e20523 Background: Gastrointestinal stromal tumours (GISTS) are the most common mesenchymal tumours of the gastrointestinal tract. Over 90% of the GISTS have either KIT or PDGFRA mutations. The pediatric/adolescent/wild-type/syndromic (PAWS) GISTS are a heterogeneous group of patients with a different biology and natural history compared with the adult sporadic GISTS. Standard therapeutic approaches using tyrosine kinase inhibitors like imatinib or sunitinib have been less successful in PAWS-GIST patients. Better understanding of the biology and newer therapeutic strategies are needed to make any advances in the treatment of these extremely rare cancers. Methods: A joint collaborative group comprising of GIST specialists (Oncologists, pathologists, surgeons, radiologists, paediatricians, specialist nurses) and patients/carers was established in October 2010 to form the PAWS-GIST Focus group in UK. The first meeting took place in London in November 2010. The aims of the group are the following: (a) clinically gather UK-wide information on the number of patients, natural history, treatment, etc; (b) act as a facilitator between various specialist groups treating these patients; (c) provide information and support to patients and carers and opportunities for fundraising activities; and (d) encourage collaborative research work leading to better understanding of the biology, exploring newer small molecules/kinase inhibitors and explore establishing GIST cell lines. Results: As of January 2011, we have been able to identify 13 paediatric/adolescent GIST patients in United Kingdom. Work is in progress as part of the UK national GIST registry to prospectively identify all PAWS-GIST patients in the UK. Conclusions: PAWS-GIST initiative is the first of its kind in UK and is fully supported by the UK GIST professional and patient groups. Future plans include establishing national clinics based on the NIH USA model. Fund raising activities are ongoing.
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