Which patients with myelofibrosis should receive ruxolitinib therapy? ELN-SIE evidence-based recommendations

Leukemia

Volumn 31, Issue 4, 2017, Pages 882-888

  Marchetti, M ^a   Barosi, G ^b   Cervantes, F ^c   Birgegard G ^d   Griesshammer, M ^e   Harrison, C ^f   Hehlmann, R ^g   Kiladjian, J J ^h   Kroger N ⁱ   McMullin, M F ^j   Passamonti, F ^k   Vannucchi, A ^l   Barbui, T ^m  

^a Ospedale Cardinal Massaia   (Italy)

^b FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^c HOSPITAL CLÍNIC   (Spain)

^d UPPSALA UNIVERSITY HOSPITAL   (Sweden)

^e KLINIKUM MINDEN   (Germany)

^f GUY'S AND ST THOMAS' NHS FOUNDATION TRUST   (United Kingdom)

^g UNIVERSITY OF HEIDELBERG   (Germany)

^h Centre d'Investigations Cliniques CIC 1427   (France)

ⁱ UNIVERSITY MEDICAL CENTER HAMBURG EPPENDORF   (Germany)

^j QUEEN'S UNIVERSITY BELFAST   (United Kingdom)

^k UNIVERSITY OF FLORENCE   (Italy)

^l OSPEDALE DI CIRCOLO   (Italy)

^m PAPA GIOVANNI XXIII HOSPITAL   (Italy)

more..

Author keywords

[No Author keywords available]

Indexed keywords

RUXOLITINIB; JANUS KINASE 1; JANUS KINASE 2; PROTEIN KINASE INHIBITOR; PYRAZOLE DERIVATIVE;

ARTICLE; BLEEDING; CANCER CHEMOTHERAPY; CANCER PROGNOSIS; CANCER SURVIVAL; EVIDENCE BASED MEDICINE; HUMAN; INFECTION RISK; INTERNATIONAL PROGNOSTIC SCORING SYSTEM; MEDICAL SOCIETY; MYELOFIBROSIS; PATIENT SAFETY; PHASE 2 CLINICAL TRIAL (TOPIC); PORTAL HYPERTENSION; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); SPLEEN SIZE; SPLENOMEGALY; THROMBOCYTOPENIA; VEIN THROMBOSIS; ANTAGONISTS AND INHIBITORS; COMORBIDITY; HEMORRHAGE; HYPERTENSION, PORTAL; INFECTION; METABOLISM; MOLECULARLY TARGETED THERAPY; MORTALITY; PHENOTYPE; PRIMARY MYELOFIBROSIS; TREATMENT OUTCOME;

COMORBIDITY; HEMORRHAGE; HUMANS; HYPERTENSION, PORTAL; INFECTION; JANUS KINASE 1; JANUS KINASE 2; MOLECULAR TARGETED THERAPY; PHENOTYPE; PRIMARY MYELOFIBROSIS; PROTEIN KINASE INHIBITORS; PYRAZOLES; SPLENOMEGALY; TREATMENT OUTCOME;

EID: 84995776521     PISSN: 08876924     EISSN: 14765551     Source Type: Journal    
DOI: 10.1038/leu.2016.283     Document Type: Article

References (46)

