PIRACETAM IS USEFUL IN THE TREATMENT OF CHILDREN WITH SICKLE-CELL DISEASE

The management of children suffering from sickle cell disease [sickle cell anaemia (SCA) and sickle cell beta(o)-thalassaemia (S beta(o)-tha l.)] has been the concern of all clinicians caring for these patients. Several agents have been tried for treatment, often limited by toxic side effects. Piracetam (2-oxo-1-pyrrolidine acetamide, Nootropyl(R)), a cyclic derivative of gamma-amino butyrate, used for the treatment o f psychosenescent syndromes with no known side effects, was considered as a possible therapeutic agent for sickle cell disease. Interest was focused on the use of piracetam when it was shown that it had an anti sickling effect, both in vivo and in vitro. We initiated multicentre d ouble-blind investigations in two groups of children suffering from si ckle cell disease ranging in age from 3-6 to 6-12 years. The total num ber of patients included in the study were 87 (SCA = 79 and Hb S beta( o)-thal. = 8) in 13 centres in 10 different regions of Saudi Arabia. C oded boxes of the drugs were received from the company (UCB) and were administered as intravenous infusion during crises and orally during t he follow-up, for a period of up to 1 year. After decoding the code at the end of the study, the patients were grouped into those receiving placebo (n = 39), i.e. controls, or piracetam (n = 48), i.e. study cas es. In terms of age, weight, height and severity index, number of bloo d transfusions received and number of hospitalization, both groups wer e statistically homogenous. Data analysis showed that the clinical sev erity of the disease, the number of crises, the extent of hospitalizat ion and the blood transfusion requirements significantly decreased dur ing piracetam treatment (p < 0.001), though no statistically significa nt changes occurred in the placebo group. However, in the levels of th e haematological and biochemical parameters no significant changes wer e documented in both groups. In addition, the improvement in the clini cal presentation of the disease continued even several months after di scontinuation of the drug in the majority of the children, as judged f rom the low severity index value. Though our results point to the reco mmendation that piracetam can be used for the treatment of children su ffering from sickle cell disease, both SCA asnd S beta(o)-thal, it is advisable to conduct long-term and close follow-up treatment programme s using piracetam to establish its therapeutic value particularly in a dults and to ascertain that there are no long-term toxic side effects.