SFEBES2023 Poster Presentations RET (5 abstracts)
National Experience of the use of the highly selective RET tyrosine kinase inhibitor Selpercatinib in children with multiple endocrine neoplasia type 2 and advanced medullary thyroid cancer: updated experience
Sara Stoneham 1 , Elizabeth A Corley 2 , Emma Ross 3 , Evelien Gevers 4 , Kate Newbold 5 , Tim Beale 6 , Kee Wong 5 , Caroline Brain 7 , Assunte Albanese 8 , Colin Butler 9 , Tarek Abdel-Aziz 10 , Ian Proctor 11 , Tom R Kurzawinsky 10 & Lynley V Marshall 2
1Children and Young People’s Cancer Services, University College London Hospitals NHS Foundation Trust, London, United Kingdom. 2Department of Paediatric and Adolescent Oncology, The Royal Marsden NHS Foundation Trust, Sutton and The Institute of Cancer Research, London, United Kingdom. 3Department of Paediatric Oncology, University Hospitals of Leicester NHS Trust, Leicester, United Kingdom. 4Centre for Endocrinology, William Harvey Research Institute, Queen Mary University of London and Department of Paediatric Endocrinology, Barts Health NHS Trust, London, United Kingdom. 5Department of Oncology, The Royal Marsden NHS Foundation Trust, Sutton and The Institute of Cancer Research, London, United Kingdom. 6Department of Head and Neck Radiology, University College London Hospitals NHS Foundation Trust, London, United Kingdom. 7Department of Paediatric Endocrinology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom. 8Department of Endocrinology, The Royal Marsden NHS Foundation Trust, Sutton and The Institute of Cancer Research, London, United Kingdom. 9Department of Endocrine Surgery, Great Ormond Street Hospital, London, United Kingdom. 10Department of Endocrine Surgery, University College London Hospitals NHS Foundation Trust, London, United Kingdom. 11Department of Cellular Pathology, University College London Hospitals NHS Foundation Trust, London, United Kingdom
Background and Demographics: Medullary thyroid carcinoma (MTC), in the context of Multiple Endocrine Neoplasia type 2 (MEN2), is caused by mutations in the RET proto-oncogene. For children with MEN2, both 2A and 2B subtypes, and advanced MTC, the RET tyrosine kinase (TK) pathway is a target for treatment with selpercatinib, a selective RET TK inhibitor (TKI). In the United Kingdom, 7 paediatric patients have been receiving named -patient, compassionate access treatment with selpercatinib for the management of advanced MTC, as primary systemic therapy after surgery. This updates our 2021 publication with an additional 48-month experience and an additional patient.
Results: 7 MTC MEN2 (2A x2; 2B x 5) patients were reviewed; aged between age 9 and 17 years and with a male: female ratio of 4: 3. 6 remain taking selpercatinib. One patient demonstrated radiological and biochemical evidence of tumour progression within 23 months of continuous therapy, after an initial good response. The remaining 6 patients continue to demonstrate ongoing biochemical and radiological response. The average duration of therapy is 33 months (range 16 - 49). Pre-treatment calcitonin ranged between 146 - 36 000ng/l. Current calcitonin levels range between 1.6 - 176ng/l. All patients achieved a maximal reduction in tumour markers of >90%. Overall, treatment was well tolerated. Two patients developed bilateral slipped capital femoral epiphyses (SCFE) requiring surgery. Another patient developed bowel lymphangiectasis responsive to dietary change. Two had intercurrent viral chest infections requiring hospital admission, oxygen and a temporary cessation of selpercatinib. One patient developed a prolonged QTc(F) which self-resolved without the need for dose modification.
Conclusions: Selpercatinib is deliverable, tolerable and efficacious at providing a period of disease control in a paediatric cohort. Further understanding of selpercatinib drug interactions and any causality between selpercatinib and SCFE for patients at known risk with MEN2, is required.
Volume 94
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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