108 Chapter 3 ALL MR patients: 275 Eligible patients: 234 Informed Consent: 152 Patients enrolled part 1: 137 Neurobehavioral problems - : 82 Neurobehavioral problems + : 55 Randomization Exclusion • Age (<3): 5% • Other (language barrier, on hydrocortisone, severe infection etc.): 10% ALL patients: 367 Exclusion • Standard risk: 20% • High risk: 5% Exclusion • Refusal: 35% 40% 60% Dropout: 10% Group 1: 25 Group 2: 25 Dropout: 10% Figure 2. Flow chart of expected number of acute lymphoblastic leukemia (ALL) patients in the DexaDays-2 study. Group 1: starting with two courses hydrocortisone, thereafter cross over to placebo. Group 2: starting with two courses placebo, thereafter cross over to hydrocortisone Statistical analysis The effect of treatment (n = 50) is assessed by comparing placebo with hydrocortisone on SDQ Total Difficulties Score (delta scores; subtracting the score on treatment day 1 from the score after treatment day 5) by employing a Paired Student’s T-test or Wilcoxon Signed Rank test in case of violation of normality assumption. SDQ sub scores will also be compared between the two groups. The effect of hydrocortisone on sleep and HRQoL (total and sub scores) is evaluated in the same way. Due to the presence of repeated measures in the design of the RCT a generalized mixed model will be estimated to study the effect of therapy on neurobehavioral outcomes. This model explicitly accounts for the correlations between repeated measurements within each patient. Results from this analysis will provide information about the longitudinal effect of the treatment. A treatment period interaction will be included in the model to investigate the groups effect over time.
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