International Journal of Hematology

DOI: 10.1007/s12185-017-2275-4 Pages: 508-516

Efficacy and safety of eltrombopag in persistent and newly diagnosed ITP in clinical practice

1. Hospital Universitario de Burgos, Department of Hematology

2. Hospital Universitario Insular de Gran Canaria, Department of Hematology

3. Hospital Infanta Leonor, Department of Hematology

4. Hospital del Mar, Department of Hematology

5. Hospital de León, Department of Hematology

6. Hospital Universitario La Paz, Department of Hematology

7. Hospital General Universitario Gregorio Marañón, Department of Hematology

8. Hospital de La Plana, Department of Hematology

9. Hospital Universitario de Burgos, Department of Statistics

10. Hospital de Soria, Department of Hematology

11. Hospital de Avila, Department of Hematology

12. Hospital Clínico de Valladolid, Department of Hematology

13. Hospital Universitario Vall de Hebron, Department of Hematology

14. Hospital Universitario Central de Asturias, Department of Hematology

15. Hospital del Bierzo, Department of Hematology

16. Hospital Universitario Río Hortega, Department of Hematology

17. IBSAL-Hospital Universitario de Salamanca, Department of Hematology

Correspondence to:
Tomás José González-López
Email: tjgonzalez@saludcastillayleon.es

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Abstract

Eltrombopag is safe and effective in primary chronic ITP. However, lack of clinical trials avoids a clear demonstration of its utility in newly diagnosed and persistent ITP. Our aim here is to report Spanish results for this type of patients. We retrospectively evaluated 220 adult primary ITP patients. According to standard definition, patients were allocated to newly diagnosed (n = 30), persistent (n = 30), and chronic (n = 160) ITP. Groups were homogenous regarding most relevant parameters. 180 (90%) of 220 patients achieved a platelet response (R) with 167 (75.9%) complete responses (CR) after a 15-month follow-up. No statistical significant differences among groups but a trend towards a greater efficacy in newly diagnosed ITP were observed (93.3% of responses with 86.7% of CR). Efficacy in persistent ITP (83.3% of responses with 80.0% of CR) and chronic ITP (79.4% of responses with 73.1% of CR) was similar. 70 patients (31.8%) experienced adverse events. 15 of them were grade 3–4. Most common adverse effects were headache and hepatobiliary laboratory abnormalities (HBLAs). One persistent ITP had a venous thrombosis and one chronic ITP had grade II myelofibrosis. We consider Eltrombopag use for the early stage ITP as effective and safe as it is in chronic ITP.

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