Department of Pharmacy, Mayo Clinic, Rochester, MN, USA.
Department of Pharmacy, Northwestern Memorial Hospital, Chicago, IL, USA.
Leuk Lymphoma. 2020 Jul;61(7):1592-1600. doi: 10.1080/10428194.2020.1737684. Epub 2020 Mar 11.
Hemophagocytic lymphohistiocytosis (HLH) is a rare syndrome of pathologic immune activation in children that is increasingly being recognized in adults. Efficacy data for the HLH-04 protocol in adults is lacking. This study retrospectively analyzed 31 adult patients, median age 46 years, who received HLH-04 from 1/1/2004 to 5/1/2018. HLH etiology included malignancy ( = 9), autoimmune ( = 8), infection ( = 8), and idiopathic ( = 6). Eighteen patients were evaluable for response at week 4 with 7 having no response, 11 reaching partial response, and 0 reaching complete response (CR). Six patients eventually achieved CR at a median 195 days. The 1-year overall survival (OS) was 35% and median OS was 3.2 months. Univariate analysis showed shorter survival for hemoglobin <9 g/dL (HR 4.29, = 0.003), platelets <100 × 10/L (HR 4.06, = 0.027), ANC <1 × 10/L (HR 5.24, = 0.001), and total bilirubin >1.2 mg/dL (HR 3.30, = 0.022). Outcomes of adults treated with HLH-04 remain dismal and newer treatment modalities are needed.
噬血细胞性淋巴组织细胞增生症（HLH）是一种罕见的儿童病理性免疫激活综合征，在成人中越来越被认识。HLH-04 方案在成人中的疗效数据缺乏。本研究回顾性分析了 31 例成年患者，中位年龄 46 岁，自 2004 年 1 月 1 日至 2018 年 5 月 1 日接受 HLH-04 治疗。HLH 的病因包括恶性肿瘤（ = 9）、自身免疫性疾病（ = 8）、感染（ = 8）和特发性（ = 6）。18 例患者在第 4 周时可评估反应，其中 7 例无反应，11 例部分缓解，0 例完全缓解（CR）。6 例患者最终在中位 195 天后达到 CR。1 年总生存率（OS）为 35%，中位 OS 为 3.2 个月。单因素分析显示血红蛋白 <9 g/dL（HR 4.29， = 0.003）、血小板 <100×10/L（HR 4.06， = 0.027）、中性粒细胞计数 <1×10/L（HR 5.24， = 0.001）和总胆红素 >1.2 mg/dL（HR 3.30， = 0.022）的患者生存期更短。接受 HLH-04 治疗的成年人的结局仍然不佳，需要新的治疗方法。
