Repeated short-term use of eltrombopag in patients with chronic immune thrombocytopenia (ITP)

British Journal of Haematology

Volumn 160, Issue 4, 2013, Pages 538-546

  Bussel, James B ^a   Saleh, Mansoor N ^b   Vasey, Sandra Y ^c   Mayer, Bhabita ^d   Arning, Michael ^c   Stone, Nicole L ^c  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

^b GlaxoSmithKline ^*

^c GLAXOSMITHKLINE   (United States)

^d GLAXOSMITHKLINE   (United Kingdom)

Author keywords

Autoimmunity; Bleeding; Intermittent dosing; Platelet; Safety

Indexed keywords

CORTICOSTEROID; DANAZOL; ELTROMBOPAG; IMMUNOGLOBULIN;

ADULT; AGED; ARTICLE; CONTUSION; COUGHING; DIARRHEA; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG INTERMITTENT THERAPY; DRUG RESPONSE; DRUG SAFETY; DRUG WITHDRAWAL; DYSPNEA; FATIGUE; FEMALE; GLUCOSE BLOOD LEVEL; HEPATOBILIARY DISEASE; HUMAN; IDIOPATHIC THROMBOCYTOPENIC PURPURA; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; MULTIPLE CYCLE TREATMENT; OPEN STUDY; PANCREAS CANCER; PETECHIA; PHASE 2 CLINICAL TRIAL; PNEUMONIA; PRIORITY JOURNAL; RHINOPHARYNGITIS; SIDE EFFECT; SINUS CONGESTION; SPLENECTOMY; TACHYCARDIA; THROMBOCYTE COUNT; THROMBOCYTE TRANSFUSION; THROMBOCYTOPENIA; THROMBOEMBOLISM; UPPER ABDOMINAL PAIN;

ADULT; AGED; BENZOATES; CHRONIC DISEASE; DOUBLE-BLIND METHOD; DRUG ADMINISTRATION SCHEDULE; FEMALE; HEMATINICS; HUMANS; HYDRAZINES; MALE; MIDDLE AGED; PLATELET COUNT; PURPURA, THROMBOCYTOPENIC, IDIOPATHIC; PYRAZOLES; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84873082420     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/bjh.12169     Document Type: Article

References (26)

