Case report of ATRA and VAH regimen in refractory EVI1-positive AML

This publication is a case report with a brief literature review concerning a 42-year-old patient with EVI1-positive acute myeloid leukemia harboring the MLL-AF6 fusion gene. The patient had failed standard 'IA' induction therapy and was treated with all-trans retinoic acid combined with the VAH regimen (venetoclax, azacitidine, and homoharringtonine). The primary outcome was complete remission with incomplete hematologic recovery (CRh), which was achieved after 24 days of treatment. A secondary outcome was a marked reduction in EVI1 expression relative to baseline levels.

The authors note that the patient had an acceptable safety profile, but specific adverse events, serious adverse events, and discontinuations were not reported. The report synthesizes this single-patient experience and a brief review of the literature, suggesting promising efficacy for this combination in patients with EVI1-positive AML who are refractory to conventional chemotherapy.

Key limitations acknowledged by the authors include the need for further clinical studies to confirm its wider applicability. The practice relevance is restrained, as this is a single case and the findings are not generalizable. The certainty of the evidence is low, as indicated by the case report design.