Myelofibrosis
Revision as of 18:51, 8 November 2015 by Jwarner (talk | contribs) (→Regimen #2 {{#subobject:6 |Variant=1}})
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9 regimens on this page
11 variants on this page
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Lenalidomide (Revlimid)
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Regimen
| Study | Evidence |
| Tefferi et al. 2006 | Phase II |
- Lenalidomide (Revlimid) 10 mg PO once per day
References
- Tefferi A, Cortes J, Verstovsek S, Mesa RA, Thomas D, Lasho TL, Hogan WJ, Litzow MR, Allred JB, Jones D, Byrne C, Zeldis JB, Ketterling RP, McClure RF, Giles F, Kantarjian HM. Lenalidomide therapy in myelofibrosis with myeloid metaplasia. Blood. 2006 Aug 15;108(4):1158-64. Epub 2006 Apr 11. link to original article contains protocol PubMed
Placebo
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Regimen
| Study | Evidence | Comparator | Efficacy |
| Verstovsek et al. 2012 (COMFORT-I) | Phase III | Ruxolitinib | Might have inferior OS |
| Pardanani et al. 2015 | Phase III | Fedratinib | Inferior RR |
No active treatment; included here because it was used as a comparator in one or more randomized controlled trials. Note that although fedratinib had superior efficacy to placebo per Pardanani et al. 2015, it had unacceptable toxicity and is no longer under active development.
References
- 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 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 Sep 13. [Epub ahead of print] link to original article PubMed
- 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
Pomalidomide (Pomalyst)
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Regimen #1
| Study | Evidence | Comparator |
| Tefferi et al. 2009 | Randomized Phase II, >20 in this arm | Pomalidomide (2 mg/d) & Prednisone Pomalidomide (0.5 mg/d) & Prednisone Prednisone |
- Pomalidomide (Pomalyst) 2 mg PO once per day
28-day cycles
Regimen #2
| Study | Evidence |
| Begna et al. 2010 | Phase II |
- Pomalidomide (Pomalyst) 0.5 mg PO once per day, escalation up to 2 mg PO once per day allowed after first six cycles
28-day cycles
References
- Tefferi A, Verstovsek S, Barosi G, Passamonti F, Roboz GJ, Gisslinger H, Paquette RL, Cervantes F, Rivera CE, Deeg HJ, Thiele J, Kvasnicka HM, Vardiman JW, Zhang Y, Bekele BN, Mesa RA, Gale RP, Kantarjian HM. Pomalidomide is active in the treatment of anemia associated with myelofibrosis. J Clin Oncol. 2009 Sep 20;27(27):4563-9. Epub 2009 Aug 3. link to original article PubMed
- Begna KH, Mesa RA, Pardanani A, Hogan WJ, Litzow MR, McClure RF, Tefferi A. A phase-2 trial of low-dose pomalidomide in myelofibrosis. Leukemia. 2011 Feb;25(2):301-4. Epub 2010 Nov 5. trial protocol contains verified protocol PubMed
Pomalidomide & Prednisone
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Regimen #1
| Study | Evidence | Comparator |
| Tefferi et al. 2009 | Randomized Phase II, <20 in this arm | Pomalidomide Pomalidomide (0.5 mg/d) & Prednisone Prednisone |
- Pomalidomide (Pomalyst) 2 mg PO once per day
- Prednisone (Sterapred) as follows:
- Cycle 1: 30 mg PO once per day
- Cycle 2: 15 mg PO once per day
- Cycle 3: 15 mg PO every other day
28-day cycles
Regimen #2
| Study | Evidence | Comparator |
| Tefferi et al. 2009 | Randomized Phase II, >20 in this arm | Pomalidomide Pomalidomide (2 mg/d) & Prednisone Prednisone |
- Pomalidomide (Pomalyst) 0.5 mg PO once per day
- Prednisone (Sterapred) as follows:
- Cycle 1: 30 mg PO once per day
- Cycle 2: 15 mg PO once per day
- Cycle 3: 15 mg PO every other day
28-day cycles
References
