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

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Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation : An international multicenter study. / Aldenhoven, Mieke; Wynn, Robert F.; Orchard, Paul J.; O'Meara, Anne; Veys, Paul; Fischer, Alain; Valayannopoulos, Vassili; Neven, Benedicte; Rovelli, Attilio; Prasad, Vinod K.; Tolar, Jakub; Allewelt, Heather; Jones, Simon A.; Parini, Rossella; Renard, Marleen; Bordon, Victoria; Wulffraat, Nico M.; De Koning, Tom J.; Shapiro, Elsa G.; Kurtzberg, Joanne; Boelens, Jaap Jan.

In: Blood, Vol. 125, No. 13, 26.03.2015, p. 2164-2172.

Research output: Contribution to journalArticle

Harvard

Aldenhoven, M, Wynn, RF, Orchard, PJ, O'Meara, A, Veys, P, Fischer, A, Valayannopoulos, V, Neven, B, Rovelli, A, Prasad, VK, Tolar, J, Allewelt, H, Jones, SA, Parini, R, Renard, M, Bordon, V, Wulffraat, NM, De Koning, TJ, Shapiro, EG, Kurtzberg, J & Boelens, JJ 2015, 'Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: An international multicenter study', Blood, vol. 125, no. 13, pp. 2164-2172. https://doi.org/10.1182/blood-2014-11-608075

APA

Aldenhoven, M., Wynn, R. F., Orchard, P. J., O'Meara, A., Veys, P., Fischer, A., ... Boelens, J. J. (2015). Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: An international multicenter study. Blood, 125(13), 2164-2172. https://doi.org/10.1182/blood-2014-11-608075

CBE

Aldenhoven M, Wynn RF, Orchard PJ, O'Meara A, Veys P, Fischer A, Valayannopoulos V, Neven B, Rovelli A, Prasad VK, Tolar J, Allewelt H, Jones SA, Parini R, Renard M, Bordon V, Wulffraat NM, De Koning TJ, Shapiro EG, Kurtzberg J, Boelens JJ. 2015. Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: An international multicenter study. Blood. 125(13):2164-2172. https://doi.org/10.1182/blood-2014-11-608075

MLA

Vancouver

Author

Aldenhoven, Mieke ; Wynn, Robert F. ; Orchard, Paul J. ; O'Meara, Anne ; Veys, Paul ; Fischer, Alain ; Valayannopoulos, Vassili ; Neven, Benedicte ; Rovelli, Attilio ; Prasad, Vinod K. ; Tolar, Jakub ; Allewelt, Heather ; Jones, Simon A. ; Parini, Rossella ; Renard, Marleen ; Bordon, Victoria ; Wulffraat, Nico M. ; De Koning, Tom J. ; Shapiro, Elsa G. ; Kurtzberg, Joanne ; Boelens, Jaap Jan. / Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation : An international multicenter study. In: Blood. 2015 ; Vol. 125, No. 13. pp. 2164-2172.

RIS

TY - JOUR

T1 - Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation

T2 - An international multicenter study

AU - Aldenhoven, Mieke

AU - Wynn, Robert F.

AU - Orchard, Paul J.

AU - O'Meara, Anne

AU - Veys, Paul

AU - Fischer, Alain

AU - Valayannopoulos, Vassili

AU - Neven, Benedicte

AU - Rovelli, Attilio

AU - Prasad, Vinod K.

AU - Tolar, Jakub

AU - Allewelt, Heather

AU - Jones, Simon A.

AU - Parini, Rossella

AU - Renard, Marleen

AU - Bordon, Victoria

AU - Wulffraat, Nico M.

AU - De Koning, Tom J.

AU - Shapiro, Elsa G.

AU - Kurtzberg, Joanne

AU - Boelens, Jaap Jan

PY - 2015/3/26

Y1 - 2015/3/26

N2 - Mucopolysaccharidosis type I-Hurler syndrome (MPS-IH) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Although hematopoietic cell transplantation (HCT) has been performed in these patients for more than 30 years, large studies on the long-term outcome of patients with MPS-IH after HCT are lacking. The goal of this international study was to identify predictors of the long-term outcome of patients with MPS-IH after successful HCT. Two hundred seventeen patients with MPS-IH successfully engrafted with a median follow-up age of 9.2 years were included in this retrospective analysis. Primary endpoints were neurodevelopmental outcomes and growth. Secondary endpoints included neurologic, orthopedic, cardiac, respiratory, ophthalmologic, audiologic, and endocrinologic outcomes. Considerable residual disease burden was observed in the majority of the transplanted patients with MPS-IH, with high variability between patients. Preservation of cognitive function at HCT andayoungerageat transplantationweremajorpredictors for superior cognitivedevelopmentposttransplant. Anormal a-L-iduronidase enzyme level obtained post-HCT was another highly significant predictor for superior long-term outcome in most organ systems. The long-termprognosisofpatientswithMPS-IH receivingHCTcanbe improvedbyreducingtheage atHCTthroughearlierdiagnosis, aswell as using exclusively noncarrier donors and achieving complete donor chimerism. (Blood. 2015;125(13):2164-2172)

AB - Mucopolysaccharidosis type I-Hurler syndrome (MPS-IH) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Although hematopoietic cell transplantation (HCT) has been performed in these patients for more than 30 years, large studies on the long-term outcome of patients with MPS-IH after HCT are lacking. The goal of this international study was to identify predictors of the long-term outcome of patients with MPS-IH after successful HCT. Two hundred seventeen patients with MPS-IH successfully engrafted with a median follow-up age of 9.2 years were included in this retrospective analysis. Primary endpoints were neurodevelopmental outcomes and growth. Secondary endpoints included neurologic, orthopedic, cardiac, respiratory, ophthalmologic, audiologic, and endocrinologic outcomes. Considerable residual disease burden was observed in the majority of the transplanted patients with MPS-IH, with high variability between patients. Preservation of cognitive function at HCT andayoungerageat transplantationweremajorpredictors for superior cognitivedevelopmentposttransplant. Anormal a-L-iduronidase enzyme level obtained post-HCT was another highly significant predictor for superior long-term outcome in most organ systems. The long-termprognosisofpatientswithMPS-IH receivingHCTcanbe improvedbyreducingtheage atHCTthroughearlierdiagnosis, aswell as using exclusively noncarrier donors and achieving complete donor chimerism. (Blood. 2015;125(13):2164-2172)

UR - http://www.scopus.com/inward/record.url?scp=84926200256&partnerID=8YFLogxK

U2 - 10.1182/blood-2014-11-608075

DO - 10.1182/blood-2014-11-608075

M3 - Article

VL - 125

SP - 2164

EP - 2172

JO - Blood

JF - Blood

SN - 1528-0020

IS - 13

ER -