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Research in Review

CLL Long-Term Survival Likely to Improve With Novel Therapies

July 26, 2017

Novel agents are predicted to significantly improve lifetime overall survival (OS) and quality-adjusted life-years (QALYs) compared with traditional therapies for patients with chronic lymphocytic leukemia (CLL), according to research published in Journal of Medical Economics (online July 19, 2017; doi:10.1080/13696998.2017.1357563).


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Most patients who are symptomatic of CLL and eligible for frontline treatment are unfit for fludarabine-based chemoimmunotherapy. While traditional treatment includes chlorambucil (Chl), bendamustine plus rituximab (BR), and a chlorambucil plus rituximab combination (ChlR), current clinical guidelines recommend novel agents—such as ibrutinib—in both frontline and relapse settings. Clinical results have been favorable for novel agents, but long-term follow-up data is lacking.

A group of British researchers led by Stuart Mealing, MSc, ICON Health Economics, conducted a study to evaluate long-term benefits of ibrutinib compared with traditional therapies for CLL. Researchers developed a simulation model to simulate treatment specific lifetime estimates of OS and QALYs for treatment with BR, Chl, ChlR, and ibrutinib. Multiple potential clinical scenarios were tested, including with and without access to novel agents for treating CLL. Researchers based their projections on health data relating to first- and second-line progression-free survival (PFS), post-progression survival, and mortality.

Researchers found treatment with ibrutinib to improve long-term outcomes in both clinical scenarios. When novel agents were unavailable, mean OS ranged from 5.4 years to 8.5 years and QALYs from 3.5 years to 6.1 years. Conversely, when novel agents were available, the mean OS increased to 10.0 years and QALYs to 7.6 years.

Additionally, frontline ibrutinib followed by physician’s choice therapy—including novel agents at relapse—resulted in an estimated OS increase between 1.5 years (18%) and 4.6 years (85%), corresponding to a 25% to 117% increase in QALYs, compared with traditional therapies.

Authors of the study concluded that the use of novel agents is predicted to yield substantial gains in predicted lifetime OS and QALY improvements compared with traditional therapies in patients with CLL who are ineligible for fludarabine-based chemoimmunotherapy.

Authors acknowledged limitations of the study, including immature OS data and the preemptive assumption of equivalent efficacy among all novel agents regarding their impact on PFS and OS. Further research is necessary to validate the claims of this study as long-term OS data continue to be made available.—Zachary Bessette

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