☆ 4.5 Article

Efficacy of eltrombopag with immunosuppressive therapy for children with acquired aplastic anemia

FRONTIERS IN PEDIATRICS (2023)

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

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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.

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Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia

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

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Review Hematology

Aplastic anemia: Pathophysiology

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Summary: Bone marrow failure syndromes are a group of hematological conditions characterized by low blood cell counts, which can be classified as constitutional or acquired based on the underlying causes. Constitutional syndromes result from genetic mutations in the hematopoietic stem cells, while acquired syndromes can be caused by external cytotoxic agents or autoimmune attacks. Aplastic anemia is an acquired autoimmune syndrome in which immune cells attack the hematopoietic stem cells, leading to marrow disruption.

SEMINARS IN HEMATOLOGY (2022)

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Article Hematology

Immunosuppressive therapy in severe aplastic anemia

Bhavisha A. Patel et al.

Summary: Severe aplastic anemia, a disease characterized by pancytopenia and a hypocellular marrow, can be treated with immunosuppressive therapy (IST) or hematopoietic stem cell transplant. Adding eltrombopag (EPAG) to the IST regimen improves treatment response rates without increasing the risk of myeloid malignancies. However, relapse remains a challenge, and further optimization of treatment protocols is needed.

SEMINARS IN HEMATOLOGY (2022)

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Article Hematology

Long-term outcomes in patients with severe aplastic anemia treated with immunosuppression and eltrombopag: a phase 2 study

Bhavisha A. Patel et al.

Summary: Patients with severe aplastic anemia (SAA) are treated with bone marrow transplant (BMT) or immunosuppression (IST) depending on individual factors. The addition of eltrombopag (EPAG) to standard IST for SAA improves hematologic responses. However, the rates and characteristics of long-term complications, relapse, and clonal evolution with this new regimen are not yet known. This study reports a cumulative relapse rate of 39% in responding patients and a clonal evolution rate of 15% in all treated patients at 4 years. Relapse occurred at distinct timepoints and most relapsed patients responded to retreatment. Clonal evolution to high-risk abnormalities was noted in 5.7% of patients and was associated with poorer overall survival.

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Editorial Material Hematology

Pediatric severe aplastic anaemia treatment: where to start?

Timothy S. Olson

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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.

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Acquired severe aplastic anaemia: how medical therapy evolved in the 20th and 21st centuries

Phillip Scheinberg

Summary: The advances in the management of aplastic anaemia (AA) have been successful, with better understanding of the mechanisms underlying the reduction of hematopoietic stem cells (HSCs) and improvements in supportive care. Efforts to increase immunosuppression potency and stimulate HSCs have not achieved better outcomes, but the use of thrombopoietin receptor agonists (Tpo-RA) has been effective in stimulating early HSCs despite high endogenous Tpo levels.

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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.

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Longitudinal evaluation of eltrombopag in paediatric acquired severe aplastic anaemia

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Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study

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Upfront eltrombopag monotherapy induces stable hematologic remission in pediatric patients with nonsevere idiopathic aplastic anemia

Wenliang Geng et al.

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Review Medicine, General & Internal

Aplastic Anemia

Neal S. Young

NEW ENGLAND JOURNAL OF MEDICINE (2018)

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Meeting Abstract Hematology

Eltrombopag Rescue for Childhood Severe Acquired Aplastic Anemia after Poor Response to Immunosuppressive Treatment: A Nationwide Study on Behalf of the French Reference Center for Aplastic Anemia

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BLOOD (2018)

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Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia

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Article Hematology

How I treat acquired aplastic anemia

Andrea Bacigalupo

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How I treat acquired aplastic anemia

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BLOOD (2012)

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Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia

Matthew J. Olnes et al.

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Long-Term Outcome of Pediatric Patients with Severe Aplastic Anemia Treated with Antithymocyte Globulin and Cyclosporine

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