The US Food and Drug Administration has given a green signal to the sale of eltrombopag (Promacta for oral suspension, Novartis) for treating thrombocytopenia. The drug is, however, meant to treat thrombocytopenia in paediatric patients one year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have not received any help from corticosteroids, immunoglobulins, or splenectomy

The FDA nodded for the approval of the drug after looking into results of two randomised, double-blinded, placebo-controlled trials enrolling paediatric patients with chronic ITP. The PETIT trial involved paediatric patients with thrombocytopenia from ITP. It was a phase II trial and showed that the patients in the trial achieved platelet counts greater than or equal to 50 x109/L at least once between days eight and 43 of the randomised period of the study.

The trial results showed that Promacta has high efficacy of increasing blood platelet production. The drug will now be available in the form of a tablet meant to be taken orally once-daily by children aged one to five.

Platelets play a key role in allowing blood to clot; the problem in people with ITP is the risk of developing bleeding under the skin or in mucous membranes such as the mouth.

Doctors should prescribe the drug to only those patients at his risk of bleeding due to thrombocytopenia and clinical condition.

The first trial also showed that Promacta boosted and sustained platelet counts among pediatric patients with chronic ITP. These patients didn’t get any benefit from prior chronic ITP therapies.