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

Publication ,  Journal Article
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 ...
Published in: Blood Adv
January 9, 2018

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.

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Published In

Blood Adv

DOI

EISSN

2473-9537

Publication Date

January 9, 2018

Volume

2

Issue

1

Start / End Page

49 / 60

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Time Factors
  • Survival Analysis
  • Risk Factors
  • Male
  • Leukodystrophy, Metachromatic
  • Leukodystrophy, Globoid Cell
  • Infant
  • Humans
  • Female
 

Citation

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van den Broek, B. T. A., Page, K., Paviglianiti, A., Hol, J., Allewelt, H., Volt, F., … Boelens, J. J. (2018). Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies. Blood Adv, 2(1), 49–60. https://doi.org/10.1182/bloodadvances.2017010645
Broek, Brigitte T. A. van den, Kristin Page, Annalisa Paviglianiti, Janna Hol, Heather Allewelt, Fernanda Volt, Gerard Michel, et al. “Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.Blood Adv 2, no. 1 (January 9, 2018): 49–60. https://doi.org/10.1182/bloodadvances.2017010645.
van den Broek BTA, Page K, Paviglianiti A, Hol J, Allewelt H, Volt F, et al. Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies. Blood Adv. 2018 Jan 9;2(1):49–60.
van den Broek, Brigitte T. A., et al. “Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.Blood Adv, vol. 2, no. 1, Jan. 2018, pp. 49–60. Pubmed, doi:10.1182/bloodadvances.2017010645.
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. Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies. Blood Adv. 2018 Jan 9;2(1):49–60.

Published In

Blood Adv

DOI

EISSN

2473-9537

Publication Date

January 9, 2018

Volume

2

Issue

1

Start / End Page

49 / 60

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Time Factors
  • Survival Analysis
  • Risk Factors
  • Male
  • Leukodystrophy, Metachromatic
  • Leukodystrophy, Globoid Cell
  • Infant
  • Humans
  • Female