Optimising maintenance therapy after transplantation: sorafenib's role in patients with FLT3-ITD acute myeloid leukaemia

In The Lancet Haematology, Li Xuan and colleagues 1 Xuan L Wang Y Yang K et al. Sorafenib maintenance after allogeneic haemopoietic stem-cell transplantation in patients with FLT3-ITD acute myeloid leukaemia: long-term follow-up of an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. 2023; 10: e600-e611 Google Scholar present the 5-year follow-up data of a randomised phase 3 study that assessed the effectiveness of sorafenib maintenance therapy versus non-maintenance after allogeneic haematopoietic stem-cell transplantation (HSCT) in patients aged 18–60 years with FLT3 internal tandem duplication (FLT3-ITD) acute myeloid leukaemia. 2 Xuan L Wang Y Huang F et al. Sorafenib maintenance in patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised phase 3 trial. Lancet Oncol. 2020; 21: 1201-1212 Summary Full Text Full Text PDF PubMed Scopus (168) Google Scholar Previous evidence from the randomised phase 2 SORMAIN trial showed the benefit of sorafenib maintenance compared with placebo in reducing the risk of relapse or death. 3 Burchert A Bug G Fritz LV et al. Sorafenib maintenance after allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia with FLT3-internal tandem duplication mutation (SORMAIN). J Clin Oncol. 2020; 38: 2993-3002 Crossref PubMed Scopus (248) Google Scholar However, the study by Xuan and colleagues 1 Xuan L Wang Y Yang K et al. Sorafenib maintenance after allogeneic haemopoietic stem-cell transplantation in patients with FLT3-ITD acute myeloid leukaemia: long-term follow-up of an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. 2023; 10: e600-e611 Google Scholar , 2 Xuan L Wang Y Huang F et al. Sorafenib maintenance in patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised phase 3 trial. Lancet Oncol. 2020; 21: 1201-1212 Summary Full Text Full Text PDF PubMed Scopus (168) Google Scholar provides new and clinically relevant evidence by not only showing the previously noted benefit in reduction of relapse risk but also an improvement in overall survival, which has not been previously shown in a phase 3 setting. Sorafenib maintenance after allogeneic haemopoietic stem-cell transplantation in patients with FLT3-ITD acute myeloid leukaemia: long-term follow-up of an open-label, multicentre, randomised, phase 3 trialWith extended follow-up, sorafenib maintenance after transplantation is associated with improved long-term survival and reduced relapse rates compared with non-maintenance, further supporting this strategy as a standard of care for patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic HSCT. Full-Text PDF Recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in patients with mild and moderate von Willebrand disease in the USA (VWDMin): a phase 3, open-label, randomised, crossover trialThese interim data suggest that recombinant VWF is not superior to tranexamic acid in reducing heavy menstrual bleeding in patients with mild or moderate von Willebrand disease. These findings support discussion of treatment options for heavy menstrual bleeding with patients based on their preferences and lived experience. Full-Text PDF