Effects of eltrombopag: A Synthesis of Findings from 20 Studies

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Original Abstract of the Article

Major Research Findings

Eltrombopag is an oral medication used to treat low platelet counts, a condition known as thrombocytopenia, in various conditions like chronic immune thrombocytopenia (ITP), aplastic anemia (AA), and myelodysplastic syndromes (MDS). 14 , 11 , 20 Studies have shown that eltrombopag can increase platelet counts and reduce the risk of bleeding in patients with these conditions. 15 Additionally, eltrombopag has demonstrated potential effectiveness in treating immune-related thrombocytopenia caused by immune checkpoint inhibitors like atezolizumab. 4 However, it's important to note that eltrombopag can have side effects, including liver problems, skin discoloration, and kidney issues. 10 , 13 , 8

Benefits and Risks

Benefit Summary

Eltrombopag has been shown to effectively increase platelet counts and reduce the risk of bleeding in patients with various blood disorders like ITP, AA, and MDS. 15 Furthermore, it shows promise in treating thrombocytopenia related to immune checkpoint inhibitors. 4

Risk Summary

Eltrombopag can cause side effects such as liver problems, skin discoloration, and kidney issues. 10 , 13 , 8

Comparison of Studies

Similarities in Studies

Multiple studies have consistently demonstrated the effectiveness of eltrombopag in treating thrombocytopenia. 14 , 11 , 20 , 15 , 13 These studies have consistently shown that eltrombopag increases platelet counts and reduces the risk of bleeding associated with these conditions. 15

Differences in Studies

There are variations in the observed effects and potential side effects of eltrombopag across different studies. 15 For instance, some studies have suggested a higher risk of liver-related side effects with eltrombopag. 10 , 13 Additionally, while eltrombopag shows potential for treating thrombocytopenia associated with immune checkpoint inhibitors, more research is needed to confirm its efficacy in this specific area. 4

Consistency and Contradictions in Findings

While multiple studies have confirmed the effectiveness of eltrombopag in treating thrombocytopenia, some studies have highlighted potential concerns regarding its side effects. 10 , 13 Furthermore, the efficacy of eltrombopag in treating thrombocytopenia associated with immune checkpoint inhibitors requires further investigation through larger clinical trials. 4

Considerations for Real-World Application

While eltrombopag is an effective medication for treating thrombocytopenia, it's important to use it cautiously and be aware of its potential side effects. 10 , 13 Although eltrombopag shows promise in managing thrombocytopenia related to immune checkpoint inhibitors, more research is necessary to confirm its effectiveness in this particular area. 4

Limitations of Current Research

Further research is required to fully understand the long-term benefits and risks of eltrombopag. 4 Large-scale clinical trials are particularly needed to confirm its effectiveness in treating thrombocytopenia associated with immune checkpoint inhibitors. 4

Future Directions for Research

Further research is needed to assess the long-term benefits and risks of eltrombopag. 14 Additionally, large clinical trials are needed to confirm eltrombopag's effectiveness in treating thrombocytopenia related to immune checkpoint inhibitors. 4

Conclusion

Eltrombopag has demonstrated effectiveness in treating thrombocytopenia associated with various conditions, including ITP, AA, and MDS. 14 , 11 , 20 , 15 , 13 However, it's important to be aware of potential side effects, and further research is needed to thoroughly understand its long-term benefits and risks, especially in the context of immune checkpoint inhibitor-related thrombocytopenia. 10 , 13 , 4 Larger clinical trials are crucial to confirm its efficacy in this specific area. 4

Literature analysis of 20 papers
Positive Content
18
Neutral Content
0
Negative Content
1
Article Type
1
0
0
2
18
1.

Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia.

Author: de LatourRégis Peffault, KulasekararajAustin, IacobelliSimona, GriffinMorag, HalkesConstantijn Jm, DufourCarlo, RisitanoAntonio M

Improved platelet levels

This summary investigated the results of standard immunosuppression treatment versus standard immunosuppression treatment with eltrombopag in participants with severe aplastic anemia (SAA) and very severe aplastic anemia (vSAA). Eltrombopag is a medication that improves blood platelet levels.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

2.

Safety and efficacy of eltrombopag in patients with post-CAR T cytopenias.

Author: WessonWilliam, AhmedNausheen, RashidAliya, TabakCarine, LoganEmerson, Marchena-BurgosJose, NelsonMaggie, DavisJames A, McGannMary, ShuneLeyla, HoffmannMarc, AbdallahAl-Ola, HashmiHamza

CAR T-cell therapy efficacy
Cytopenia management
Post-treatment cytopenias

This study describes the management of post-CAR T cytopenias using eltrombopag, a thrombopoietin mimetic, in patients receiving chimeric antigen receptor (CAR) T-cell therapy for relapsed/refractory hematological malignancies.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

3.

Romiplostim use for thrombocytopenia following allogeneic hematopoietic stem cell transplantation: a case series from a single center in Qatar.

