Eltrombopag in pediatric chronic and refractory ITP: data from a retrospective multicenter study from Lebanon

Noun, Peter; Inati, Adlette; Raffoul, Rana; Younes, Jennifer Abi; Mardini, Joelle; Khalifé, Hassan

doi:10.1007/s00277-022-04804-z

Cited by 4 publications

(3 citation statements)

References 18 publications

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“…Currently, data have confirmed that approximately 30% of adults with p/cITP would attain a sustained response off treatment (SRoT) for platelets off TRA treatment 11,12 . Although the sustained response after TRA withdrawal has also been reported in several case (series) reports in the paediatric population, 13,14 data on large cohorts of children with p/cITP are lacking. In this study, we collected data from three tertiary children's hospitals in China, aiming to explore SRoT of ELT in children with p/cITP.…”

Section: Introductionmentioning

confidence: 99%

Sustained response off treatment in eltrombopag for children with persistent/chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China

Wang

Liu

et al. 2023

Br J Haematol

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Immune thrombocytopenia (ITP) is an acquired immune disorder characterized by reduced platelet (Plt) production and increased platelet destruction, which increases bleeding risk, with a prevalence of 5 in 100 000 children. 1,2 Although it does have a high rate of spontaneous remission, 30%-40% of cases of persistent or chronic immune thrombocytopenia (p/cITP) lead to heavy bleeding. 3,4 Thrombopoietin receptor agonists (TRAs) have emerged as the best treatment to elevate and maintain the Plt count for children with p/cITP. [5][6][7] Eltrombopag (ELT) increases

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Section: Introductionmentioning

confidence: 99%

Sustained response off treatment in eltrombopag for children with persistent/chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China

Wang

Liu

et al. 2023

Br J Haematol

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“… 80 – 83 As for hematological diseases, one retrospective multicenter observational study confirmed that Eltrombopag, a medication used to treat thrombocytopenia and severe aplastic anemia may be used as second- or third-line therapy in pediatric patients with chronic and refractory idiopathic thrombocytopenia. 84 Another phase 2 trial confirmed that, a new hemoglobin oxygen-affinity modulator, improves Hemoglobin levels and markers of hemolysis in pediatric patients with sickle cell disease and has the potential to mitigate sickle cell disease-related complications in patients aged ⩾4 years. 85 …”

Section: Resultsmentioning

confidence: 96%

Identifying health research in the era of COVID-19: A scoping review

Samah

2023

SAGE Open Medicine

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Background: Health improvements are considered one of the most important fields of research. Since the coronavirus disease 2019 was declared a pandemic, it might have impacted clinical and public health research in various forms. Objectives: The goal of this study is to explore health research approaches in the era of coronavirus disease 2019. Methods: In this scoping review, we reviewed published medical full-text studies and identified potential areas of interest of health research in the era the coronavirus disease 2019 pandemic during the last 3 years within a higher educational setting. A bibliometric analysis was used to compare among published works. Results: Among the 93 studies that met the inclusion criteria, most focused on mental health ( n = 23; 24.7%). Twenty-one publications targeted coronavirus disease 2019 and its consequences on general health. Other studies have described hemato-oncological, cardiovascular, respiratory, and endocrinological diseases. 42 studies were cross-sectional or cohort studies and most of them published in Q1 journals. Almost half of them belonged to the Faculty of Medicine (49.5%) followed by the School of Arts, Sciences, and Psychology (26.9%). Conclusions: Health research has been demonstrated to be important, at all times, especially during crises. Therefore, researchers need to invest more efforts into seeking new medical updates in various health-related fields, regardless of their correlation with coronavirus disease 2019.

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“…45 The results of the meta-analysis contrast to reports of nonrandomized (mostly retrospective observational) cohorts successfully treated with eltrombopag as second-line treatment of persistent/chronic ITP in childhood and adolescence. 46 47 48 49 50 51 This raises the question, how previous first-line treatment with other medication or their combination with eltrombopag affects treatment response. In adults, a meta-analysis on multiple drugs for the treatment of ITP showed that the efficacy of eltrombopag plus rituximab was significantly superior than placebo or dexamethasone alone.…”

Section: Methodsmentioning

confidence: 99%

Update on the Use of Thrombopoietin-Receptor Agonists in Pediatrics

Gebetsberger,

Streif,

Dame

2024

Hamostaseologie

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This review summarizes the rationale and current data on the use of thrombopoietin receptor agonists (TPO-RAs) for treating severe thrombocytopenia in infants, children, and adolescents. It focuses on substances that have been approved by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for pediatric patients. Romiplostim and eltrombopag are already established as second-line treatment for persistent or chronic immune thrombocytopenia (ITP). As in adults, TPO-RAs are currently also evaluated in severe aplastic anemia (SAA), chemotherapy-induced thrombocytopenia (CIT), myelodysplastic syndromes (MDS), and poor engraftment after hematopoietic stem cell transplantation in pediatric and adolescent patients. Moreover, studies on the implication of TPO-RA in treating rare inherited thrombocytopenias, such as Wiskott-Aldrich syndrome (WAS), congenital amegakaryocytic thrombocytopenia (CAMT), or MYH9-associated thrombocytopenia, deserve future attention. Current developments include testing of avatrombopag and lusutrombopag that are approved for the treatment of thrombocytopenia associated with chronic liver disease (CLD) in adult patients. In pediatric and adolescent medicine, we expect in the near future a broader use of TPO-RAs as first-line treatment in primary ITP, thereby considering immunomodulatory effects that increase the rate of sustained remission off-treatment, and a selective use in rare inherited thrombocytopenias based on current clinical trials.

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Eltrombopag in pediatric chronic and refractory ITP: data from a retrospective multicenter study from Lebanon

Cited by 4 publications

References 18 publications

Sustained response off treatment in eltrombopag for children with persistent/chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China

Sustained response off treatment in eltrombopag for children with persistent/chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China

Identifying health research in the era of COVID-19: A scoping review

Update on the Use of Thrombopoietin-Receptor Agonists in Pediatrics

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