National Institute of Allergy and Infectious Diseases, Laboratory of Parasitic Diseases, National Institutes of Health, Bethesda, MD 20892, USA.
J Allergy Clin Immunol. 2009 Dec;124(6):1319-25.e3. doi: 10.1016/j.jaci.2009.09.022.
Hypereosinophilic syndrome (HES) is a heterogeneous group of rare disorders defined by persistent blood eosinophilia > or =1.5 x 10(9)/L, absence of a secondary cause, and evidence of eosinophil-associated pathology. With the exception of a recent multicenter trial of mepolizumab (anti-IL-5 mAb), published therapeutic experience has been restricted to case reports and small case series.
The purpose of the study was to collect and summarize baseline demographic, clinical, and laboratory characteristics in a large, diverse cohort of patients with HES and to review responses to treatment with conventional and novel therapies.
Clinical and laboratory data from 188 patients with HES, seen between January 2001 and December 2006 at 11 institutions in the United States and Europe, were collected retrospectively by chart review.
Eighteen of 161 patients (11%) tested were Fip1-like 1-platelet-derived growth factor receptor alpha (FIP1L1-PDGFRA) mutation-positive, and 29 of 168 patients tested (17%) had a demonstrable aberrant or clonal T-cell population. Corticosteroid monotherapy induced complete or partial responses at 1 month in 85% (120/141) of patients with most remaining on maintenance doses (median, 10 mg prednisone equivalent daily for 2 months to 20 years). Hydroxyurea and IFN-alpha (used in 64 and 46 patients, respectively) were also effective, but their use was limited by toxicity. Imatinib (used in 68 patients) was more effective in patients with the FIP1L1-PDGFRA mutation (88%) than in those without (23%; P < .001).
This study, the largest clinical analysis of patients with HES to date, not only provides useful information for clinicians but also should stimulate prospective trials to optimize treatment of HES.
嗜酸性粒细胞增多综合征（HES）是一组罕见疾病的异质性群体，其特征为持续性血嗜酸性粒细胞计数 >或=1.5 x 10(9)/L，无继发性原因，并且存在与嗜酸性粒细胞相关的病理学证据。除了最近一项关于美泊利单抗（抗 IL-5 mAb）的多中心试验外，已发表的治疗经验仅限于病例报告和小病例系列。
本研究的目的是收集和总结大量、多样化的 HES 患者的基线人口统计学、临床和实验室特征，并回顾常规和新型治疗方法的治疗反应。
通过病历回顾，收集了 2001 年 1 月至 2006 年 12 月期间在美国和欧洲的 11 家机构就诊的 188 例 HES 患者的临床和实验室数据。
在 161 例接受检测的患者中，有 18 例（11%）为 Fip1 样 1-血小板衍生生长因子受体α（Fip1L1-PDGFRA）突变阳性，在 168 例接受检测的患者中有 29 例（17%）存在可检测的异常或克隆 T 细胞群体。在 141 例患者中，有 85%（120/141）在 1 个月时接受皮质类固醇单药治疗后出现完全或部分缓解，大多数患者（中位数为 10 mg 泼尼松等效剂量，持续 2 个月至 20 年）仍在维持剂量。羟基脲和 IFN-α（分别用于 64 例和 46 例患者）也有效，但由于毒性限制了其使用。伊马替尼（用于 68 例患者）在 Fip1L1-PDGFRA 突变患者中的疗效（88%）优于无突变患者（23%；P <.001）。
这项迄今为止对 HES 患者进行的最大规模临床分析不仅为临床医生提供了有用的信息，而且还应刺激前瞻性试验来优化 HES 的治疗。
