Long-term adaptive functioning outcomes of children with inherited metabolic and genetic diseases treated with hematopoietic stem cell transplantation in a single large pediatric center: Parents' perspective

Journal of Pediatric Hematology/Oncology

Volumn 33, Issue 3, 2011, Pages 216-220

  Gassas, Adam ^a   Raiman, Julian ^a   White, Laura ^a   Schechter, Tal ^a   Clarke, Joe ^a   Doyle, John ^a  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

genetic metabolic disease; hematopoietic stem cell transplantation; long term outcome

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN; ETOPOSIDE; FLUCONAZOLE; GANCICLOVIR; IMMUNOGLOBULIN; METHOTREXATE; METHYLPREDNISOLONE; PENICILLIN G; PNEUMOCOCCUS VACCINE; THYMOCYTE ANTIBODY;

ADRENOLEUKODYSTROPHY; ALBERS SCHOENBERG DISEASE; ARTICLE; CLINICAL ARTICLE; CYTOMEGALOVIRUS INFECTION; DONOR; GANGLIOSIDOSIS; GAUCHER DISEASE; GENETIC DISORDER; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HYPERSALIVATION; METABOLIC DISORDER; METACHROMATIC LEUKODYSTROPHY; MYCOSIS; NEUROPSYCHOLOGICAL TEST; NIEMANN PICK DISEASE; OUTCOME ASSESSMENT; PNEUMOCYSTIS PNEUMONIA; PRIORITY JOURNAL; QUALITY OF LIFE; SURVIVAL; WHOLE BODY RADIATION; X CHROMOSOME LINKED DISORDER;

ADRENOLEUKODYSTROPHY; CHILD, PRESCHOOL; FEMALE; GENETIC DISEASES, INBORN; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; MALE; METABOLISM, INBORN ERRORS; MUCOPOLYSACCHARIDOSIS I; OSTEOPETROSIS; TREATMENT OUTCOME;

EID: 79954415147     PISSN: 10774114     EISSN: 15363678     Source Type: Journal    
DOI: 10.1097/MPH.0b013e3182050945     Document Type: Article

References (16)

1. Gerritsen EJ, Vossen JM, Fasth A, et al. A report from the Working Party on Inborn Errors of the European Bone Marrow Transplantation Group. Bone marrow transplantation for autosomal recessive osteopetrosis. J Pediatr. 1994;125:896-902.
2. Peters C, Shapiro EG, Anderson J, et al. The Storage Disease Collaborative Study Group. Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. Blood. 1998;91:2601-2608.
3. Krivit W. Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases. Springer Semin Immunopathol. 2004;26:119-132. (Pubitemid 40021961)
4. Staba SL, Escolar ML, Poe M, et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004;350:1960-1969. (Pubitemid 38580199)
5. Peters C, Charnas LR, Tan Y, et al. Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood. 2004;104:881-888.
6. Bjoraker KJ, Delaney K, Peters C, et al. Long-term outcomes of adaptive functions for children with mucopolysaccharidosis I. J Dev Behav Pediatr. 2006;27:290-296.
7. Sparrow SS, Balla DA, Cicchetti DV. Vineland Adaptive Behavior Scales. Circle Pines, MN: American Corporation; 1991;6:52-56.
8. Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HLA-matched sibling donors. Transplantation. 1974;18:295-304.
9. Atkinson K, Horowitz MM, Gale RP, et al. Consensus among bone marrow transplanters for diagnosis, grading and treatment of chronic graft-versus-host disease. Bone Marrow Transplant. 1989;4:247-254. (Pubitemid 19152518)
10. Morel CF, Gassas A, Doyle J, et al. Unsuccessful treatment attempt: cord blood stem cell transplantation in a patient with Niemann-Pick disease type A. J Inherit Metab Dis. 2007;30:987.
11. Kasow KA, Bonfim C, Asch J, et al. Malignant infantile osteopetrosis and primary pulmonary hypertension: a new combination? Pediatr Blood Cancer. 2004;42:190-194.
12. Steward CG, Pellier I, Mahajan A, et al. The Working Party on Inborn Errors of the European Blood and Marrow Transplantation Group. Severe pulmonary hypertension: a frequent complication of stem cell transplantation for malignant infantile osteopetrosis. Br J Haematol. 2004;124:63-71. (Pubitemid 38021624)
13. Corbacioglu S, Hönig M, Lahr G, et al. Stem cell transplantation in children with infantile osteopetrosis is associated with a high incidence of VOD, which could be prevented with defibrotide. Bone Marrow Transplant. 2006;38:547-553. (Pubitemid 44509547)
14. Tolar J, Bonfim C, Grewal S, et al. Engraftment and survival following hematopoietic stem cell transplantation for osteopetrosis using a reduced intensity conditioning regimen. Bone Marrow Transplant. 2006;38:783-787. (Pubitemid 44830837)
15. Driessen GJ, Gerritsen EJ, Fischer A, et al. Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report. Bone Marrow Transplant. 2003;32:657-663.
16. Loes DJ, Peters C, Krivit W. Globoid cell leukodystrophy: distinguishing early-onset from late-onset disease using a brain MR imaging scoring method. AJNR Am J Neuroradiol. 1999;20:316-323. (Pubitemid 30418723)