Treatment of idiopathic pulmonary hemosiderosis in children: a systematic review

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.