Androgens in childhood acquired aplastic anaemia in Chandigarh, India

This study assessed the efficacy of stanozolol in acquired aplastic anaemia (AA) in children, who were unable to opt for alternative therapies due to financial constraints. It uses a retrospective case-record analysis. Medical records of children with AA who received stanozolol were analysed. Their clinical and haematological profile was studied and the outcome assessed. Forty-nine children with acquired AA received stanozolol (1 mg/kg/day) in the period between January 1991 and December 2000. The median age at diagnosis was 9 years (range: 1.5-13). Thirteen (26.5%) defaulted therapy after periods of 1-4 weeks and 11 (22.4%) died within 2 months of starting treatment. Ten (20.4%) responded, whilst 15 (30.6%) did not. None of the patients with very severe AA responded, two (28.6%) with severe AA responded and eight (38%) with non-severe AA had a response. All responses were partial except one. Median time taken to respond was 11 weeks (range: 1-20). Responders were treated for a median duration of 25 weeks (range: 13-155), and they were followed-up for a median duration of 25 months (range: 3-144). Eight (80%) responders were off drugs for a median duration of 18 months (range: 6-110). Stanazolol is ineffective for severe and very severe AA. As it induced remission in 38% of our patients with non-severe AA, it can be tried in this group if other methods of treatment are unaffordable or have failed. A short history of presentation (< 3 months) and > or = 70% lymphoid cells in marrow correlated with poor prognosis.