Disappointing recruitment despite promising results

Sickle cell disease is the commonest inherited haemoglobinopathy in Britain and affects over 9000 people.1 Clinical severity varies considerably, but patients with the most severe disease have a life expectancy of just over 40 years. Conventional management of the disease is largely supportive, highlighting a pressing need for approaches that can alter the course of the disease. Trials in America have suggested that hydroxyurea can have a significant impact on the course of the disease, but in Britain it is proving virtually impossible to recruit patients into trials to confirm these results in a British population.

The fact that the clinical severity of the sickle cell disease varies even within groups of patients with the same β globin genotype2 has led to the concept that the disease is a multigene disorder, with inheritance of α thalassemia and genes controlling the concentration of fetal haemoglobin, among others, modulating disease expression. …