Bohn Jan-Paul, Neururer Sabrina, Pirklbauer Markus, Pircher Andreas, Wolf Dominik
Department of Internal Medicine V, Hematology and Oncology, Medical University of Innsbruck, A-6020 Innsbruck, Austria.
Department of Medical Statistics, Informatics and Health Economics, Medical University of Innsbruck, A-6020 Innsbruck, Austria.
Cancers (Basel). 2022 Feb 28;14(5):1242. doi: 10.3390/cancers14051242.
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.
经典毛细胞白血病（HCL）是一种罕见的血液系统恶性肿瘤，自20世纪90年代引入嘌呤类似物（PA），如克拉屈滨（2-CdA）以来，其预后良好。然而，大多数关于长期预后的数据是从接受PA一线治疗的患者中收集的，或者包含的既往治疗结果信息有限，即干扰素-α（IFN-α）治疗结果。既往系列研究的生存曲线未达到平台期，这表明几乎所有患者最终都会复发。然而，总体生存（OS）数据很少根据一般人群的预期寿命进行校正。我们在此报告了1983年至2017年期间在奥地利因斯布鲁克大学中心接受治疗的83例连续HCL患者。中位随访时间为170个月（1 - 498个月）。1990年前的一线治疗选择IFN-α，应用于24例患者，总缓解率（ORR）为86%，未确认的完全缓解（CRu）率为23%。所有这些患者在中位无进展生存期（PFS）30个月（3 - 80个月）后复发，但再次接受IFN-α治疗时仍对药物敏感，或成功接受PA挽救治疗。所有42例接受一线2-CdA治疗的患者均有反应（ORR为100%）。16例患者接受了两到四个连续疗程的PA治疗，缓解质量持续下降（CRu率一线治疗为85.7%，三线治疗为41.5%）。初治患者和首次复发后接受再治疗的患者均未达到中位PFS。虽然PA治疗前使用IFN-α与PA治疗后中位PFS缩短至81个月（43 - 118个月）相关，但这种PFS较差的趋势并未导致OS较差。83例患者的OS均良好，与来自蒂罗尔一般人群的年龄、性别和诊断期匹配的对照组相当（标准化死亡率为0.8），无论其诊断时的年龄或是否在2000年之前或之后被诊断。这些结果证实，无论预处理史如何，HCL患者接受PA治疗后可能期待正常寿命。
