Long-Term Follow up of Children with Apalstic Anemia Treated with Immunosuppressive Therapy.

Background: Immunosuppressive therapy (IST) is the alternative first line treatment in children with aplastic anemia (AA) who have no HLA match siblings available. The long-term outcome of patients with AA who survive after IST is unknown. We evaluated outcomes of children with AA treated with IST at long-term follow up.

Methods: we retrospectively reviewed the hospital records of children with AA from 1984 to 2004, treated at our institution with horse-derived antithymocyte globulin (hATG) 160 mg/Kg over 4 days, short course of prednisone and cyclosporine (CS).

Results: Forty two patients were treated with IST (25 boys, 18 girls). The median age at diagnosis was 8.5 years. Twenty nine (69%), eight (19%) and five (12%) patients were diagnosed with severe, very severe, and moderate AA, respectively. Nine patients (21%) had hepatitis associated AA. Twenty seven patients (64%) received one course of ATG and fifteen (36%) received 2 courses (8 received 2 courses of hATG and 7 received 1 course hATG and 1 course rabbit derived ATG). Eleven patients (26%) required G-CSF. Median follow up time was 53.3 months (range 3–244 months). Twenty six patients (62%) had a complete response (CR), eight (19%) had a partial response (PR) and eight (19%) had no response (NR). Two patients relapsed after one course of IST and needed a second course of IST and both of them had a partial response. Median time to discontinuation of CS was 13 months. Nine patients (21%) died (7 with NR and 2 with PR) and 33 patients (79%) are alive. Two patients developed myelodysplastic syndrome (MDS) 21 and 19 months post IST; both received long-term G-CSF (18 and 14 months) and had PR after 2 courses of IST. Five of 33 patients (15%) who survived had significant hypertension after CS was discontinued and one required continuous antihypertensive medication.

Conclusion: The results of this study shows promising response in children with AA treated with IST. Hypertension and MDS are late complication. Longer follow up in these patients is warranted to definite the accurate rates of the late complications and risk factors.