title: Androgens in childhood acquired aplastic anaemia in Chandigarh, India.
abstract: 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.
authors.affiliation: Division of Pediatric Hematology-Oncology, Advanced Pediatric Center, Chandigarh-160012, India. rammarwaha1@rediffmail.com
authors.forename: R K
authors.initials: RK
authors.lastname: Marwaha
authors.forename: Deepak
authors.initials: D
authors.lastname: Bansal
authors.forename: Amita
authors.initials: A
authors.lastname: Trehan
authors.forename: Neelam
authors.initials: N
authors.lastname: Varma
mesh_terms: D000293:Adolescent; D000728:Androgens / Q000627:therapeutic use*; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000453:epidemiology* / Q000209:etiology / Q000473:pathology; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007194:India / Q000453:epidemiology; D007223:Infant; D008297:Male; D008499:Medical Records; D012189:Retrospective Studies; D012720:Severity of Illness Index; D013197:Stanozolol / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D000728:Androgens; D013197:Stanozolol