Personalization of regorafenib treatment in metastatic gastrointestinal stromal tumours in real-life clinical practice

Margherita Nannini, Maria Concetta Nigro, Bruno Vincenzi, Elena Fumagalli, Giovanni Grignani, Lorenzo D’Ambrosio, Giuseppe Badalamenti, Lorena Incorvaia, Raffaella Bracci, Silvia Gasperoni, Maristella Saponara, Lidia Gatto, Valentina Indio, Annalisa Astolfi, Valerio Di Scioscio, Paolo G. Casali, Giuseppe Tonini, Massimo Aglietta, Antonio Russo, Guido BiascoMaria A. Pantaleo

Research output: Contribution to journalArticlepeer-review


Background: Regorafenib (REG) has now been approved as the standard third-line therapy in metastatic gastrointestinal stromal tumour (GIST) patients at the recommended dose and schedule of 160 mg once daily for the first 3 weeks of each 4-week cycle. However, it has a relevant toxicity profile that mainly occurs within the first cycles of therapy, and dose and schedule adjustments are often required to reduce the frequency or severity of adverse events and to avoid early treatment discontinuation. To date, large amounts of data on the use of REG in metastatic GIST patients in daily clinical practice are not available, and we lack information about how this treatment personalization really affects the quality of life (QoL) of patients. The aim of the present retrospective study is to build a comprehensive picture of all alternative REG strategies adopted in daily clinical practice for use in metastatic GIST patients. Methods: Metastatic GIST patients treated with dose adjustment or alternative schedules of REG at seven reference Italian centres were retrospectively included. Results: For a total of 62 metastatic GIST patients, we confirmed that REG treatment adjustment is common in clinical practice and that it is very heterogeneous, with approximately 20 different strategies being adopted. Independent of which strategy is chosen, treatment personalization has led to a clinical benefit defined as complete or partial resolution of side effects in almost all patients, affecting the duration of REG treatment. Conclusions: The personalization of REG, even if it is heterogeneous, seems to be crucial to maximize the overall treatment duration.

Original languageEnglish
Pages (from-to)731-739
Number of pages9
JournalTherapeutic Advances in Medical Oncology
Issue number12
Publication statusPublished - Dec 1 2017


  • GIST
  • personalized treatment
  • quality of life
  • referral centres
  • regorafenib
  • tyrosine kinase inhibitors

ASJC Scopus subject areas

  • Oncology


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