Compassionate Use of Ripretinib for Patients With Metastatic Gastrointestinal Stromal Tumors: Taiwan and Hong Kong Experience

Li-Ching Lin; Wen-Kuan Huang; Chueh-Chuan Yen; Ching-Yao Yang; Meng-Ta Sung; Natalie S.M. Wong; Daniel T T Chua; Sarah W M Lee; Jen-Shi Chen; Chun-Nan Yeh

doi:10.3389/fonc.2022.883399

Compassionate Use of Ripretinib for Patients With Metastatic Gastrointestinal Stromal Tumors: Taiwan and Hong Kong Experience

Front Oncol. 2022 Jun 29:12:883399. doi: 10.3389/fonc.2022.883399. eCollection 2022.

Authors

Li-Ching Lin¹, Wen-Kuan Huang², Chueh-Chuan Yen^{3

4}, Ching-Yao Yang⁵, Meng-Ta Sung⁶, Natalie S.M. Wong⁷, Daniel T T Chua⁸, Sarah W M Lee⁹, Jen-Shi Chen², Chun-Nan Yeh¹

Affiliations

¹ Department of Surgery and GIST Team, Chang Gung Memorial Hospital at Linkou, Chang Gung University College of Medicine, Taoyuan, Taiwan.
² Division of Hematology-Oncology, Department of Internal Medicine and GIST Team, Chang Gung Memorial Hospital at Linkou, Chang Gung University College of Medicine, Taoyuan, Taiwan.
³ Division of Clinical Research, Department of Medical Research and Division of Medical Oncology, Center for Immuno-oncology, Department of Oncology, Taipei Veterans General Hospital, Taipei, Taiwan.
⁴ National Yang Ming Chiao Tung University School of Medicine, Taipei, Taiwan.
⁵ Department of Surgery, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, Taiwan.
⁶ Department of Oncology, Mennonite Christian Hospital, Haulien, Taiwan.
⁷ Department of Clinical Oncology, Tuen Mun Hospital, Hong Kong, Hong Kong SAR, China.
⁸ Department of Medicine, Hong Kong Sanatorium and Hospital, Hong Kong, Hong Kong SAR, China.
⁹ Department of Clinical Oncology, Pamela Youde Nethersole Eastern Hospital, Hong Kong, Hong Kong SAR, China.

Abstract

Background: Ripretinib was recently approved for the fourth-line targeted therapy for advanced gastrointestinal stromal tumor (GIST) refractory to imatinib, sunitinib, and regorafenib based on the pivotal INVICTUS phase III study. The INVICTUS study demonstrated significantly improved median progression-free survival (PFS) of 6.3 months and an overall survival (OS) insignificant benefit of ripretinib of 15.1 months as compared with placebo in 85 patients with advanced metastatic GIST. However, treatment outcome for the Chinese population, including in Taiwan and Hong Kong, was lacking.

Material and method: A compassionate study regarding ripretinib use for patients with advanced/metastatic GIST was conducted from March 2020 to March 2021 to assess the treatment efficacy and safety in Taiwan and Hong Kong patients.

Result: Twenty evaluable patients (16 men and 4 women) with heavily pretreated metastatic GIST receiving ripretinib from March 2020 to March 2021 were enrolled to evaluate the treatment outcome. The response and clinical benefit rates to ripretinib were 25% (5/20) and 60% (12/20), respectively. The median PFS and OS in this compassionate cohort receiving ripretinib were 6.1 months and not reachable, respectively. Albumin less than 3.5 and disease progression after ripretinib use were the two independent unfavorable factors for PFS. There were 14 out of 20 (70%) experiencing any grade adverse event (AE). Loss of hair is the most common grade I to II AE with an incidence of 55%. Grade III AEs included diarrhea, skin rash, and anemia with one patient (5%) for each AE.

Conclusions: Late-line ripretinib use in pretreated Taiwan and Hong Kong patients with advanced GIST showed efficacy consistent with the INVICTUS study. Albumin less than 3.5 and disease progression after ripretinib use were the two independent unfavorable factors for PFS. Ripretinib is generally tolerable, with loss of hair being the most common AE.

Keywords: GIST; advanced; compassionate use; metastatic; ripretinib.