High-dose methylprednisolone therapy in pure red cell aplasia

OBJECTIVE: To report our experience using high-dose methylprednisolone (HDMP) treatment in a patient with primary acquired pure red cell aplasia (PRCA) who failed to respond to conventional prednisone therapy. CASE SUMMARY: A 29-year-old woman reported weakness, was easily fatigued, and had developed palpitations. On physical examination, pallor and splenomegaly were detected. On blood smear, mild macrocytic anemia was seen. Bone marrow aspiration and biopsy revealed normocellularity, erythroid hypoplasia (E/M: 1/10), reduction in erythroid precursors, and normal megakaryocytes and myeloid series. No disease associated with secondary PRCA was detected. Oral prednisone 1 mg/kg (total 60 mg/d) was started as conventional treatment. However, the patient's status deteriorated and the hemoglobin concentration fell from 6.5 to 5.5 g/dL within the first week of hospitalization. HDMP was then begun. Treatment protocol consisted of methylprednisolone 30 mg/kg for 4 days, 20 mg/kg for 3 days, 10 mg/kg for 3 days, 5 mg/kg for 4 days, and 1 mg/kg for 2 weeks. The patient's hemoglobin concentration increased from 5.5 to 14.2 g/dL over a period of 9 weeks. Transient hyperglycemia and cushingoid appearance were seen during prednisone treatment. DISCUSSION: Exactly how steroids enhance erythropoiesis in PRCA is unknown. It seems likely that steroids render abnormal erythroid progenitors more sensitive to marrow growth factors, thereby permitting them to differentiate to functional precursors. HDMP treatment had been rarely used in patients with primary acquired PRCA. Limited studies using HDMP have shown variable results. CONCLUSIONS: HDMP treatment may be considered safe and effective in patients with primary acquired PRCA who do not respond to conventional steroid therapy.