Author: Al-MashdaliAbdulrahman F, GuliedAmaal, BakrMohammed M, YassinMohamed A

Safe and effective
Thrombocytopenia treatment

This article discusses the use of thrombopoietin receptor agonists (TPO-RAs) for the treatment of post-hematopoietic stem cell transplantation (HSCT) thrombocytopenia.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

4.

A case of successful treatment with eltrombopag for severe immune-related thrombocytopenia induced by atezolizumab:Case report.

Author: HaradaSaki, ImakuraTakeshi, SatoSeidai, NokiharaHiroshi, NishiokaYasuhiko

Improved thrombocytopenia
Severe ITP

A patient with non-small-cell lung cancer who developed severe immune-related thrombocytopenia (ITP) after atezolizumab treatment was successfully treated with eltrombopag, a thrombopoietin receptor agonist. This suggests eltrombopag as a potential treatment option for severe ITP in patients receiving immune checkpoint inhibitors.

Case ReportsIn the medical field, a report that describes in detail a unique case, an unusual medical condition, or the effect of a treatment. Provides new insights and possibilities for treatment through individual patient cases.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

5.

Real-world outcomes with immunosuppressive therapy for aplastic anemia in patients treated at the University of Michigan.

Author: FoucarCharles E, FoleyDaniel H, AldousJessica, BurkePatrick W, PettitKristen R, BenitezLydia L, PerissinottiAnthony J, MariniBernard L, BoonstraPhilip, BixbyDale L

AA treatment
Infectious complications

A retrospective review of patients with aplastic anemia (AA) treated with immunosuppressive therapy (IST) showed that dose-attenuated IST was feasible and safe in older patients (>60) with concerns about tolerability. Younger patients receiving IST, especially in combination with eltrombopag, showed favorable outcomes.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

6.

Thrombopoietin receptor agonists for refractory thrombocytopenia in patients after autologous hematopoietic stem cell transplantation.

Author: WangDan, LangTao, ZengHanqing, ZouZhongmin, YangShijie, ChengTing, LiuHuanfeng, ZhuLidan, XiangXixi, YaoHan, TangShuhan, KongPeiyan, WeiJin, XiongJingkang, GaoLei, ZhangXi, FengYimei

ASCT treatment

This study investigated the use of thrombopoietin receptor agonists (TPORAs) to treat thrombocytopenia, a common side effect of autologous hematopoietic stem cell transplantation (ASCT). TPORAs are a potential therapy for managing thrombocytopenia after ASCT, offering an effective way to manage the complication while patients undergo treatment for their underlying condition.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

7.

Multicenter evaluation of the addition of eltrombopag to immunosuppressive therapy for adults with severe aplastic anemia.

Author: ShinnLauren T, BenitezLydia L, PerissinottiAnthony J, ReidJustin H, BuhlingerKaitlyn M, van DeventerHendrik, BarthDylan, WagnerCharlotte B, ZacholskiKyle, DesaiRuchi, SouleAshley, StumpSarah E, WeisTaylor M, BixbyDale, BurkePatrick, PettitKristen, MariniBernard L

Aplastic anemia treatment
Deeper responses
Hepatotoxicity

This study compared outcomes in patients with severe aplastic anemia receiving immunosuppressive therapy with or without eltrombopag. While overall response rates were similar, eltrombopag was associated with significantly higher complete response rates. Eltrombopag did not significantly impact overall survival or disease-free survival.

Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

8.

Coffee Colored Serum, Adverse Reaction of Eltrombopag.

Author: RojasCarlos Rodríguez, SantamaríaCelia Juez, Pons BeldaOscar David, NogueroEmilia Moreno

Cutaneous hyperpigmentation
Serum discoloration

Eltrombopag, a medication for severe aplastic anemia, can cause brown serum due to its brown color in solution at high concentrations. This case report describes a patient who experienced brown serum and cutaneous hyperpigmentation due to high-dose Eltrombopag therapy.

Case ReportsIn the medical field, a report that describes in detail a unique case, an unusual medical condition, or the effect of a treatment. Provides new insights and possibilities for treatment through individual patient cases.

Language : English

9.

Fostamatinib or Thrombopoietic Receptor Agonists for the Treatment of Chronic Immune Thrombocytopenia in Adult Patients: A Real-World Assessment of Safety, Effectiveness and Cost.

Author: DranitsarisGeorge, PeevyhouseAaron, WoodTiffany, KreychmanYuliya, NeuhalfenHeather, MoeziMehdi

ITP treatment

This real-world study compared the safety, effectiveness, and cost of fostamatinib with thrombopoietic agents (TPOs) for the treatment of chronic immune thrombocytopenia purpura (ITP) in adults.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

10.

Clinical Care Team's Guide for Awareness on Risk Assessment of Eltrombopag Complicating Acute Kidney Injury in Relapsed Immune Thrombocytopenic Patients: A Case Report.

