Sorafenib maintenance after hematopoietic stem cell transplantation improves outcome of FLT3-ITD-mutated acute myeloid leukemia

Int J Hematol. 2022 Dec;116(6):883-891. doi: 10.1007/s12185-022-03427-4. Epub 2022 Aug 9.

Abstract

In a retrospective analysis, 21 acute myeloid leukemia patients receiving single-agent sorafenib maintenance therapy in complete remission (CR) after hematopoietic stem cell transplantation (HSCT) were compared with a control group of 22 patients without maintenance. Sorafenib was initiated a median of 3 months (IQR: 2.3-3.5) after allogeneic HSCT with a median daily dosage of 400 mg (range: 200-800) orally, and lasted a median of 11.3 months (IQR: 3.3-24.4). No significant increase in graft versus host disease or toxicity was observed. Adverse events were reversible with dose adjustment or temporary discontinuation in 19/19 cases. With a median follow-up of 34.7 months (IQR: 16.9-79.5), sorafenib maintenance significantly improved cumulative incidence of relapse (p = 0.028) as well as overall survival (OS) (p = 0.016), especially in patients undergoing allogeneic HSCT in CR1 (p < 0.001). In conclusion, sorafenib maintenance after allogeneic HSCT is safe and may improve cumulative incidence of relapse and OS in FLT3-ITD-mutated AML.

Keywords: Acute myeloid leukemia; FLT3–ITD mutation; Hematopoietic stem cell transplantation; Sorafenib maintenance.

MeSH terms

  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Humans
  • Leukemia, Myeloid, Acute* / drug therapy
  • Leukemia, Myeloid, Acute* / therapy
  • Mutation
  • Niacinamide / therapeutic use
  • Phenylurea Compounds / adverse effects
  • Recurrence
  • Retrospective Studies
  • Sorafenib / therapeutic use
  • Transplantation, Homologous
  • fms-Like Tyrosine Kinase 3 / genetics

Substances

  • Sorafenib
  • Phenylurea Compounds
  • Niacinamide
  • fms-Like Tyrosine Kinase 3
  • FLT3 protein, human