Study objectives: Previously, IPH patients have been reported to have an average survival of 2.5 years. However, at our institution, many IPH patients have survived longer than that. Therefore, we conducted this study to determine the clinical course and current mortality of pediatric IPH patients treated with immunosuppressants.
Design: Retrospective chart review.
Setting: Children's hospital.
Participants: Seventeen patients in whom IPH was diagnosed between 1972 and 1998.
Measurements and results: Mean age at diagnosis was 4.5 +/- 3.5 years, and 12 patients were female. At diagnosis, all patients had anemia and pulmonary infiltrates; 85% had hypoxemia, 65% had hemoptysis, and 70% had fever. The diagnosis was made by open lung biopsy in 13 patients (76%), hemosiderin-laden macrophages in BAL fluid in 1 patient (6%), hemosiderin-laden macrophages in gastric aspirate in 2 patients (12%), or by clinical presentation alone in 1 patient (6%). The mean duration of follow-up for all patients was 3.6 +/- 3.4 years (range, 0.7 to 10.2). Initial treatment consisted of prednisone only in 14 patients (82%), and prednisone and hydroxychloroquine in two patients (12%). Thirteen patients (76%) required long-term corticosteroids because of recurrent hemoptysis. Eight patients (47%) required other immunosuppressants (hydroxychloroquine or azathioprine) in addition to prednisone to control their hemoptysis. One patient who was not treated with prednisone remained asymptomatic for 1.8 years. Three patients (17%) died of acute massive pulmonary hemorrhage (4.1 +/- 5.0 years postdiagnosis).
Conclusion: Five-year survival for IPH patients in our study was 86% (by Kaplan-Meier method). We conclude that these IPH patients who received long-term treatment had a better outcome than those previously reported who were not treated with extended courses of immunosuppressive therapy. We speculate that long-term immunosuppression therapy may improve the prognosis in IPH.