Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study

Tarantino, Michael D.; Bussel, James B.; Blanchette, Victor S.; Despotovic, Jenny M.; Bennett, Carolyn M.; Raj, Ashok; Williams, Bronwyn; Beam, Donald; Morales, Jaime; Rose, Melissa J.; Carpenter, Nancy; Nie, Kun; Eisen, Melissa

doi:10.1016/s0140-6736(16)00279-8

Cited by 121 publications

(154 citation statements)

References 28 publications

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“…Due to reduced steroid prescriptions and the new thrombopoietin receptor agonists (TRAs), the prognosis has improved significantly in recent years. Mortality in current pediatric studies is unchanged at 0%; in adults it has halved to 0-7% [34,[48][49][50][51][52] (table 5).…”

Section: Prognosis and Risk Factorsmentioning

confidence: 99%

See 1 more Smart Citation

Immune Thrombocytopenia - Current Diagnostics and Therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI

Matzdorff¹,

Meyer²,

Ostermann³

et al. 2018

Oncol Res Treat

104

156

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Section: Prognosis and Risk Factorsmentioning

confidence: 99%

“…There is no standard of care. TRAs are effective in chronic ITP in children and adolescents [49,52,84,[131][132][133]. Eltrombopag has been approved for this indication since 2016.…”

Section: Degree Of Recommendation: Ec Evidence Level:mentioning

confidence: 99%

Immune Thrombocytopenia - Current Diagnostics and Therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI

Matzdorff¹,

Meyer²,

Ostermann³

et al. 2018

Oncol Res Treat

104

156

View full text Add to dashboard Cite

“…There are currently two US Food and Drug Administration (FDA)-approved TPO-RAs, eltrombopag and romiplostim. These agents have both been extensively studied in adults and children with chronic ITP [24][25][26][27][28][29][30][31][32][33] ; however, their use as therapy for newly diagnosed patients remains unclear. Although the primary mechanism of the TPO-RAs is not immunomodulatory, changes in the immune profile with increase in T regulatory cells have been shown after use.…”

Section: Thrombopoietin-receptor Agonistsmentioning

confidence: 99%

Management of newly diagnosed immune thrombocytopenia: can we change outcomes?

Neunert

2017

Blood Advances

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Immune thrombocytopenia resulting from antibody-mediated platelet destruction combined with impaired platelet production is a common cause of thrombocytopenia. The decision to treat newly diagnosed patients is based on several factors including ceasing hemorrhagic manifestations, increasing the platelet count, prevention of bleeding, and inducing remission.Current standard first-line therapy is a course of corticosteroids. Although this treatment paradigm increases the platelet count in the majority of patients, a high percentage relapse after discontinuation of corticosteroid therapy. For this reason, intensification of first-line therapy that results in superior long-term remission rates would be desirable. This manuscript focuses primarily on adults with idiopathic thrombocytopenic purpura (ITP), highlighting pediatric data and practice when applicable. The primary aim is to outline upfront strategies for treatment-naive patients with ITP to enhance remission rates, taking into account assessment of the risks and benefits of these approaches.

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“…Durch Zurückhaltung bei der Steroidtherapie und durch die neuen Thrombopoetinrezeptor-Agonisten (TRA) ist die Prognose in den letzten Jahren deutlich besser geworden. Die Mortalität liegt in aktuellen pädiatrischen Studien unverändert bei 0%, bei Erwachsenen hat sie sich auf 0-7% halbiert [34,[48][49][50][51][52] (Tab. 5).…”

Section: Prognose Und Risikounclassified

“…Es gibt keinen Therapiestandard. TRA sind bei chronischer ITP im Kindes-und Jugendalter wirksam [49,52,84,[131][132][133] Bei Patienten, die eine ITP in der Anamnese haben und die jetzt in Remission sind, oder bei Patienten, die aktuell unter einer chronischen ITP leiden, scheinen Impfungen keinen Rückfall oder eine Verschlimmerung der Thrombozytopenie zu induzieren. Wenn die Impfung unterlassen wird und der Patient dann an der Infektion erkrankt, hat er möglicherweise ein höheres Risiko, dass diese Infektion die Thrombozytopenie verschlimmert [136].…”

Section: Empfehlungsgrad Ek Evidenzlevelunclassified

Immunthrombozytopenie - aktuelle Diagnostik und Therapie: Empfehlungen einer gemeinsamen Arbeitsgruppe der DGHO, ÖGHO, SGH, GPOH und DGTI

Matzdorff

Meyer²,

Ostermann³

et al. 2018

Oncol Res Treat

View full text Add to dashboard Cite

Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study

Cited by 121 publications

References 28 publications

Immune Thrombocytopenia - Current Diagnostics and Therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI

Immune Thrombocytopenia - Current Diagnostics and Therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI

Management of newly diagnosed immune thrombocytopenia: can we change outcomes?

Immunthrombozytopenie - aktuelle Diagnostik und Therapie: Empfehlungen einer gemeinsamen Arbeitsgruppe der DGHO, ÖGHO, SGH, GPOH und DGTI

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