Difference between revisions of "Myelofibrosis - null regimens"
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===References=== | ===References=== | ||
| − | # '''COMFORT-I:''' Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger M, Miller C, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner E, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Koumenis IL, Sun W, Sandor V, Kantarjian HM. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012 Mar 1;366(9):799-807. [https://www.nejm.org/doi/full/10.1056/NEJMoa1110557 link to original article] [https://www.nejm.org/doi/suppl/10.1056/NEJMoa1110557/suppl_file/nejmoa1110557_appendix.pdf supplementary appendix] [https://www.nejm.org/doi/suppl/10.1056/NEJMoa1110557/suppl_file/nejmoa1110557_protocol.pdf trial protocol] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4822164/ link to PMC article] [https:// | + | # '''COMFORT-I:''' Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger M, Miller C, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner E, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Koumenis IL, Sun W, Sandor V, Kantarjian HM. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012 Mar 1;366(9):799-807. [https://www.nejm.org/doi/full/10.1056/NEJMoa1110557 link to original article] [https://www.nejm.org/doi/suppl/10.1056/NEJMoa1110557/suppl_file/nejmoa1110557_appendix.pdf supplementary appendix] [https://www.nejm.org/doi/suppl/10.1056/NEJMoa1110557/suppl_file/nejmoa1110557_protocol.pdf trial protocol] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4822164/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/22375971 PubMed] |
| − | ## '''Update:''' Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH, Arcasoy MO, Hexner EO, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Sun W, Sandor V, Kantarjian HM. Efficacy, safety and survival with ruxolitinib treatment in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I. Haematologica. 2013 Dec;98(12):1865-71. Epub 2013 Sep 13. [http://www.haematologica.org/content/98/12/1865.full link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3856961/ link to PMC article] [https:// | + | ## '''Update:''' Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH, Arcasoy MO, Hexner EO, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Sun W, Sandor V, Kantarjian HM. Efficacy, safety and survival with ruxolitinib treatment in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I. Haematologica. 2013 Dec;98(12):1865-71. Epub 2013 Sep 13. [http://www.haematologica.org/content/98/12/1865.full link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3856961/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/24038026 PubMed] |
| − | ## '''Update:''' Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH, Arcasoy MO, Hexner EO, Lyons RM, Raza A, Vaddi K, Sun W, Peng W, Sandor V, Kantarjian H. Three-year efficacy, overall survival, and safety of ruxolitinib therapy in patients with myelofibrosis from the COMFORT-I study. Haematologica. 2015 Apr;100(4):479-88. Epub 2015 Jan 23. [http://www.haematologica.org/content/100/4/479.long link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4380721/ link to PMC article] [https:// | + | ## '''Update:''' Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH, Arcasoy MO, Hexner EO, Lyons RM, Raza A, Vaddi K, Sun W, Peng W, Sandor V, Kantarjian H. Three-year efficacy, overall survival, and safety of ruxolitinib therapy in patients with myelofibrosis from the COMFORT-I study. Haematologica. 2015 Apr;100(4):479-88. Epub 2015 Jan 23. [http://www.haematologica.org/content/100/4/479.long link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4380721/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/25616577 PubMed] |
