Standard versus personalized schedule of regorafenib in metastatic gastrointestinal stromal tumors: a retrospective, multicenter, real-world study

ESMO Open

article

M. Nannini¹  A. Rizzo¹  M.C. Nigro¹  B. Vincenzi²  A. Mazzocca²  G. Grignani³  A. Merlini³  L. D’Ambrosio³  F. Tolomeo³  G. Badalamenti⁴  L. Incorvaia⁴  A. Bonasera⁴  E. Fumagalli⁵  D. Miliziano⁵  F. Ligorio⁵  A. Brunello⁶  B. Chiusole⁶  S. Gasperoni⁷  M. Novelli⁸  M.A. Pantaleo¹ 

[1] Division of Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna;Department of Oncology, University Campus Bio-Medico;Division of Medical Oncology, Candiolo Cancer Institute;Section of Medical Oncology, Department of Surgical, Oncological and Oral Sciences, University of Palermo;Adult Mesenchymal Tumour and Rare Cancer Medical Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori;Medical Oncology 1, Department of Oncology, Istituto Oncologico Veneto IOV - IRCCS;Translational Oncology Unit, Azienda Ospedaliero-Universitaria Careggi;Department of Statistical Sciences, University of Bologna;Department of Experimental, Diagnostic and Specialty Medicine, Sant’Orsola-Malpighi University Hospital

关键词: GIST;    regorafenib;    personalized treatment;    toxicity;    quality of life;   

DOI  :  10.1016/j.esmoop.2021.100222

学科分类：社会科学、人文和艺术（综合）

来源: BMJ Publishing Group

【 摘 要 】

Background Despite its proven activity as third-line treatment in gastrointestinal stromal tumors (GIST), regorafenib can present a poor tolerability profile which often leads to treatment modifications and transient or permanent discontinuation; thus, in clinical practice physicians usually adopt various dosing and interval schedules to counteract regorafenib-related adverse events and avoid treatment interruption. The aim of this real-world study was to investigate the efficacy and safety of personalized schedules of regorafenib in patients with metastatic GIST, in comparison with the standard schedule (160 mg daily, 3-weeks-on, 1-week-off).Patients and methods Institutional registries across seven Italian reference centers were retrospectively reviewed and data of interest retrieved to identify patients with GIST who had received regorafenib from February 2013 to January 2021. The Kaplan–Meier method was used to estimate survival and the log-rank test to make comparisons.Results Of a total of 152 patients with GIST, 49 were treated with standard dose, while 103 received personalized schedules. At a median follow-up of 36.5 months, median progression-free survival was 5.6 months [95% confidence interval (CI) 3.73-11.0 months] versus 9.7 months (95% CI 7.9-14.5 months) in the standard-dose and the personalized schedule groups, respectively [hazard ratio (HR) 0.51; 95% CI 0.34-0.75; P = 0.00052]. Median overall survival was 16.6 months (95% CI 14.1-21.8 months) versus 20.5 months (95% CI 15.0-25.4 months), respectively (HR 0.75; 95% CI 0.49-1.22; P = 0.16).Conclusions Regorafenib-personalized schedules are commonly adopted in daily clinical practice of high-volume GIST expert centers and correlate with significant improvement of therapeutic outcomes. Therefore, regorafenib treatment optimization in patients with GIST may represent the best strategy to maximize long-term therapy.

【 授权许可】

CC BY|CC BY-NC-ND