Romiplostim or standard of care in patients with immune thrombocytopenia

New England Journal of Medicine

Volumn 363, Issue 20, 2010, Pages 1889-1899

  Kuter, David J ^a   Rummel, Mathias ^b   Boccia, Ralph ^d   Macik, B Gail ^e   Pabinger, Ingrid ^c   Selleslag, Dominik ^f   Rodeghiero, Francesco ^g   Chong, Beng H ^h   Wang, Xuena ⁱ   Berger, Dietmar P ⁱ  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b UNIVERSITY HOSPITAL GIESSEN   (Germany)

^c MEDICAL UNIVERSITY OF VIENNA   (Austria)

^d Center for Cancer and Blood Disorders   (United States)

^e UNIVERSITY OF VIRGINIA   (United States)

^f General Hospital St Jan   (Belgium)

^g SAN BORTOLO HOSPITAL   (Italy)

^h UNIVERSITY OF NEW SOUTH WALES   (Australia)

ⁱ AMGEN INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AZATHIOPRINE; DANAZOL; GLUCOCORTICOID; IMMUNOGLOBULIN; RETICULIN; RITUXIMAB; ROMIPLOSTIM;

ADULT; AGED; ARTICLE; BLEEDING; BLOOD TRANSFUSION; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG SAFETY; DRUG TREATMENT FAILURE; FEMALE; HEALTH CARE QUALITY; HEMATOLOGIC MALIGNANCY; HUMAN; LYMPHOMA; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; MYELODYSPLASTIC SYNDROME; OPEN STUDY; OUTCOME ASSESSMENT; PRIORITY JOURNAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; SIDE EFFECT; SPLENECTOMY; THROMBOCYTE COUNT; THROMBOCYTE TRANSFUSION; THROMBOCYTOPENIA; THROMBOSIS; TREATMENT DURATION; TREATMENT RESPONSE;

EID: 78149479585     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1002625     Document Type: Article

References (27)

1. Nugent D, McMillan R, Nichol JL, Slichter SJ. Pathogenesis of chronic immune thrombocytopenia: increased platelet destruction and/or decreased platelet production. Br J Haematol 2009;146:585-96.
2. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood 2010; 115:168-86.
3. George JN. Treatment options for chronic idiopathic (immune) thrombocytopenic purpura. Semin Hematol 2000;37: Suppl 1:31-4.
4. George JN, Kojouri K, Perdue JJ, Vesely SK. Management of patients with chronic, refractory idiopathic thrombocytopenic purpura. Semin Hematol 2000;37:290-8.
5. Kaznelson P. Verschwinden der hämorrhagische Diathese bei einem Falle von "essentieller Thrombopenie" (Frank) nach Milzextirpation: splenogene thrombolytische Purpura. Wien Klin Wochenschr 1916;29:1451-4.
6. George JN. Diagnosis, clinical course, and management of idiopathic thrombocytopenic purpura. Curr Opin Hematol 1996;3:335-40.
7. Mikhael J, Northridge K, Lindquist K, Kessler C, Deuson R, Danese M. Shortterm and long-term failure of laparoscopic splenectomy in adult immune thrombocytopenic purpura patients: a systematic review. Am J Hematol 2009;84:743-8.
8. Kojouri K, Vesely SK, Terrell DR, George JN. Splenectomy for adult patients with idiopathic thrombocytopenic purpura: a systematic review to assess longterm platelet count responses, prediction of response, and surgical complications. Blood 2004;104:2623-34.
9. Brasseur P, Gorenflot A. Human babesial infections in Europe. Rocz Akad Med Bialymst 1996;41:117-22.
10. Deodhar HA, Marshall RJ, Barnes JN. Increased risk of sepsis after splenectomy. BMJ 1993;307:1408-9.
11. Schwartz PE, Sterioff S, Mucha P, Melton LJ III, Offord KP. Postsplenectomy sepsis and mortality in adults. JAMA 1982;248:2279-83.
12. Crary SE, Buchanan GR. Vascular complications after splenectomy for hematologic disorders. Blood 2009;114:2861-8.
13. Robinette CD, Fraumeni JF Jr. Splenectomy and subsequent mortality in veterans of the 1939-45 war. Lancet 1977;2: 127-9.
14. Purcell PL, Crary SE, Adix LM, Alder AC, Buchanan GR. Postsplenectomy vascular complications: feasibility of studying patients with splenectomy following trauma. Am J Hematol 2009;84:316-7.
15. Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet 2008;371:395-403.
16. Bussel JB, Provan D, Shamsi T, et al. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet 2009;373:641-8.
17. Gernsheimer TB, George JN, Aledort LM, et al. Evaluation of bleeding and thrombotic events during long-term use of romiplostim in patients with chronic immune thrombocytopenia (ITP). J Thromb Haemost 2010;8:1372-82.
18. Bussel J, Kuter DJ, Newland A, et al. Long-term efficacy and safety of romiplostim for the treatment of patients with chronic immune thrombocytopenia (ITP): 5-year update from an open-label extension study. Blood 2009;114:Suppl. abstract.
19. Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood 2009;113:2161-71. [Erratum, Blood 2009;113:4822.]
20. George JN, Woolf SH, Raskob GE, et al. Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood 1996;88:3-40.
21. George JN, Mathias SD, Go RS, et al. Improved quality of life for romiplostimtreated patients with chronic immune thrombocytopenic purpura: results from two randomized, placebo-controlled trials. Br J Haematol 2009;144:409-15.
22. Mathias SD, Gao SK, Rutstein M, Snyder CF, Wu AW, Cella D. Evaluating clinically meaningful change on the ITP-PAQ: preliminary estimates of minimal important differences. Curr Med Res Opin 2009; 25:375-83.
23. Godeau B, Porcher R, Fain O, et al. Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood 2008;112:999-1004.
24. Hernández F, Linares M, Colomina P, et al. Dapsone for refractory chronic idiopathic thrombocytopenic purpura. Br J Haematol 1995;90:473-5.
25. Cooper N, Woloski BM, Fodero EM, et al. Does treatment with intermittent infusions of intravenous anti-D allow a proportion of adults with recently diagnosed immune thrombocytopenic purpura to avoid splenectomy? Blood 2002;99: 1922-7.
26. Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood 2009;114:3748-56.
27. Saleh MN, Bussel JB, Meyer O, et al. Results of bone marrow examinations in patients with chronic immune thrombocytopenic purpura treated with eltrombopag. Blood 2009;114:1326.