Eltrombopag in Immune Thrombocytopenia, Aplastic Anemia, and Myelodysplastic Syndrome: From Megakaryopoiesis to Immunomodulation

Fattizzo, Bruno; Levati, Giorgia; Cassin, Ramona; Barcellini, Wilma

doi:10.1007/s40265-019-01159-0

Cited by 39 publications

(40 citation statements)

References 114 publications

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“…It has been identified that the thrombopoietin (TPO) receptor is expressed not only on megakaryocytes, but also on hematopoietic stem cells [2]. So it is easy to understand that eltrombopag (ELT), an oral TPO receptor agonist, which has been first designed for immune thrombocytopenia [3], has been proved to promote tri-lineage hematopoiesis [4] in patients with AA [5,6]. Olnes et al [7] did the first trial of eltrombopag on 25 patients with refractory AA, and found that 11 of them had at least one lineage response at 12 weeks' treatment.…”

Section: Introductionmentioning

confidence: 99%

Eltrombopag is effective in patients with relapse/refractory aplastic anemia—report from a single center in China

et al. 2020

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Eltrombopag has shown a promising effect on patients with refractory/relapsed aplastic anemia (AA). However, data in Asian patients are limited due to the short launching time. Data from relapsed/refractory AA patients treated with eltrombopag in Peking Union Medical College Hospital from December 2017 to May 2019 were analyzed retrospectively. Totally 41 patients including 37 non-severe AA and 4 severe AA were analyzed with a median age of 47 (13~81) years old. The dosage of eltrombopag varies from 25 mg every other day (qod) to 100 mg every day (qd) (median 75 mg qd) with the duration of 13 (3~23) months. The overall response rates were 51.2%, 58.5%, and 55.2% at 3-month, 6-month, and 1-year follow-up. Seventy-five percent of patients achieved the best response below the dosage of 75 mg qd at 3 (1–6) months. Two patients stopped eltrombopag after achieving complete remission and remained stable. Two patients relapsed and evolved to myelodysplastic syndrome. Adverse events included gastrointestinal discomfort, hepatic toxicity, and skin reactions, which were mild and controllable. Eltrombopag is effective for Chinese relapsed/refractory AA patients with a relatively lower dose and acceptable side effects.

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Section: Introductionmentioning

confidence: 99%

Eltrombopag is effective in patients with relapse/refractory aplastic anemia—report from a single center in China

et al. 2020

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“…were reported, which indicated potentially better compliance of hetrombopag compared to eltrombopag. 12,13 Increased alanine aminotransferase was reported in two patients. Aspartate amino transferase transient elevation was reported in one patient in the absorption of eltrombopag occurs along the gastrointestinal tract.…”

Section: Discussionmentioning

confidence: 97%

First‐in‐patient study of hetrombopag in patients with chronic idiopathic thrombocytopenic purpura

Wang

Chen

Zhang

et al. 2020

Journal of Thrombosis and Haemostasis

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Background: Idiopathic thrombocytopenic purpura (ITP) especially refractory and (or) relapsed ITP, is a serious and global health burden and its clinical treatment is far from being satisfied. Hetrombopag is a novel, small-molecule thrombopoietin receptor agonist for the treatment of chronic idiopathic thrombocytopenic purpura (CITP). Objectives: This first-in-patient study aimed to investigate the safety, pharmacokinetics, and anticipated therapeutic dose of hetrombopag in CITP patients. Methods: In this multicenter, first-in-patient study, CITP patients received hetrombopag in a dose escalation (2.5 mg/day, 5 mg/day, or 7.5 mg/day) cohort. All patients received hetrombopag in fasting condition once daily for 2 weeks. Results: Of 44 patients screened, 32 were enrolled and treated. Most adverse events were graded 1 to 2 (ie, mild to moderate), and the incidence and severity were similar for three study cohorts. The pharmacokinetics of hetrombopag were found to be nonlinear with greater than dose-proportional: 12.5% of patients (1/8) in the 2.5 mg/d cohort, 58.3% of patients (7/12) in the 5 mg/d cohort, 66.7% of patients (8/12) in the 7.5 mg/d cohort reached the primary study endpoint of a platelet count exceeding 50 × 10 9 /L on day 28. Conclusion: Hetrombopag was well tolerated and preliminarily efficacious. Efficacy, safety, and pharmacokinetic data suggest that 7.5 mg hetrombopag once daily was the anticipated therapeutic dose of hetrombopag in CITP patients and has been recommended for investigation in a later confirmatory clinical study of hetrombopag.

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“…Eltrombopag was approved to treat SAA, in both refractory and newly diagnosed patients, combined with IST. 4 Obvious effects were noticed on the platelets, white blood cells, and Hgb. Based on these properties, Busca et al .…”

Section: Discussionmentioning

confidence: 99%

Effective treatment of refractory acquired pure red blood cell aplasia with eltrombopag and sirolimus: a case report

Huang

Jiang

Han

2020

Therapeutic Advances in Hematology

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Acquired pure red cell aplasia (aPRCA) is a kind of anemia characterized by severe reticulocytopenia and reduced bone marrow erythroblastic cells. For patients who are refractory to the first-line therapy (cyclosporin A with/without glucocorticoids), second-line therapy is considered less effective. We report on a patient with primary aPRCA who was refractory to cyclosporin A, glucocorticoids, and several second-line regimens. The patient was treated with sirolimus for 10 months with no improvement in hemoglobin but complete response was achieved after adding eltrombopag at a dosage of 25 mg/day. Eltrombopag was well tolerated with no evidence of clonal evolution at the end of follow up. This case provided a new attempt at treating patients with refractory/relapse aPRCA with eltrombopag, probably in combination with sirolimus.

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Eltrombopag in Immune Thrombocytopenia, Aplastic Anemia, and Myelodysplastic Syndrome: From Megakaryopoiesis to Immunomodulation

Cited by 39 publications

References 114 publications

Eltrombopag is effective in patients with relapse/refractory aplastic anemia—report from a single center in China

Eltrombopag is effective in patients with relapse/refractory aplastic anemia—report from a single center in China

First‐in‐patient study of hetrombopag in patients with chronic idiopathic thrombocytopenic purpura

Effective treatment of refractory acquired pure red blood cell aplasia with eltrombopag and sirolimus: a case report

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