1,598
Views
3
CrossRef citations to date
0
Altmetric
Hematology

Pharmacoeconomics of ruxolitinib therapy in patients with myelofibrosis

, , &
Pages 424-431 | Accepted 15 Dec 2015, Published online: 08 Jan 2016

References

  • Cervantes F, Vannucchi AM, Kiladjian JJ, et al. Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood 2013;122:4047-53
  • Verstovsek S, Mesa RA, Gotlib J, et al. Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis: results of a median 3-year follow-up of COMFORT-I. Haematologica 2015;100:479-88
  • Harrison C, Kiladjian JJ, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med 2012;366:787-98
  • Mesa RA, Niblack J, Wadleigh M, et al. The burden of fatigue and quality of life in myeloproliferative disorders (MPDs): an international Internet-based survey of 1179 MPD patients. Cancer 2007;109:68-76
  • Geyer HL, Mesa RA. Therapy for myeloproliferative neoplasms: when, which agent, and how? Blood 2014;124:3529-37
  • Cervantes F, Dupriez B, Pereira A, et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 2009;113:2895-901
  • Tefferi A, Siragusa S, Hussein K, et al. Transfusion-dependency at presentation and its acquisition in the first year of diagnosis are both equally detrimental for survival in primary myelofibrosis–prognostic relevance is independent of IPSS or karyotype. Am J Hematol 2010;85:14-17
  • Passamonti F, Rumi E, Elena C, et al. Incidence of leukaemia in patients with primary myelofibrosis and RBC-transfusion-dependence. Br J Haematol 2010;150:719-21
  • Cervantes F, Pereira A. Prognostication in primary myelofibrosis. Curr Hematol Malig Rep 2012;7:43-9
  • Ostojic A, Vrhovac R, Verstovsek S. Ruxolitinib for the treatment of myelofibrosis: its clinical potential. Ther Clin Risk Manag 2012;8:95-103
  • Tefferi A. Primary myelofibrosis. Cancer Treat Res 2008;142:29-49
  • Mehta J, Wang H, Fryzek JP, et al. Health resource utilization and cost associated with myeloproliferative neoplasms in a large United States health plan. Leuk Lymphoma 2014;55:2368-74
  • Price G, Pohl G, Xie J, et al. A retrospective observational study of annual healthcare costs for patients with forms of Myeloproliferative Neoplasms (MPN). Blood (ASH Annual Meeting Abstracts) 2011;118:2060
  • Gimenez E, Besses C, Boque C, et al. Indirect and non-medical economic burden, quality-of-life, and disabilities of the myelofibrosis disease in Spain. J Med Econ 2014;17:435-41
  • Infarmed - Autoridade Nacional do Medicamento e Produtos de Saúde IP. Orientações Metodológicas para Estudos de Avaliação Económica de Medicamentos. http://www.infarmed.pt; 1999
  • Jonsson L, Sandin R, Ekman M, et al. Analyzing overall survival in randomized controlled trials with crossover and implications for economic evaluation. Value Health 2014;17:707-13
  • Robins J, Tsiatis A. Correcting for non-compliance in randomized trials using rank preserving structural failure time models. Communications in Statistics - Theory and Methods 1991;20:2609-31
  • Myelofibrosis Audit: clinical management, resource utilisation and outcome in primary and secondary myelofibrosis. Haematological Malignancy Research Network, 2012. http://www.hmrn.org/Resources/Reports/Documents/HMRN_Myelofibrosis_Audit.pdf.
  • NICE. Ruxolitinib for disease-related splenomegaly or symptoms in adults with myelofibrosis. NICE technology appraisal guidance 289. guidance.nice.org.uk/ta289. June 2013
  • RTI Health Solutions. Treatment patterns, clinical outcomes, and the associated economic burden of myelofibrosis in the United States.: Novartis data on file JAK-001-12, 2011
  • Ministério da Saúde. Diário da República, 1.a série — N.° 80 — Portaria n.° 163/2013 de 24 de Abril de 2013. : Ministério da Saúde, 2013
  • Vannucchi AM, Kantarjian HM, Kiladjian JJ, et al. A pooled analysis of overall survival in COMFORT-I and COMFORT-II, 2 randomized phase 3 trials of ruxolitinib for the treatment of myelofibrosis. Haematologica 2015;Sep 100(9):1139-45
  • Wade R, Rose M, Neilson AR, et al. Ruxolitinib for the treatment of myelofibrosis: a NICE single technology appraisal. Pharmacoeconomics 2013;31:841-52
  • Evidence Review Group Report. Ruxolitinib for the treatment of myelofibrosis. CRD and CHE Technology Assessment Group University of York, 2012. http://www.nice.org.uk/guidance/ta289/documents/myelofibrosis-splenomegaly-symptoms-ruxolitinib-evidence-review-group-report2.
  • Passamonti F, Maffioli M, Cervantes F, et al. Impact of ruxolitinib on the natural history of primary myelofibrosis: a comparison of the DIPSS and the COMFORT-2 cohorts. Blood 2014;123:1833-5
  • Caro JJ, Briggs AH, Siebert U, et al. Modeling good research practices–overview: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force–1. Value Health 2012;15:796-803
  • Russell L, Siegel J, Daniels N, et al. Cost-effectiveness analysis as a guide to resource allocation in health: roles and limitations. Cost-effectiveness in health and medicine. Oxford: Oxford University Press, 1996
  • Latimer NR. Survival analysis for economic evaluations alongside clinical trials–extrapolation with patient-level data: inconsistencies, limitations, and a practical guide. Med Decis Making 2013;33:743-54
  • Mesa RA, Schwager S, Radia D, et al. The Myelofibrosis Symptom Assessment Form (MFSAF): an evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis. Leuk Res 2009;33:1199-203
  • Harrison CN, Mesa RA, Kiladjian JJ, et al. Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy. Br J Haematol 2013;162:229-39
  • Kontodimopoulos N, Aletras VH, Paliouras D, et al. Mapping the cancer-specific EORTC QLQ-C30 to the preference-based EQ-5D, SF-6D, and 15D instruments. Value Health 2009;12:1151-7
  • Mesa R, Miller CB, Thyne M, et al. Impact of Myeloproliferative Neoplasms (MPNs) on patients’ overall health and productivity: results from the MPN LANDMARK SURVEY in the United States. Blood 2014;124:3183
  • Jaekel N, Behre G, Behning A, et al. Allogeneic hematopoietic cell transplantation for myelofibrosis in patients pretreated with the JAK1 and JAK2 inhibitor ruxolitinib. Bone Marrow Transpl 2014;49:179-84
  • Ballinger TJ, Savani BN, Gupta V, et al. How we manage JAK inhibition in allogeneic transplantation for myelofibrosis. Eur J Haematol 2015;94:115-9
  • Gupta V, Gotlib J, Radich JP, et al. Janus kinase inhibitors and allogeneic stem cell transplantation for myelofibrosis. Biol Blood Marrow Transpl 2014;20:1274-81
  • Saret CJ, Winn AN, Shah G, et al. Value of innovation in hematologic malignancies: a systematic review of published cost-effectiveness analyses. Blood 2015;125:1866-9

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.