single death was accidental; therefore, there was no transplant-related mortality. Grade II-IV acute GVHD occurred in two patients (7%) and chronic GVHD in one (3%). GVHD-free rejection-free survival was 87%. No graft failure occurred, and 70% achieved complete donor chimerism. No statistically significant age threshold for post-transplant complications was identified, although PRES tended to be more frequent from 8 years. Cerebral vasculopathy was stable or improved in all evaluated patients. In the haploidentical group, one primary graft failure occurred despite thiotepa and post-transplant cyclophosphamide. Conclusions: This cohort demonstrates excellent survival and low morbidity following MSD-HCT in children with SCD and justifies a larger registry-based analysis of age and complications. The growing role of haploidentical HCT warrants evaluation in larger prospective cohorts, with particular attention to conditioning strategies.
SN - 2077-0383
ER -