Myelofibrosis

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9 regimens on this page 11 variants on this page

Guidelines

NCCN

• NCCN Guidelines - Myeloproliferative Neoplasms

All lines of treatment

Lenalidomide (Revlimid)

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Regimen

Study	Evidence
Tefferi et al. 2006	Phase II

Chemotherapy

• Lenalidomide (Revlimid) 10 mg PO once per day

References

1. 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
Tefferi et al. 2016 (RESUME)	Phase III	Pomalidomide	Seems not superior

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

1. 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
   1. 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
2. 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
3. Tefferi A, Al-Ali HK, Barosi G, Devos T, Gisslinger H, Jiang Q, Kiladjian JJ, Mesa R, Passamonti F, 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

Pomalidomide (Pomalyst)

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Regimen #1

Study	Evidence	Comparator	Efficacy
Begna et al. 2010	Phase II
Tefferi et al. 2016 (RESUME)	Phase III	Placebo	Seems not superior

Chemotherapy

• Pomalidomide (Pomalyst) 0.5 mg PO once per day
  • In Begna et al. 2010, escalation up to 2 mg PO once per day allowed after first six cycles

28-day cycles

Regimen #2

Study	Evidence	Comparator	Efficacy
Tefferi et al. 2009	Randomized Phase II	Pomalidomide (2 mg/d) & Prednisone Pomalidomide (0.5 mg/d) & Prednisone Prednisone	Not reported

Chemotherapy

• Pomalidomide (Pomalyst) 2 mg PO once per day

28-day cycles

References

1. 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 link to PMC article PubMed
2. 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
3. Tefferi A, Al-Ali HK, Barosi G, Devos T, Gisslinger H, Jiang Q, Kiladjian JJ, Mesa R, Passamonti F, 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

Pomalidomide & Prednisone

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Regimen #1

Study	Evidence	Comparator	Efficacy
Tefferi et al. 2009	Randomized Phase II, <20 in this arm	Pomalidomide Pomalidomide (0.5 mg/d) & Prednisone Prednisone	Not reported

Chemotherapy

• 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	Efficacy
Tefferi et al. 2009	Randomized Phase II	Pomalidomide Pomalidomide (2 mg/d) & Prednisone Prednisone	Not reported

Chemotherapy

• 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

1. 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 link to PMC article PubMed

Prednisone (Sterapred)

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Regimen

Study	Evidence	Comparator	Efficacy
Tefferi et al. 2009	Randomized Phase II	Pomalidomide Pomalidomide (2 mg/d) & Prednisone Pomalidomide (0.5 mg/d) & Prednisone	Not reported

Chemotherapy

• 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 for 3 cycles

References

1. 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 link to PMC 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
Al-Ali et al. 2016 (JUMP)	Phase 3b

Patients in COMFORT-II who received "best available therapy" generally received no active treatment, Hydroxyurea (Hydrea), or glucocorticoids.

Chemotherapy

• Ruxolitinib (Jakafi) 10 to 25 mg PO twice per day

Given until progression

References

1. 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 link to PMC article PubMed
2. 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
   1. 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
   2. 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
3. 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
   1. 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 Dec 12;122(25):4047-53. Epub 2013 Oct 30. Erratum in: Blood. 2016 Dec 22;128(25):3013. link to original article PubMed
   2. Update: Harrison CN, Vannucchi AM, Kiladjian JJ, Al-Ali HK, Gisslinger H, Knoops L, Cervantes F, Jones MM, Sun K, McQuitty M, Stalbovskaya V, Gopalakrishna P, Barbui T. Long-term findings from COMFORT-II, a phase 3 study of ruxolitinib vs best available therapy for myelofibrosis. Leukemia. 2016 Aug;30(8):1701-7. Epub 2016 May 23. link to original article link to PMC article PubMed
4. 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 Jul;170(1):29-39. Epub 2015 Mar 30. link to original article contains protocol PubMed
5. Al-Ali HK, Griesshammer M, le Coutre P, Waller CF, Liberati AM, Schafhausen P, Tavares R, Giraldo P, Foltz L, Raanani P, Gupta V, Tannir B, Ronco JP, Ghosh J, Martino B, Vannucchi AM. 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 Sep;101(9):1065-73. Epub 2016 May 31. link to original article link to PMC article PubMed