Efficacy of methylprednisolone pulses in neuroendocrine cells hyperplasia of infancy: an early phase study (CORTICONEHI).

简要总结

主要结局

次要结局

• Efficacy endpoint, number of children requiring only sleep oxygen at M18.
• Efficacy endpoint, number of children with a normal respiratory rate at rest at M18
• Efficacy endpoint, difference of Fan’s severity score assessing the severity of children with ILD (respiratory symptoms, SpO2<90% at sleep or exertion, SpO2<90% at rest and pulmonary hypertension) between M0) and M18
• Efficacy endpoint, patients and family impact PedsQL and the chILD QoL scores; the chILDPQoL score, a parents QoL score (submitted for publication) at M0 and M18
• Efficacy endpoint, number of patients requiring at least one enteral nutrition episode (based on the physician appreciation in case of abnormal weight curve) between M0 and M18
• Safety endpoint, number of unexpected hospitalizations for a respiratory exacerbation between M0 and M18
• Safety endpoint, number of antibiotic courses for an infectious lung exacerbation between M0 and M18
• Safety endpoint, at M6, the number of patients with at least once systolic or diastolic arterial blood pressure (AP) over 2SD for the age at the days of methylprednisolone pulses
• Safety endpoints, the number of patients with at least one an elevated fasting glycaemia before the first day of methylprednisolone pulses
• Exploratory endpoint, correlation between family impact PedsQL questionnaire and the newly described chILD-PQoL questionnaire at each time of measurement
• Exploratory endpoint, number of patients with an extension, a stability, a decrease or an absence of the GGO lesions on the CT-scan at M6 and M18