1. Afdhal, N., Dusheiko, G., Giannini, E.G., Chen, P.-J., Han, K.-H., Moshin, A., Rodriguez-Torres, M., Rugina, S., Lawitz, E., Shiffman, M.L., Tayyab, G., Poordad, F., Mostafa Kamel, Y., Brainsky, A., Geib, J., Vasey, S.Y., Patwardhan, R., Campbell, F. & Theodore, D. (2011) Final results of ENABLE 1, a phase 3, multicenter study of eltrombopag as an adjunct for antiviral treatment of hepatitis C virus-related chronic liver disease associated with thrombocytopenia. Hepatology, 54(suppl. 1), 1427A.abstract LB-3.
2. AREDS (2001) The age-related eye disease study (AREDS) system for classifying cataracts from photographs: AREDS report no. 4. American Journal of Ophthalmology, 131, 167-175.
3. British Committee for Standardization in Haematology (2003) Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. British Journal of Haematology, 120, 574-596.
4. Bussel, J.B., Kuter, D.J., George, J.N., McMillan, R., Aledort, L.M., Conklin, G.T., Lichtin, A.E., Lyons, R.M., Nieva, J., Wasser, J.S., Wiznitzer, I., Kelly, R., Chen, C.F. & Nichol, J.L. (2006) AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP. New England Journal of Medicine, 355, 1672-1681.
5. Bussel, J.B., Cheng, G., Saleh, M.N., Psaila, B., Kovaleva, L., Meddeb, B., Kloczko, J., Hassani, H., Mayer, B., Stone, N.L., Arning, M., Provan, D. & Jenkins, J.M. (2007) Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. New England Journal of Medicine, 357, 2237-2247.
6. Bussel, J.B., Provan, D., Shamsi, T., Cheng, G., Psaila, B., Kovaleva, L., Salama, A., Jenkins, J.M., Roychowdhury, D., Mayer, B., Stone, N. & Arning, M. (2009a) Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet, 373, 641-648.
7. Bussel, J.B., Kuter, D.J., Pullarkat, V., Lyons, R.M., Guo, M. & Nichol, J.L. (2009b) Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood, 113, 2161-2171.
8. Bussel, J.B., Zhang, J., Tang, S., McIntosh, J. & Kuter, D.J. (2010a) A randomized, double-blind, placebo-controlled phase II trial on the efficacy, safety and tolerability of E5501 (AKR501) in subjects with chronic immune thrombocytopenia (ITP). Blood (ASH Annual Meeting Abstracts), 116, 38, abstract #71.
9. Bussel, J.B., Frelinger, III, A.L., Mitchell, W.B., Pinheiro, M.P., Barnard, M.R., Lampa, M., Psaila, B., Boulad, N., Tamary, H. & Michelson, A.D. (2010b) Platelet function and response to thrombopoietin mimetics in Wiskott-Aldrich syndrome/X-linked thrombocytopenia. Blood (ASH Annual Meeting Abstracts), 116, 613. abstract 1429.
10. Chawla, S.P., Staddon, A.P., Messam, C., Graham, A.M. & Mostafa Kamel, Y.M. (2010) An open-label, dose-ranging study to assess the safety, efficacy, and pharmacokinetics of eltrombopag in treating thrombocytopenia in patients with advanced sarcomas receiving doxorubicin and ifosfamide. Journal of Clinical Oncology, 28(suppl. 15), 64s, abstract # TPS334.
11. Cheng, G., Saleh, M.N., Marcher, C., Vasey, S., Mayer, B., Aivado, M., Arning, M., Stone, N.L. & Bussel, J.B. (2011) Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet, 377, 393-402.
12. Cines, D.B. & Blanchette, V.S. (2002) Immune thrombocytopenic purpura. New England Journal of Medicine, 346, 995-1008.
13. Cines, D.B. & Bussel, J.B. (2005) How I treat idiopathic thrombocytopenic purpura (ITP). Blood, 106, 2244-2251.
14. Erickson-Miller, C.L., Delorme, E., Tian, S.S., Hopson, C.B., Landis, A.J., Valoret, E.I., Sellers, T.S., Rosen, J., Miller, S.G., Luengo, J.I., Duffy, K.J. & Jenkins, J.M. (2009) Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells, 27, 424-430.
15. Garnock-Jones, K.P. & Keam, S.J. (2009) Eltrombopag. Drugs, 69, 567-576.
16. George, J.N., Woolf, S.H., Raskob, G.E., Wasser, J.S., Aledort, L.M., Ballem, P.J., Blanchette, V.S., Bussel, J.B., Cines, D.B., Kelton, J.G., Lichtin, A.E., McMillan, R., Okerbloom, J.A., Regan, D.H. & Warrier, I. (1996) Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood, 88, 3-40.
17. Kalota, A., Brennan, K., Erickson-Miller, C.L., Danet, G., Carroll, M. & Gewirtz, A.M. (2004) Effects of SB559457, a novel small molecule thrombopoietin receptor (TpoR) agonist, on human hematopoietic cell growth and differentiation. Blood (ASH Annual Meeting Abstracts), 104, 796a, abstract 2913.
18. Kuter, D.J., Bussel, J.B., Lyons, R.M., Pullarkat, V., Gernsheimer, T.B., Senecal, F.M., Aledort, L.M., George, J.N., Kessler, C.M., Sanz, M.A., Liebman, H.A., Slovick, F.T., de Wolf, J.T., Bourgeois, E., Guthrie, Jr, T.H., Newland, A., Wasser, J.S., Hamburg, S.I., Grande, C., Lefrère, F., Lichtin, A.E., Tarantino, M.D., Terebelo, H.R., Viallard, J.F., Cuevas, F.J., Go, R.S., Henry, D.H., Redner, R.L., Rice, L., Schipperus, M.R., Guo, D.M. & Nichol, J.L. (2008) Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet, 371, 395-403.
19. Kuter, D.J., Rummel, M., Boccia, R., Macik, B.G., Pabinger, I., Selleslag, D., Rodeghiero, F., Chong, B.H., Wang, X. & Berger, D.P. (2010) Romiplostim or standard of care in patients with immune thrombocytopenia. New England Journal of Medicine, 363, 1889-1899.
20. McHutchison, J.G., Dusheiko, G., Shiffman, M.L., Rodriguez-Torres, M., Sigal, S., Bourliere, M., Berg, T., Gordon, S.C., Campbell, F.M., Theodore, D., Blackman, N., Jenkins, J., Afdhal, N.H. & TPL102357 Study Group (2007) Eltrombopag for thrombocytopenia in patients with cirrhosis associated with hepatitis C. New England Journal of Medicine, 357, 2227-2236.
21. Olnes, M.J., Scheinberg, P., Calvo, K.R., Desmond, R., Tang, Y., Dumitriu, B., Parikh, A.R., Soto, S., Biancotto, A., Feng, X., Lozier, J., Wu, C.O., Young, N.S. & Dunbar, C.E. (2012) Eltrombopag and improved hematopoiesis in refractory aplastic anemia. New England Journal of Medicine, 367, 11-19.
22. Pecci, A., Gresele, P., Klersy, C., Savoia, A., Noris, P., Fierro, T., Bozzi, V., Mezzasoma, A.M., Melazzini, F. & Balduini, C.L. (2010) Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations. Blood, 116, 5832-5837.
23. Provan, D., Stasi, R., Newland, A.C., Blanchette, V.S., Bolton-Maggs, P., Bussel, J.B., Chong, B.H., Cines, D.B., Gernsheimer, T.B., Godeau, B., Grainger, J., Greer, I., Hunt, B.J., Imbach, P.A., Lyons, G., McMillan, R., Rodeghiero, F., Sanz, M.A., Tarantino, M., Watson, S., Young, J. & Kuter, D.J. (2010) International consensus report on the investigation and management of primary immune thrombocytopenia. Blood, 115, 168-186.
24. Saleh, M.N., Cheng, G., Bussel, J.B., Burgess, P., Marcello, L., Bailey, C.K. & Brainsky, A. (2011) Safety and efficacy of extended treatment with eltrombopag in adults with chronic immune thrombocytopenia (ITP) from June 2006 to February 2011. Blood (ASH Annual Meeting Abstracts), 118, 1417-1418, abstract 3296.
25. Stasi, R. & Provan, D. (2004) Management of immune thrombocytopenic purpura in adults. Mayo Clinic Proceedings, 79, 504-522.
26. Wroblewski, S., Shi, W., Mudd, Jr, P. & Aivado, M. (2010) Eltrombopag in thrombocytopenic patients with advanced myelodysplastic syndromes (MDS) or secondary myeloid leukemia after MDS: a phase I/II study. Journal of Clinical Oncology, 28(suppl. 15), 27s, abstract TPS184.