Hairy Cell Leukemia Patients Have a Normal Life Expectancy—A 35-Year Single-Center Experience and Comparison with the General Population

SIMPLE SUMMARY: Classic hairy cell leukemia (HCL) is a rare, chronic B-cell malignancy with long-term remissions after standard treatment with purine analogues introduced in the 1990s. Although nearly all patiently ultimately relapse after up to 20 years from therapy requiring retreatment, overall survival prognosis remains insufficiently defined as life expectancyhas rarely been corrected for that of the general population. The aim of this single-center retrospective analysis was to report the long-term clinical outcomes of 83 consecutive HCL patients and compare overall survival with a matched cohort from the general population. We show that HCL patients may look forward to a normal lifespan when treated with purine analogues irrespectively of their pretreatment history, age at diagnosis, or whether they were treated until or after the year 2000. ABSTRACT: Classic hairy cell leukemia (HCL) is an uncommon hematologic malignancy characterized by an excellent prognosis since purine analogues (PA), such as cladribine (2-CdA), have been introduced in the 1990s. However, most data on long-term outcomes is gathered from patients treated with PA first-line or include limited information on previous treatment outcomes, i.e., Interferon-α (IFN-α). Survival curves from previous series did not reach a plateau, indicating that nearly all patients ultimately relapse. Yet, overall survival (OS) data were rarely corrected for life expectancy of the general population. We here report 83 consecutive HCL patients treated between 1983 and 2017 at the University Center in Innsbruck, Austria. Median follow-up was 170 months (1–498). IFN-α, the first-line treatment of choice before 1990, was administered to 24 patients, achieving an overall response rate (ORR) of 86% and an unconfirmed complete remission (CRu) in 23%. All these patients relapsed after a median progression-free survival (PFS) of 30 months (3–80), but either remained drug-sensitive upon re-exposure to IFN-α or were successfully salvaged with PA. All 42 patients exposed to first-line 2-CdA responded (ORR of 100%). Sixteen patients received two to four successive courses of PA with a continuous decrease in the response quality (CRu rate 85.7% 1st-line vs. 41.5% 3rd-line treatment). Median PFS was not reached in both treatment-naïve patients and those retreated at first relapse. Although pretreatment with IFN-α was associated with a shortened median PFS of 81 months (43–118) after PA therapy, this tendency of inferior PFS did not result in inferior OS. OS of all 83 patients was excellent and equivalent to that of age-, sex-, and diagnostic period-matched controls from the Tyrolean general population (standardized mortality ratio 0.8), regardless of their age at diagnosis or whether they were diagnosed until or after the year 2000. These results confirm that HCL patients may look forward to a normal lifespan when treated with PA irrespective of their pretreatment history.