Results at a single centre of immunosuppression with cyclosporine A in 66 children with aplastic anaemia.

A retrospective analysis of cyclosporine A (CsA) monotherapy administered to 66 children with aplastic anaemia (AA) at a single centre was carried out. The study was conducted on 66 children (F34/M32) with a median age of 10.5 years. 30 children (45%) achieved complete or partial remission within a median of 8 weeks. The response rate was 3/19 (16%) in cases of very severe aplastic anaemia (vSAA), 16/34 (47%) in severe aplastic anaemia (SAA) and 11/13 (85%) in nonSAA. 10 remitters (33%) relapsed after CsA cessation or dose tapering and six of those responded again on CsA alone. The only variable predictive for response was granulocyte count before treatment. The actuarial probability of survival was 51% at 4 years (85% in moderate AA, 50% in SAA and 32% in vSAA). The optimal dosage of CsA has yet to be established.