- Tefferi A, Verstovsek S, Barosi G, Passamonti F, Roboz GJ, Gisslinger H, Paquette RL, Cervantes F, Rivera CE, Deeg HJ, Thiele J, Kvasnicka HM, Vardiman JW, Zhang Y, Bekele BN, Mesa RA, Gale RP, Kantarjian HM. Pomalidomide is active in the treatment of anemia associated with myelofibrosis. J Clin Oncol. 2009 Sep 20;27(27):4563-9. Epub 2009 Aug 3. link to original article PubMed
Prednisone (Sterapred)
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Regimen
| Study | Evidence | Comparator | |
| Tefferi et al. 2009 | Randomized Phase II, >20 in this arm | Pomalidomide | |
| Tefferi et al. 2009 | Randomized Phase II, >20 in this arm | Pomalidomide (2 mg/d) & Prednisone | |
| Tefferi et al. 2009 | Randomized Phase II, >20 in this arm | Pomalidomide (0.5 mg/d) & Prednisone | |
- Prednisone (Sterapred) as follows:
- Cycle 1: 30 mg PO once per day
- Cycle 2: 15 mg PO once per day
- Cycle 3: 15 mg PO every other day
28-day cycle x 3 cycles
References
- Tefferi A, Verstovsek S, Barosi G, Passamonti F, Roboz GJ, Gisslinger H, Paquette RL, Cervantes F, Rivera CE, Deeg HJ, Thiele J, Kvasnicka HM, Vardiman JW, Zhang Y, Bekele BN, Mesa RA, Gale RP, Kantarjian HM. Pomalidomide is active in the treatment of anemia associated with myelofibrosis. J Clin Oncol. 2009 Sep 20;27(27):4563-9. Epub 2009 Aug 3. link to original article PubMed
Ruxolitinib (Jakafi)
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Regimen
COMFORT: COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Treatment
| Study | Evidence | Comparator | Efficacy |
| Verstovsek et al. 2010 | Phase II | ||
| Verstovsek et al. 2012 (COMFORT-I) | Phase III | Placebo | Might have superior OS |
| Harrison et al. 2012 (COMFORT-II) | Phase III | "Best available therapy" | Seems to have superior OS |
| Mead et al. 2015 (ROBUST) | Phase II | ||
Patients in COMFORT-II who received "best available therapy" generally received no active treatment, Hydroxyurea (Hydrea), or glucocorticoids.
- Ruxolitinib (Jakafi) 10 to 25 mg PO twice per day
Given until progression
References
- Verstovsek S, Kantarjian H, Mesa RA, Pardanani AD, Cortes-Franco J, Thomas DA, Estrov Z, Fridman JS, Bradley EC, Erickson-Viitanen S, Vaddi K, Levy R, Tefferi A. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med. 2010 Sep 16;363(12):1117-27. link to original article supplementary appendix contains verified protocol PubMed
- 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 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 Sep 13. [Epub ahead of print] link to original 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 Jan 23. [Epub ahead of print] link to original article PubMed
- Harrison C, Kiladjian JJ, Al-Ali HK, Gisslinger H, Waltzman R, Stalbovskaya V, McQuitty M, Hunter DS, Levy R, Knoops L, Cervantes F, Vannucchi AM, Barbui T, Barosi G. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med. 2012 Mar 1;366(9):787-98. link to original article contains verified protocol PubMed content property of HemOnc.org
- Update: Cervantes F, Vannucchi AM, Kiladjian JJ, Al-Ali HK, Sirulnik A, Stalbovskaya V, McQuitty M, Hunter DS, Levy RS, Passamonti F, Barbui T, Barosi G, Harrison CN, Knoops L, Gisslinger H. Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood. 2013 Oct 30. [Epub ahead of print] link to original article PubMed
- Mead AJ, Milojkovic D, Knapper S, Garg M, Chacko J, Farquharson M, Yin J, Ali S, Clark RE, Andrews C, Ktiouet Dawson M, Harrison C. Response to ruxolitinib in patients with intermediate-1-, intermediate-2-, and high-risk myelofibrosis: results of the UK ROBUST Trial. Br J Haematol. 2015 Mar 30. [Epub ahead of print] link to original article contains protocol PubMed