1. Sant M, Minicozzi P, Mounier M, Anderson LA, Brenner H, Holleczek B et al. EUROCARE-5 Working Group. Survival for haematological malignancies in Europe between 1997 and 2008 by region and age: results of EUROCARE-5, a populationbased study. Lancet Oncol 2014; 15: 931-942
2. Lichtenberg FR. The impact of pharmaceutical innovation on premature cancer mortality in Switzerland, 1995-2012. Eur J Health Econ 2016; 17: 833-854
3. Lichtenberg FR. The impact of pharmaceutical innovation on premature cancer mortality in Canada, 2000-2011. Int J Health Econ Manag 2016; 5: 339-359
4. Harrison C, Kiladjian JJ, Al-Ali HK, Gisslinger H, Waltzman R, Stalbovskaya V et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med 2012; 366: 787-798
5. Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med 2012; 366: 799-807
6. Martí-Carvajal AJ, Anand V, Solà I. Janus kinase-1 and Janus kinase-2 inhibitors for treating myelofibrosis. Cochrane Database Syst Rev 2015; 4: CD010298
7. Reilly JT, McMullin MF, Beer PA, Butt N, Conneally E, Duncombe AS et al. Use of JAK inhibitors in the management of myelofibrosis: a revision of the British Committee for Standards in Haematology Guidelines for Investigation and Management of Myelofibrosis 2012. Br J Haematol 2014; 167: 418-420
8. Vannucchi AM, Barbui T, Cervantes F, Harrison C, Kiladjian JJ, Kroger N et al. Philadelphia chromosome-negative chronic myeloproliferative neoplasms: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol 2015, v85-v99
9. Ho PJ, Marlton P, Tam C, Stevenson W, Ritchie D, Bird R et al. Practical management of myelofibrosis with ruxolitinib. Intern Med J 2015; 45: 1221-1230
10. Atkins D, Best D, Briss PA, Eccels M, Falck-Ytter Y, Flottrop S et al. Grading quality of evidence and strength of recommendations. BMJ 2004; 328: 1490-1498
11. NICE appraisal 2016: (accessed on 25 April 2016) https://www.nice.org.uk/gui dance/TA386/documents/final-appraisal-determination-document
12. William PL, Webb C. The Delphi technique: a methodological discussion. J Adv Nurs 1994; 19: 180-186
13. Harrison CN, Vannucchi AM, Kiladjian JJ, Al-Ali HK, Gisslinger H, Knoops L et al. Long-term findings from COMFORT-III, a phase 3 study of ruxolitinib vs best available therapy for myelofibrosis. Leukemia 2016; 30: 1701-1707
14. Mesa RA, Kiladjian JJ, Verstovsek S, Al-Ali HK, Gotlib J, Gisslinger H et al. Comparison of placebo and best available therapy for the treatment of myelofibrosis in the phase 3 COMFORT studies. Haematologica 2014; 99: 292-298
15. Mead AJ, Milojkovic D, Knapper S, Garg M, Chacko J, Farquharson M et al. Response to ruxolitinib in patients with intermediate-1-, intermediate-2-, and high-risk myelofibrosis: results of the UK ROBUST Trial. Br J Haematol 2015; 170: 29-39
16. Al-Ali Haifa, Griesshammer M, Coutre P, Waller CF, Liberati AM, Schafausen P et al. Safety and efficacy of ruxolitinib in an open-label, multicenter, single-arm phase 3b expanded-access study in patients with myelofibrosis: a snapshot of 1144 patients in the JUMP trial. Haematologica 2016; 101: 1065-1073
17. Kiladjian JJ, Giraudier S, Cassinat B. Interferon-alpha for the therapy of myeloproliferative neoplasms: targeting the malignant clone. Leukemia 2016; 30: 776-781
18. Ianotto JC, Boyer-Perrard F, Gyan E, Laribi K, Cony-Makhoul P, Demory JL et al. Efficacy and safety of pegylated-interferon a-2a in myelofibrosis: a study by the FIM and GEM French cooperative groups. Br J Haematol 2013; 162: 783-791
19. Silver RT, Vandris K, Goldman JJ. Recombinant interferon-A may retard progression of early primary myelofibrosis: a preliminary report. Blood 2011; 117: 6669-6672
20. Gowin K, Thapaliya P, Samuelsson J, Harrison C, Radia D, Andreasson B et al. Experience with pegylated Interferon alpha-2a in advanced myeloproliferative neoplasms in an international cohort of 118 patients. Haematologica 2012; 97: 1570-1573
21. Scherber R, Duecka A, Geyer H, Kosiorek H, Kiladjian JJ, Slots S et al. Symptoms, Risk Classification, and Spleen Size in JAK2 Inhibitor-Naive Myelofibrosis: Implications for JAK2 Inhibitor Treatment. E1345. 21st EHA Meeting; 9-12 June 2016; Copenhagen, Denmark. Available at: http://learningcenter.ehaweb.org/eha/2016/ 21st/137125/eha.annual.congress.eha21.abstract.book.html?f=p5m6l12006
22. Vannucchi AM, Kantarjian HM, Kiladjian JJ, Gotllib J, Cervantes F, Mesa RA et al. A pooled analysis of overall survival in COMFORT-I and COMFORT-II, 2 randomized phase III trials of ruxolitinib for the treatment of myelofibrosis. Haematologica 2015; 100: 1139-1145
23. Verstovsek S, Kantarjian HM, Estrov Z, Cortes JA, Thoams DA, Kadia T et al. Longterm outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls. Blood 2012; 120: 1202-1209
