Myelofibrosis

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Section editor
Sanjay mohan.png
 Sanjay R. Mohan, MD, MSCI
Vanderbilt University
Nashville, TN, USA

Are you looking for a regimen but can't find it here? For placebo or observational studies in this condition, please visit this page. If you still can't find it, please let us know so we can add it!

9 regimens on this page
11 variants on this page

This page covers the treatment of both primary and secondary myelofibrosis.


Guidelines

Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.

ESMO

NCCN

All lines of therapy

Danazol monotherapy

Regimen

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Cervantes et al. 2005 1995-11 to 2004-08 Non-randomized
Awaiting publication (PACIFICA) 2017-ongoing Phase 3 (C) Pacritinib TBD if different co-primary endpoints of spleen volume/TSS response rate at 24 weeks
Verstovsek et al. 2023 (MOMENTUM) 2020-04-24 to 2021-12-03 Phase 3 (C) Momelotinib Inferior TSS response rate at 24 weeks

Endocrine therapy

Continued indefinitely

References

  1. Cervantes F, Alvarez-Larrán A, Domingo A, Arellano-Rodrigo E, Montserrat E. Efficacy and tolerability of danazol as a treatment for the anaemia of myelofibrosis with myeloid metaplasia: long-term results in 30 patients. Br J Haematol. 2005 Jun;129(6):771-5. link to original article contains dosing details in abstract PubMed
  2. MOMENTUM: Verstovsek S, Gerds AT, Vannucchi AM, Al-Ali HK, Lavie D, Kuykendall AT, Grosicki S, Iurlo A, Goh YT, Lazaroiu MC, Egyed M, Fox ML, McLornan D, Perkins A, Yoon SS, Gupta V, Kiladjian JJ, Granacher N, Lee SE, Ocroteala L, Passamonti F, Harrison CN, Klencke BJ, Ro S, Donahue R, Kawashima J, Mesa R; MOMENTUM Study Investigators. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study. Lancet. 2023 Jan 28;401(10373):269-280. link to original article contains dosing details in abstract PubMed NCT04173494
    1. Update: Gerds AT, Verstovsek S, Vannucchi AM, Al-Ali HK, Lavie D, Kuykendall AT, Grosicki S, Iurlo A, Goh YT, Lazaroiu MC, Egyed M, Fox ML, McLornan D, Perkins A, Yoon SS, Gupta V, Kiladjian JJ, Granacher N, Lee SE, Ocroteala L, Passamonti F, Harrison CN, Oh S, Klencke BJ, Yu J, Donahue R, Kawashima J, Mesa R. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis previously treated with a JAK inhibitor (MOMENTUM): an updated analysis of an international, double-blind, randomised phase 3 study. Lancet Haematol. 2023 Sep;10(9):e735-e746. Epub 2023 Jul 27. link to original article PubMed
  3. PACIFICA: NCT03165734

Fedratinib monotherapy

Regimen

FDA-recommended dose
Study Dates of enrollment Evidence Comparator Comparative Efficacy
Pardanani et al. 2015 (JAKARTA) 2011-12 to 2012-09 Phase 3 (E-RT-esc) 1. Fedratinib; 500 mg/d Not reported
2. Placebo Superior spleen response (primary endpoint)

Targeted therapy

28-day cycles

References

  1. 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 contains dosing details in manuscript PubMed NCT01437787

Hydroxyurea monotherapy

Regimen

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Mesa et al. 2017 (PERSIST-1) 2013-01-08 to 2014-08-01 Phase 3 (C) Pacritinib Inferior spleen volume reduction
Awaiting publication (PACIFICA) 2017-ongoing Phase 3 (C) Pacritinib TBD if different co-primary endpoints of spleen volume/TSS response rate at 24 weeks

Note: this was the most commonly used "best available therapy" (BAT) in PERSIST-1.

Chemotherapy

References

  1. PERSIST-1: Mesa RA, Vannucchi AM, Mead A, Egyed M, Szoke A, Suvorov A, Jakucs J, Perkins A, Prasad R, Mayer J, Demeter J, Ganly P, Singer JW, Zhou H, Dean JP, Te Boekhorst PA, Nangalia J, Kiladjian JJ, Harrison CN. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial. Lancet Haematol. 2017 May;4(5):e225-e236. Epub 2017 Mar 20. link to original article link to PMC article does not contain dosing details PubMed NCT01773187
  2. PACIFICA: NCT03165734

Lenalidomide monotherapy

Regimen

Study Dates of enrollment Evidence
Tefferi et al. 2006a 2004-03-05 to 2005-06-15 Phase 2
Tefferi et al. 2006b 2004-07-21 to 2005-07-08 Phase 2

Note: this manuscript reports on two independently conducted phase 2 studies, with some differences in the duration of therapy.

