OBJECTIVES: Venetoclax, the first BCL-2 targeted therapy, was approved in China in Dec 2020 to treat newly diagnosed acute myeloid leukemia (AML) patients who are unfit for intensive chemotherapy. This study aims to investigate the real-world effectiveness and usage of venetoclax and associated difference in health care resource utilization (HCRU) in China.

METHODS: A two-stage nationwide survey was conducted. Convenience sampling was used to recruit hematologists with rich AML treatment and venetoclax experience. In stage one, 15 hematologists were recruited to participate in in-depth interviews using a semi-structured questionnaire. In stage two, 70 hematologists were invited to finish an online survey. Descriptive statistics and non-parametric median tests were conducted to summarize and compare results.

RESULTS: 85 questionnaires, representing >2,000 AML cases, were collected from 68 tertiary hospitals. Hematologists reported a similar rate of composite complete remission (CRc) to the VIALE-A trial of venetoclax. In the maintenance phase, venetoclax was given in the dosage of 400mg (46%), 200mg (28%), 100mg (20%) or 70mg (7%) per day. The most common reason for lower dosage was concomitantly taking azole antifungal medications (75%). 50% and 60% of patients took venetoclax less than 28 days per cycle before and after CRc. Considering the reduced dosage and shorter duration of taking venetoclax per cycle, the median venetoclax daily dose was 191mg.

Among patients achieving CRc, compared with low-intensity chemotherapy, the proportion of patients taking blood transfusion and anti-infection and the frequency of blood transfusion were reduced by 50% (P<0.05) in those taking venetoclax + azacitidine. The days of hospitalization per month was also significantly reduced by 30% (P<0.05).

CONCLUSIONS: Dose modifications aligned with the label of venetoclax were common in real-world practice. Despite this, a high CRc rate was observed. Additionally, venetoclax + azacitidine was associated with less HCRU particularly among patients achieving CRc.