All-trans retinoic acid combined with the VAH regimen for EVI1-positive acute myeloid leukemia: a case report with a brief literature review

EVI1 positivity is a well-recognized high-risk factor in acute myeloid leukemia (AML), typically characterized by complex molecular genetic abnormalities, suboptimal responses to conventional chemotherapy, and a poor prognosis. We report a case of a 42-year-old patient with EVI1-positive AML harboring the MLL-AF6 fusion gene, who failed to achieve remission after undergoing standard "IA" induction therapy and was then treated with VAH (venetoclax, azacitidine, and homoharringtonine) consolidation chemotherapy. The patient subsequently received all-trans retinoic acid (ATRA) (20 mg twice daily, administered orally) and achieved complete remission with incomplete hematologic recovery (CRh) after 24 days, accompanied by a marked reduction in EVI1 expression relative to baseline levels. Following this, consolidation therapy consisting of one cycle of "VAH plus ATRA" led to complete remission (CR). This case suggests that the combination of ATRA with the VAH regimen may demonstrate promising efficacy and an acceptable safety profile in patients with EVI1-positive AML who are refractory to conventional chemotherapy. However, further clinical studies are required to confirm its wider applicability.