TY - JOUR
T1 - A phase 2 trial of sunitinib in patients with progressive paraganglioma or pheochromocytoma
T2 - the SNIPP trial
AU - O’Kane, Grainne M.
AU - Ezzat, Shereen
AU - Joshua, Anthony M.
AU - Bourdeau, Isabelle
AU - Leibowitz-Amit, Raya
AU - Olney, Harold J.
AU - Krzyzanowska, Monika
AU - Reuther, Dean
AU - Chin, Soo
AU - Wang, Lisa
AU - Brooks, Kelly
AU - Hansen, Aaron R.
AU - Asa, Sylvia L.
AU - Knox, Jennifer J.
N1 - Publisher Copyright:
© 2019, Cancer Research UK.
PY - 2019/6/11
Y1 - 2019/6/11
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85066079262&partnerID=8YFLogxK
U2 - 10.1038/s41416-019-0474-x
DO - 10.1038/s41416-019-0474-x
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C2 - 31105270
AN - SCOPUS:85066079262
SN - 0007-0920
VL - 120
SP - 1113
EP - 1119
JO - British Journal of Cancer
JF - British Journal of Cancer
IS - 12
ER -
