Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Author:

Aldenhoven Mieke1,Wynn Robert F.2,Orchard Paul J.3,O’Meara Anne4,Veys Paul5,Fischer Alain6,Valayannopoulos Vassili7,Neven Benedicte6,Rovelli Attilio8,Prasad Vinod K.9,Tolar Jakub3,Allewelt Heather9,Jones Simon A.10,Parini Rossella11,Renard Marleen12,Bordon Victoria13,Wulffraat Nico M.14,de Koning Tom J.15,Shapiro Elsa G.16,Kurtzberg Joanne9,Boelens Jaap Jan1

Affiliation:

1. Blood and Marrow Transplantation Program, Department of Pediatrics, University Medical Center Utrecht, Utrecht, The Netherlands;

2. Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, United Kingdom;

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

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

5. Blood and Marrow Transplantation Program, Great Ormond Street Hospital for Children National Health Service Trust, London, United Kingdom;

6. Département de Biothérapie and

7. Reference Center for Inherited Metabolic Diseases and Imagine Institute, Assistance Publique-Hopiteaux de Paris, Hôpital Universitaire Necker-Enfants Malades, Paris, France;

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

9. The Pediatric Blood and Marrow Transplant Program, Duke University, Durham, NC;

10. Willink Unit, Manchester Centre for Genomic Medicine, Central Manchester University Hospitals, University of Manchester, Manchester, United Kingdom;

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

12. Pediatric Hematology and Oncology, University Hospital Leuven, Leuven, Belgium;

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

14. Department of Pediatric Immunology and Rheumatology, University Medical Center Utrecht, Utrecht, The Netherlands;

15. University Groningen, University Medical Center Groningen, Department of Genetics, Groningen, The Netherlands; and

16. Department of Pediatrics and Neurology, Division of Clinical Behavioral Neuroscience, University of Minnesota, Minneapolis, MN

Abstract

Key Points Patients with Hurler syndrome show significant residual disease burden despite HCT. Early referral for HCT, using noncarrier donors and regimens designed to achieve full-donor chimerism, offers the best long-term prognosis.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Reference27 articles.

1. The Mucopolysaccharidoses.;Neufeld,2001

2. Center for International Blood and Marrow Transplant Research CIBMTR Working Committee Meetings. Primary Immune Deficiencies, Inborn Errors of Metabolism and Other Non-Malignant Marrow Disorders Working Committee Meeting Materials. http://www.cibmtr.org/Meetings/Materials/WorkingCommittees/pages/WorkingCommitteeMaterialsDetail.aspx?CID=a0IE000000DAt93MAD. Accessed April 10, 2014

3. Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler’s syndrome after pharmacological enzyme replacement therapy.;Saif;Haematologica,2012

4. Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy.;Wynn;J Pediatr,2009

5. Reversal of clinical features of Hurler’s disease and biochemical improvement after treatment by bone-marrow transplantation.;Hobbs;Lancet,1981

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