| − | ## '''Pooled update:''' Vannucchi AM, Kantarjian HM, Kiladjian JJ, Gotlib J, Cervantes F, Mesa RA, Sarlis NJ, Peng W, Sandor V, Gopalakrishna P, Hmissi A, Stalbovskaya V, Gupta V, Harrison C, Verstovsek S; COMFORT Investigators. 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 Sep;100(9):1139-45. Epub 2015 Jun 11. [http://www.haematologica.org/content/100/9/1139.long link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4800694/ link to PMC article] [https:// | + | ## '''Pooled update:''' Vannucchi AM, Kantarjian HM, Kiladjian JJ, Gotlib J, Cervantes F, Mesa RA, Sarlis NJ, Peng W, Sandor V, Gopalakrishna P, Hmissi A, Stalbovskaya V, Gupta V, Harrison C, Verstovsek S; COMFORT Investigators. 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 Sep;100(9):1139-45. Epub 2015 Jun 11. [http://www.haematologica.org/content/100/9/1139.long link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4800694/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/26069290 PubMed] |
| − | ## '''Update:''' Verstovsek S, Mesa RA, Gotlib J, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner EO, Lyons RM, Paquette R, Raza A, Jones M, Kornacki D, Sun K, Kantarjian H; COMFORT-I investigators. Long-term treatment with ruxolitinib for patients with myelofibrosis: 5-year update from the randomized, double-blind, placebo-controlled, phase 3 COMFORT-I trial. J Hematol Oncol. 2017 Feb 22;10(1):55. [https://jhoonline.biomedcentral.com/articles/10.1186/s13045-017-0417-z link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5322633/ link to PMC article] [https:// | + | ## '''Update:''' Verstovsek S, Mesa RA, Gotlib J, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner EO, Lyons RM, Paquette R, Raza A, Jones M, Kornacki D, Sun K, Kantarjian H; COMFORT-I investigators. Long-term treatment with ruxolitinib for patients with myelofibrosis: 5-year update from the randomized, double-blind, placebo-controlled, phase 3 COMFORT-I trial. J Hematol Oncol. 2017 Feb 22;10(1):55. [https://jhoonline.biomedcentral.com/articles/10.1186/s13045-017-0417-z link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5322633/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/28228106 PubMed] |
| − | # '''JAKARTA:''' Pardanani A, Harrison C, Cortes JE, Cervantes F, Mesa RA, Milligan D, Masszi T, Mishchenko E, Jourdan E, Vannucchi AM, Drummond MW, Jurgutis M, Kuliczkowski K, Gheorghita E, Passamonti F, Neumann F, Patki A, Gao G, Tefferi A. Safety and efficacy of fedratinib in patients with primary or secondary myelofibrosis: A randomized clinical trial. JAMA Oncol. 2015 Aug 1;1(5):643-51. [http://oncology.jamanetwork.com/article.aspx?articleid=2330618 link to original article] [https:// | + | # '''JAKARTA:''' Pardanani A, Harrison C, Cortes JE, Cervantes F, Mesa RA, Milligan D, Masszi T, Mishchenko E, Jourdan E, Vannucchi AM, Drummond MW, Jurgutis M, Kuliczkowski K, Gheorghita E, Passamonti F, Neumann F, Patki A, Gao G, Tefferi A. Safety and efficacy of fedratinib in patients with primary or secondary myelofibrosis: A randomized clinical trial. JAMA Oncol. 2015 Aug 1;1(5):643-51. [http://oncology.jamanetwork.com/article.aspx?articleid=2330618 link to original article] [https://pubmed.ncbi.nlm.nih.gov/26181658 PubMed] |
| − | # '''RESUME:''' Tefferi A, Al-Ali HK, Barosi G, Devos T, Gisslinger H, Jiang Q, Kiladjian JJ, Mesa R, Passamonti F, McMullin MF, Ribrag V, Schiller G, Vannucchi AM, Zhou D, Reiser D, Zhong J, Gale RP. A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. Leukemia. 2017 Apr;31(4):896-902. Epub 2016 Oct 24. [https://www.nature.com/leu/journal/v31/n4/full/leu2016300a.html link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5383927/ link to PMC article] [https:// | + | # '''RESUME:''' Tefferi A, Al-Ali HK, Barosi G, Devos T, Gisslinger H, Jiang Q, Kiladjian JJ, Mesa R, Passamonti F, McMullin MF, Ribrag V, Schiller G, Vannucchi AM, Zhou D, Reiser D, Zhong J, Gale RP. A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. Leukemia. 2017 Apr;31(4):896-902. Epub 2016 Oct 24. [https://www.nature.com/leu/journal/v31/n4/full/leu2016300a.html link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5383927/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/27773929 PubMed] |
[[Category:Myelofibrosis regimens]] | [[Category:Myelofibrosis regimens]] | ||
Revision as of 01:19, 25 February 2020
The purpose of this page is to provide references to "null therapy" such as placebo and observation. While not clinically relevant, these references provide further insight into the historical development of the treatment landscape. See the main myelofibrosis page for current regimens.
