Survival After Immunosuppressive Therapy in Children with Aplastic Anemia.

ABSTRACT

Objective: To determine the survival of children ≤18y, treated with immunosuppresive therapy (IST) using equine antithymocyte globulin (e-ATG) and cyclosporine (CsA). Design: Prospective data entry as per a specified format. Setting: Tertiary care hospital. Patients From January 1998 to December 2009, 40 children were diagnosed with acquired aplastic anemia; 33 patients, who received IST, were analyzed. 31 children (94%) received one course of e-ATG and CsA. 2 patients (6%) received two courses of ATG. Intervention Immunosuppressive therapy using equine ATG and cyclosporine. Main Outcome Measures: Overall response and overall survival. Results: The overall response (complete response + partial response) to IST at 6 months was 87.9%. 8 (24.2%) patients achieved CR, 21 (63.6%) patients had PR and 4 (12.1%) patients did not respond to IST. Median follow-up was 24 (6-102) months. Overall survival at 24 months was 90%, with an acturial survival of 85.4% at 5 years. Seventeen patients (51.5%) received G-CSF for a median duration of 32 (23-64) days. The patients who received G-CSF had fewer infectious complications (P=0.002), but G-CSF administration did not influence survival/ outcome. No patient developed myelodysplastic syndrome or acute leukemia. Conclusions: The survival of patients who respond to IST is excellent. Also, G-CSF reduces the infectious complications without conferring any survival advantage.