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2018 ; 2
(4
): 454-461
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Eltrombopag for use in children with immune thrombocytopenia
#MMPMID29487060
Kim TO
; Despotovic J
; Lambert MP
Blood Adv
2018[Feb]; 2
(4
): 454-461
PMID29487060
show ga
Eltrombopag is currently the only US Food and Drug Administration-approved
thrombopoietin receptor agonist for the treatment of chronic immune
thrombocytopenia (ITP) in children. This oral, once-per-day therapy has shown
favorable efficacy and adverse effect profiles in children. Two multicenter,
double-blind, placebo controlled clinical trials (PETIT [Efficacy and Safety
Study of Eltrombopag in Pediatric Patients With Thrombocytopenia From Chronic
Idiopathic Thrombocytopenic Purpura (ITP)] and PETIT2 [Study of a New Medication
for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low
Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding
Inside the Body]) demonstrated efficacy in raising platelet counts, reducing
bleeding, and reducing the need for concomitant ITP therapies with relatively few
adverse effects. The most commonly reported drug-related adverse effects include
headache, nausea, and hepatobiliary laboratory abnormalities. Long-term safety
data in children are limited, and studies in adults have not revealed a
clinically significant increased incidence of thrombosis, marrow fibrosis, or
cataract formation. Eltrombopag has also been approved for treating refractory
severe aplastic anemia (AA) and has potential for expanded use in ITP and severe
AA as well as in other conditions associated with thrombocytopenia.
|Benzoates/adverse effects/*therapeutic use
[MESH]
|Child
[MESH]
|Hemorrhage/prevention & control
[MESH]
|Humans
[MESH]
|Hydrazines/adverse effects/*therapeutic use
[MESH]