Service Communautaire d'Information sur la Recherche et le Développement - CORDIS

FP7

CHILD-EU Résumé de rapport

Project ID: 305653
Financé au titre de: FP7-HEALTH
Pays: Germany

Periodic Report Summary 2 - CHILD-EU (Orphans Unite: chILD better together – European Management Platform for Childhood Interstitial Lung Diseases)

Project Context and Objectives:
Childhood interstitial lung diseases (chILD) are rare diseases, frequently not diagnosed because the presentation is non-specific, and comprise a large group of childhood specific entities as well as the spectrum known from adult ILD. There are many unmet needs which we aim to address in this project, including the (a) low diagnostic awareness and isolated treatment across Europe; (b) the lack of uniform characterization, expert verification of diagnoses by international panels of clinicians, radiologists, geneticists and pathologists, (c) a missing pan-European database and bio-bank compatible with others worldwide, (d) no knowledge on currently used treatments; (e) undefined relevant clinical outcomes; (f) complete lack of quality controlled observations of all interventions; or (g) randomised and controlled interventions of off-label treatments, and (h) lack of evidence-based guidelines and treatment protocols for chILD in Europe.

Project Results:
We have successfully implemented the European database and biobank based on SecuTrial®, a web-based data capture and biobank system. After notification of a case via a web-site Fig. 1 or by email contact the participant is registered, trained through a training database and then can enter pseudonymized patient data and submit biomaterials to the central site. Support and help with entering data is provided by the central site. The Data dictionaries of the databases from UK, Germany, France and the United States were evaluated, discussed in consensus meetings, and the child-EU database was accomplished. The data safety concept of the register and biobank was approved by the technical and methodological platform of research networks (TMF). International harmonized biomaterial handling and storage procedures were agreed upon and are available on our website.
Patient data entry started in March 2014. End of December 2015 438 cases from sites in Germany, UK, Italy, Turkey and other sites were included. After being appropriately entered, those cases ready for peer-review were reviewed by clinician experts, pediatric radiology experts, and if necessary specialized pathology and genetic experts. Up to now 180 cases are finally reviewed and followed prospectively.
We generated a “Best practice Checklist” for the diagnostics in suspected chILD. This list is available on the chILD EU project website. In addition, we complied “Standard Operating Procedures (SOPs)” based on the results of collected current clinical practice in Europe, closely linked with American and Canadian chILD groups. A major step was to set up the observational trial on incident and prevalent chILD cases and establish all necessary Ethics approvals for the participating centers.
Following several rounds of discussion and document evaluation among the participants, key parameters for outcome follow-up were defined. Key respiratory parameters like respiratory rate, oxygen saturation, retractions, dyspnea assessed by Borg scale, coughing or eating behavior were assessed by nurses or lay caregivers following one instruction in infants and young children with no differences detected. The complex variable acute exacerbation was defined as a sustained/unexplained worsening of the patient’s condition from stable state and beyond normal day to day variations and a list of seven acute exacerbation criteria was suggested. Other major parameters (chest imaging and quality of life) are still under evaluation.
In a high rank respiratory journal we published “European protocols for the diagnosis and initial treatment of interstitial lung disease in children”; Thorax 2015.
From a caregiver point of view, family experience were assessed and up to now unrecognized and unmet needs identified, like feeding issues, identified.
Patient care was directly assessed and improved by development of a freely available patient information booklet. This was produced in German, French, Danish, Turkish and English. For patient education we developed and evaluated instruments and initiated a pilot study of a structured patient education program.
An important objective of this project was to set up randomized therapeutic interventions. After extensive internal discussions and a world-wide Delphi process to reflect on the needs and wishes of the community, we decided to focus on the investigations of two different compounds and 3 clinical situations:
1. Initiation of treatment with hydroxychloroquine (HCQ) (4 weeks randomized and placebo controlled) (Fig. 2)
2. Hydroxychloroquine withdrawal (HCQ) (3 months, randomized and placebo controlled) (Fig. 3)
3. Medium term tapered oral systemic steroid treatment, randomized against placebo (EAA) (Fig. 4)
Trials are available in Germany since 2015, open sites include Munich, Hannover, Essen, Giessen, Leipzig, Frankfurt. Sites in UK, Italy and Turkey are pending.

Potential Impact:
ChILD-EU program will achieve the following results, impacts and implications, including:
(i) Unite European clinicians caring for isolated orphan cases of chILD into a critical mass of expertise that no individual European country can provide.
(ii) Actively pull together currently isolated cases into a critical mass of patients that will enable us to evaluate and elucidate the clinical, radiological and prognostic course of these rare diseases.
(iii) Offer biobank of specimens from well characterized patients to elucidate the mechanisms of these rare diseases
(iv) Enable individual clinicians to be more effective through a supportive peer review of diagnosis with recommendations for best practice, and subsequent outcome review by anonymised data provided by the common European database.
(v) Improve the quality and consistency of diagnostic accuracy; benefitting patients and clinicians.
(vi) Define and assess the definition of appropriate biomarkers as outcome measures.
(vii) Test disease outcomes based on current clinical practice variance across Europe
(viii) Include subjects into therapeutic double blind randomized controlled trials of currently off-label used medicines and to evaluate their effectiveness and adverse effects.
(ix) Use this pan European chILD communication network to better support patients and their families by providing knowledge and evidence-based clinical guideline frameworks for clinician practice based on the outcomes of our work.
(x) Use the pan European management platform for children´s interstitial lung disease as a solid base for future clinical and research networks
(xi) Continue our partnership work with European chILD parent network to ensure practice reflects and incorporates outcomes of importance to the quality of life for children and their parents.
(xii) Lead to a better care of patients afflicted by rare chILD diseases for which no dedicated treatment is currently available.

Publication: Bush A, Anthony G, Barbato A, Cunningham S, Clement A, Epaud R, Gilbert C, Goldbeck L, Kronfeld K, Nicholson AG, Schwerk N, Griese M. Research in progress: put the orphanage out of business. Thorax. 2013 Oct;68(10):971-3. Thorax
List of Websites:
www.childeu.net

Informations connexes

Contact

Petra Slamova, (Administrative financial officer)
Tél.: +49 89 5160 8341
E-mail
Numéro d'enregistrement: 184095 / Dernière mise à jour le: 2016-06-08
Source d'information: SESAM