Targeted therapy

28-day cycle for at least 3 up to 24 cycles

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 dosing details in abstract PubMed

Pacritinib monotherapy

Regimen

FDA-recommended dose
Study Dates of enrollment Evidence Comparator Comparative Efficacy
Komrokji et al. 2015 (SB1518-2008-003) 2010 Phase 2
Mesa et al. 2017 (PERSIST-1) 2013-01-08 to 2014-08-01 Phase 3 (E-switch-ic) "Best available therapy" Superior spleen volume reduction (primary endpoint)
Mascarenhas et al. 2018 (PERSIST-2) 2014-2016 Phase 3 (E-RT-switch-ic) "Best available therapy" Superior spleen volume reduction (co-primary endpoint)

Targeted therapy

Continued indefinitely

References

  1. SB1518-2008-003: Komrokji RS, Seymour JF, Roberts AW, Wadleigh M, To LB, Scherber R, Turba E, Dorr A, Zhu J, Wang L, Granston T, Campbell MS, Mesa RA. Results of a phase 2 study of pacritinib (SB1518), a JAK2/JAK2(V617F) inhibitor, in patients with myelofibrosis. Blood. 2015 Apr 23;125(17):2649-55. Epub 2015 Mar 11. link to original article link to PMC article PubMed NCT00745550
  2. PERSIST-1: Mesa RA, Vannucchi AM, Mead A, Egyed M, Szoke A, Suvorov A, Jakucs J, Perkins A, Prasad R, Mayer J, Demeter J, Ganly P, Singer JW, Zhou H, Dean JP, Te Boekhorst PA, Nangalia J, Kiladjian JJ, Harrison CN. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial. Lancet Haematol. 2017 May;4(5):e225-e236. Epub 2017 Mar 20. link to original article link to PMC article PubMed NCT01773187
  3. PERSIST-2: Mascarenhas J, Hoffman R, Talpaz M, Gerds AT, Stein B, Gupta V, Szoke A, Drummond M, Pristupa A, Granston T, Daly R, Al-Fayoumi S, Callahan JA, Singer JW, Gotlib J, Jamieson C, Harrison C, Mesa R, Verstovsek S. Pacritinib vs best available therapy, including ruxolitinib, in patients with myelofibrosis: a randomized clinical trial. JAMA Oncol. 2018 May 1;4(5):652-659. Epub 2018 Mar 8. link to original article link to PMC article contains dosing details in abstract PubMed NCT02055781
  4. PACIFICA: NCT03165734

Pomalidomide monotherapy

Regimen variant #1

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Begna et al. 2010 (PO-MMM-PI-0007) NR Phase 2
Tefferi et al. 2016 (RESUME) 2010-2012 Phase 3 (E-esc) Placebo Did not meet primary endpoint of RBC-transfusion independence within 6 mo

Targeted therapy

28-day cycles

Dose and schedule modifications

  • In PO-MMM-PI-0007, escalation up to 2 mg PO once per day allowed after first six cycles


Regimen variant #2, 2 mg/day

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Tefferi et al. 2009 (CC-4047-MMM-001) 2007-NR Randomized Phase 2 (E-esc) 1. Pomalidomide & Prednisone; 2 mg/day
2. Pomalidomide & Prednisone; 0.5 mg/day
3. Prednisone 		Not reported

Targeted therapy

28-day cycles

References

  1. CC-4047-MMM-001: 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 contains dosing details in manuscript PubMed NCT00463385
  2. PO-MMM-PI-0007: 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 dosing details in manuscript PubMed
  3. 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 dosing details in manuscript link to PMC article PubMed NCT01178281

Pomalidomide & Prednisone

Regimen variant #1, 2 mg/day

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Tefferi et al. 2009 (CC-4047-MMM-001) 2007-NR Randomized Phase 2, <20 in this arm (E-esc) 1. Pomalidomide
2. Pomalidomide & Prednisone; 0.5 mg/day
3. Prednisone 		Not reported

Targeted therapy

Glucocorticoid therapy

  • Prednisone (Sterapred) as follows:
    • Cycle 1: 30 mg PO once per day on days 1 to 28
    • Cycle 2: 15 mg PO once per day on days 1 to 28
    • Cycle 3: 15 mg PO once every other day on days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27

28-day cycles


Regimen variant #2, 0.5 mg/day

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Tefferi et al. 2009 (CC-4047-MMM-001) 2007-NR Randomized Phase 2 (E-esc) 1. Pomalidomide
2. Pomalidomide & Prednisone; 2 mg/day
3. Prednisone 		Not reported

Targeted therapy

Glucocorticoid therapy

  • Prednisone (Sterapred) as follows:
    • Cycle 1: 30 mg PO once per day on days 1 to 28
    • Cycle 2: 15 mg PO once per day on days 1 to 28
    • Cycle 3: 15 mg PO once every other day on days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27

28-day cycles

References

  1. CC-4047-MMM-001: 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 contains dosing details in manuscript PubMed NCT00463385

Prednisone monotherapy

Regimen

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Tefferi et al. 2009 (CC-4047-MMM-001) 2007-NR Randomized Phase 2 (C) 1. Pomalidomide
2. Pomalidomide & Prednisone; 2 mg/day
3. Pomalidomide & Prednisone; 0.5 mg/day 		Not reported
Awaiting publication (PACIFICA) 2017-ongoing Phase 3 (C) Pacritinib TBD if different co-primary endpoints of spleen volume/TSS response rate at 24 weeks

