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Review
. 2023 Jun 6;12(12):3872.
doi: 10.3390/jcm12123872.

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

Affiliations
Review

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

Danielle Francisco Honorato de Barros Torelli et al. J Clin Med. .

Abstract

Eltrombopag is an agonist that binds to the membrane-bound domain of the thrombopoietin receptor used in immune thrombocytopenic purpura (ITP). We conducted a meta-analysis of randomized controlled trials to assess the efficacy and safety of eltrombopag in adults and children with refractory ITP. Adults who received eltrombopag had a significantly better platelet response (relative risk [RR], 3.65; 95% confidence interval [CI], 2.39-5.55), but there were no differences in the incidence of bleeding (RR, 0.8; 95% CI, 0.52-1.22) and adverse effects (RR, 0.99; 95% CI, 0.55-1.78) compared with the placebo. In children, there was no difference between eltrombopag and placebo for a platelet response >50,000/mm3 (RR, 3.93; 95% CI, 0.56-27.79) and the number of adverse events (RR, 0.99; 95% CI, 0.25-1.49); however, a lower incidence of bleeding was observed (RR, 0.47; 95% CI, 0.27-0.83). Treatment with eltrombopag protected adults and children from severe disease and death.

Keywords: blood coagulation; erythropoietin receptors; hematologic drugs; immune thrombocytopenic purpura; systematic review.

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Conflict of interest statement

The authors report that there are no competing interest to declare.

Figures

Figure 1
Figure 1
Flowchart of the studies included in the systematic review. ITP, immune thrombocytopenic purpura.
Figure 2
Figure 2
Risk of bias summary. The authors’ judgment for each study. Green, low risk of bias; yellow, uncertain risk [7,8,21,22,23,24,25,26,27].
Figure 3
Figure 3
Comparison of the lasting response with eltrombopag and placebo in adults and children with immune thrombocytopenic purpura [7,21,24].
Figure 4
Figure 4
Comparison between the occurrence of adverse events with eltrombopag and placebo in adults and children with immune thrombocytopenic purpura [7,8,22,23,24,25,26].
Figure 5
Figure 5
Comparison between the platelet response with eltrombopag and placebo in adults and children with immune thrombocytopenic purpura [7,8,21,22,23,24,25,26,27].
Figure 6
Figure 6
Comparison between the use of eltrombopag rescue therapy and placebo in adults and children with immune thrombocytopenic purpura [7,21,23,24,25,27].
Figure 7
Figure 7
Comparison between the occurrence of bleeding with eltrombopag and placebo in adults and children with immune thrombocytopenic purpura [7,8,21,22,23,24,25,26].

References

    1. Gonzalez-Porras J.R., Bastida J.M. Eltrombopag in immune thrombocytopenia: Efficacy review and update on drug safety. Ther. Adv. Drug. Saf. 2018;9:263–285. doi: 10.1177/2042098618769587. - DOI - PMC - PubMed
    1. Matzdorff A., Meyer O., Ostermann H., Kiefel V., Eberl W., Kühne T., Pabinger I., Rummel M. Immune thrombocytopenia—Current diagnostics and therapy: Recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI. Oncol. Res. Treat. 2018;41((Suppl. S5)):1–30. doi: 10.1159/000492187. - DOI - PubMed
    1. Ozelo M.C., Colella M.P., Vinicius de Paula E., Virgílio do Nascimento A.C.K., Villaça P.R., Bernardo W.M. Guideline on immune thrombocytopenia in adults: Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular. Project guidelines: Associação Médica Brasileira—2018. Hematol. Transfus. Cell Ther. 2018;40:50–74. doi: 10.1016/j.htct.2017.11.001. - DOI - PMC - PubMed
    1. Provan D., Arnold D.M., Bussel J.B., Chong B.H., Cooper N., Gernsheimer T., Ghanima W., Godeau B., González-López T.J., Grainger J., et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3:3780–3817. doi: 10.1182/bloodadvances.2019000812. - DOI - PMC - PubMed
    1. Kistangari G., McCrae K.R. Immune thrombocytopenia. Hematol. Oncol. Clin. N. Am. 2013;27:495–520. doi: 10.1016/j.hoc.2013.03.001. - DOI - PMC - PubMed

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