Author: HamedEman Mostafa, MeabedMohamed Hussein, IbrahimAhmed R N, KhalafAhmed M, El DemerdashDoaa Mohamed, ElgendyMarwa O, SaeedHaitham, MahmoudTamer M, SalemHeba F, RabeaHoda

Eltrombopag efficacy
ITP treatment
Acute kidney injury
Eltrombopag side effects

This case report describes a patient who experienced acute kidney injury during Eltrombopag therapy for immune thrombocytopenia (ITP). This highlights the importance of vigilant kidney function screening in patients receiving Eltrombopag.

Case ReportsIn the medical field, a report that describes in detail a unique case, an unusual medical condition, or the effect of a treatment. Provides new insights and possibilities for treatment through individual patient cases.

Language : English

13.

The efficacy and safety of eltrombopag in treating TKI-induced thrombocytopenia in patients with chronic myeloid leukemia.

Author: LiuLi, ChenYilin, LiangYan, MengLi, GuoJingming, LiuChuancai, ZhaoZhe, ZouJing, HeWenjuan, ZhangJiangzhao, HongZhenya, LiangCaixia, FuXianjie, WuHui, ZhangYoushan, ZhangYanli, LiWeiming

यो अध्ययनले CML भएका बिरामीहरूमा TKI-सम्बन्धित थ्रम्बोसाइटोपेनिआ को उपचार को लागि एलट्रोम्बोपागको प्रयोग को अनुसन्धान गर्छ।

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

14.

Long term use of eltrombopag in children with chronic immune thrombocytopenia: extended real life retrospective multicenter experience of the Italian Association of Pediatric Hematology and Oncology.

Author: GiordanoPaola, LassandroGiuseppe, BaroneAngelica, CesaroSimone, FotziIlaria, GionaFiorina, GorioChiara, MaggioAngela, MianoMaurizio, MarzolloAntonio, NardiMargherita, PessionAndrea, RuggieroAntonio, RussoGiovanna, SaraccoPaola, SpinelliMarco, TolvaAlessandra, TorneselloAssunta, PalladinoValentina, Del VecchioGiovanni Carlo

Platelet count improvement
Reduced use of additional therapies

Eltrombopag treatment demonstrated benefits in terms of platelet count improvement and use of additional therapies in children with chronic ITP, with benefits identifiable from the first 6 months of treatment.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

15.

Eltrombopag for Adults and Children with Immune-Refractory Thrombocytopenic Purpura: A Systematic Review.

Author: de Barros TorelliDanielle Francisco Honorato, OliveiraCrystian Bitencourt Soares, NaiGisele Alborghetti, TrindadeEvelinda Marramon, Prestes-CarneiroLuiz Euribel

Disease prevention
Platelet response
Adverse effects
Bleeding

This meta-analysis assessed the efficacy and safety of eltrombopag in adults and children with refractory immune thrombocytopenic purpura (ITP). Eltrombopag improved platelet response in adults and reduced bleeding in children.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

16.

[Effect of Eltrombopag on Response to Immunosuppressive Therapy in Patients with Transfusion-Dependent Non-Severe Aplastic Anemia].

Author: LinYing, ZhangRong-Dong, LinZeng-Hua, ChenQi, ChenRen-Li

Hematopoietic response
Platelet recovery

Eltrombopag combined with cyclosporine A (CsA) significantly improved hematological response and platelet recovery in patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA) compared to CsA alone.

English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : Chinese

17.

Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial.

Author: WanZiqi, ChenMiao, HanBing

Effective treatment
Well-tolerated

Avatrombopag (AVA) was found to be effective and well-tolerated in patients with non-severe aplastic anemia (NSAA) who were refractory, relapsed, or intolerant to standard treatments.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

20.

Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS).

Author: OlivaEsther Natalie, RivaMarta, NiscolaPasquale, SantiniValeria, BrecciaMassimo, GiaiValentina, PoloniAntonella, PatriarcaAndrea, CrisàElena, CapodannoIsabella, SalutariPrassede, RedaGianluigi, CascavillaNicola, FerreroDario, GuariniAttilio, TripepiGiovanni, IannìGiuseppe, RussoEmilio, CastelliAndrea, FattizzoBruno, BeltramiGermana, BocchiaMonica, MolteniAlfredo, FenauxPierre, GermingUlrich, RiccoAlessandra, PalumboGiuseppe A, ImperaStefana, Di RenzoNicola, RivelliniFlavia, BuccisanoFrancesco, Stamatoullas-BastardAspasia, LiberatiAnna Marina, CandoniAnna, DelfinoIlaria Maria, ArcadiMaria Teresa, CufariPatrizia, RizzoLorenzo, BovaIrene, D'ErrigoMaria Grazia, ZiniGina, LatagliataRoberto

Effective treatment
Safe treatment

Eltrombopag was effective and relatively safe in patients with low-risk myelodysplastic syndromes (MDS) and severe thrombocytopenia. In a randomized, placebo-controlled trial of 169 patients, platelet response (PLT-R) occurred in 42.3% of eltrombopag patients versus 11.1% in placebo patients.

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

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