The impact of Treosulfan‐based conditioning for inborn errors of immunity: Is dose monitoring crucial?

Author:

Ersoy Gizem Zengin1ORCID,Çipe Funda2,Fışgın Tunç1,Aksoy Basak Adakli1,Öner Özlem Başoğlu1,Hashemi Nazlı1,Aydoğdu Selime3,Erdem Melek4,Dikme Gürcan5,Murat Koza6,Bozkurt Ceyhun7

Affiliation:

1. Altınbaş University Medical Park Bahçelievler Hospital Pediatric Hematology Oncology & Pediatric Bone Marrow Transplantation Unit İstanbul Turkey

2. Altınbaş University Medical Park Bahçelievler Hospital Pediatric Allergy‐Immunology & Pediatric Bone Marrow Transplantation Unit İstanbul Turkey

3. Umraniye Research & Training Hospital Pediatric Hematology & Oncology Department Medical Sciences University İstanbul Turkey

4. İstinye University Pediatric Hematology Oncology Gaziosmanpaşa Medical Park Hospital İstanbul Turkey

5. Aydin University Pediatric Hematology Oncology Florya Medical Park Hospital İstanbul Turkey

6. Duzen Laboratory İstanbul Turkey

7. Medical Park Bahçelievler Hospital Pediatric Hematology Oncology & Pediatric Bone Marrow Transplantation Unit İstinye University Pediatric Hematology Oncology İstanbul Turkey

Abstract

AbstractIntroductionIn children with inborn errors of immunity (IEI) who will receive a hematopoietic stem cell transplant (HSCT) treosulfan‐based conditioning is currently preferred. The aim of this study was to investigate early and late outcomes in pediatric IEI patients receiving pre‐HSCT treosulfan and to examine the effect of treosulfan dose monitoring on outcomes.MethodsSeventy‐three pediatric patients receiving this management between 2015 and 2022 were included.ResultsOverall survival rate was 80%, and event‐free survival was 67.8%. A larger treosulfan dose AUC after first application increased the rate of early toxicity (p = .034) and slowed lymphocyte engraftment (r = .290; p = .030). Underlying disease, treosulfan AUC, donor type, stem cell type, number of immunosuppressive agents, the dose of anti‐thymocyte globulin, and post‐transplantation cyclophosphamide did not to increase risk of acute graft‐versus‐host disease. The risk of mixed chimerism (MC) in patients with autoimmune lymphoproliferative syndrome and leukocyte adhesion deficiency were higher than those with severe combined immunodeficiency (p = .021 and p = .014, respectively). The risk of MC was lower in those receiving peripheral blood stem cells (SC) compared with bone marrow derived SC (OR = .204, p = .022).ConclusionThe AUC of the treosulfan dose was not associated with poorer late outcomes. Treosulfan is an agent that can be used safely in the IEI patient group,  level measurement appears essential to identify early toxicities. Prospective studies with more extended follow‐up periods are needed.

Publisher

Wiley

Subject

Transplantation

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