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IDH1-mutated relapsed or refractory AML: current challenges and future prospects.

Authors
  • Megías-Vericat, Juan Eduardo1
  • Ballesta-López, Octavio1
  • Barragán, Eva2, 3
  • Montesinos, Pau2, 3
  • 1 Servicio de Farmacia, Área del Medicamento, Hospital Universitari i Politècnic La Fe, Valencia, Spain. , (Spain)
  • 2 Servicio de Hematología y Hemoterapia, Hospital Universitari i Politècnic La Fe, Valencia, Spain. , (Spain)
  • 3 CIBERONC, Instituto Carlos III, Madrid, Spain. , (Spain)
Type
Published Article
Journal
Blood and lymphatic cancer : targets and therapy
Publication Date
Jan 01, 2019
Volume
9
Pages
19–32
Identifiers
DOI: 10.2147/BLCTT.S177913
PMID: 31413655
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

The prognosis of patients with relapsed or refractory acute myeloid leukemia (R/R AML) is discouraging with salvage standard approaches. Mutations of isocitrate dehydrogenase 1 (IDH1 mut ), present in 7-14% of AML patients, have been discovered recently, opening the door to targeted agents aiming to improve the outcomes in this setting. Several oral selective IDH1 mut inhibitors are under investigation, ivosidenib being the first approved for R/R AML. We performed a systematic review to analyze the clinical outcomes and safety reported with IDH1 mut inhibitors and other agents in adult patients with IDH1 mut R/R AML. Ivosidenib in monotherapy achieved complete remission (CR) of 24%, overall response of 42%, and median overall survival of 9 months in R/R AML, and promising outcomes were reported with IDH305 and FT-2102. IDH1 mut inhibitors were generally well tolerated, but some therapy-related toxicities should be monitored, including IDH-differentiation syndrome, prolongation of the QT interval, and leukocytosis, all manageable and reversible. Also, venetoclax, CB-839, PARP inhibitors, and IDH1 peptide vaccine are being studied in IDH1mut AML. The results of the ongoing and upcoming clinical trials will bring new evidence to establish the role of IDH1 mut inhibitors in therapeutic strategies of AML.

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