Abstract / Summary
Background: Although chronic lymphocytic leukemia (CLL) typically follows an indolent course involving blood, lymph nodes, and bone marrow, extranodal organ involvement is rare and poorly characterized.
Methods: We retrospectively analyzed 19 patients with histologically confirmed extranodal CLL or small lymphocytic lymphoma (SLL), assessing organ involvement, clinical features, genetic risk factors, and response to therapy.
Results: Different organs were affected, most frequently the kidneys (7/19). Median age at initial CLL diagnosis was 54 years, median time from CLL diagnosis to extranodal manifestation was 3 years. Genetic risk factors were comparable to typical CLL cohorts. Per treatment episode, exploratory overall response rate (ORR) for Bruton tyrosine kinase inhibitors (BTKi) was 88.9% (8/9); median progression-free survival (PFS) from treatment initiation was 29 months. For venetoclax-based regimens, ORR was 83.3% (5/6) and median PFS 52 months.
Conclusions: These data suggest meaningful activity of BTK and BCL-2 inhibitors in CLL/SLL with extranodal organ involvement.