Hypereosinophilic syndrome: a multicenter, retrospective analysis of clinical characteristics and response to therapy.
Hypereosinophilic syndrome: a multicenter, retrospective analysis of clinical characteristics and response to therapy.
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DOI：
10.1016/j.jaci.2009.09.022
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发表时间：
2009-12
影响因子：
14.2
通讯作者：
Klion, Amy D.
中科院分区：
文献类型：
--
作者：
Ogbogu, Princess U.;Bochner, Bruce S.;Butterfield, Joseph H.;Gleich, Gerald J.;Huss-Marp, Johannes;Kahn, Jean Emmanuel;Leiferman, Kristin M.;Nutman, Thomas B.;Pfab, Florian;Ring, Johannes;Rothenberg, Marc E.;Roufosse, Florence;Sajous, Marie-Helene;Sheikh, Javed;Simon, Dagmar;Simon, Hans-Uwe;Stein, Miguel L.;Wardlaw, Andrew;Weller, Peter F.;Klion, Amy D.
嗜酸性粒细胞增多综合征 (HES) 是一组异质性罕见疾病，其定义为持续性血液嗜酸性粒细胞增多≥1.5 × 109/L、缺乏继发原因以及嗜酸性粒细胞相关病理证据。除了最近的美泊利单抗（抗 IL-5 单克隆抗体）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。该研究的目的是收集和总结大量不同 HES 患者的基线人口统计学、临床和实验室特征，并审查对传统和新型疗法治疗的反应。通过图表审查回顾性收集了 2001 年 1 月至 2006 年 12 月期间在美国和欧洲 11 个机构观察到的 188 名 HES 患者的临床和实验室数据。接受测试的 161 名患者中有 18 名 (11%) 呈 FIP1L1-PDGFRA 突变阳性，29/168 名接受测试的患者 (17%) 具有明显的异常或克隆性 T 细胞群。皮质类固醇单一疗法在 1 个月时在 85% (120/141) 的患者中诱导完全或部分缓解，其中大多数患者仍维持维持剂量（中位 10 mg 强的松当量，每日 2 个月至 20 年）。羟基脲和干扰素-α（分别用于 64 名和 46 名患者）也有效，但其使用受到毒性的限制。伊马替尼（用于 68 名患者）对 FIP1L1-PDGFRA 突变患者 (88%) 比无 FIP1L1-PDGFRA 突变患者 (23%；p<0.001) 更有效。这项研究是迄今为止对 HES 患者最大规模的临床分析，不仅为临床医生提供了有用的信息，而且应该激发前瞻性试验来优化 HES 的治疗。
Hypereosinophilic syndromes (HES) are a heterogeneous group of rare disorders defined by persistent blood eosinophilia ≥1.5 × 109/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 monoclonal antibody), 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 eleven institutions in the United States and Europe, were collected retrospectively by chart review. Eighteen of 161 patients (11%) tested were FIP1L1-PDGFRA mutation-positive and 29/168 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-20 years). Hydroxyurea and interferon-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<0.001). This study, the largest clinical analysis of patients with HES to date, not only provides useful information for clinicians but should stimulate prospective trials to optimize treatment of HES.
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影响因子：
2.7
作者：
Vaklavas, C.;Tefferi, A.;Pardanani, A.
通讯作者：
Pardanani, A.
影响因子：
14.2
作者：
Stein, Miguel L.;Villanueva, Joyce M.;Rothenberg, Marc E.
通讯作者：
Rothenberg, Marc E.
影响因子：
158.5
作者：
Rothenberg, Marc E.;Klion, Amy D.;Gleich, Gerald J.
通讯作者：
Gleich, Gerald J.
影响因子：
11.4
作者：
Roche-Lestienne, C;Lepers, S;Preudhomme, C
通讯作者：
Preudhomme, C
影响因子：
158.5
作者：
Simon, HU;Plötz, SG;Blaser, K
通讯作者：
Blaser, K