Sildenafil as a selective pulmonary vasodilator in childhood primary pulmonary hypertension

Abstract

Primary pulmonary hypertension is a rare disease of childhood, which carries a poor prognosis. Patients often present with severe exercise limitation, and untreated life expectancy is less than 1 year. Pharmacological intervention is directed towards reduction of the raised pulmonary artery pressure with vasodilator treatment, initially with calcium antagonists, although more recently long term prostacyclin treatment has shown benefit in some patients. Heart-lung transplantation remains an option for children with severe disease refractory to therapeutic treatment. A 4 year old Bangladeshi girl with dyspnoea, cyanosis, and signs of a low cardiac output, is described. Initial treatment with prostacyclin was gradually reduced, and maintenance treatment with oral sildenafil (Viagra; Pfizer) instituted. At follow up 3 months later, her exercise capacity was greatly improved and she continues to enjoy a good quality of life without obvious side effects. In view of the encouraging initial results, this may become an acceptable adjunct in treating this patient group.

Figure 1

The mechanism of action of sildenafil. cGMP, cyclic guanosine monophosphate; GTP, guanosine triphosphate; GMP, guanosine monophosphate.