Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura.
BACKGROUND
The pathogenesis of chronic idiopathic thrombocytopenic purpura (ITP) involves antibody-mediated platelet destruction and reduced platelet production. Stimulation of platelet production may be an effective treatment for this disorder.


METHODS
We conducted a trial in which 118 adults with chronic ITP and platelet counts of less than 30,000 per cubic millimeter who had had relapses or whose platelet count was refractory to at least one standard treatment for ITP were randomly assigned to receive the oral thrombopoietin-receptor agonist eltrombopag (30, 50, or 75 mg daily) or placebo. The primary end point was a platelet count of 50,000 or more per cubic millimeter on day 43.


RESULTS
In the eltrombopag groups receiving 30, 50, and 75 mg per day, the primary end point was achieved in 28%, 70%, and 81% of patients, respectively. In the placebo group, the end point was achieved in 11% of patients. The median platelet counts on day 43 for the groups receiving 30, 50, and 75 mg of eltrombopag were 26,000, 128,000, and 183,000 per cubic millimeter, respectively; for the placebo group the count was 16,000 per cubic millimeter. By day 15, more than 80% of patients receiving 50 or 75 mg of eltrombopag daily had an increased platelet count. Bleeding also decreased during treatment in these two groups. The incidence and severity of adverse events were similar in the placebo and eltrombopag groups.


CONCLUSIONS
Eltrombopag increased platelet counts in a dose-dependent manner in patients with relapsed or refractory ITP. (ClinicalTrials.gov number, NCT00102739.)
Weill Cornell Medical College of Cornell University, New York, NY 10065, USA. jbussel@med.cornell.edu
James B
JB
Bussel
Gregory
G
Cheng
Mansoor N
MN
Saleh
Bethan
B
Psaila
Lidia
L
Kovaleva
Balkis
B
Meddeb
Janusz
J
Kloczko
Habib
H
Hassani
Bhabita
B
Mayer
Nicole L
NL
Stone
Michael
M
Arning
Drew
D
Provan
Julian M
JM
Jenkins
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001565:Benzoates / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D002908:Chronic Disease; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D006470:Hemorrhage / Q000139:chemically induced / Q000453:epidemiology; D006801:Humans; D006834:Hydrazines / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000097:blood / Q000188:drug therapy*; D011720:Pyrazoles / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D011788:Quality of Life; D053628:Receptors, Thrombopoietin / Q000819:agonists*; D012008:Recurrence; D013926:Thrombopoietin / Q000032:analysis
D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; D053628:Receptors, Thrombopoietin; D013926:Thrombopoietin; C520809:eltrombopag