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Název česky Sunitinib v pediatrické onkologii - současný klinický výzkum


Rok publikování 2018
Druh Konferenční abstrakty
Fakulta / Pracoviště MU

Lékařská fakulta

Popis Paediatric clinical trials represent a minor part of the clinical research in oncology, despite the needs of the daily clinical practice. The use of targeted therapeutics in children may be associated with long-term or irreversible consequences seen after the intensive chemotherapy. Sunitinib is a multiple tyrosine kinase inhibitor used in the treatment of renal carcinoma (RCC), gastrointestinal stromal tumours (GIST), and pancreatic neuroendocrine tumours in adults. Up-to-date, there is no official indication for sunitinib in paediatrics and the drug is mostly used off-label. The aim of this study is to summarize the published and ongoing paediatric-oriented clinical research focused on the use of sunitinib and to supplement it with case reports from the clinical practice. The literature search was carried out in PubMed and Web of Science databases, and and registries. Promises and pitfalls of the offlabel use of sunitinib are described on case reports of child patients treated with sunitinib at the Department of Paediatric Oncology, University Hospital Brno. We have identified 11 paediatric clinical trials or clinical data analyses published in medical journals. Five (45.5%) were focused on sarcoma incl. GIST, others on solid tumours in general, nervous system tumours, or RCC. In the registries, 19 paediatric trials with sunitinib were identified. Five ongoing trials use sunitinib to treat solid tumours, RCC, and sarcomas. The literature and our experience as well show that sunitinib is used in a variety of doses and regimens in child patients. The most frequent therapeutic response is stable disease, with a few partial remissions described. The safety profile of sunitinib is similar as in the adult population. The most common adverse reactions are gastrointestinal symptoms, skin and hair 86 discolouration, hypothyroidism, hand-foot syndrome, or hypoglycaemia. Possible influence on growth has to be clarified, as growth plates’ pathologies have been noted. Altogether, there is an urgent need of paediatric clinical research to answer the questions of the optimal indication, dose, regimen, or safety aspects of sunitinib as well as other targeted drugs. As childhood cancer is rare condition, pharmaceutical companies do not extensively focus on this topic. It is more suitable for an investigator-initiated clinical trial. The non-profit Czech Clinical Research Infrastructure Network (CZECRIN) offers help and consultations for investigators with the conduct of non-commercial clinical trials.
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