Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience

Author:

Morillo-Gutierrez Beatriz1,Beier Rita2,Rao Kanchan3,Burroughs Lauri45,Schulz Ansgar6,Ewins Anna-Maria7,Gibson Brenda7,Sedlacek Petr8,Krol Ladislav8,Strahm Brigitte9,Zaidman Irina10,Kalwak Krzysztof11,Talano Julie-An12,Woolfrey Ann45,Fraser Chris13,Meyts Isabelle14,Müller Ingo15,Wachowiak Jacek16,Bernardo Maria Ester17,Veys Paul3,Sykora Karl-Walter2,Gennery Andrew R.118,Slatter Mary118

Affiliation:

1. Department of Paediatric Immunology, Great North Children´s Hospital, Newcastle Upon Tyne, United Kingdom;

2. Hannover Medical School, Hannover, Germany;

3. Paediatric Bone Marrow Transplant, Great Ormond Street Hospital, London, United Kingdom;

4. Fred Hutchinson Cancer Research Center, Seattle, WA;

5. The University of Washington School of Medicine, Seattle, WA;

6. Department of Pediatrics, University Medical Center Ulm, Ulm, Germany;

7. Paediatric Bone Marrow Transplantation Schiehallion Unit, Royal Hospital for Sick Children, Glasgow, United Kingdom;

8. Department of Pediatric Hematology and Oncology, Teaching Hospital Motol, Prague, Czech Republic;

9. Pediatric Haematology/Oncology, University Medical Centre, Freiburg, Germany;

10. Rambam Hospital, Haifa, Israel;

11. Wroclaw Medical University, Wroclaw, Poland;

12. Medical College of Wisconsin, Milwaukee, WI;

13. Lady Cilento Children’s Hospital, Brisbane, Australia;

14. Department of Microbiology and Immunology, Department of Pediatrics, University Hospitals Leuven, KU Leuven, Leuven, Belgium;

15. University Medical Center Hamburg-Eppendorf, Hamburg, Germany;

16. University of Medical Sciences, Poznan, Poland;

17. San Raffaele Telethon Institute for Gene Therapy, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy; and

18. Institute of Cellular Medicine, Newcastle University, Newcastle Upon Tyne, United Kingdom

Abstract

Key Points Treosulfan, a low-toxicity alkylating agent, can be used effectively as part of conditioning for HSCT in children with CGD. Long-term follow-up is required to ascertain effects, particularly on gonadal function and compare with other regimens.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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