Thrombopoietic Agents for the Treatment of Persistent and Chronic Immune Thrombocytopenia in Children

Ramaswamy, Kavitha; Hsieh, Loan; Leven, Emily; Thompson, Michaela V.; Nugent, Diane J.; Bussel, James B.

doi:10.1016/j.jpeds.2014.03.060

Cited by 46 publications

(30 citation statements)

References 19 publications

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“…treatment for more than 3 years. 45 Furthermore, adults with increased grades of fibrosis have demonstrated regression of the fibrosis when eltrombopag was stopped. These results suggest that long-term use of eltrombopag does not substantially increase BM fibrosis and that, if it occurs, it is reversible.…”

Section: Discussionmentioning

confidence: 99%

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Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia

Gerrits

Leven

Frelinger

et al. 2015

Blood

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Section: Discussionmentioning

confidence: 99%

“…46,47 Use of romiplostim or eltrombopag in a retrospective analysis of 33 children with ITP appeared to be safe, effective, and tolerable. 45 Eltrombopag has recently been FDA-approved for the treatment of children 6 years and older with chronic ITP. For personal use only.…”

Section: Discussionmentioning

confidence: 99%

Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia

Gerrits

Leven

Frelinger

et al. 2015

Blood

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“…Adolescent patients with ITP may be at similar increased risk of thrombosis in the setting of acquired additional risk factors for thrombosis; the pediatric patients who have been reported with thrombosis have had additional risk factors, including immobilization from a fracture, antiphospholipid antibody syndrome, and use of estrogen-containing birth control. 35,36 Risk for iron deficiency. The structure of eltrombopag mimics that of currently available iron chelators.…”

Section: February 2018mentioning

confidence: 99%

Eltrombopag for use in children with immune thrombocytopenia

2018

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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.

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“…The most commonly used second line treatments include therapeutics such as thrombopoietic agents, rituximab, azathioprine, mycophenolate mofetil, and splenectomy. 11–15 …”

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confidence: 99%

Treatment of Children with Persistent and Chronic Idiopathic Thrombocytopenic Purpura: 4 Infusions of Rituximab and Three 4-Day Cycles of Dexamethasone

Oved

Lee

Bussel

2017

The Journal of Pediatrics

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Objectives To assess initial and long-term outcome of children with persistent/chronic idiopathic thrombocytopenic purpura (ITP) treated with 4 infusions of rituximab and three 4-day cycles of dexamethasone (4R+3Dex) including cohorts with most benefit and/or treatment associated toxicity. Study design All pediatric patients with ITP at Weill-Cornell who received 4R+3Dex were included in this retrospective study. Duration was median time from first rituximab infusion to treatment failure. Patient cohort included 33 children ages 1-18 years with persistent/chronic ITP; 19 were female, 10 of whom were adolescents. Every patient had failed more than 1 and usually several ITP treatments. Results Children were treated with rituximab, 375 mg/m2 weekly for 4 weeks and three 4-day courses of dexamethasone 28 mg/m2 (40 mg max). Average age of nonresponders was 7.75 years, and initial responders averaged 12.7 years (P =.0073); 30% maintained continuing response at 60 months or last check-up. Eight of the 10 patients who underwent remission were female with ITP <24 months prior to initiating 4R+3Dex. All responding male patients except 2 relapsed. Conclusions Durable unmaintained ITP remission after 4R+3Dex was seen almost exclusively in female adolescents with <24 months duration of ITP. This provides a new therapeutic paradigm for a subpopulation with hard-to-treat chronic ITP. The pathophysiology of ITP underlying this distinction requires further elucidation.

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Thrombopoietic Agents for the Treatment of Persistent and Chronic Immune Thrombocytopenia in Children

Cited by 46 publications

References 19 publications

Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia

Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia

Eltrombopag for use in children with immune thrombocytopenia

Treatment of Children with Persistent and Chronic Idiopathic Thrombocytopenic Purpura: 4 Infusions of Rituximab and Three 4-Day Cycles of Dexamethasone

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