CHILD-EU

Orphans Unite: chILD better together – European Management Platform for Childhood Interstitial Lung Diseases

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 Obiettivo del progetto (Objective)

'Children with diffuse lung disease, also called childhood interstitial lung diseases (chILD), may have one of more than 200 entities, the biggest group of respiratory orphan lung diseases. Frequently undiagnosed because of lack of awareness or complex differential diagnosis, they lead to much morbidity, mortality (about 15%) and psychosocial stress for the families. Current lack of evidence based guidelines reflects the absence of any real scientific evidence for management. All current therapeutic options are off label. We propose that leading European clinical scientists and paediatric pulmonologists collaborate to assemble cohorts in which children with well defined disease entities, verified by international panels of clinicians, radiologists, geneticists and pathologists are followed in a pan-European database and biobank compatible with others worldwide to allow common projects. Outcomes and treatment schemes will be rigorously defined and their value systematically assessed. We will put defined treatment protocols systematically into practice to allow their evaluation and perform a randomised controlled trial in line with the EU recommendations, to put prescribing for children on an evidence based footing. This will give evidence to use medicines available based on their objectively determined effects and side effects. The project will lead to accepted evidence-based and consensus-agreed diagnostic and management clinical guidelines, to a better care of patients afflicted by rare chILD and lead to improved quality of life for children with these incurable diseases.'

Introduzione (Teaser)

Childhood interstitial lung disease (chILD) refers to a group of over 100 rare lung diseases that affect babies, children and teenagers. Symptoms include chronic cough, shortness of breath and rapid breathing.

Descrizione progetto (Article)

Annually, in Europe, there are one or so cases of CHILD for every 100 000. Such patients often have an indeterminate, sometimes poor, prognosis and quality of life.

Diagnosis is difficult as chILD is rare and complex with no currently approved cures in existence. Besides lack of standardised diagnostic criteria, treatment requires use of off-label drugs as meeting the requirements for conducting clinical trials is too difficult for such rare diseases.

Through a pan-European network, the EU-funded http://www.childeu.net (CHILD-EU) project will conduct randomised clinical trials for chILD and implement a standardised patient database and biobank. Their goal is to fill the gaps in knowledge and develop evidence-based guidelines for faster diagnosis and effective clinical management of chILD.

CHILD-EU members successfully established a web-based database and biobank system. chILD cases can be registered and their data anonymised to ensure long-term monitoring as well as biomaterial and data availability. Researchers also harmonised biomaterial handling and storage procedures at the international level. These protocols and a Best Practice Checklist for diagnosis are currently available on the project website.

Patient data entry began in March 2014 and over 300 cases from different sites are already included in the database. Most importantly, key parameters for outcome follow-up were selected for the forthcoming observational trials on chILD cases. Upon obtaining the requisite approvals, researchers will assess the efficacy of hydroxychloroquine and oral systemic steroid treatment in alleviating chILD symptoms.

At the end of the CHILD-EU project, data obtained from well-characterised chILD patients should provide insight into the aetiology of these diseases. This should aid in optimising protocols for faster diagnosis and effective treatment of such patients. Such evidence-based guidelines should also aid in obtaining approvals for currently off-label drugs. Ultimately, this should improve the quality of life of chILD patients as well as their families.