Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres
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- 作者：Arunabha Ghosha ; c ; arunabha.ghosh@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author ; Weston Millerb ; mill4991@umn.edu" class="auth_mail" title="E-mail the corresponding author ; Paul J. Orchardb ; orcha001@umn.edu" class="auth_mail" title="E-mail the corresponding author ; Simon A. Jonesa ; simon.jones@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author ; Jean Mercera ; jean.mercer@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author ; Heather J. Churcha ; heather.church@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author ; Karen Tyleea ; karen.tylee@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author ; Troy Lundb ; lundx072@umn.edu" class="auth_mail" title="E-mail the corresponding author ; Brian W. Biggerc ; brian.bigger@manchester.ac.uk" class="auth_mail" title="E-mail the corresponding author ; Jakub Tolarb ; tolar003@umn.edu" class="auth_mail" title="E-mail the corresponding author ; Robert F. Wynnd ; robert.wynn@cmft.nhs.uk" class="auth_mail" title="E-mail the corresponding author
- 关键词：EFS ; event-free survival ; ERT ; enzyme replacement therapy ; GAG ; glycosaminoglycan ; GvHD ; graft versus host disease ; HSCT ; haematopoietic stem cell transplantation ; LVEF ; left ventricular ejection fraction ; MPS I ; Mucopolysaccharidosis Type I ; OS ; overall survival ; STR ; short tandem repeats ; VNTR ; variable number tandem repeats
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：March 2016
- 年：2016
- 卷：117
- 期：3
- 页码：373-377
- 全文大小：384 K
文摘
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This is the first large multi-centre report of peri-transplant ERT in Hurler syndrome.
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Overall survival and engrafted survival were favourable compared to other reported cohorts.
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The majority of graft failures were successfully managed with a second HSCT.
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ERT did not have any apparent detrimental effects on transplant outcomes.
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ERT significantly improved the condition of several individuals prior to HSCT.