Gilteritinib (XOSPATA®) in Turkey: Early Access Program Results

Dogu M. H., Emre Tekgunduz A. I., Deveci B., Korkmaz G., Comert M., SEVİNDİK Ö. G., ...More

Mediterranean Journal of Hematology and Infectious Diseases, vol.15, no.1, 2023 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 15 Issue: 1
  • Publication Date: 2023
  • Doi Number: 10.4084/mjhid.2023.031
  • Journal Name: Mediterranean Journal of Hematology and Infectious Diseases
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, EMBASE, Directory of Open Access Journals
  • Keywords: Gilteritinib, Acute myeloid leukemia (AML), Early access, Real-life data, Response, Prognosis
  • Istanbul Medipol University Affiliated: Yes


Background And Objectives: Gilteritinib (XOSPATA ®, Astellas) is a type I oral FLT3 inhibitor, a tyrosine kinase AXL inhibitor, involved in both c-Kit and FMS-like tyrosine kinase 3 (FLT3) resistance. In the phase 3 ADMIRAL trial, gilteritinib was compared with the standard of care in (R/R) acute myeloid leukemia (AML) patients who harbored any FLT3 mutation and showed superior efficacy with regard to response and survival. Objectives: This research aimed to investigate the real-life efficacy and safety of gilteritinib in FLT3-positive R/R AML patients who were treated as a part of an early access program held in Turkey in April 2020 (NCT03409081). Results: The research included 17 R/R AML patients who had received gilteritinib from seven centers. The overall response rate was 100%. The most common adverse events were anemia and hypokalemia (7 patients, 41.2%). Grade 4 thrombocytopenia was observed in one patient only (5.9%), leading to permanent treatment discontinuation. Patients with peripheral edema had a 10.47 (95% CI: 1.64-66.82) times higher risk of death than those without peripheral edema (p<0.05). Conclusion: This research showed that patients with febrile neutropenia and peripheral edema were at a high risk of death when compared to patients without febrile neutropenia and peripheral edema.