24. Passamonti F, Maffioli M, Cervantes F, Vannucchi AM, Morra E, Barbui T 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: 1833-1835
25. Talpaz M, Paquette R, Afrin L, Hamburg SI, Prchal JT, Jamieson K et al. Interim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts. J Hematol Oncol 2013; 6: 81
26. Griesshammer M, Vannucci AM, Le Coutre P, Tavares RS, Al-Ali HK, Raani P et al. Safety and efficacy of ruxolitinib in patients with low platelets enrolled in a phase 3b expanded-access study in myelofibrosis (MF) ASH 2014. 1859
27. Vannucchi AM, Gisslinger H, Harrison CN, Al-Ali HK, Pungolino E, Kiladjiean J-J et al. EXPAND: a phase 1b, open-label, dose-finding study of ruxolitinib in patients with myelofibrosis (MF) and low platelet counts (50 × 109/L to 99 × 109/L) at baseline. ASH 2015. 2817
28. Galli S, McLornan D, Harrison C. Safety evaluation of ruxolitinib for treating myelofibrosis. Expert Opin Drug Saf 2014; 13: 967-976
29. Harrison C, Mesa R, Ross D, Mead A, Keohane C, Gotlib J et al. Practical management of patients with myelofibrosis receiving ruxolitinib. Expert Rev Hematol 2013; 6: 511-523
30. Pieri L, Paoli C, Arena U, Marra F, Mori F, Zucchini M et al. Long-term follow-up of a phase 2 study of ruxolitinib in patients with splanchnic vein thrombosis associated with myeloproliferative neoplasms, ASH 2015: 2803
31. Colaizzo D, Amitrano L, Guardascione MA, Tiscia GL, D'Andrea G, Longo VA et al. Outcome of patients with splanchnic venous thrombosis presenting without overt MPN: a role for the JAK2 V617F mutation re-evaluation. Thromb Res 2013; 132: e99-e104
32. Ageno W, Riva N, Schulman S, Beyer-Westendorf J, Bang SM, Senzolo M et al. Long-term clinical outcomes of splanchnic vein thrombosis: results of an International Registry. JAMA Intern Med 2015; 175: 1474-1480
33. Benjamini O, Jain P, Estrov Z, Kantarjian HM, Verstovsek S. Therapeutic effects of ruxolitinib in patients with myelofibrosis without clinically significant splenomegaly. Blood 2012; 120: 2768-2769
34. Verstovsek S, Kantanrjian H, Mesa RA, Pardanani AD, Cortes-Franco J, Thomas DA et al. Safety and efficacy of INCB18424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med 2010; 363: 1117-1127
35. Isfort S, Kaifie A, Bennemann K, Jost E, Panse J, Bruemmerndorf TH et al. Comorbidity scales in patients with myeloproliferative neoplasms: which one fits best? Blood 2014; 124: 1828
36. EPAR EMA. http://www.ema.europa.eu/ema/index.jsp?curl = pages/medicines/ human/medicines/002464/human-med-001568.jspandmid = vvv WC0b01ac058 001d124. Accessed on 4 July 2016
37. FDA: ruxolitinib prescribing information www.accessdata.fda.gov/drugsatfda- docs/label/2011/202192lbl.pdf. Accessed on 4 July 2016
38. Tefferi A, Cervantes F, Mesa R, Passamonti F, Verstovsek S, Vannucchi AM et al. Revised response criteria for myelofibrosis: International Working Group-Myeloproliferative neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus report. Blood 2013; 122: 1395-1398
39. Pardanani A, Tefferi A. Definition and management of ruxolitinib treatment failure in myelofibrosis. Blood Cancer J 2014; 4: e268
40. Kroger NM, Deeg JH, Olavarria E, Niederwieser D, Bacigalupo A, Barbui T et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by EBMT/ELN international working group. Leukemia 2015; 29: 2126-2133
41. Mehta J, Wang H, Fryzek JP, Iqbal SU, Mesa R. Health resource utilization and cost associated with myeloproliferative neoplasms in a large United States health plan. Leuk Lymphoma 2014; 55: 2368-2374
42. Ouagari K, Knight CJ, Mendelson ET. Cost-effectiveness of ruxolitinib versus bestavailable therapy for medical treatment of myelofibrosis: Canadian societal perspective. Am Soc Hematol Meeting 2012; 4255
43. Loblaw DA, Prestrud AA, Somerfield MR, Oliver TK, Brouwers MC, Nam RK et al. American Society of Clinical Oncology Clinical Practice Guidelines: formal systematic review-based consensus methodology. J Clin Oncol 2012; 30: 3136-3140
44. Passamonti F, Cervantes F, Vannucchi AM, Morra E, Rumi E, Pereira A. A dynamic prognostic model to predict survival in primary myelofibrosis: a study by the IWGMRT (International Working Group for Myeloproliferative Neoplasms Research and Treatment). Blood 2010; 115: 1703-1708
45. Cervantes F, Dupriez B, Pereira A, Passamonti F, Reilly JT, Morra E 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: 2895-2901
46. Scherber R, Dueck AC, Johansson P, Barbui T, Barosi G, Vannucchi AM et al. The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): international prospective validation and reliability trial in 402 patients. Blood 2011; 118: 401-408