Treatment of Pediatric Idiopathic Pulmonary Hemosiderosis with Low-Dose Cyclophosphamide

OBJECTIVE:To report the safety and efficacy of long-term, low-dose cyclophosphamide therapy in a child with idiopathic pulmonary hemosiderosis (IPH).CASE SUMMARY:A 7-year-old boy diagnosed with IPH 4 years previously was initially prescribed prednisolone. Because he only had a transient response to prednisolone, oral cyclophosphamide 2 mg/kg/d was later added. A dramatic improvement was noted during the subsequent follow-up. One year after cyclophosphamide therapy, the patient suddenly developed thrombocytopenia (platelet count 75 times 103/mm3), with the platelet count decreasing to 10 times 103/mm3 over the following 10 months. Cyclophosphamide was tapered to an alternating daily dosage of 1 mg/kg. The tapering resulted in a subsequent increase in the platelet count, which was maintained between 20 and 50 times 103/mm3 without occurrence of petechiae or spontaneous bleeding. Under this reduced dosing regimen, the disease has remained in remission for >1 year.DISCUSSION:Due to the low prevalence of IPH, ...