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Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies. Blood Adv 2018 Jan 09;2(1):49-60

Date

01/19/2018

Pubmed ID

29344584

Pubmed Central ID

PMC5761624

DOI

10.1182/bloodadvances.2017010645

Scopus ID

2-s2.0-85049559395 (requires institutional sign-in at Scopus site)   30 Citations

Abstract

Leukodystrophies (LD) are devastating inherited disorders leading to rapid neurological deterioration and premature death. Hematopoietic stem cell transplantation (HSCT) can halt disease progression for selected LD. Cord blood is a common donor source for transplantation of these patients because it is rapidly available and can be used without full HLA matching. However, precise recommendations allowing care providers to identify patients who benefit from HSCT are lacking. In this study, we define risk factors and describe the early and late outcomes of 169 patients with globoid cell leukodystrophy, X-linked adrenoleukodystrophy, and metachromatic leukodystrophy undergoing cord blood transplantation (CBT) at an European Society for Blood and Marrow Transplantation center or at Duke University Medical Center from 1996 to 2013. Factors associated with higher overall survival (OS) included presymptomatic status (77% vs 49%; P = .006), well-matched (≤1 HLA mismatch) CB units (71% vs 54%; P = .009), and performance status (PS) of >80 vs <60 or 60 to 80 (69% vs 32% and 55%, respectively; P = .003). For patients with PS≤60 (n = 20) or 60 to 80 (n = 24) pre-CBT, only 4 (9%) showed improvement. Of the survivors with PS >80 pre-CBT, 50% remained stable, 20% declined to 60 to 80, and 30% to <60. Overall, an encouraging OS was found for LD patients after CBT, especially for those who are presymptomatic before CBT and received adequately dosed grafts. Early identification and fast referral to a specialized center may lead to earlier treatment and, subsequently, to improved outcomes.

Author List

van den Broek BTA, Page K, Paviglianiti A, Hol J, Allewelt H, Volt F, Michel G, Diaz MA, Bordon V, O'Brien T, Shaw PJ, Kenzey C, Al-Seraihy A, van Hasselt PM, Gennery AR, Gluckman E, Rocha V, Ruggeri A, Kurtzberg J, Boelens JJ

Author

Kristin Page MD, MHS, MEd Associate Professor in the Pediatrics department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Adrenoleukodystrophy
Child
Child, Preschool
Cord Blood Stem Cell Transplantation
Female
Humans
Infant
Leukodystrophy, Globoid Cell
Leukodystrophy, Metachromatic
Male
Risk Factors
Survival Analysis
Time Factors
Treatment Outcome