Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: Safety and efficacy

British Journal of Haematology

Volumn 161, Issue 3, 2013, Pages 411-423

  Kuter, David J ^a   Bussel, James B ^b   Newland, Adrian ^c   Baker, Ross I ^d   Lyons, Roger M ^e   Wasser, Jeffrey ^f   Viallard, Jean Francois ^g   Macik, Gail ^h   Rummel, Mathias ⁱ   Nie, Kun ^j   Jun, Susie ^j  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b WEILL CORNELL MEDICAL COLLEGE   (United States)

^c ROYAL LONDON HOSPITAL   (United Kingdom)

^d MURDOCH UNIVERSITY   (Australia)

^e Developmental Therapeutics Committee and Co Chair of the Genitourinary Committee   (United States)

^f UNIVERSITY OF CONNECTICUT SCHOOL OF MEDICINE   (United States)

^g UNIVERSITÉ DE BORDEAUX   (France)

^h UNIVERSITY OF VIRGINIA   (United States)

ⁱ UNIVERSITY HOSPITAL GIESSEN   (Germany)

^j AMGEN INC   (United States)

more..

Author keywords

Autoimmunity; Platelets; Thrombopoiesis; Thrombopoietic agents; TPO receptor agonists

Indexed keywords

ROMIPLOSTIM;

ADULT; ARTICLE; BLEEDING; CATHETER THROMBOSIS; CEREBROVASCULAR ACCIDENT; CHRONIC LYMPHATIC LEUKEMIA; CONTUSION; DEEP VEIN THROMBOSIS; DIARRHEA; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG SAFETY; DRUG SELF ADMINISTRATION; DRUG TOLERABILITY; EPISTAXIS; FATIGUE; FEMALE; FIBROSIS; HEART INFARCTION; HEMIPARESIS; HUMAN; IDIOPATHIC THROMBOCYTOPENIC PURPURA; LATERAL SINUS THROMBOSIS; LONG TERM CARE; LUNG EMBOLISM; LYMPHOMA; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; OUTCOME ASSESSMENT; PORTAL VEIN THROMBOSIS; PRIORITY JOURNAL; RHINOPHARYNGITIS; SIDE EFFECT; THROMBOCYTE COUNT; THROMBOCYTOPENIA; THROMBOPHLEBITIS; THROMBOSIS; TRANSIENT ISCHEMIC ATTACK; TRANSITIONAL BLINDNESS; UPPER RESPIRATORY TRACT INFECTION;

ADULT; AGED; BONE MARROW DISEASES; COMBINED MODALITY THERAPY; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HEMORRHAGE; HUMANS; INJECTIONS, SUBCUTANEOUS; MALE; MIDDLE AGED; MYOCARDIAL INFARCTION; PLATELET COUNT; PURPURA, THROMBOCYTOPENIC, IDIOPATHIC; RECEPTORS, FC; RECOMBINANT FUSION PROTEINS; SELF ADMINISTRATION; SPLENECTOMY; THROMBOCYTOPENIA; THROMBOPOIETIN; THROMBOSIS;

EID: 84876198697     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/bjh.12260     Document Type: Article

References (32)

