A physician survey reveals differences in management of idiopathic pulmonary hemosiderosis

作者：Chin Chana I C; Kohn Shirleen Loloyan^*; Keens Thomas G; Margetis Monique F; Kato Roberta M

来源：Orphanet Journal of Rare Diseases, 2015, 10(1): 98.

DOI：10.1186/s13023-015-0319-5

• Interstitial lung disease
• Lung pathology
• Pulmonary vascular disorders
• Vasculitis
• Idiopathic pulmonary hemosiderosis
• Diffuse alveolar hemorrhage
• Capilliritis
• Lung biopsy

Background: Idiopathic pulmonary hemosiderosis (IPH) is a rare disorder of unknown etiology characterized by chronic pulmonary hemorrhage and presents with a triad of anemia, hemoptysis and pulmonary infiltrates. IPH is a diagnosis of exclusion with a variable and disparate clinical course. Despite existing therapies, few children achieve full remission while others have recurrent hemorrhage, progressive lung damage, and premature death.
Methods: We surveyed physicians who care for patients with IPH via a web-based survey to assess the most common practices. 88 providers responded, caring for 274 IPH patients from five continents.
Results: 63.3 % of respondents had patients that were initially misdiagnosed with anemia (60.0 %) or gastrointestinal bleed (18.2 %). Respondents varied in diagnostic tools used for evaluation. The key difference was in the use of lung biopsy (51.9 %) for diagnosis. Common medications respondents used for treatment at initial presentation and chronic maintenance therapy were corticosteroids (98.7 and 84.0 %, initial and chronic therapy respectively), hydroxychloroquine (33.3 and 64.0 %), azathioprine (8.0 and 37.3 %), and cyclophosphamide (4.0 and 16.0 %). There was agreement on the use of corticosteroids for exacerbation amongst all respondents. Reported deaths before adulthood occurred in 7.3 % of patients. We conclude that there were common features and specific variations in physician management of IPH. Respondents were divided on whether to perform lung biopsy for diagnosis.
Conclusion: Despite the availability of various immunomodulators, corticosteroids remained the primary therapy. We speculate that the standardization of care for diffuse alveolar hemorrhage will improve patient outcomes.

• 出版日期2015-8-20