Long-term outcomes in MPS-IH: Throwing stars

Blood

Volumn 125, Issue 13, 2015, Pages 2016-2017

  Pulsipher, Michael A ^a,b,c  

^a UNIVERSITY OF UTAH   (United States)

^b UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^c Primary Children's Hospital   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

LEVO IDURONIDASE;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ALLOTRANSPLANTATION; CHILD; CHILDHOOD DISEASE; CHIMERA; HUMAN; HURLER SYNDROME; INTELLIGENCE QUOTIENT; LONG TERM CARE; OUTCOME ASSESSMENT; PRIORITY JOURNAL; QUALITY OF LIFE; REVIEW; RISK FACTOR; SIBLING; UNRELATED DONORS; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; MALE; MUCOPOLYSACCHARIDOSIS I;

FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; MUCOPOLYSACCHARIDOSIS I;

EID: 84926222121     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2015-02-626507     Document Type: Review

References (8)

1. Aldenhoven M, Wynn RF, Orchard PJ, et al. Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study. Blood. 2015;125(13):2164-2172.
2. Eiseley L. The Star Thrower. In: The Unexpected Universe. Harcourt, Brace and World, London; 1969.
3. Aldenhoven M, Boelens JJ, de Koning TJ. The clinical outcome of Hurler syndrome after stem cell transplantation. Biol Blood Marrow Transplant. 2008;14(5):485-498.
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(19):1960-1969.
5. Prasad VK, Mendizabal A, Parikh SH, et al. Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood. 2008;112(7):2979-2989.
6. Peters C, Shapiro EG, Anderson J, et al The Storage Disease Collaborative Study Group. Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLAhaploidentical related donor bone marrow transplantation in fifty-four children. Blood. 1998;91(7):2601-2608.
7. Boelens JJ, Aldenhoven M, Purtill D, et al; Eurocord; Inborn Errors Working Party of European Blood and Marrow Transplant group; Duke University Blood and Marrow Transplantation Program; Centre for International Blood and Marrow Research. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. Blood. 2013;121(19):3981-3987.
8. Church H, Tylee K, Cooper A, et al. Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease. Bone Marrow Transplant. 2007;39(4):207-210.