TY - JOUR

T1 - Real-World Outcomes for 205 Patients with Chronic Lymphocytic Leukemia Treated with Ibrutinib

AU - Aarup, Kathrine

AU - Rotbain, Emelie Curovic

AU - Enggaard, Lisbeth

AU - Pedersen, Robert Schou

AU - Bergmann, Olav Jonas

AU - Thomsen, Rasmus Heje

AU - Frederiksen, Mikael

AU - Frederiksen, Henrik

AU - Nielsen, Tine

AU - Christiansen, Ilse

AU - Andersen, Michael Asger

AU - Niemann, Carsten Utoft

N1 - © 2020 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

PY - 2020/11/1

Y1 - 2020/11/1

N2 - Ibrutinib has now been approved for treatment of chronic lymphocytic leukemia (CLL) in both front-line setting and as later-line treatment. However, knowledge about the outcomes and adverse events (AE) among patients at a population-based level is still limited. Objectives: To report outcomes and AEs in a population-based cohort treated with ibrutinib outside clinical trials. Methods: We conducted a multicenter, retrospective cohort study including all patients with CLL treated with ibrutinib. Results: In total, 205 patients were included of whom 39 (19%) were treatment-naïve. The median follow-up was 21.4 months (interquartile range (IQR), 11.9,32.8), the estimated overall survival at 12 months was 88.8% (95% confidence interval (CI); 84.3%, 93.3%), and the estimated progression-free survival at 12 months was 86.3% (95% CI; 81.3%, 91.2%). During follow-up, 200 (97.6%) patients had at least one AE and 100 (48.8%) patients had at least one grade ≥3 AE. Eighty-six patients (42.0%) discontinued ibrutinib, hereof 47 (54.7%) due to AEs and 19 (22.1%) had progression of CLL or Richter transformation. Conclusions: In our study, we find comparable, though slightly inferior, overall, and progression-free survival, and discontinuation due to toxicity was higher compared with clinical trials. Patient training and information may improve treatment adherence outside clinical trials.

AB - Ibrutinib has now been approved for treatment of chronic lymphocytic leukemia (CLL) in both front-line setting and as later-line treatment. However, knowledge about the outcomes and adverse events (AE) among patients at a population-based level is still limited. Objectives: To report outcomes and AEs in a population-based cohort treated with ibrutinib outside clinical trials. Methods: We conducted a multicenter, retrospective cohort study including all patients with CLL treated with ibrutinib. Results: In total, 205 patients were included of whom 39 (19%) were treatment-naïve. The median follow-up was 21.4 months (interquartile range (IQR), 11.9,32.8), the estimated overall survival at 12 months was 88.8% (95% confidence interval (CI); 84.3%, 93.3%), and the estimated progression-free survival at 12 months was 86.3% (95% CI; 81.3%, 91.2%). During follow-up, 200 (97.6%) patients had at least one AE and 100 (48.8%) patients had at least one grade ≥3 AE. Eighty-six patients (42.0%) discontinued ibrutinib, hereof 47 (54.7%) due to AEs and 19 (22.1%) had progression of CLL or Richter transformation. Conclusions: In our study, we find comparable, though slightly inferior, overall, and progression-free survival, and discontinuation due to toxicity was higher compared with clinical trials. Patient training and information may improve treatment adherence outside clinical trials.

KW - chronic lymphocytic leukemia

KW - epidemiology

KW - targeted therapy

U2 - 10.1111/ejh.13499

DO - 10.1111/ejh.13499

M3 - Journal article

C2 - 32736410

VL - 105

SP - 646

EP - 654

JO - European Journal of Haematology

JF - European Journal of Haematology

SN - 0902-4441

IS - 5

ER -