Case report: Venetoclax plus Azacitidine in treatment of acute undifferentiated leukemia.

Objectives: Acute undifferentiated leukemia (AUL) is a clinical rare leukemia with an overall poor prognosis. Currently, there are no well-established treatment guidelines for AUL, further exploration of optimal treatment options is now required.

Methods: We report an AUL patient who was complicated by a NRAS mutation and del5q was admitted to our hospital and we present the clinical features. In addition, we conducted a literature review.

Results: The "VA" scheme combines agents Venetoclax and Azacitidine that have synergistic therapeutic effect with a tolerable safety profile. There is no previous report of the "VA" scheme employed in AUL treatment. An AUL patient who was complicated by a NRAS mutation and del5q was admitted to our hospital. The "VA" scheme was administrated, and complete remission (CR) was achieved at the end of the first cycle. The patient then underwent HLA-identical sibling allogeneic hematopoietic stem cell transplantation.

Discussion: The "VA" scheme has been extensively used in AML treatment, but its application in AUL treatment has not yet been reported. This study is the first to report an AUL patient treated with the "VA" scheme and achieved CR. Our result preliminarily suggested the effectiveness and safety of the "VA" scheme in AUL treatment, but validation is required in more clinical samples. The "VA" scheme provides a new treatment option for AUL patients and deserves further clinical promotion.