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

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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 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.

Chromosomal abnormalities in childhood acute nonlymphocytic leukemia (M4).

A new reciprocal, apparently balanced translocation between chromosomes 7 and 22, i.e., t(7;22)(p22;q13), in association with inv(16)(p13q22) in a child with acute nonlymphocytic leukemia (M4) is reported. Five percent of her bone marrow cells contained both of these aberrations while 90% of her cells have the pericentric inversion of chromosome 16 as the sole abnormality. A clonal evolution of the unusual cell line may be associated with atypical clinical presentation. The presence of inversion 16 in adults has a better prognosis as compared with children. A concise review on the cytogenetic findings in children with ANLL(M4) is also provided.

Hemoglobin D-beta (0) thalassemia. A case report and family study.

Findings are presented on a Pakistani family in Doha, Qatar with hemoglobin D-Los Angeles (alpha 2 beta 3 121 (glutamine----glycine) and thalassemia trait. The propositus, a child, has hemoglobin D-beta(0) thalassemia, and suffers from moderately severe hemolytic anemia. The father has beta-thalassemia trait, and the mother is heterozygous for hemoglobin D-Los Angeles. This, the eighth confirmed case in the medical literature, is reported to emphasize its clinical manifestations and genetic basis.

High prevalence of human immunodeficiency virus infection in children with thalassemia exposed to blood imported from the United States.

PIP: 38.5% or 10 of 26 children with thalassemia who had received blood transfusion have tested positive for HIV at the Hamad General Hospital, Doha, Qatar. Blood for transfusion was obtained from commercial suppliers in Miami, Florida, since late 1970. Since December 1985 all blood for transfusion has been tested for HIV for ELISA kits from Abbott Laboratories, Chicago, Ill, and confirmed by Western Blot. These children ranged from 3-13 years old, mean 7.7, and had received an average of 63.5 transfusions. All had been given at least 30 transfusions. Children testing negative for HIV averaged 35.0 lifetime transfusions. 4 of the affected children had persistent generalized lymphadenopathy, but no other symptoms were recorded.^ieng

Ganglioneuroblastoma and fetal hydantoin-alcohol syndromes.

A ganglioneuroblastoma developed in a 35-month-old boy with both the fetal hydantoin and fetal alcohol syndromes. Our case, plus two recent reports in the literature, would very likely establish the relationship between fetal hydantoin syndrome and the development of neural crest tumors. Infants exposed in utero to hydantoins should be closely observed for the development of these tumors.