title: Childhood Cerebral Adrenoleukodystrophy: MR Perfusion Measurements and Their Use in Predicting Clinical Outcome after Hematopoietic Stem Cell Transplantation.
abstract: MR perfusion has shown abnormalities of affected WM in cerebral X-linked adrenoleukodystrophy, but serial data is needed to explore the import of such findings after hematopoietic stem cell transplantation. Our aim was to prospectively measure MR perfusion parameters in patients with cerebral adrenoleukodystrophy pre- and post-hematopoietic stem cell transplantation, and to correlate those measurements with clinical outcome.
  
  
  
  Ten patients with cerebral adrenoleukodystrophy prospectively underwent DSC-MR perfusion imaging at <45 days pre- (baseline), 30-60 days post-, and 1 year post-hematopoietic stem cell transplantation. MR perfusion measurements in the 10 patients and 8 controls were obtained from the parieto-occipital WM, splenium of the corpus callosum, leading enhancing edge, and normal-appearing frontal white matter. MR imaging severity scores and clinical neurologic function and neurocognitive scores were also obtained. MR perfusion values were analyzed in the patients with cerebral adrenoleukodystrophy at each time point and compared with those in controls. Correlations were calculated between the pre-hematopoietic stem cell transplantation MR perfusion values and 1-year clinical scores, with P value adjustment for multiple comparisons.
  
  
  
  At baseline in patients with cerebral adrenoleukodystrophy, both relative CBV and relative CBF within the splenium of the corpus callosum and parieto-occipital WM significantly differed from those in controls (P = .005-.031) and remained so 1 year post-hematopoietic stem cell transplantation (P = .003-.005). Meanwhile, no MR perfusion parameter within the leading enhancing edge differed significantly from that in controls at baseline or at 1 year (P = .074-.999) or significantly changed by 1 year post-hematopoietic stem cell transplantation (P = .142-.887). Baseline Loes scores correlated with 1-year clinical neurologic function (r = 0.813, P < .0001), while splenium of the corpus callosum relative CBV also significantly correlated with 1-year neurologic function scale and the neurocognitive full-scale intelligence quotient and performance intelligence quotient scores (r = -0.730-0.815, P = .007-.038).
  
  
  
  Leading enhancing edge measurements likely remain normal post-hematopoietic stem cell transplantation in cerebral adrenoleukodystrophy, suggesting local disease stabilization. Meanwhile, parieto-occipital WM and splenium of the corpus callosum relative CBV and relative CBF values worsened; this change signified irreversible injury. Baseline splenium of the corpus callosum relative CBV may predict clinical outcomes following hematopoietic stem cell transplantation.
authors.affiliation: From the Departments of Radiology (A.M.M., J.B., D.R.N., M.B.S.) mckinrad@umn.edu.
authors.forename: A M
authors.identifier: 0000-0003-2530-5970
authors.initials: AM
authors.lastname: McKinney
authors.affiliation: From the Departments of Radiology (A.M.M., J.B., D.R.N., M.B.S.).
authors.forename: J C
authors.identifier: 0000-0002-4038-5422
authors.initials: JC
authors.lastname: Benson
authors.affiliation: From the Departments of Radiology (A.M.M., J.B., D.R.N., M.B.S.).
authors.forename: D R
authors.identifier: 0000-0002-2468-7359
authors.initials: DR
authors.lastname: Nascene
authors.affiliation: Pediatrics (J.E., G.R.), Division of Clinical Behavioral Neuroscience, University of Minnesota Masonic Children's Hospital, Minneapolis, Minnesota.
authors.forename: J
authors.identifier: 0000-0001-5340-017X
authors.initials: J
authors.lastname: Eisengart
authors.affiliation: From the Departments of Radiology (A.M.M., J.B., D.R.N., M.B.S.).
authors.forename: M B
authors.identifier: 0000-0002-2850-6041
authors.initials: MB
authors.lastname: Salmela
authors.affiliation: Suburban Radiologic Consultants (D.J.L.), Minneapolis, Minnesota.
authors.forename: D J
authors.identifier: 0000-0001-7661-3442
authors.initials: DJ
authors.lastname: Loes
authors.affiliation: Clinical and Translational Science Institute (L.Z.), University of Minnesota, Minneapolis, Minnesota.
authors.forename: L
authors.identifier: 0000-0002-3067-305X
authors.initials: L
authors.lastname: Zhang
authors.affiliation: Radiology Associates of the Fox Valley (K.P.), Neenah, Wisconsin.
authors.forename: K
authors.identifier: 0000-0002-4863-8957
authors.initials: K
authors.lastname: Patel
authors.affiliation: Pediatrics (J.E., G.R.), Division of Clinical Behavioral Neuroscience, University of Minnesota Masonic Children's Hospital, Minneapolis, Minnesota.
authors.forename: G V
authors.identifier: 0000-0002-7528-1906
authors.initials: GV
authors.lastname: Raymond
authors.affiliation: Department of Pediatrics (W.P.M.), Pediatric Blood and Marrow Transplantation Division, University of Minnesota Amplatz Children's Hospital, Minneapolis, Minnesota.
authors.forename: W P
authors.identifier: 0000-0003-3168-3337
authors.initials: WP
authors.lastname: Miller
mesh_terms: D000293:Adolescent; D000326:Adrenoleukodystrophy / Q000000981:diagnostic imaging* / Q000503:physiopathology / Q000628:therapy*; D002560:Cerebrovascular Circulation; D002648:Child; D002675:Child, Preschool; D003337:Corpus Callosum / Q000000981:diagnostic imaging; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007361:Intelligence Tests; D008297:Male; D009483:Neuropsychological Tests; D011237:Predictive Value of Tests; D011379:Prognosis; D011446:Prospective Studies; D016896:Treatment Outcome; D066127:White Matter / Q000000981:diagnostic imaging