Annelienke van Hulst

180 Chapter 6 METHODS Study design This phase three, double-blind, placebo-controlled, RCT with cross-over design, the DexaDays-2 study, was conducted in the Princess Máxima Center for pediatric oncology in the Netherlands (national tertiary hospital). The study was approved by the Medical Ethical Committee of Rotterdam (NL62388.078.17) and was included in the Netherlands Trial Register (NTR6695/NL6507).12 Detailed methods have been published previously11 and an additional relevant method section is available as Supplement. Participants Medium Risk Group (MRG) ALL patients, aged 3–18 years, treated according to the Dutch Childhood Oncology Group ALL-11 protocol who received dexamethasone during maintenance treatment were eligible. All included parents and/or patients gave written informed consent to participate in the study. Patients were assessed before and after one dexamethasone course, whereafter patients with an increase of ≥5 points (clinically relevant dexamethasone-induced problems)10,13 on the parent-reported Strengths and Difficulties Questionnaire (SDQ) were eligible for the RCT (Figure 1). Intervention The intervention consisted of oral physiological dosage of liquid hydrocortisone: 10 mg/ m2/day in a circadian rhythm; 5 mg/m2 in the morning directly after awakening, 3 mg/ m2 in the afternoon and 2 mg/m2 in the evening. Hydrocortisone was administered for five consecutive days, in addition to dexamethasone. Placebo was administered similarly and had the same appearance and taste as hydrocortisone. Patients were randomized using the method of a prefixed randomization list, prepared by the pharmacy, to receive two courses hydrocortisone followed by two courses placebo, or vice versa (Figure 1). The administration of study medication was blinded for physicians, parents, patients and research personnel. At the close-out visit, parents were asked whether they thought their child had started with hydrocortisone or with placebo during the RCT. Outcomes The primary outcome was measured at all timepoints (T1-T10). Secondary outcomes were measured on T1/T2, T3/T4 and T7/T8, except for health-related quality of life and objective sleep through actigraphy, which were measured at T3/T4 and T7/T8 only, to minimize patient burden (Figure 1).

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