1. Ballem, P.J., Segal, G.M., Stratton, J.R., Gernsheimer, T., Adamson, J.W. & Slichter, S.J. (1987) Mechanisms of thrombocytopenia in chronic autoimmune thrombocytopenic purpura. Evidence of both impaired platelet production and increased platelet clearance. Journal of Clinical Investigation, 80, 33-40.
2. Bottiger, L.E. & Westerholm, B. (1972) Thrombocytopenia. I. Incidence and aetiology. Acta Medica Scandinavica, 191, 535-540.
3. Brynes, R., Orazi, S., Verma, S., Brainsky, A., Bailey, C. & Bakshi, K. (2011) Evaluation of bone marrow reticulin in patients with chronic immune thrombocytopenic purpura (ITP) treated with eltrombopag -data from the EXTEND study. Blood (ASH Annual Meeting Abstracts), 118, 528.
4. Brynes, R., Orazi, A., Wong, R., Bakshi, K., Bailey, C. & Brainsky, A. (2012) A longitudinal prospective study evaluating the effects of eltrombopag treatment on bone marrow in patients with chronic immune thrombocytopenia: interim analysis at 1 year. Blood (ASH Annual Meeting Abstracts), 120, 2195.
5. 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.
6. 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.
7. Bussel, J.B., Kuter, D.J., Pullarkat, V., Lyons, R.M., Guo, M. & Nichol, J.L. (2009a) Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood, 113, 2161-2171.
8. 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. (2009b) 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.
9. Bussel, J., Rodeghiero, F., Lyons, R., Firstenberg, B., Joseph, J., Kessler, C., Terriou, L., Stasi, R., Chang, P. & Jun, S. (2011) Sustained hemostatic platelet counts in adults with immune thrombocytopenia (ITP) following cessation of treatment with the TPO receptor agonist romoiplostim: report of 9 cases. Blood (ASH Annual Meeting Abstracts), 118, 3281.
10. Chang, M., Nakagawa, P.A., Williams, S.A., Schwartz, M.R., Imfeld, K.L., Buzby, J.S. & Nugent, D.J. (2003) Immune thrombocytopenic purpura (ITP) plasma and purified ITP monoclonal autoantibodies inhibit megakaryocytopoiesis in vitro. Blood, 102, 887-895.
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. & Bussel, J.B. (2005) How I treat idiopathic thrombocytopenic purpura (ITP). Blood, 106, 2244-2251.
13. 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.
14. Gernsheimer, T.B., George, J.N., Aledort, L.M., Tarantino, M.D., Sunkara, U., Matthew Guo, D. & Nichol, J.L. (2010) Evaluation of bleeding and thrombotic events during long-term use of romiplostim in patients with chronic immune thrombocytopenia (ITP). Journal of Thrombosis and Haemostasis, 8, 1372-1382.
15. Karpatkin, S. (1997) Autoimmune (idiopathic) thrombocytopenic purpura. Lancet, 349, 1531-1536.
16. Kelton, J.G. & Gibbons, S. (1982) Autoimmune platelet destruction: idiopathic thrombocytopenic purpura. Seminars in Thrombosis and Hemostasis, 8, 83-104.
17. Khellaf, M., Michel, M., Quittet, P., Viallard, J.F., Alexis, M., Roudot-Thoraval, F., Cheze, S., Durand, J.M., Lefrere, F., Galicier, L., Lambotte, O., Panelatti, G., Slama, B., Damaj, G., Sebahoun, G., Gyan, E., Delbrel, X., Dhedin, N., Royer, B., Schleinitz, N., Rossi, J.F., Mahevas, M., Languille, L., Bierling, P. & Godeau, B. (2011) Romiplostim safety and efficacy for immune thrombocytopenia in clinical practice: 2-year results of 72 adults in a romiplostim compassionate-use program. Blood, 118, 4338-4345.
18. Kuter, D.J. (2009) Thrombopoietin and thrombopoietin mimetics in the treatment of thrombocytopenia. Annual Review of Medicine, 60, 193-206.
19. 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, T.H. Jr, Newland, A., Wasser, J.S., Hamburg, S.I., Grande, C., Lefrere, 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.
20. Kuter, D.J., Mufti, G.J., Bain, B.J., Hasserjian, R.P., Davis, W. & Rutstein, M. (2009) Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood, 114, 3748-3756.
21. 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.
22. Kuter, D.J., Mathias, S.D., Rummel, M., Mandanas, R., Giagounidis, A.A., Wang, X. & Deuson, R.R. (2012) Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care. American Journal of Hematology, 87, 558-561.
23. Li, J., Yang, C., Xia, Y., Bertino, A., Glaspy, J., Roberts, M. & Kuter, D.J. (2001) Thrombocytopenia caused by the development of antibodies to thrombopoietin. Blood, 98, 3241-3248.
24. McMillan, R. (1981) Chronic idiopathic thrombocytopenic purpura. New England Journal of Medicine, 304, 1135-1147.
25. McMillan, R., Wang, L., Tomer, A., Nichol, J. & Pistillo, J. (2004) Suppression of in vitro megakaryocyte production by antiplatelet autoantibodies from adult patients with chronic ITP. Blood, 103, 1364-1369.
26. Neunert, C., Lim, W., Crowther, M., Cohen, A., Solberg, L. Jr. & Crowther, M.A. (2011) The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood, 117, 4190-4207.
27. Newland, A., Caulier, M.T., Kappers-Klunne, M., Schipperus, M.R., Lefrere, F., Zwaginga, J.J., Christal, J., Chen, C.F. & Nichol, J.L. (2006) An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. British Journal of Haematology, 135, 547-553.
28. Newland, A., Cervinek, L., Eggermann, J., Lefrere, F. & Kreutzbauer, G. (2011) Sustained hemostatic platelet counts in adult patients with primary immune thrombocytopenia (ITP) following cessation of romiplostim - four European case studies. Haematologica, 96(Suppl. 2), 98, abstract 237.
29. Page, L.K., Psaila, B., Provan, D., Michael Hamilton, J., Jenkins, J.M., Elish, A.S., Lesser, M.L. & Bussel, J.B. (2007) The immune thrombocytopenic purpura (ITP) bleeding score: assessment of bleeding in patients with ITP. British Journal of Haematology, 138, 245-248.
30. 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.
31. Rodeghiero, F., George, J., Rummel, M., Anderson, D., Chong, B., Boda, Z., Hellmann, A., Wang, X. & Woodard, P. (2012) Results from a phase IV open-label study evaluating changes in bone marrow morphology in adult immune thrombocytopenia (ITP) patients receiving romiplostim: analysis of the 1-year romiplostim cohort. Haematologia, 97, 611.
32. Venna, N., Gonzalez, R.G. & Camelo-Piragua, S.I. (2010) Case records of the Massachusetts General Hospital. Case 11-2010. A 69-year-old woman with lethargy, confusion, and abnormalities on brain imaging. New England Journal of Medicine, 362, 1431-1437.