International Journal of Hematology

DOI: 10.1007/s12185-019-02683-1 Pages: 187-196

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

1. Kanazawa University Hospital, Division of Transfusion Medicine

2. Hematology Ohta Clinic

3. National Hospital Organization Nagoya Medical Center

4. Japanese Red Cross Osaka Hospital

5. University of Tsukuba

6. Itami City Hospital

7. Osaka University Hospital

8. NTT Medical Center Tokyo

9. Okayama City Hospital

10. Japanese Red Cross Nagoya Daiichi Hospital

11. Miyagi Cancer Center

12. Novartis Pharma K.K

13. Saitama Medical University, International Medical Center

14. Kanazawa University Institute of Medical Pharmaceutical and Health Sciences

Correspondence to:
Hirohito Yamazaki
Tel: +81 (0) 76-265-2275



Eltrombopag, an oral thrombopoietin-receptor agonist, stimulates hematopoiesis in patients with acquired aplastic anemia (AA) and has higher exposure in patients of East Asian origin. We evaluated the pharmacokinetics, efficacy, and safety of eltrombopag in Japanese patients with AA refractory or intolerant to immunosuppressive therapy (IST). Twenty-one patients (15 with non-severe AA, six with severe AA) with platelet counts < 30,000/µL received eltrombopag in a dose-escalation fashion (25, 50, 75, or 100 mg once daily) depending on individual platelet responses; the responders continued eltrombopag treatment beyond 6 months. The primary endpoint was hematologic response at 6 months, defined as improvements in blood counts or transfusion requirements. Ten (48%) patients achieved hematologic responses in at least one lineage at 6 months. Six patients achieved tri- and/or bi-lineage responses with continuation of eltrombopag treatment, with two patients no longer requiring eltrombopag treatment. The most common adverse events were nasopharyngitis and abnormal hepatic function, with the majority being grade 1 or 2. Cytogenetic abnormalities were observed in three patients; however, no progression to myelodysplastic syndrome/other malignancy was observed. Eltrombopag can safely restore multi-lineage hematopoiesis in Japanese patients with AA refractory or intolerant to IST.

Clinical Trial registration NCT02148133.

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