Volume of Gadolinium Enhancement and Successful Repair of the Blood-Brain Barrier in Cerebral Adrenoleukodystrophy

Troy C. Lund, Michelle Ng, Paul J. Orchard, Daniel J. Loes, Gerald V. Raymond, Ashish Gupta, Dan Kenny-Jung, David R. Nascene

Research output: Contribution to journalArticlepeer-review

Abstract

Up to 40% of boys with adrenoleukodystrophy develop a severe central nervous system demyelinating form (cALD) characterized by white matter changes and gadolinium enhancement on magnetic resonance imaging (MRI). Hematopoietic cell transplant (HCT) is the only proven means to attenuate cALD progression. The elimination of active neuroinflammation is indicated radiographically by the resolution of gadolinium (Gd) enhancement and correlates to speed of donor neutrophil recovery. We analyzed 66 boys with cALD undergoing HCT for biomarkers correlating with early (30 days post-HCT) Gd signal resolution. We found that log Gd volume (cm3) on pre-HCT MRI strongly positively correlated to day 30 Gd resolution (P = .0003) with smaller volume correlating to higher proportion resolved, as was the baseline gadolinium intensity score (P = .04), plasma chitotriosidase activity (P = .04), and faster absolute neutrophil count recovery (P = .03). In multivariate analysis, log Gd volume remained superior in determining which patients would have Gd signal resolution by 30 days post-HCT (P = .016). A final analysis indicated that early Gd resolution also correlated with less neurologic progression from baseline to 1 year following HCT (P = .006). MRI Gd volume may serve as a contributing biomarker to better delineate outcomes and an important metric in comparing therapies in the treatment of cALD.

Original languageEnglish (US)
Pages (from-to)1894-1899
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Volume26
Issue number10
DOIs
StatePublished - Oct 2020

Keywords

  • Adrenoleukodystrophy
  • Blood-brain barrier
  • Demyelination
  • Gadolinium
  • Transplant

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Fingerprint Dive into the research topics of 'Volume of Gadolinium Enhancement and Successful Repair of the Blood-Brain Barrier in Cerebral Adrenoleukodystrophy'. Together they form a unique fingerprint.

Cite this