Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation

Author:

Aldenhoven Mieke1,van den Broek Brigitte T. A.123,Wynn Robert F.4,O’Meara Anne5,Veys Paul6,Rovelli Attilio7,Jones Simon A.8,Parini Rossella9,van Hasselt Peter M.123,Renard Marleen10,Bordon Victoria11,de Koning Tom J.12,Boelens Jaap Jan123

Affiliation:

1. Blood and Marrow Transplantation Program, Department of Pediatrics, and

2. Laboratory for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands;

3. Sylvia Toth Center for Multidisciplinary Follow up after Hematopoietic Stem Cell Transplantation, Utrecht, The Netherlands;

4. Blood and Marrow Transplant Unit, Royal Manchester Children’s Hospital, Manchester, United Kingdom;

5. Department of Hematology and Bone Marrow Transplantation, Our Lady’s Children’s Hospital, Dublin, Ireland;

6. Blood and Marrow Transplantation Program, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom;

7. Pediatric Bone Marrow Transplant Unit, Department of Pediatrics, San Gerardo University Hospital, Monza, Italy;

8. Willink Unit, Manchester Centre for Genomic Medicine, Central Manchester NHS Foundation Trust, University of Manchester, Manchester, United Kingdom;

9. Rare Metabolic Disorders Unit, Department of Pediatrics, San Gerardo University Hospital, Monza, Italy;

10. Pediatric Hematology and Oncology, University Hospitals Leuven, Leuven, Belgium;

11. Pediatric Hematology and Oncology, Ghent University Hospital, Ghent, Belgium; and

12. Department of Genetics, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands

Abstract

Key Points Functional health of transplanted HS patients was significantly diminished. Patients with higher enzyme levels showed favorable results. Psychosocial health appeared unaffected compared with healthy peers.

Publisher

American Society of Hematology

Subject

Hematology

Reference15 articles.

1. The mucopolysaccharidosis;Neufeld,2001

2. The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK;Moore;Orphanet J Rare Dis,2008

3. The clinical outcome of Hurler syndrome after stem cell transplantation;Aldenhoven;Biol Blood Marrow Transplant,2008

4. Predictors of long-term clinical outcome in Hurler syndrome patients after successful hematopoietic stem cell transplantation: an international, multi-center study;Aldenhoven;Blood,2015

5. World Health Organization. Child Growth Standards2006. http://www.who.int/childgrowth. Accessed September 2014.

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