☆ 4.5 Review

The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review

FRONTIERS IN PEDIATRICS (2023)

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Article Hematology

Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia

Olga Goronkova et al.

Summary: This study compared the efficacy and safety of eltrombopag combined with immunosuppressive therapy and immunosuppressive therapy alone in treatment-naive children with severe and very severe aplastic anemia. The results showed that adding eltrombopag to standard immunosuppressive therapy increased the complete response rate and overall response rate compared to immunosuppressive therapy alone. The greatest benefit from eltrombopag combined with immunosuppressive therapy was observed in patients with severe aplastic anemia, but not in those with very severe aplastic anemia.

BLOOD ADVANCES (2023)

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Article Medicine, General & Internal

Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia

R. Peffault de Latour et al.

Summary: Eltrombopag improves the efficacy of standard immunosuppressive therapy for severe aplastic anemia, leading to higher complete response rates within 3 months.

NEW ENGLAND JOURNAL OF MEDICINE (2022)

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Article Hematology

Predicting response of severe aplastic anemia to immunosuppression combined with eltrombopag

Yoshitaka Zaimoku et al.

Summary: This study aimed to investigate the relationship between response to immunosuppressive therapy plus eltrombopag and response to immunosuppressive therapy alone in severe aplastic anemia patients. The study found that blood counts remained the best predictors of response to nontransplant therapies in severe aplastic anemia, and the addition of eltrombopag improved treatment response.

HAEMATOLOGICA (2022)

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Review Biophysics

Haploidentical stem cell transplantation for aplastic anemia: the current advances and future challenges

Zheng-Li Xu et al.

Summary: HSCT is a curative option for SAA, with haploidentical donors providing expanded choices. Recent advancements in haplo-HSCT have overcome previous challenges and achieved inspiring survival outcomes in SAA. Different regimens such as G-CSF and ATG, PT-Cy, and TCD have been successful in promoting haplo-HSCT as a priority option for SAA patients without matched donors in China.

BONE MARROW TRANSPLANTATION (2021)

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Article Hematology

Eltrombopag added to immunosuppression for children with treatment-naive severe aplastic anaemia

Emma M. Groarke et al.

Summary: The study compared pediatric patients under 18 years of age who received EPAG plus standard IST with a historical cohort who received IST alone, indicating that EPAG did not improve outcomes in children with treatment-naive SAA. However, in adults, EPAG significantly improved overall response rate. Younger children had lower response rates compared to adolescents.

BRITISH JOURNAL OF HAEMATOLOGY (2021)

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Article Biochemistry & Molecular Biology

Effects of Eltrombopag on In Vitro Macrophage Polarization in Pediatric Immune Thrombocytopenia

Alessandra Di Paola et al.

Summary: Immune Thrombocytopenia (ITP) is an autoimmune disease characterized by platelet destruction mediated by autoantibodies. Eltrombopag (ELT) can potentially switch macrophage phenotype from pro-inflammatory to anti-inflammatory, reducing inflammation and restoring immune function in pediatric patients with ITP.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

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Article Oncology

Eltrombopag in children with severe aplastic anemia

Harry Lesmana et al.

Summary: This study evaluated the efficacy and safety of standard immunosuppressive therapy and combination therapy with eltrombopag in pediatric severe aplastic anemia patients. The results showed a higher objective response rate in the treatment group with eltrombopag added, and no significant differences in infections between the two therapies.

PEDIATRIC BLOOD & CANCER (2021)

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Article Oncology

Efficacy and safety of eltrombopag in the first-line therapy of severe aplastic anemia in children

Ma Jie et al.

Summary: The study evaluated the efficacy and safety of eltrombopag as a first-line therapy in pediatric patients with aplastic anemia. Results showed significantly increased complete response rates without intolerable side effects compared to historical cohort after 6 months of treatment.

PEDIATRIC HEMATOLOGY AND ONCOLOGY (2021)

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Article Oncology

Efficacy and safety of immunosuppressive therapy with or without eltrombopag in pediatric patients with acquired aplastic anemia: A Chinese retrospective study

Meixin Fang et al.

Summary: The study found that the combination of E-PAG and IST therapy was more effective than IST alone in the treatment of pediatric SAA after 6 months, with good tolerability and compliance.

PEDIATRIC HEMATOLOGY AND ONCOLOGY (2021)

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Editorial Material Pediatrics

Role of eltrombopag in severe aplastic anemia treatment in children

Min-Yu Su et al.

PEDIATRICS AND NEONATOLOGY (2021)

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Review Hematology

Current evidence and the emerging role of eltrombopag in severe aplastic anemia

Beatrice Drexler et al.

Summary: Eltrombopag as a TPO receptor agonist shows beneficial effects on expanding and maintaining HSCs in the treatment of AA. When used in combination with horse antithymocyte globulin and cyclosporine, it can significantly improve treatment response rates. However, high-dose usage may lead to adverse events and clonal evolution with chromosomal abnormalities.

THERAPEUTIC ADVANCES IN HEMATOLOGY (2021)

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Letter Hematology

Longitudinal evaluation of eltrombopag in paediatric acquired severe aplastic anaemia

Maria Filippidou et al.

BRITISH JOURNAL OF HAEMATOLOGY (2020)

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Meeting Abstract Hematology

Low Dose of Eltrombopag with Long-Term Response in Children with Severe Aplastic Anemia, Less Morbi-Mortality Better Quality of Life

Norma C. Lopez-Santiago et al.

BLOOD (2019)

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Meeting Abstract Hematology

Outcomes in Pediatric Patients with Severe Aplastic Anemia Treated with Standard Immunosuppression and Eltrombopag

Emma M. Groarke et al.

BLOOD (2019)

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Article Multidisciplinary Sciences

Iron chelating properties of Eltrombopag: Investigating its role in thalassemia-induced osteoporosis

Francesca Punzo et al.

PLOS ONE (2018)

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Article Medicine, General & Internal

Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia

Danielle M. Townsley et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

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Article Hematology

Eltrombopag safety and efficacy for primary chronic immune thrombocytopenia in clinical practice

Tomas Jose Gonzalez-Lopez et al.

EUROPEAN JOURNAL OF HAEMATOLOGY (2016)

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Article Medicine, General & Internal

The PRISMA Extension Statement for Reporting of Systematic Reviews Incorporating Network Meta-analyses of Health Care Interventions: Checklist and Explanations

Brian Hutton et al.

ANNALS OF INTERNAL MEDICINE (2015)

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Article Medicine, General & Internal

Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia

Matthew J. Olnes et al.

NEW ENGLAND JOURNAL OF MEDICINE (2012)

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