Pediatric Pulmonology 13:176-180 (1992) zy Case Reports - Long-Term Prednisone and Azathioprine Treatment of a Patient With Idiopathic Pulmonary Hemosiderosis Giovanni A. Rossi, MD,~ Emma Balzano, MD,~ Elena Battistini, MD,~ Susanna Oddera, PhD,l Pier Giorgio Mori, MD* Patrizia Marchese, phD,2 Maura Acquila, Bruno Fregonese, MD,~ and INTRODUCTION zyxwvutsrq Idiopathic pulmonary hemosiderosis is an uncommon disorder of unknown etiology, occurring in the majority of patients in the first decade of life and manifested by iron deficiency anemia, recurrent or chronic pulmonary symptoms (including cough, hemoptysis, and dyspnea), and transient parenchymal infiltrates or a miliary pattern on chest radiography. I-’ This disorder is characterized morphologically by focal intraalveolar hemorrhage and thickening of the alveolar basement membrane with inter- stitial fibrosis.”‘ In contrast with many other pulmonary hemorrhagic syndromes, which are mediated by immune mechanisms, in idiopathic pulmonary hemosiderosis there zyxwvutsrqp is no evidence of anti-basement membrane antibod- ies or of immune complexes deposition in the lung.4.’ Therefore, although the association with cow milk al- lergy6 or celiac disease’ has been described in some pa- tients, and although a number of familial cases have been reported’, suggesting a genetic mechanism, the etiology of the repeated intraaveolar hemorrhage and of the fi- brotic changes is still unknown. Corticosteroids appear to be helpful in the management of the acute hemorrhagic episodes but, because of the intermittent nature of the disorder and the lack of randomized trials, the efficacy of this therapy in the long term (in combination or not with immunosuppressive agents’) is difficult to assess. We report the case of a girl in whom idiopathic pulmonary hemosiderosis was diagnosed at the age of 8 years and who has been treated for 15 years with different doses of prednisone and azathioprine. The results of this therapy are discussed based on clinical symptoms, on pulmonary function tests, and, lately, on bronchoalveolar lavage. CASE REPORT An 8-year-old white girl was referred for evaluation in January 1977, because of a 12 month history of hy- zyxwvu 0 1992 Wiley-Liss, Inc. pochromic anemia and recurrent episodes of shortness of breath. Physical examination of the chest revealed bilat- eral basilar rales without wheezing. Blood tests revealed an iron deficiency anemia with hemoglobin 4.5 g/lOO mL, hematocrit of 18% and striking hypochromia, micro- cytosis, and anisopoikilocytosis in peripheral blood smear. The white blood cell count and differential leuko- cyte counts were normal, the platelet count 350,000/ mm3, and reticulocyte count 20%. The serum iron level was 27 pg1100 mL, total bilirubin 1.4 mg/l00 mL, and the direct Coombs’ test was negative. Bilateral alveolar infiltrates were found on chest roentgenograph (Fig. 1). The results of pulmonary function studies were as fol- lows: forced expiratory volume in 1 second: (FEV,), 1.310 Lls (89% of predicted); forced vital capacity (FVC), 1.5 10 L (80% of predicted); FEV,/FVC, 86%.” Arterial blood gases at rest (breathing room air) were: oxygen partial pressure, (Pa,,,) 85 mmHg; carbon dioxide partial pressure, (Paco2) 35 mmHg. Tests for antinuclear antibodies (ANA), rheumatoid factors, and lupus erythe- matosus (LE) preparations were negative, serum immune complexes were absent, and tests of complement within normal limits. Iron-laden macrophages were demon- strated in the gastric juice and in the bronchial washing performed during bronchoscopy. No association with cow’s milk allergy was found. Treatment with pred- nisone, 1.5 mglkglday and azathioprine, 1.5 mglkglday From the Divisions of Pneumology’ and of Oncology and Hematol- ogy’, zyxwvuts G. Gaslini Institute, and the First Division of Pneumology’, San Martino Hospital, Genova, Italy. Received August 23. 1991; (revision) accepted for publication k b N - ary 27, 1992. Addrcss correspondence and reprint requests to Dr. G. A. Rossi, Division of Pneumology, lstituto G. Gaslini, Largo G. Gaslini. 4, 16148 Genovd, Italy.