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Holly R. Martin, Michele D. Poe, James M

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1 Neurodevelopmental Outcomes of Umbilical Cord Blood Transplantation in Metachromatic Leukodystrophy 
Holly R. Martin, Michele D. Poe, James M. Provenzale, Joanne Kurtzberg, Adam Mendizabal, Maria L. Escolar  Biology of Blood and Marrow Transplantation  Volume 19, Issue 4, Pages (April 2013) DOI: /j.bbmt Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

2 Figure 1 Overall survival of all patients (A) and according to age at onset (B) through the follow-up period. Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

3 Figure 2 Gross motor and cognitive function versus Loes score. (A) Cognitive function and (B) gross motor at the last evaluation (ratio of functional age-to-chronologic age) are plotted against pretransplant modified Loes scores. Patients are represented by red markers (late-infantile-onset MLD) and blue markers (juvenile-onset MLD). Red and blue lines indicate the least-squares regression lines for each group. Symptom status at the time of transplantation is indicated by hollow markers (asymptomatic) versus solid markers (symptomatic). Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

4 Figure 3 Developmental outcomes are shown by plotting age-equivalent score (developmental age) against actual age (calendar age) for late-infantile and juvenile patients. (A) Cognitive and (B) gross motor development for the late-infantile group. (C) Cognitive and (D) gross motor development for the juvenile group. Red and blue lines indicate individual values of children who were symptomatic (red) and asymptomatic (blue) at time of transplantation. Gray lines represent the mean and approximate variability (95% CI) observed in typically developing children. Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

5 Figure 4 The average developmental trajectory for each developmental domain is presented for the patients who were asymptomatic at the time of transplantation. Developmental outcomes for each developmental domain are shown by plotting age-equivalent score (developmental age) against actual age (calendar age). (A) Developmental trajectories for late-infantile onset. (B) Developmental trajectories for juvenile onset. Gray lines represent the mean and approximate variability (95% CI) observed in typically developing children. Biology of Blood and Marrow Transplantation  , DOI: ( /j.bbmt ) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions


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