Piracetam is useful in the treatment of children with sickle cell disease
Related Experiment Videos
M A el-Hazmi1, A S Warsy, I al-Fawaz
1College of Medicine, King Saud University, Riyadh, Saudi Arabia.
Insights
Piracetam significantly reduced sickle cell disease severity, crises, hospitalizations, and transfusion needs in children. This promising treatment showed sustained benefits even after discontinuation, warranting further investigation.
Area of Science:
- Hematology
- Pediatrics
- Pharmacology
Background:
- Sickle cell disease (SCD) management is challenging due to limited treatment options and toxic side effects of current agents.
- Piracetam, a cyclic derivative of gamma-amino butyrate with a favorable safety profile, was investigated for its potential antisickling effects in SCD.
Purpose of the Study:
- To evaluate the efficacy and safety of piracetam in managing children with sickle cell anaemia (SCA) and sickle cell beta degree-thalassaemia (S beta degree-thal.).
Main Methods:
- A multicentre, double-blind investigation involving 87 children (aged 3-12 years) with SCD in Saudi Arabia.
- Patients received either piracetam or placebo intravenously during crises and orally for up to 1 year.
- Clinical outcomes, including disease severity, crisis frequency, hospitalizations, and blood transfusions, were compared between groups.
Main Results:
- Piracetam treatment led to a significant decrease in clinical disease severity, number of crises, hospitalizations, and blood transfusion requirements (p < 0.001).
- No statistically significant changes were observed in the placebo group for these parameters.
- Hematological and biochemical parameters showed no significant changes in either group.
- Clinical improvement persisted for months after piracetam discontinuation.
Conclusions:
- Piracetam demonstrates therapeutic value in treating childhood sickle cell disease (SCA and S beta degree-thal.), significantly improving clinical outcomes.
- Long-term studies in adults are recommended to establish piracetam's full therapeutic potential and confirm the absence of long-term toxicity.
Abstract:
The management of children suffering from sickle cell disease [sickle cell anaemia (SCA) and sickle cell beta degree-thalassaemia (S beta degree-thal.)] 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-l-pyrrolidine acetamide, Nootropyl), a cyclic derivative of gamma-amino butyrate, used for the treatment of 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 antisickling effect, both in vivo and in vitro. We initiated multicentre double-blind investigations in two groups of children suffering from sickle cell disease ranging in age from 3-6 to 6-12 years. The total number of patients included in the study were 87 (SCA = 79 and Hb S beta degree-thal. = 8) in 13 centres in 10 different regions of Saudi Arabia. Coded boxes of the drugs were received from the company (UCB) and were administered as intravenous infusion during crises and orally during the 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 cases. In terms of age, weight, height and severity index, number of blood transfusions received and number of hospitalization, both groups were statistically homogenous. Data analysis showed that the clinical severity of the disease, the number of crises, the extent of hospitalization and the blood transfusion requirements significantly decreased during piracetam treatment (p < 0.001), though no statistically significant changes occurred in the placebo group. However, in the levels of the haematological and biochemical parameters no significant changes were documented in both groups. In addition, the improvement in the clinical presentation of the disease continued even several months after discontinuation of the drug in the majority of the children, as judged from the low severity index value. Though our results point to the recommendation that piracetam can be used for the treatment of children suffering from sickle cell disease, both SCA and S beta degree-thal, it is advisable to conduct long-term and close follow-up treatment programmes using piracetam to establish its therapeutic value particularly in adults and to ascertain that there are no long-term toxic side effects.