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Development of the SIOPE DIPG network, registry and imaging repository: a collaborative effort to optimize research into a rare and lethal disease.

ABSTRACT: Diffuse intrinsic pontine glioma (DIPG) is a rare and deadly childhood malignancy. After 40 years of mostly single-center, often non-randomized trials with variable patient inclusions, there has been no improvement in survival. It is therefore time for international collaboration in DIPG research, to provide new hope for children, parents and medical professionals fighting DIPG. In a first step towards collaboration, in 2011, a network of biologists and clinicians working in the field of DIPG was established within the European Society for Paediatric Oncology (SIOPE) Brain Tumour Group: the SIOPE DIPG Network. By bringing together biomedical professionals and parents as patient representatives, several collaborative DIPG-related projects have been realized. With help from experts in the fields of information technology, and legal advisors, an international, web-based comprehensive database was developed, The SIOPE DIPG Registry and Imaging Repository, to centrally collect data of DIPG patients. As for April 2016, clinical data as well as MR-scans of 694 patients have been entered into the SIOPE DIPG Registry/Imaging Repository. The median progression free survival is 6.0 months (95% Confidence Interval (CI) 5.6-6.4 months) and the median overall survival is 11.0 months (95% CI 10.5-11.5 months). At two and five years post-diagnosis, 10 and 2% of patients are alive, respectively. The establishment of the SIOPE DIPG Network and SIOPE DIPG Registry means a paradigm shift towards collaborative research into DIPG. This is seen as an essential first step towards understanding the disease, improving care and (ultimately) cure for children with DIPG.

SUBMITTER: Veldhuijzen van Zanten SEM

PROVIDER: S-EPMC5378734 | biostudies-literature | 2017 Apr

REPOSITORIES: biostudies-literature

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Development of the SIOPE DIPG network, registry and imaging repository: a collaborative effort to optimize research into a rare and lethal disease.

Veldhuijzen van Zanten Sophie E M SEM Baugh Joshua J Chaney Brooklyn B De Jongh Dennis D Sanchez Aliaga Esther E Barkhof Frederik F Noltes Johan J De Wolf Ruben R Van Dijk Jet J Cannarozzo Antonio A Damen-Korbijn Carin M CM Lieverst Jan A JA Colditz Niclas N Hoffmann Marion M Warmuth-Metz Monika M Bison Brigitte B Jones David T W DTW Sturm Dominik D Gielen Gerrit H GH Jones Chris C Hulleman Esther E Calmon Raphael R Castel David D Varlet Pascale P Giraud Géraldine G Slavc Irene I Van Gool Stefaan S Jacobs Sandra S Jadrijevic-Cvrlje Filip F Sumerauer David D Nysom Karsten K Pentikainen Virve V Kivivuori Sanna-Maria SM Leblond Pierre P Entz-Werle Natasha N von Bueren Andre O AO Kattamis Antonis A Hargrave Darren R DR Hauser Péter P Garami Miklos M Thorarinsdottir Halldora K HK Pears Jane J Gandola Lorenza L Rutkauskiene Giedre G Janssens Geert O GO Torsvik Ingrid K IK Perek-Polnik Marta M Gil-da-Costa Maria J MJ Zheludkova Olga O Shats Liudmila L Deak Ladislav L Kitanovski Lidija L Cruz Ofelia O Morales La Madrid Andres A Holm Stefan S Gerber Nicolas N Kebudi Rejin R Grundy Richard R Lopez-Aguilar Enrique E Zapata-Tarres Marta M Emmerik John J Hayden Tim T Bailey Simon S Biassoni Veronica V Massimino Maura M Grill Jacques J Vandertop William P WP Kaspers Gertjan J L GJL Fouladi Maryam M Kramm Christof M CM van Vuurden Dannis G DG

Journal of neuro-oncology 20170121 2

Diffuse intrinsic pontine glioma (DIPG) is a rare and deadly childhood malignancy. After 40 years of mostly single-center, often non-randomized trials with variable patient inclusions, there has been no improvement in survival. It is therefore time for international collaboration in DIPG research, to provide new hope for children, parents and medical professionals fighting DIPG. In a first step towards collaboration, in 2011, a network of biologists and clinicians working in the field of DIPG wa ...[more]

PMID: 28110411

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Project description:Background: The Rare Disease Research Partnership (RAinDRoP) was established in 2018 to bring together a wide variety of diverse voices in the rare disease community in Ireland and form a research partnership. This approach enabled clinicians, patients, carers and researchers to work together to identify top research priorities for rare diseases, which focused on a life-course perspective rather than a disease-specific need. Methods: A participatory multiple phase approach was used to identify research priorities for rare diseases. The research process involved three main phases: Phase I, Public Consultation Survey on Research in Rare Diseases in Ireland (PCSRRDI); Phase II, Research Prioritisation Workshop (RPW); Phase III, Follow-up Public Consultation and Prioritisation Survey (FWPCPS). Results: In total, 240 individuals completed the phase I PCSRRDI, which comprised of a cross-section of health care professionals, researchers and people living with rare diseases. One thousand and fifteen statements were collected, reflecting issues and shared challenges in rare diseases. A shortlisting step by step was used to identify any statements that had received a total score of above 50% into 10-12 researchable questions or statements per the theme for the phase II workshop. Phase II was focused on three main themes: (1) Route to Diagnosis, (2) Living with Rare Disease, (3) Integrated and Palliative Care. In total, 62 individuals attended the overall workshop; 42 participated in the prioritisation sessions. A cross-section of health care professionals, researchers and people living with rare diseases were engaged at each workshop. Seventy-five individuals completed the final phase III public ranking by priority responses, and they ranked the top 15 research priorities defined by the multi-stakeholders at the phase II consensus meeting. Conclusions: This study identified priorities for rare diseases research aimed at improving the health and wellbeing of people living with rare diseases.

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Development of the SIOPE DIPG network, registry and imaging repository: a collaborative effort to optimize research into a rare and lethal disease.

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Development of the SIOPE DIPG network, registry and imaging repository: a collaborative effort to optimize research into a rare and lethal disease.

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