Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy
Abstract Context Mucopolysaccharidosis (MPS) requires urgent treatment to prevent neurological damage. While gene therapy holds promise for effectively treating these diseases with minimal toxicity, access remains limited for most patients. Consequently, advancing allogeneic hematopoietic stem cell...
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2024-12-01
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| Online Access: | https://doi.org/10.1002/jha2.1056 |
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| author | Laura Danhardt Arnaud Wiedemann Gerard Michel Jean‐Hugues Dalle Fanny Rialland Cécile Renard Charlotte Jubert Johan Maertens Anne Sirvent Nimrod Buchbinder Christine Devalck Bénédicte Brichard Catherine Paillard Stephanie Nguyen Angelo Paci David Combarel Martin Castelle Simona Pagliuca Cecile Pochon |
| author_facet | Laura Danhardt Arnaud Wiedemann Gerard Michel Jean‐Hugues Dalle Fanny Rialland Cécile Renard Charlotte Jubert Johan Maertens Anne Sirvent Nimrod Buchbinder Christine Devalck Bénédicte Brichard Catherine Paillard Stephanie Nguyen Angelo Paci David Combarel Martin Castelle Simona Pagliuca Cecile Pochon |
| author_sort | Laura Danhardt |
| collection | DOAJ |
| description | Abstract Context Mucopolysaccharidosis (MPS) requires urgent treatment to prevent neurological damage. While gene therapy holds promise for effectively treating these diseases with minimal toxicity, access remains limited for most patients. Consequently, advancing allogeneic hematopoietic stem cell transplantation (HSCT) for young children is crucial. Since the 2010s, cord blood (CB) transplants with reduced‐toxicity conditioning (RTC) have become the standard of care. Patients and methods Recent reports in France indicate a significant incidence of graft failures (GF), prompting a large‐scale retrospective study from the French‐speaking bone marrow transplantation society's registry, to understand GF risks, guide clinicians in selecting transplant platforms, and describe outcomes of second HSCT in young patients. Results This report analyses 93 children who underwent HSCT for MPS between 2000 and 2020. The GF rate was notably high (22.6% at day 100), primarily associated with the donor's HLA compatibility and the recipient's age. Well‐matched CB and RTC were not found to be risk factors for GF. This study also details the procedures for second and third transplants in patients who rejected their first HSCT. Conclusion In the era of RTC, CB remains a viable and expedient option for MPS transplantation. |
| format | Article |
| id | doaj-art-eb2c802a3f8a43d8b58b177679a882ab |
| institution | OA Journals |
| issn | 2688-6146 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | Wiley |
| record_format | Article |
| series | eJHaem |
| spelling | doaj-art-eb2c802a3f8a43d8b58b177679a882ab2025-08-20T02:36:31ZengWileyeJHaem2688-61462024-12-01561295130010.1002/jha2.1056Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular TherapyLaura Danhardt0Arnaud Wiedemann1Gerard Michel2Jean‐Hugues Dalle3Fanny Rialland4Cécile Renard5Charlotte Jubert6Johan Maertens7Anne Sirvent8Nimrod Buchbinder9Christine Devalck10Bénédicte Brichard11Catherine Paillard12Stephanie Nguyen13Angelo Paci14David Combarel15Martin Castelle16Simona Pagliuca17Cecile Pochon18Pediatric Onco‐Hematology DepartmentNancy University HospitalNancyFrancePediatric Intensive Care DepartmentNancy University Hospital, Unité INSERM u1256 N‐GERENancy FranceDepartment of Pediatric Hematology and OncologyLa Timone HospitalMarseilleFranceImmuno‐Hematology DepartmentRobert Debré Hospital, APHPParisFrancePediatric Onco‐Hematology DepartmentChildren HospitalNantesFranceInstitute of Hematology and Pediatric OncologyUniversité Lyon 1, Hospices Civils de LyonLyonFrancePediatric Onco‐Hematology DepartmentChildren Hospital, Pellegrin GroupBordeauxFranceDepartment of HematologyUniversity Hospital LeuvenLeuvenBelgiumPediatric Onco‐Hematology DepartmentMontpellier HospitalMontpellierFrancePediatric Immunology and Onco‐HematologyCharles‐Nicolle HospitalRouenFranceDepartment of Pediatric OncologyChildren Hospital Reine FabiolaBrusselsBelgiumDepartment of Pediatric Hematology and OncologyCliniques Universitaires Saint‐LucBrusselsBelgiumPediatric Onco‐Hematology DepartmentHautepierre HospitalStrasbourgFranceDepartment of HematologyCentre Hospitalier Universitaire Pitié‐Salpêtrière, Assistance Publique‐Hôpitaux de ParisParisFrancePharmacology departmentGustave Roussy InstituteVillejuif FrancePharmacology departmentGustave Roussy InstituteVillejuif FrancePediatric Hematology‐Immunology Department Necker Enfants Malades Hospital, APHP Paris FranceDepartment of Hematology Nancy University Hospital Nancy FrancePediatric Onco‐Hematology DepartmentNancy University HospitalNancyFranceAbstract Context Mucopolysaccharidosis (MPS) requires urgent treatment to prevent neurological damage. While gene therapy holds promise for effectively treating these diseases with minimal toxicity, access remains limited for most patients. Consequently, advancing allogeneic hematopoietic stem cell transplantation (HSCT) for young children is crucial. Since the 2010s, cord blood (CB) transplants with reduced‐toxicity conditioning (RTC) have become the standard of care. Patients and methods Recent reports in France indicate a significant incidence of graft failures (GF), prompting a large‐scale retrospective study from the French‐speaking bone marrow transplantation society's registry, to understand GF risks, guide clinicians in selecting transplant platforms, and describe outcomes of second HSCT in young patients. Results This report analyses 93 children who underwent HSCT for MPS between 2000 and 2020. The GF rate was notably high (22.6% at day 100), primarily associated with the donor's HLA compatibility and the recipient's age. Well‐matched CB and RTC were not found to be risk factors for GF. This study also details the procedures for second and third transplants in patients who rejected their first HSCT. Conclusion In the era of RTC, CB remains a viable and expedient option for MPS transplantation.https://doi.org/10.1002/jha2.1056cord bloodgraft failureGVHDHSCTmucopolysaccharidosisoutcomes |
| spellingShingle | Laura Danhardt Arnaud Wiedemann Gerard Michel Jean‐Hugues Dalle Fanny Rialland Cécile Renard Charlotte Jubert Johan Maertens Anne Sirvent Nimrod Buchbinder Christine Devalck Bénédicte Brichard Catherine Paillard Stephanie Nguyen Angelo Paci David Combarel Martin Castelle Simona Pagliuca Cecile Pochon Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy eJHaem cord blood graft failure GVHD HSCT mucopolysaccharidosis outcomes |
| title | Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy |
| title_full | Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy |
| title_fullStr | Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy |
| title_full_unstemmed | Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy |
| title_short | Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy |
| title_sort | incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort a study on behalf of the francophone society of bone marrow transplantation and cellular therapy |
| topic | cord blood graft failure GVHD HSCT mucopolysaccharidosis outcomes |
| url | https://doi.org/10.1002/jha2.1056 |
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