Hematologic recovery induced by eltrombopag in Japanese patients with aplastic anemia refractory or intolerant to immunosuppressive therapy

Yamazaki, Hirohito; Ohta, Kensuke; Iida, Hiroatsu; Imada, Kazunori; Obara, Naoshi; Tokumine, Yukihiro; Tomiyama, Yoshiaki; Usuki, Kensuke; Imajo, Kento; Miyamura, Koichi; Sasaki, Osamu; Zhang, Fanghong; Hattori, Takuya; Tajima, Tsuyoshi; Matsuda, Akihisa; Nakao, Shinji

doi:10.1007/s12185-019-02683-1

Cited by 29 publications

(27 citation statements)

References 23 publications

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“…Since the optimal dosage of eltrombopag is unknown for patients in China, we started at 25 mg qd and the maximum dosage was 100 mg qd in our patients. This dosage was lower than that reported in Hong Kong and Europe (50~300 mg, median 150 mg) [13], but similar to that in Japan (25~100 mg, mean 84 mg) [15]. We found that the median response time was 3 months and 33.3% of patients achieved the best response at the dosage of 75 mg qd and 75% of them had response below the dosage of 75 mg qd.…”

Section: Discussionsupporting

confidence: 57%

“…Hwang et al [13] reported an ORR of 50% in 10 refractory patients combining eltrombopag and IST in Hong Kong. While two Japanese teams reported an ORR of 48~55% in patients refractory or intolerant to IST [14,15].…”

Section: Discussionmentioning

confidence: 99%

“…It has been shown that patients from Asian countries have different pharmacokinetics of eltrombopag; however, very few data have been published 13 . Reports of 20 cases in Hong Kong [13] and totally 32 cases in Japan [14,15] are the current available data so far. Furthermore, although CsA+ATG is the standard first-line treatment for patients with SAA or transfusiondependent NSAA, many patients are not willing to use ATG due to the high cost, potential risks, and hospitalization difficulties in China.…”

Section: Introductionmentioning

confidence: 99%

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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: 57%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

et al. 2020

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“…In Europe and North America, the incidence is 2-3 per million per year, which is 2-to 3-fold lower than in East Asia (7). In Japan and some other countries, the standard treatment for AA is immunosuppressive therapy (IST) with antithymocyte globulin (ATG), and cyclosporine A (CsA) in patients not selected for potentially curative allogeneic hematopoietic stem cell transplantation (allo-HSCT) (8,9). There are limitations associated with ATG/CsA treatment, mainly a "ceiling" response rate of 60%-70%, with one-third relapse rates by 2 years, incomplete responses, and clonal evolution in nearly 15% of patients after long-term treatment (7,10).…”

Section: Introductionmentioning

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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“…Os resultados obtidos com a droga nos estudos clínicos pivotais, inclusive, já têm sido reproduzidos em outras populações, consolidando sua relevância (ECSEDI et al, 2019;YAMAZAKI et al, 2019). Mais do que isso, tornaram evidente que estratégias direcionadas à expansão do compartimento de CTPH exaurido pelo dano imune na AAa podem atuar de modo adjuvante à terapia imunossupressora.…”

Section: Falência Da Célula-tronco Hematopoética: Anemia Aplásticaunclassified

Efeito do derivativo de purina SR1 e do derivativo pirimidoindólico UM171 na expansão de células-tronco e progenitoras hematopoéticas na anemia aplástica adquirida

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Hematologic recovery induced by eltrombopag in Japanese patients with aplastic anemia refractory or intolerant to immunosuppressive therapy

Cited by 29 publications

References 23 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

Efeito do derivativo de purina SR1 e do derivativo pirimidoindólico UM171 na expansão de células-tronco e progenitoras hematopoéticas na anemia aplástica adquirida

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