儿童特发性肺含铁血黄素沉着症的治疗：一项系统评价。
完整摘要 · 中文译文
背景：特发性肺含铁血黄素沉着症（IPH）是一种以儿童反复弥漫性肺泡出血为特征的罕见疾病。糖皮质激素是传统的一线治疗，免疫抑制剂也构成管理的支柱。然而，证据几乎完全来自小型观察性研究，尚无标准化建议。 方法：我们系统检索了PubMed、Embase、Web of Science、Cochrane对照试验中心注册库以及中文数据库（CNKI和万方），检索时间自建库至2026年9月24日，无语言限制。纳入报告儿童（0-18岁）IPH治疗的研究；进行了叙述性综合。 结果：共纳入149项研究。糖皮质激素是一线治疗，在最大的多中心队列（n = 567）中，494例IPH或病因未明儿童中有78.7%接受糖皮质激素单药治疗。在小型单中心队列中，总体复发率约为35%至60%，最常由呼吸道感染和糖皮质激素减量触发。免疫抑制剂，包括硫唑嘌呤、羟氯喹、环磷酰胺、来氟米特和6-巯基嘌呤，被用于难治性、激素依赖性或不缓解/复发性疾病。生物制剂如利妥昔单抗也作为有前景的减少激素用量选项出现。辅助治疗包括静脉注射免疫球蛋白、ECMO、动脉栓塞和铁螯合。在一个41例儿童的单中心IPH队列中，死亡率为27.3 %（随访22名儿童中的6名）。 结论：糖皮质激素仍是儿童特发性肺含铁血黄素沉着症（IPH）治疗的基石，免疫抑制剂或生物制剂保留用于难治性疾病。鉴于缺乏高质量证据，迫切需要采用标准化方案的多中心前瞻性研究。 系统综述注册：https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD420261486860，PROSPERO CRD420261486860。
完整摘要 · 英文原文
BACKGROUND: Idiopathic pulmonary hemosiderosis (IPH) is a rare disease characterized by recurrent diffuse alveolar hemorrhage in children. Corticosteroids are the conventional first-line treatment, and immunosuppressive agents also constitute a mainstay of management. However, evidence is derived almost entirely from small observational studies, and no standardized recommendations exist. METHODS: We systematically searched PubMed, Embase, Web of Science, the Cochrane Central Register of Controlled Trials, and Chinese databases (CNKI and Wanfang) from inception to 24 September 2026, with no language restrictions. Studies reporting treatment for IPH in children (0-18 years) were included; a narrative synthesis was performed. RESULTS: A total of 149 studies were included. Corticosteroids were the first-line treatment, with 78.7% of the 494 children with IPH or undetermined etiology in the largest multicenter cohort (n = 567) receiving corticosteroid monotherapy. The overall relapse rate ranged from approximately 35% to 60% across small single-center cohorts, most commonly triggered by respiratory infections and corticosteroid tapering. Immunosuppressive agents, including azathioprine, hydroxychloroquine, cyclophosphamide, leflunomide, and 6-mercaptopurine, were added for refractory, steroid-dependent, or relapsing disease. Biological agents such as rituximab also emerged as promising steroid-sparing options. Adjunctive therapies included intravenous immunoglobulin, ECMO, arterial embolization, and iron chelation. In a single-center IPH cohort of 41 children, mortality was 27.3% (6 of 22 children followed). CONCLUSIONS: Corticosteroids remain the cornerstone of pediatric IPH treatment, with immunosuppressive or biologic agents reserved for refractory disease. Given the absence of high-quality evidence, multicenter prospective studies with standardized protocols are urgently needed. SYSTEMATIC REVIEW REGISTRATION: https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD420261486860, PROSPERO CRD420261486860.