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Blood Adv. 2018 Jan 4;2(1):49-60. doi: 10.1182/bloodadvances.2017010645. eCollection 2018 Jan 9.

Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.

Author information

1
Blood and Marrow Transplantation Program.
2
Laboratory for Translational Immunology, and.
3
Sylvia Toth Center for Multidisciplinary Follow Up After Hematopoietic Cell Transplantation, UMC Utrecht, Utrecht, The Netherlands.
4
Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC.
5
Eurocord, Paris, France.
6
AP-HM Hôpital de la Timone, Marseille, France.
7
Hospital Infantil Universitario Niño Jesús, Madrid, Spain.
8
Blood and Marrow Transplantation Program, Universiteits Ziekenhuis Gent, Gent, Belgium.
9
Sydney Children's Hospital, Sydney, Australia.
10
Children's Hospital at Westmead, Sydney, Australia.
11
King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.
12
Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom; and.
13
Duke University Medical Center, Durham, NC.

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.

PMID:
29344584
PMCID:
PMC5761624
DOI:
10.1182/bloodadvances.2017010645
[Indexed for MEDLINE]
Free PMC Article

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