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Outcomes of Venetoclax Treatment for CLL Patients

In science and medicine, information is constantly changing and may become out-of-date as new data emerge. All articles and interviews are informational only, should never be considered medical advice, and should never be acted on without review with your health care team.

Medically reviewed by Dr. Brian Koffman

The Bottom Line:

Venetoclax is a very effective treatment for chronic lymphocytic leukemia (CLL) in patients with no prior exposure to BTK inhibitors (BTKi). Still, it is less effective for patients whose disease has progressed on prior BTKi therapy.

Who Performed the Research and Where Was it Presented:

Dr. Paul Hampel and colleagues from the Mayo Clinic presented the results at the American Society for Hematology (ASH) Annual Meeting 2023.


Venetoclax is a BCL2 inhibitor that has been shown to be a very effective treatment for CLL / SLL in clinical trials both as a first-line treatment and for relapsed / refractory disease. However, in the real world, patient outcomes can differ from those seen in clinical trials partially due to the eligibility criteria used to select patients for clinical trials. This study looked at outcomes for CLL patients treated with venetoclax in routine clinical practice.

Methods and Participants:

This study used data from the Mayo Clinic CLL Database and included patients who received venetoclax therapy for CLL between 2021 and 2023. Patients were divided into three categories based on their treatment status:

  1. Venetoclax as a first-line therapy (n=55)
  2. Venetoclax for relapsed / refractory CLL with no previous exposure to BTKi (n=17)
  3. Venetoclax for relapsed / refractory CLL and prior treatment with BTKi (n=83)

One of the primary outcomes that researchers measured was treatment-free survival (the amount of time before patients needed another treatment or died). Treatment-free survival was used as a surrogate for progression-free survival.


  • Outcomes for the first line and relapsed with no previous BTKi groups were very good and similar to what was previously seen in clinical trials.
  • Two years after starting venetoclax, 91% of patients in the first-line therapy group were treatment-free, and 73% of the patients in the relapsed with no previous BTKi group were treatment-free.
  • Overall survival after two years was 91% in the first-line therapy group and 100% in the relapsed with no previous BTKi group.
  • Outcomes for patients in the relapsed with previous BTKi group were not as good, with only half of patients remaining treatment-free after two years.
  • Patients whose disease had progressed on previous BTKi therapy had a median treatment-free survival time of 2 years, which was much shorter than patients who had stopped previous BTKi therapy due to toxicity (about 3.5 years).
  • Complex karyotype (defined as more than three chromosomal aberrations) was associated with shorter treatment-free survival.


Venetoclax was very effective for CLL patients with no prior BTKi exposure. However, it was less effective for patients whose disease had progressed on prior BTKi therapy. This highlights the importance of understanding how the sequencing of different treatments may affect patient outcomes and reminds us of the need for more possible therapies for patients who may run out of good, approved options.

Links and Resources:

Watch the interview on the abstract here:

Clinical Outcomes of Venetoclax Treatment in CLL Patients – Dr. Paul Hampel ASH 2023

You can read the actual ASH abstract: Clinical Outcomes with Venetoclax-Based Treatment Regimens in Patients with Chronic Lymphocytic Leukemia (CLL)

Take care of yourself first.

Ann Liu, PhD