Idelalisib addition has neutral to beneficial effects on quality of life in bendamustine/rituximab-treated patients: Results of a phase 3, randomized, controlled trial

Marco Montillo, Árpád Illés, Tadeusz Robak, Alexander S. Pristupa, Malgorzata Wach, Miklós Egyed, Julio Delgado, Wojciech Jurczak, Franck Morschhauser, Anna Schuh, Herbert Eradat, Sanatan Shreay, Jacqueline C. Barrientos, Andrew D. Zelenetz

Research output: Contribution to journalArticle

Abstract

Background: In a phase 3 randomized, double-blind, placebo-controlled trial, treatment with idelalisib, a phosphoinositol-3 kinase δinhibitor, + bendamustine/rituximab improved progression-free survival (PFS) and overall survival (OS) in adult patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL). Here we report the results of health-related quality of life (HRQL) analyses from this study. Methods: From June 15, 2012 to August 21, 2014, 416 patients with R/R CLL were enrolled; 207 patients were randomized to the idelalisib arm and 209 to the placebo arm. In the 416 patients randomized to receive bendamustine/rituximab and either oral idelalisib 150 mg twice-daily or placebo, HRQL was assessed at baseline and throughout the blinded part of the study using the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) and EuroQoL Five-Dimension (EQ-5D) visual analogue scale (VAS) questionnaires. The assessments were performed at scheduled patient visits; every 4 weeks for the first 6 months from the initiation of treatment, then every 8 weeks for the next 6 months, and every 12 weeks thereafter until end of study. Least-squares mean changes from baseline were estimated using a mixed-effects model by including treatment, time, and treatment-by-time interaction, and stratification factors as fixed effects. Time to first symptom improvement was assessed by Kaplan-Meier analysis. Results: In mixed-effects model analysis, idelalisib + bendamustine/rituximab treatment led to clinically meaningful improvements from baseline in leukemia-associated symptoms. Moreover, per Kaplan-Meier analysis, the proportion of patients with symptom improvement was higher and time to improvement was shorter among patients in the idelalisib-containing arm compared with those who did not receive idelalisib. The physical and social/family FACT-Leu subscale scores, along with the self-rated health assessed by EQ-VAS, showed improvement with idelalisib over placebo, but the difference did not reach statistical significance. The functional and emotional FACT-Leu subscale scores remained similar to placebo. Conclusions: Addition of idelalisib to bendamustine/rituximab, apart from improving PFS and OS, had a neutral to beneficial impact on HRQL in patients with R/R CLL, particularly by reducing leukemia-specific disease symptoms.

Original languageEnglish
Article number173
JournalHealth and Quality of Life Outcomes
Volume17
Issue number1
DOIs
Publication statusPublished - Nov 15 2019

Keywords

  • Health-related quality of life
  • Idelalisib
  • Patient-related outcomes
  • Randomized phase 3 study
  • Relapsed/refractory CLL

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health

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    Montillo, M., Illés, Á., Robak, T., Pristupa, A. S., Wach, M., Egyed, M., Delgado, J., Jurczak, W., Morschhauser, F., Schuh, A., Eradat, H., Shreay, S., Barrientos, J. C., & Zelenetz, A. D. (2019). Idelalisib addition has neutral to beneficial effects on quality of life in bendamustine/rituximab-treated patients: Results of a phase 3, randomized, controlled trial. Health and Quality of Life Outcomes, 17(1), [173]. https://doi.org/10.1186/s12955-019-1232-8