Glucocorticoid therapy

  • Prednisone (Sterapred) as follows:
    • Cycle 1: 30 mg PO once per day on days 1 to 28
    • Cycle 2: 15 mg PO once per day on days 1 to 28
    • Cycle 3: 15 mg PO once every other day on days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27

28-day cycle for 3 cycles

References

  1. CC-4047-MMM-001: 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 contains dosing details in manuscript PubMed NCT00463385
  2. PACIFICA: NCT03165734

Ruxolitinib monotherapy

Regimen

COMFORT: COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Treatment

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Verstovsek et al. 2010 (INCB 18424-251) 2007-NR Phase 2
Verstovsek et al. 2012 (COMFORT-I) 2009-09 to 2010-04 Phase 3 (E-RT-esc) Placebo Seems to have superior OS1 (secondary endpoint)
Median OS: NYR vs 200 weeks
(HR 0.69, 95% CI 0.50-0.96)

Superior spleen volume reduction of at least 35% at week 24 (primary endpoint)
Harrison et al. 2012 (COMFORT-II) 2009-07-01 to 2010-01-22 Phase 3 (E-RT-switch-ooc) "Best available therapy" Might have superior OS2 (secondary endpoint)
Median OS: NYR vs 4.1 y
(HR 0.67, 95% CI 0.44-1.02)

Superior spleen volume reduction of at least 35% at week 48 (primary endpoint)
Al-Ali et al. 2016 (JUMP) 2011-2014 Phase 3b
Mead et al. 2015 (UK ROBUST) 2012-NR Phase 2
Mesa et al. 2017 (SIMPLIFY-1) 2013-2016 Phase 3 (C) Momelotinib Non-inferior spleen response
Harrison et al. 2017 (SIMPLIFY-2) 2014-2016 Phase 3 (C) Momelotinib Did not meet primary endpoint of spleen volume reduction
Mascarenhas et al. 2018 (PERSIST-2) 2014-2016 Phase 3 (C) Pacritinib Inferior spleen volume reduction
Awaiting publication (PACIFICA) 2017-ongoing Phase 3 (C) Pacritinib TBD if different co-primary endpoints of spleen volume/TSS response rate at 24 weeks

1Reported efficacy for COMFORT-I is based on the 2017 update.
2Reported efficacy for COMFORT-II is based on the 2016 update.
Note: Patients in COMFORT-II who received "best available therapy" (BAT) generally received no active treatment (33%), Hydroxyurea (Hydrea) (47%), or glucocorticoids (16%). Patients in SIMPLIFY-2 had been previously treated with ruxolitinib but were allowed to receive ruxolitinib as BAT. This was the most commonly used BAT in PERSIST-2.

Targeted therapy

Continued indefinitely

References

  1. INCB 18424-251: 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 dosing details in manuscript link to PMC article PubMed NCT00509899
  2. 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 dosing details in manuscript link to PMC article PubMed NCT00952289
    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. 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
    4. 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
  3. COMFORT-II: 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 dosing details in manuscript PubMed content property of HemOnc.org NCT00934544
    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. 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
    3. 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. UK ROBUST: 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 dosing details in abstract PubMed NCT01558739
  5. JUMP: 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 NCT01493414
  6. SIMPLIFY-1: Mesa RA, Kiladjian JJ, Catalano JV, Devos T, Egyed M, Hellmann A, McLornan D, Shimoda K, Winton EF, Deng W, Dubowy RL, Maltzman JD, Cervantes F, Gotlib J. SIMPLIFY-1: a phase III randomized trial of momelotinib versus ruxolitinib in Janus kinase inhibitor-naïve patients with myelofibrosis. J Clin Oncol. 2017 Dec 1;35(34):3844-3850. Epub 2017 Sep 20. link to original article link to PMC article PubMed NCT01969838
  7. SIMPLIFY-2: Harrison CN, Vannucchi AM, Platzbecker U, Cervantes F, Gupta V, Lavie D, Passamonti F, Winton EF, Dong H, Kawashima J, Maltzman JD, Kiladjian JJ, Verstovsek S. Momelotinib versus best available therapy in patients with myelofibrosis previously treated with ruxolitinib (SIMPLIFY 2): a randomised, open-label, phase 3 trial. Lancet Haematol. 2018 Feb;5(2):e73-e81. Epub 2017 Dec 20. link to original article PubMed NCT02101268
  8. PERSIST-2: Mascarenhas J, Hoffman R, Talpaz M, Gerds AT, Stein B, Gupta V, Szoke A, Drummond M, Pristupa A, Granston T, Daly R, Al-Fayoumi S, Callahan JA, Singer JW, Gotlib J, Jamieson C, Harrison C, Mesa R, Verstovsek S. Pacritinib vs best available therapy, including ruxolitinib, in patients with myelofibrosis: a randomized clinical trial. JAMA Oncol. 2018 May 1;4(5):652-659. Epub 2018 Mar 8. link to original article link to PMC article PubMed NCT02055781
  9. PACIFICA: NCT03165734
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