Abstract
The present study was done to determine the response and survival analysis in upfront hypomethylating agents-Venetoclax (HMA-Ven) regimen in Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) patients unfit for chemotherapy. This cross sectional study was conducted at National Institute of Blood Diseases and Bone Marrow Transplantation (NIBD-BMT), Karachi, Pakistan from January 2020 to December 2023. The primary endpoint was overall response rate (ORR) defined as Complete Remission (CR), Complete Count Recovery (CCR), Partial Count Recovery (PCR) with or without blasts. Secondary outcomes included median overall survival (OS) and event-free survival (FFS) analysed by Kaplan-Meier method. A total of 33 (22 AML and 11 MDS) patients were included. AML patients had median age of 62 and 48years was observed in MDS. After 4 cycles of HMA + Ven, ORR was reported as 93.7%. Median OS was 14 months, with deeper (CR vs. CCR; 20 vs. 14 months; p < 0.001) and quicker responses (achieved in 2 vs. 4 cycles; 14 vs. 11 months, p < 0.001) associated with improved OS. However, median EFS were 14 months. In MDS, ORR was 100%. Median OS and EFS were 11 and 10 months, respectively. Our study demonstrated the real-world applicability of HMA-Ven based regimen for unfit AML/MDS patients in low-middle income countries (LMIC) settings. Further national, multicentre, larger collaborative studies utilizing novel biomarkers are necessary to enhance risk assessment and treatment outcomes.