Patient-reported outcomes from a phase 2 study of copanlisib in patients with relapsed/refractory indolent B-cell non-Hodgkin lymphoma (iNHL).

e18123 Background: Information on the degree to which treatment of iNHL impacts health-related quality of life in terms of disease- and treatment-related symptoms is limited. Copanlisib is an investigational pan-Class I phosphatidylinositol 3-kinase (PI3K) inhibitor. In a phase II study in patients with relapsed/refractory iNHL, copanlisib treatment resulted in a high response rate and durable responses. We report here on patient-reported outcomes (PRO) from this trial (NCT01660451, part B). Methods: Patients with 4 subtypes of iNHL were eligible: follicular, marginal zone, small lymphocytic and lymphoplasmacytoid/Waldenstrӧm macroglobulinemia. Patients had to have received ≥2 prior lines of treatment, including rituximab and an alkylating agent. Copanlisib (60 mg, I.V.) was intermittently administered on days 1, 8 and 15 of a 28-day cycle. Health-related quality of life questionnaire FACT-Lym questionnaire (with focus on Total and Lymphoma subscale [FACT-LymS] and LOCF-method applied) was evaluated with Hodges-Lehmann estimate for change in median until week 16, including 95% confidence intervals. EQ-5D and visual analogue scale (EQ-5D VAS) were statistically described. All questionnaires were compiled at baseline and every other cycle in the first year of the study (aligned with radiological imaging). Results: A total of 141 iNHL patients were treated, with completed questionnaires received from 132 patients at baseline and 86 at week 16. The mean±SD (and median) for FACT-Lym LymS score at baseline was 45.27 ± 9.55 (46.5), and 47.20 ± 8.69 (49.0) at 16 weeks. The estimated change in median was +1.0 with 95% CI: [0.5; 2.5] and min: -13.0, max: 23.0, representing an improvement in lymphoma-related symptoms. Accordingly, the FACT-Lym total score was 126.59 ± 22.96 (127.5) at baseline and 127.99 ± 23.60 (130.8) at 16 weeks. Estimated change in median was 0.0 with 95% CI: [-0.5; 3.2] and min: -38.0, max 42.5. Conclusions: This study demonstrated the favorable impact of copanlisib treatment on disease-related symptoms and HRQoL in patients with relapsed/refractory iNHL.