Efficacy and Safety of Eltrombopag for Aplastic Anemia: A Systematic Review and Meta-analysis

Hong, Yaqun; Li, Xiaofan; Wan, Bo; Li, Nainong; Chen, Yuanzhong

doi:10.1007/s40261-018-0725-2

Cited by 12 publications

(10 citation statements)

References 59 publications

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“…Adverse events in our study were mild, controllable, with grade I~II gastrointestinal discomfort, mild hepatic toxicity, and skin reactions the most common ones. This result was consistent with the safety profiles in previous trials [24,25]. There were great concerns about clonal evolution during TPO-mimetics stimulation [11,26], and long-term previous exposure to the bone marrow failure environment may be a trigger to clone evolution as well [27].…”

Section: Discussionsupporting

confidence: 91%

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: Discussionsupporting

confidence: 91%

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

et al. 2020

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“…Clonal evolution or cytogenetic abnormality associated with dysplasia, including the loss of chromosome 7, was not observed in our study. A recent meta-analysis involving 11 studies indicated clonal evolution at the karyotype abnormality level in 8% of IST-naïve patients treated with eltrombopag plus IST ( 32 ). In another study, the rates of clonal evolution among patients with severe AA treated with eltrombopag and horse-ATG/CsA were similar to those of their historical cohorts at a median follow-up of 2 years; the role of eltrombopag in clonal evolution is therefore unclear ( 15 ).…”

Section: Discussionmentioning

confidence: 99%

Eltrombopag in Combination with Rabbit Anti-thymocyte Globulin/Cyclosporine A in Immunosuppressive Therapy-naïve Patients with Aplastic Anemia in Japan

et al. 2021

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Objective In Japan, immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG), and cyclosporine A (CsA) is the standard of care in patients with aplastic anemia (AA) who are not indicated for stem-cell transplantation, although some patients may experience relapse. This study assessed the efficacy and safety of eltrombopag in combination with rabbit-ATG/CsA in IST-naïve patients with non-severe or severe AA in Japan. Methods In this non-randomized, open-label, single-arm, phase II study, rabbit-ATG/CsA and eltrombopag were initiated on Days 1 and 15 (±3 days), respectively, and continued for " 26 weeks; rabbit-ATG was given for 5 days (Days 1 to 5). The primary endpoint was the overall response rate (ORR) at Week 26. Patients Patients with AA who were IST-naïve and ! 70 years old or between 71 and 75 years old based on the recommendation of the investigator were enrolled in Japan. Results Of the 11 enrolled patients, 10 started treatment with eltrombopag. The ORRs at Weeks 26 and 52 were 70.0% and 60.0%, respectively. The ORR at Week 26 was 100% (all 3 patients) in patients with nonsevere AA and 57.1% (4/7) in patients with severe AA. Among transfusion-dependent patients, 66.7% (4/6) and 62.5% (5/8) became red blood cell-and platelet-transfusion independent, respectively. The most common adverse events were nausea and headache. No deaths or hematologic malignancies were reported. A cytogenetic abnormality was reported in one patient. Conclusion This study confirmed the clinical benefit of eltrombopag plus rabbit-ATG/CsA in IST-naïve patients with non-severe or severe AA in Japan.

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“…A recent systematic review and meta-analysis of the efficacy of eltrombopag in AA revealed hematologic response in 88% of patients treated with eltrombopag and IST. 8 In the pediatric population however, data are lacking to develop a standard of care. In a recent survey from the North American Pediatric Aplastic Anemia Consortium (NAPAAC), HSCT was stated as the preferred first-line therapy in children with SAA who have human leukocyte antigen (HLA)-identical sibling.…”

Section: Introductionmentioning

confidence: 99%

“…In 2018, the Food and Drug Administration (FDA) approved the use of eltrombopag in combination with standard IST in newly diagnosed patients with SAA who are 2 years or older. A recent systematic review and meta‐analysis of the efficacy of eltrombopag in AA revealed hematologic response in 88% of patients treated with eltrombopag and IST 8 . In the pediatric population however, data are lacking to develop a standard of care.…”

Section: Introductionmentioning

confidence: 99%

Eltrombopag in children with severe aplastic anemia

Lesmana

Jacobs

Boals

et al. 2021

Pediatric Blood & Cancer

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Background: Immunosuppressive therapy with horse antithymocyte globulin and cyclosporine currently remains the standard therapy for children with severe aplastic anemia (SAA) who lack human leukocyte antigen (HLA)-identical sibling. The thrombopoietin receptor agonist eltrombopag has been recently approved for SAA patients 2 years and older. However, there are limited data on its safety and efficacy in pediatric cohorts. Methods:We conducted a retrospective study of patients ≤18 years old consecutively diagnosed with SAA between 2000 and 2018. Patients received either standard immunosuppressive therapy (IST-Std) or IST with eltrombopag (IST-Epag). The primary outcome was the objective response (OR), including partial and complete response (CR), at 6 and 12 months after starting therapy. Results:We identified 16 patients receiving IST-Std and nine IST-Epag treatment (seven of nine as upfront therapy and two of seven after previously failed IST). The OR at 6 and 12 months in IST-Std arm was 71% and 100%, with CR in 29% and 58%, respectively. Seven patients receiving upfront IST-Epag had OR at 6 and 12 months, with two of seven (29%) achieving CR at 6 and 12 months. Two patients who previously failed standard IST did not respond to eltrombopag. No significant differences were observed in both cohorts with regard to infections. One IST-Epag-treated patient developed transient grade 3 transaminitis. Finally, no changes in paroxysmal nocturnal hemoglobinuria (PNH) clone size and cytogenetic abnormalities were seen in either cohort. Conclusion:The addition of eltrombopag to standard IST was well tolerated and resulted in satisfactory hematological response at 6 and 12 months in this singleinstitution experience. A larger cohort with longer follow-up is required to assess response durability.

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Efficacy and Safety of Eltrombopag for Aplastic Anemia: A Systematic Review and Meta-analysis

Cited by 12 publications

References 59 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

Eltrombopag in Combination with Rabbit Anti-thymocyte Globulin/Cyclosporine A in Immunosuppressive Therapy-naïve Patients with Aplastic Anemia in Japan

Eltrombopag in children with severe aplastic anemia

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