How effective is Eltrombopag/Eltrombopag?

Eltrombopag/ Eltrombopag is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenia (ITP), chronic hepatitis C, and severe aplastic anemia (AA).

1. Treatment of chronic immune (idiopathic) thrombocytopenia (ITP):

Eltrombopag was compared with placebo (dummy treatment) in two major studies in the treatment of ITP in adult patients The two studies involved a total of 311 patients who had previously received treatment but had failed to respond or had disease recurrence. The first study showed that after 6 weeks of treatment, 59% of patients taking eltrombopag had platelet counts above 50,000 per microliter (which is considered sufficient to prevent bleeding complications), compared with 16% of patients taking a placebo. The second study showed that 135 patients who took eltrombopag were about eight times more likely to achieve target platelet counts of 50,000 to 400,000 per microliter during 6 months of treatment than 62 patients who took placebo.

An additional analysis examined the impact of time to diagnosis before starting treatment on drug response in adult patients. The study, which included nearly 400 patients with ITP, found that after 6 weeks of treatment, roughly the same number of patients had platelet counts of at least 50,000/microliter, whether diagnosed less than 6 months ago or more than 6 months after diagnosis.

Eltrombopag is more effective than placebo in a major study for the treatment of children with ITP The study involved 92 children aged 1-17 years who had previously been treated for ITP. The study lasted 13 weeks and looked at the proportion of patients whose platelet counts increased to at least 50,000 per microliter over an eight-week period between weeks 5 and 12 without rescue medication. The results showed that about 40% of patients taking eltrombopag experienced this condition, compared with about 3% of those taking a placebo. The study also found that eltrombopag was also effective in maintaining adequate platelet levels over the long term.

2. Hepatitis C-related thrombocytopenia:

To treat hepatitis C-related diseasesITP, conducted two major studies involving a total of 1,441 adults. These studies compared eltrombopag with placebo for initiating and maintaining antiviral therapy in patients with hepatitis C. Because platelet counts in patients with hepatitis C are initially too low to initiate this treatment (less than 75,000 platelets per microliter), the goal of these studies is to raise platelet counts to qualify for antiviral therapy. Results showed that in both studies, a higher proportion of patients who took eltrombopag achieved a negative hepatitis C test than those who took placebo (23% versus 14% in the first study and 19% versus 13% in the second study).

3. Severe aplastic anemia:

In the treatment of severe aplastic anemia, one study involving 43 patients evaluated the efficacy of eltrombopag but did not compare it with other drugs. The study's primary measure was the number of patients who responded to eltrombopag after 12 or 16 weeks of treatment, meaning their platelet, red blood cell or white blood cell count remained above a preset level. The results showed that 40% of patients in the study responded to treatment after 12 weeks, with 65% of patients having platelet counts that increased by at least 20,000 per microliter or remained stable without the need for transfusions. Preliminary data from a supporting study are also consistent with the main study's results, showing that 46% of patients responded to treatment after 12 weeks.