Piracetam for the treatment of sickle cell disease in children- a double blind test.

Sickle cell disease (SCD) occurs frequently in the Saudi population and is often associated with high morbidity and disabilitating complications. The search for drugs to ameliorate the clinical presentation of SCD has been going on for the last 3 decades. Aim: The objective of this investigation was to study the effect of piracetam on the clinical presentation, hematological and relevant biochemical parameters in children of two different age groups suffering from severe SCD. Study design: A double-blind, placebo controlled, randomised multicentre trial conducted in different regions of Saudi Arabia. Patients and methods: Children (3-12 years) suffering from severe SCD as judged from severity index of 6 or more were enrolled in the study. One hundred and one children were included of whom 87 (79 SCA and 8S beta degrees thal) completed the one year treatment protocol. The drug/placebo was received from UCB Company in coded boxes and administered as intravenous infusion during crises (300 mg/kg/day) and orally 160 mg/kg/day) during follow-up period. The base line clinical data was recorded and hematological and biochemical parameters were assessed. The patients were treated and followed-up for a one year period and the follow-up was conducted every 8-12 weeks. On completion of the study period, the codes were decoded and the patients were grouped according to whether they had received piracetam or placebo and on the basis of age i.e. 3-6 years and 7-12 years of age. Results: The results were separately analyzed for the 3-6 and 7-12 years age groups. In terms of age, weight, height, SI, number of blood transfusions received and number of hospitalization both groups (i.e. placebo and piracetam) were statistically homogeneous. The results of the placebo and Piracetam treated groups were analysed and a statistically significant decrease was observed in SI, number of crises, blood transfusion requirements and extent of hospitalization in the groups treated with piracetam compared to the placebo group. No differences were seen in the levels of hematological and biochemical parameters and Hb F level. Conclusion: Piracetam can be used for the amelioration of the clinical presentation of SCD. Even after discontinuation of the drug it's beneficial effects remain for several months.