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Sorafenib as a Salvage Therapy in FLT3-ITD Negative Relapse/ Refractory Acute Myeloid Leukemia

Nan Yang1, Zhenyang Gu1, Zhanxiang Liu1

  • 1Department of Hematology, Chinese People's Liberation Army General Hospital, Beijing, China.

Anti-Cancer Agents in Medicinal Chemistry
|March 10, 2018
PubMed
Abstract

Insights

Sorafenib shows promise as a salvage therapy for acute myeloid leukemia (AML) patients lacking the FLT3-ITD mutation. This multi-kinase inhibitor demonstrated effectiveness in two cases of refractory and relapsed AML.

Area of Science:

  • Oncology
  • Hematology
  • Pharmacology

Background:

  • Sorafenib effectively targets acute myeloid leukemia (AML) with FLT3-ITD mutations.
  • FLT3-ITD mutations are present in only 25% of AML cases, necessitating research into sorafenib's efficacy in other AML subtypes.
  • The therapeutic potential of sorafenib in AML patients without FLT3-ITD mutations requires further investigation.

Observation:

  • A case study involved a young AML patient with central nervous system (CNS) relapse treated with sorafenib and chemotherapy.
  • Another case involved a patient with refractory AML originating from chronic myelomonocytic leukemia (CMML), treated with sorafenib monotherapy.
  • Disease status was monitored using MRI, minimal residual disease (MRD) assays, and peripheral blood cell counts.

Findings:

  • The patient with CNS relapse showed significant tumor volume reduction.
  • The patient with refractory AML achieved hematological improvements.
  • These outcomes suggest sorafenib's potential efficacy in specific AML patient groups.

Implications:

  • Sorafenib may serve as a valuable salvage treatment option for refractory or relapsed AML patients who do not possess the FLT3-ITD mutation.
  • These findings warrant further clinical investigation into sorafenib's role in broader AML treatment strategies.
  • Expanding the use of sorafenib could offer new therapeutic avenues for AML patients with specific genetic profiles.
Keywords:
AMLCNS myeloid tumorFLT3-ITDSorafenibrefractoryrelapse.

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