A phase 2 trial of sunitinib in patients with progressive paraganglioma or pheochromocytoma: the SNIPP trial

Grainne M. O’Kane, Shereen Ezzat, Anthony M. Joshua, Isabelle Bourdeau, Raya Leibowitz-Amit, Harold J. Olney, Monika Krzyzanowska, Dean Reuther, Soo Chin, Lisa Wang, Kelly Brooks, Aaron R. Hansen, Sylvia L. Asa, Jennifer J. Knox

Research output: Contribution to journalArticlepeer-review

Abstract

Background: Pheochromocytoma (PCC) and paraganglioma (PGL) are uncommon neoplasms with high morbidity in advanced stages. Effective systemic treatments are limited. Methods: A multisite phase 2 trial evaluated sunitinib in patients with progressive PCC/PGL. Patients received 50 mg orally for 4–6 weeks. Results: Between May 2009 and May 2016, 25 patients were enroled. The median age was 50 years and 56% were male. Three patients (12%) received prior chemotherapy and 16 (64%) prior surgery. The DCR was 83% (95% CI: 61–95%) and median PFS 13.4 (95% CI: 5.3–24.6) months. Of 23 evaluable patients, 3 (13%) with germline mutations (SDHA, SDHB, RET) achieved a PR. The patient with mutated RET and MEN2A remains on treatment after 64 cycles. The median time on treatment was 12.4 (1–88.0) months. Grade 3 or 4 toxicities were as expected and manageable; fatigue (16%) and thrombocytopenia (16%) were most common. One patient with grade 3 hypertension and 2 with grade 3 cardiac events discontinued treatment. Conclusion: Although the primary endpoint of disease control was met, the overall response rate of sunitinib was low in unselected patients with progressive PCC/PGL. Patients with germline variants in RET or in the subunits of SDH may derive greatest benefit.

Original languageEnglish
Pages (from-to)1113-1119
Number of pages7
JournalBritish Journal of Cancer
Volume120
Issue number12
DOIs
StatePublished - 11 Jun 2019
Externally publishedYes

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