Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

Author:

Boelens Jaap Jan1,Aldenhoven Mieke1,Purtill Duncan2,Ruggeri Annalisa2,DeFor Todd3,Wynn Robert4,Wraith Ed5,Cavazzana-Calvo Marina67,Rovelli Attilio8,Fischer Alain6,Tolar Jakub3,Prasad Vinod K.9,Escolar Maria10,Gluckman Eliane2,O’Meara Anne11,Orchard Paul J.3,Veys Paul12,Eapen Mary13,Kurtzberg Joanne9,Rocha Vanderson214,Driscoll Timothy A.,Kurtzberg Joanne,Landi Daniel B.,Martin Paul L.,Page Kristin M.,Parikh Suhag H,Prasad Vinod K.

Affiliation:

1. University Medical Center Utrecht, Pediatric Blood and Marrow Transplantation Program, Utrecht, The Netherlands;

2. Eurocord/Hôpital Saint Louis, Department of Hematology/BMT, Paris, France;

3. Program in Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN;

4. Royal Manchester Children’s Hospital, Department of Hematology/BMT, Manchester, UK;

5. Manchester Academic Health Sciences Centre (Genetic Medicine), St. Mary’s Hospital, Manchester, UK;

6. Département de Biothérapie, AP-HP, Hôpital Universitaire Necker-Enfants Malades, Paris, France;

7. Université Paris Descartes, Sorbonne Paris Cité, IMAGINE Institute, Paris, France;

8. Centro Trapianto di Midollo Osseo Fondazione MBBM, Clinica Pediatrica dell'Università di Milano-Bicocca, Monza, Italy;

9. Duke University, Division of Pediatric Blood and Marrow Transplantation, Durham, NC;

10. University of Pittsburgh, Program for the Study of Neurodevelopment in Rare Disorders, Pittsburgh, PA;

11. Our Lady’s Children’s Hospital, Dept of Hematology and BMT, Dublin, Ireland;

12. Blood and Marrow Transplantation Program, Great Ormond Street Hospital, London, UK;

13. Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI; and

14. Oxford University Hospitals, Oxford, UK

Abstract

Key Points In HS, patients’ early referral for HCT, with the best available HLA-matched donor offers the best event-free survival. In HS, patients’ HCT with a well-matched unrelated cord blood unit is particularly attractive because the unit is readily available.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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