We expected the largest changes in cognition and motor activity to occur between baseline (T1) and study week 16 (T2).
Mean baseline and study end (week 12) scores for each individual ILC item are shown in Figure 4a (parents' assessments) and Figure 4b (patients' assessments), for the overall population and the 'children' and 'adolescents' subgroups.
Health-related quality of life (QOL) was evaluated using the Medical Outcomes Study 36-item Short Form Health Survey (SF-36) [ 27], which patients completed at baseline and at study weeks 12 and 28.
Safety was assessed with serial blood chemistry panels and symptom assessment, performed at baseline and study visits at weeks 1, 3, 7, 9, and 12. Adverse events were summarized by grade defined by Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 [ 30].
Computed tomography or magnetic resonance imaging was performed at baseline, on study week 4, 8, 12 and every 8 weeks thereafter.
This was true for transfusions received from baseline to study Week 13 or from baseline to the end of treatment (Table 3).
After checking for normality, Pearson's product moment correlation coefficients (r) were calculated for scores, as well as for changes in scores of rating scales at baseline, study week 16 and open-label endpoint at week 52.
Assessments of self-reported Quality of Life Inventory (QOLI) [ 44], as well as neuropsychological assessments performed by certified psychologists, were conducted at baseline (T1), at study week 16 (T2) and at endpoint study week 52 (T3), with the exception of the WAIS-III subtest Similarities [ 45], which was assessed at baseline (T1), and at study week 52 (T3) only.
Intervention (I) and control group (C). 3 Day Diet Diaries (3DDDs) were done at baseline and end of study (week 12).
It was used as a specificity measure for the assessments performed during the study, thus only administered at baseline (T1) and at study week 52 (T3).
