Hematopoietic stem cell transplantation for infantile osteopetrosis

Author:

Orchard Paul J.1,Fasth Anders L.2,Le Rademacher Jennifer34,He Wensheng3,Boelens Jaap Jan5,Horwitz Edwin M.6,Al-Seraihy Amal7,Ayas Mouhab7,Bonfim Carmem M.8,Boulad Farid9,Lund Troy1,Buchbinder David K.10,Kapoor Neena11,O’Brien Tracey A.12,Perez Miguel A. Diaz13,Veys Paul A.14,Eapen Mary3

Affiliation:

1. Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, MN;

2. Department of Pediatrics, Sahlgrenska University Hospital, Gothenburg, Sweden;

3. Center for International Blood and Marrow Transplant Research, Department of Medicine, and

4. Division of Biostatistics, Institute for Health and Society, Medical College of Wisconsin, Milwaukee, WI;

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

6. Blood and Marrow Transplant Program, Nationwide Children’s Hospital and The Ohio State University College of Medicine, Columbus, OH;

7. Department of Pediatric Hematology Oncology, King Fasial Specialist Hospital & Research Center, Riyadh, Saudi Arabia;

8. Bone Marrow Transplant Program, Hospital de Clinicas–Universidade Federal do Paraná, Curitiba, Brazil;

9. Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY;

10. Department of Pediatrics, Children’s Hospital of Orange County, Orange, CA;

11. Department of Research Immunology/Bone Marrow Transplant, Children’s Hospital of Los Angeles, Los Angeles, CA;

12. Cord & Marrow Transplant Program, Sydney Children’s Hospital, Sydney, Australia;

13. Servicio de Oncohematologia, Hospital Infantil Universitario Nino Jesus, Madrid, Spain; and

14. Blood and Marrow Transplant Program, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom

Abstract

Key Points Hematopoietic cell transplantation results in long-term survival. Primary graft failure is very high and the predominant cause of death.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Reference35 articles.

1. Osteopetrosis.;Tolar;N Engl J Med,2004

2. Autosomal recessive osteopetrosis: variability of findings at diagnosis and during the natural course.;Gerritsen;Pediatrics,1994

3. Successful bone-marrow transplantation for infantile malignant osteopetrosis.;Coccia;N Engl J Med,1980

4. Allogeneic bone-marrow transplantation in infantile malignant osteopetrosis.;Sieff;Lancet,1983

5. Bone marrow transplantation for autosomal recessive osteopetrosis. A report from the Working Party on Inborn Errors of the European Bone Marrow Transplantation Group.;Gerritsen;J Pediatr,1994

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