Sustained response to brigatinib in a patient with refractory metastatic pheochromocytoma harboring R1192P anaplastic lymphoma kinase mutation: a case report from the Austrian Group Medical Tumor Therapy next-generation sequencing registry and discussion of the literature

Goffredo, 2013, Malignant pheochromocytoma and paraganglioma: a population level analysis of long-term survival over two decades, J Surg Oncol, 107, 659, 10.1002/jso.23297 Prochilo, 2013, Targeting VEGF-VEGFR pathway by sunitinib in peripheral primitive neuroectodermal tumor, paraganglioma and epithelioid hemangioendothelioma: three case reports, Case Rep Oncol, 6, 90, 10.1159/000348429 Druce, 2009, Novel and evolving therapies in the treatment of malignant phaeochromocytoma: experience with the mTOR inhibitor everolimus (RAD001), Horm Metab Res, 41, 697, 10.1055/s-0029-1220687 Oh, 2012, Phase 2 study of everolimus monotherapy in patients with nonfunctioning neuroendocrine tumors or pheochromocytomas/paragangliomas, Cancer, 118, 6162, 10.1002/cncr.27675 Peters, 2017, Alectinib versus crizotinib in untreated ALK-positive non-small-cell lung cancer, N Engl J Med, 377, 829, 10.1056/NEJMoa1704795 Hida, 2017, Alectinib versus crizotinib in patients with ALK-positive non-small-cell lung cancer (J-ALEX): an open-label, randomised phase 3 trial, Lancet, 390, 29, 10.1016/S0140-6736(17)30565-2 Niemeijer, 2014, Chemotherapy with cyclophosphamide, vincristine and dacarbazine for malignant paraganglioma and pheochromocytoma: systematic review and meta-analysis, Clin Endocrinol, 81, 642, 10.1111/cen.12542 Averbuch, 1988, Malignant pheochromocytoma: effective treatment with a combination of cyclophosphamide, vincristine, and dacarbazine, Ann Intern Med, 109, 267, 10.7326/0003-4819-109-4-267 Kim, 2017, Brigatinib in patients with crizotinib-refractory anaplastic lymphoma kinase-positive non-small-cell lung cancer: a randomized, multicenter phase II trial, J Clin Oncol, 35, 2490, 10.1200/JCO.2016.71.5904 Camidge, 2018, Brigatinib versus crizotinib in ALK-positive non-small-cell lung cancer, N Engl J Med, 379, 2027, 10.1056/NEJMoa1810171 Richards, 2015, Standards and guidelines for the interpretation of sequence variants: a joint consensus recommendation of the American College of Medical Genetics and Genomics and the Association for Molecular Pathology, Genet Med, 17, 405, 10.1038/gim.2015.30 FMI. 2021. Available at https://www.foundationmedicine.com/test/foundationone-cdx. Accessed June 19, 2021. Eisenhauer, 2009, New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1), Eur J Cancer, 45, 228, 10.1016/j.ejca.2008.10.026 Gimenez-Roqueplo, 2008, Recent advances in the genetics of phaeochromocytoma and functional paraganglioma, Clin Exp Pharmacol Physiol, 35, 376, 10.1111/j.1440-1681.2008.04881.x Murugan, 2011, Anaplastic thyroid cancers harbor novel oncogenic mutations of the ALK gene, Cancer Res, 71, 4403, 10.1158/0008-5472.CAN-10-4041 Mossé, 2008, Identification of ALK as a major familial neuroblastoma predisposition gene, Nature, 455, 930, 10.1038/nature07261 Choi, 2010, EML4-ALK mutations in lung cancer that confer resistance to ALK inhibitors, N Engl J Med, 363, 1734, 10.1056/NEJMoa1007478 Baglivo, 2018, Dramatic response to lorlatinib in a heavily pretreated lung adenocarcinoma patient harboring G1202R mutation and a synchronous novel R1192P ALK point mutation, J Thorac Oncol, 13, e145, 10.1016/j.jtho.2018.03.017 Bourdeaut, 2012, ALK germline mutations in patients with neuroblastoma: a rare and weakly penetrant syndrome, Eur J Hum Genet, 20, 291, 10.1038/ejhg.2011.195 Schönherr, 2011, Activating ALK mutations found in neuroblastoma are inhibited by Crizotinib and NVP-TAE684, Biochem J, 440, 405, 10.1042/BJ20101796 Bresler, 2014, ALK mutations confer differential oncogenic activation and sensitivity to ALK inhibition therapy in neuroblastoma, Cancer Cell, 26, 682, 10.1016/j.ccell.2014.09.019 Lu, 2017, The second-generation ALK inhibitor alectinib effectively induces apoptosis in human neuroblastoma cells and inhibits tumor growth in a TH-MYCN transgenic neuroblastoma mouse model, Cancer Lett, 400, 61, 10.1016/j.canlet.2017.04.022 Alam, 2019, Alectinib, an anaplastic lymphoma kinase inhibitor, abolishes ALK activity and growth in ALK-positive neuroblastoma Cells, Front Oncol, 9, 579, 10.3389/fonc.2019.00579 Siaw, 2016, Brigatinib, an anaplastic lymphoma kinase inhibitor, abrogates activity and growth in ALK-positive neuroblastoma cells, Drosophila and mice, Oncotarget, 7, 29011, 10.18632/oncotarget.8508 Amin, 2016, TKI sensitivity patterns of novel kinase-domain mutations suggest therapeutic opportunities for patients with resistant ALK+ tumors, Oncotarget, 7, 23715, 10.18632/oncotarget.8173