BACKGROUND Purine nucleoside analogs (PNAs) are the recommended first-line treatment for patients with hairy cell leukemia (HCL), but they are associated with adverse events (AEs). Due to a lack of real-world evidence regarding AEs that are associated with PNAs, we used commercial data to assess AE rates, AE-related health care resource utilization (HCRU), and costs among PNA-treated patients with HCL. Adults aged ≥18 years with ≥2 claims for HCL ≥30 days apart from 1 January 2006 through 31 December 2015 were included. Included patients had ≥1 claim for HCL therapy (cladribine ± rituximab or pentostatin ± rituximab [index date: first claim date]) and continuous enrollment for a ≥ 6-month baseline and ≥ 12-month follow-up period. Patient sub-cohorts were based on the occurrence of myelosuppression and opportunistic infections (OIs). Generalized linear models were used to compare HCRU and costs. RESULTS In total, 647 PNA-treated patients were identified (mean age: 57.1 years). Myelosuppression and OI incidence were 461 and 42 per 1000 patient-years, respectively. Adjusted results indicated that those with myelosuppression had higher rates of hospitalization (47.4% vs 12.4%; P < .0001) and incurred higher mean inpatient costs ($23,517 vs $12,729; P = .011) and total costs ($57,325 vs $34,733; P = .001) as compared with those without myelosuppression. Similarly, patients with OIs had higher rates of hospitalization (53.8% vs 30.8%; P = .025) and incurred higher mean inpatient costs ($21,494 vs $11,229; P < .0001) as compared with those without OIs. CONCLUSIONS PNA therapy is highly effective but associated with significant toxicities that increase costs; these findings indicate a need for therapies with improved toxicity profiles and better risk stratification of patients at risk of developing myelosuppression and OIs.

中文翻译：

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嘌呤核苷类似物在毛细胞白血病患者中的不良事件发生率和经济负担：美国人群回顾性索赔分析。

背景技术嘌呤核苷类似物（PNAs）是毛细胞白血病（HCL）患者的推荐一线治疗方法，但它们与不良事件（AE）相关。由于缺乏与PNA相关的AE的真实证据，我们使用商业数据评估AE率，与AE相关的医疗资源利用率（HCRU）和PNA治疗的HCL患者的费用。自2006年1月1日至2015年12月31日，年龄在≥18岁且HCL≥30天的索赔要求≥2的成年人。纳入的患者具有≥1的HCL治疗要求（克拉屈滨±利妥昔单抗或喷司他丁±利妥昔单抗[索引日期：首次申领日期]）并且连续入组基线≥6个月且随访≥12个月。患者亚组基于骨髓抑制和机会性感染（OIs）的发生。使用广义线性模型比较HCRU和成本。结果总共鉴定出647名接受PNA治疗的患者（平均年龄：57.1岁）。骨髓抑制和OI发生率分别为每1000患者年461和42。调整后的结果表明，患有骨髓抑制的患者住院率更高（47.4％vs 12.4％; P <.0001），平均住院费用更高（$ 23,517 vs $ 12,729; P = .011）和总费用（$ 57,325 vs $ 34,733; P = .001）与没有骨髓抑制者比较。同样，与没有OI的患者相比，有OI的患者住院率更高（53.8％对30.8％； P = .025），平均住院费用更高（$ 21,494对$ 11,229； P <.0001）。结论PNA疗法是高度有效的，但伴随着明显的毒性，增加了费用。