Outcomes of Unrelated Umbilical Cord Blood Transplantation for X-Linked Adrenoleukodystrophy

(a) Overall survival after transplantation. Kaplan-Meier estimates of the probability of overall survival. Cumulative incidence of overall survival at 6.25 months was 66.7% (95% confidence interval 39.9%-93.3%). (b) Cumulative incidence of acute GVHD grades II-IV (n = 10, 2 events). Cumulative incidence at day 100: 40.0% (95% confidence interval 7.5%-72.5%). (c) Cumulative incidence of chronic GVHD (n = 10, 2 events). Cumulative incidence at day 365: 10.0% (95% confidence interval 0.0%-29.6%) CINC at day 730: 22.9% (95% confidence interval 0.0%-53.2%).

(a-f) Developmental function by domain. Each patient’s development is represented by a unique color line. The green diagonal line represents typical population development. The gray area represents the approximate normal 95% range in the general population.

(a) Cognitive development and pretransplant MRI Loes scores. The green line represents typical development. Each blue line represents the longitudinal cognitive course of a surviving patient with baseline Loes Scores <10. The red lines represent the longitudinal cognitive course of surviving patients with Loes Scores >10. The black and purple represent deceased patients. (b) Gross motor development and pretransplant MRI Loes scores. The green line represents typical development. Each blue line represents the longitudinal motor course of a surviving patient with baseline Loes scores <10. The red lines represent the longitudinal motor course of surviving patients with Loes scores >10. The black and purple represent deceased patients. Note that motor scores are in general lower than cognitive scores in (a).