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chILD EU: HCQ in pediatric ILD study and Stop EAA study to evaluate prednisolone treatment and course of disease

Laufzeit: 01.01.2012 - 31.12.2016

Kurzfassung


In order to better understand the natural course, risk factors, treatments and reasons for the development of childhood interstitial lung disease (chILD), we will collect and analyse details of symptoms and quality of life, clinical data and also biological material in a Register and Biobank. In the long run, this Register will serve the improved understanding of the disease and will lead to the development of new and effective approaches to treatment.

Interstitial lung disease in children...
In order to better understand the natural course, risk factors, treatments and reasons for the development of childhood interstitial lung disease (chILD), we will collect and analyse details of symptoms and quality of life, clinical data and also biological material in a Register and Biobank. In the long run, this Register will serve the improved understanding of the disease and will lead to the development of new and effective approaches to treatment.       

Interstitial lung disease in children (chILD) is a very rare condition. It is a group of over 200 different entities that affect the parenchyma of the lung and lead to a chronic lung disease. The natural course of many of these diseases is connected to a high morbidity and mortality. Often the children are dependent on oxygen for a long period of time or demand mechanical ventilation.
No successfully proven pharmacological treatments are available up-to-date. The symptomatic treatment in general consists of oxygen supplementation if necessary, adequate nutrition adapted to the high energy demand due to the increased breathing effort, as well as immunization against respiratory pathogens to prevent exacerbations through respiratory infections.
 
Two studies are to be conducted within this context: HCQ in pediatric ILD and Stop EAA.        
   
Hydroxychloroquine in pediatric ILD: START randomized controlled in parallel-group, then switch placebo to active drug, and STOP randomized controlled in parallel-group to evaluate the efficacy and safety of hydroxychloroquine (HCQ). The current pharmacological treatment regimens are mainly dependent on the treating physician’s expertise. Common treatment options include the treatment with steroids, hydroxychloroquine (HCQ) and macrolide antibiotics. All three substances are often used in chILD, although so far no controlled study has shown a benefit. In some cases treatment consist of other agents such as azathioprine, cyclophosphamide, cyclosporine or methotrexate.

Stop exogenous allergic alveolitis (EAA) in childhood: Healthy into adulthood – a randomized, double-blind, placebo-controlled, parallel-group study to evaluate prednisolone treatment and course of disease. The long term course of individuals with exogenous allergic alveolitis (EAA) cannot be predicted individually, although initial allergen removal and steroid treatment appears simple. Some children may have a complicated course; thus all should be assessed, treated and followed systematically.
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