Real‐world data on diagnostics, treatment and outcomes of patients with hairy cell leukemia: The HCL‐CLLEAR study

Abstract

AbstractHairy cell leukemia (HCL) and HCL‐like disorders have to be distinguished because of their different biology and treatment response. Thus, we conducted a retrospective study on patients with HCL and hairy cell leukemia variant (HCLv) to assess diagnostic algorithms and treatment outcomes in a real‐world setting. We analyzed 225 HCL and 26 HCLv patients with median follow‐up of 67.9 months (HCL) and 20.1 months (HCLv). Median age at diagnosis was 56.2 (HCL) and 69.5 years (HCLv), male predominance was observed in both groups (76.0% vs. 73.1%). Diagnostics was mostly based on morphological evidence of hairy cells in the peripheral blood and bone marrow. At diagnosis, BRAF V600E mutation was detected in 94.7% of examined HCL patients and in no HCLv patient. Front‐line treatment was indicated in 205 (91.1%) HCL and 18 (69.2%) HCLv patients. The majority of HCL patients were administered a cladribine‐based regimen (91.2%). Overall response rate (ORR) was higher in cladribine‐treated patients compared to those given other treatments (97.7% vs. 81.3%), the same applied with achieving Complete remission (CR) (91.2% vs. 62.5%). HCLv treatment was heterogeneous, but cladribine remained the most frequent option (44.4%) with ORR 81.3% and CR rates 43.8%. Second‐line treatment was indicated in 52 HCL and 8 HCLv patients, 25.4% and 44.4% of those treated in first‐line. In the whole HCL group, median time to next treatment (TTNT) was not reached and 10‐year TTNT was estimated at 74.1%. HCLv patients who underwent first‐line treatment had a median TTNT of 56 months. The median overall survival (OS) in HCL patients was not reached compared to HCLv with a median OS of 9.5 years. These data confirm an excellent prognosis for HCL patients treated with cladribine‐based therapy. On the contrary, HCLv with its aggressive behavior represents a group of patients in whom novel treatment approaches are needed.