All lines of therapy
Placebo
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Regimen
| Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|---|
| Verstovsek et al. 2012 (COMFORT-I) | 2009-2010 | Phase III (C) | Ruxolitinib | Seems to have inferior OS (*) |
| Tefferi et al. 2016 (RESUME) | 2010-2012 | Phase III (C) | Pomalidomide | Did not meet primary endpoint of RBC-transfusion independence within 6 mo |
| Pardanani et al. 2015 (JAKARTA) | 2011-2012 | Phase III (C) | Fedratinib | Inferior RR |
No active treatment. Reported efficacy for COMFORT-I is based on the 2017 update.
References
- COMFORT-I: Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger M, Miller C, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner E, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Koumenis IL, Sun W, Sandor V, Kantarjian HM. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012 Mar 1;366(9):799-807. link to original article supplementary appendix trial protocol contains verified protocol link to PMC article PubMed
- Update: Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH, Arcasoy MO, Hexner EO, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Sun W, Sandor V, Kantarjian HM. Efficacy, safety and survival with ruxolitinib treatment in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I. Haematologica. 2013 Dec;98(12):1865-71. Epub 2013 Sep 13. link to original article link to PMC article PubMed
- Update: Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH, Arcasoy MO, Hexner EO, Lyons RM, Raza A, Vaddi K, Sun W, Peng W, Sandor V, Kantarjian H. Three-year efficacy, overall survival, and safety of ruxolitinib therapy in patients with myelofibrosis from the COMFORT-I study. Haematologica. 2015 Apr;100(4):479-88. Epub 2015 Jan 23. link to original article link to PMC article PubMed
- Pooled update: Vannucchi AM, Kantarjian HM, Kiladjian JJ, Gotlib J, Cervantes F, Mesa RA, Sarlis NJ, Peng W, Sandor V, Gopalakrishna P, Hmissi A, Stalbovskaya V, Gupta V, Harrison C, Verstovsek S; COMFORT Investigators. 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 Sep;100(9):1139-45. Epub 2015 Jun 11. link to original article link to PMC article PubMed
- Update: Verstovsek S, Mesa RA, Gotlib J, Gupta V, DiPersio JF, Catalano JV, Deininger MW, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner EO, Lyons RM, Paquette R, Raza A, Jones M, Kornacki D, Sun K, Kantarjian H; COMFORT-I investigators. Long-term treatment with ruxolitinib for patients with myelofibrosis: 5-year update from the randomized, double-blind, placebo-controlled, phase 3 COMFORT-I trial. J Hematol Oncol. 2017 Feb 22;10(1):55. link to original article link to PMC article PubMed
- JAKARTA: Pardanani A, Harrison C, Cortes JE, Cervantes F, Mesa RA, Milligan D, Masszi T, Mishchenko E, Jourdan E, Vannucchi AM, Drummond MW, Jurgutis M, Kuliczkowski K, Gheorghita E, Passamonti F, Neumann F, Patki A, Gao G, Tefferi A. Safety and efficacy of fedratinib in patients with primary or secondary myelofibrosis: A randomized clinical trial. JAMA Oncol. 2015 Aug 1;1(5):643-51. link to original article PubMed
- RESUME: Tefferi A, Al-Ali HK, Barosi G, Devos T, Gisslinger H, Jiang Q, Kiladjian JJ, Mesa R, Passamonti F, McMullin MF, Ribrag V, Schiller G, Vannucchi AM, Zhou D, Reiser D, Zhong J, Gale RP. A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. Leukemia. 2017 Apr;31(4):896-902. Epub 2016 Oct 24. link to original article contains verified protocol link to PMC article PubMed