Results of a Prospective Non-Interventional Post-Authorization Safety Study of Idelalisib in Germany

Idelalisib demonstrated robust effectiveness and manageable safety, regardless of high-risk features, in patients with chronic lymphocytic leukemia and relapsed follicular lymphoma in routine clinical practice in Germany. This non-interventional post-authorization study supports the effectiveness and tolerability profile of idelalisib previously obtained in clinical trials. Background: In pivotal studies, idelalisib demonstrated remarkable efficacy and manageable tolerability in patients with chronic lymphocytic leukemia (CLL) and follicular lymphoma (FL). This prospective, multicenter, non-interventional post -authorization study assessed the characteristics, clinical management, and outcome of CLL and FL patients receiving idelalisib in routine clinical practice in Germany. Patients: Observational study in CLL and FL patients treated with idelalisib between September 2015 and December 2020. Results: A total of 147 patients with CLL and FL were included with a median age of 75 and 71 years, respectively. More than 80% of patients presented with comorbidity and many CLL patients with documented high-risk genetic features, including del(17p)/TP53 mutation or unmutated IGHV. The median progression-free survival (PFS) and overall survival (OS) were not reached in the CLL cohort irrespective of del(17p)/TP53 or unmutated IGHV. The estimated 6-month PFS and OS rates in CLL were 82% and 92%. The estimated 6-month PFS and OS rates for FL were 32.2% and 77.2%. Overall response rates in the CLL and FL cohorts were 70.4% and 36.4%, with the presence of high-risk genetics having no negative impact. No unexpected adverse events were observed. Most frequently reported adverse drug reactions (ADRs) were diarrhea, nausea, pneumonia, rash, and fatigue. Conclusion: This real-world study shows that idelalisib is an effective therapy for CLL and FL, regardless of age and high-risk genetic features, consistent with results from previous clinical trials . Collected safety data and the pattern of ADRs reflect those from previous studies.