Ten years after ruxolitinib approval for myelofibrosis: a review of clinical efficacy

References

• Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127(20):2391–2405.
   PubMed Web of Science ®Google Scholar
• England J, Gupta V. Novel therapies vs hematopoietic cell transplantation in myelofibrosis: who, when, how? Hematol Am Soc Hematol Educ Program. 2021;2021(1):453–462.
   PubMedGoogle Scholar
• Tefferi A. Primary myelofibrosis: 2021 update on diagnosis, risk-stratification and management. Am J Hematol. 2021;96(1):145–162.
   PubMed Web of Science ®Google Scholar
• Mesa R, Miller CB, Thyne M, et al. Myeloproliferative neoplasms (MPNs) have a significant impact on patients’ overall health and productivity: the MPN Landmark survey. BMC Cancer. 2016;16:167.
   PubMed Web of Science ®Google Scholar
• Hultcrantz M, Kristinsson SY, Andersson TM, et al. Patterns of survival among patients with myeloproliferative neoplasms diagnosed in Sweden from 1973 to 2008: a population-based study. J Clin Oncol. 2012;30(24):2995–3001.
   PubMed Web of Science ®Google Scholar
• Price GL, Davis KL, Karve S, et al. Survival patterns in United States (US) Medicare enrollees with non-CML myeloproliferative neoplasms (MPN). PLOS One. 2014;9(3):e90299.
   PubMed Web of Science ®Google Scholar
• Rampal R, Al-Shahrour F, Abdel-Wahab O, et al. Integrated genomic analysis illustrates the central role of JAK-STAT pathway activation in myeloproliferative neoplasm pathogenesis. Blood. 2014;123(22):e123-133–e133.
   Web of Science ®Google Scholar
• Guijarro-Hernandez A, Vizmanos JL. A broad overview of signaling in Ph-negative classic myeloproliferative neoplasms. Cancers. 2021;13(5):984.
   PubMed Web of Science ®Google Scholar
• Klampfl T, Gisslinger H, Harutyunyan AS, et al. Somatic mutations of calreticulin in myeloproliferative neoplasms. N Engl J Med. 2013;369(25):2379–2390.
   PubMed Web of Science ®Google Scholar
• National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Myeloproliferative Neoplasms, version 1. 2022. https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1477.
   Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012;366(9):799–807.
   PubMed Web of Science ®Google Scholar
• Harrison C, Kiladjian JJ, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med. 2012;366(9):787–798.
   PubMed Web of Science ®Google Scholar
• JAKAFI® (ruxolitinib). Full prescribing information. Wilmington (DE): Incyte Corporation; 2023.
   Google Scholar
• Raedler LA. Jakafi (ruxolitinib): first FDA-approved medication for the treatment of patients with polycythemia vera. Am Health Drug Benefits. 2015;8:75–79.
   PubMedGoogle Scholar
• Al-Ali HK, Griesshammer M, Foltz L, et al. Primary analysis of JUMP, a phase 3b, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis, including those with low platelet counts. Br J Haematol. 2020;189(5):888–903.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, for the COMFORT-I investigators, et al. 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;10(1):55.
   PubMedGoogle Scholar
• Harrison CN, Vannucchi AM, Kiladjian JJ, on behalf of the COMFORT-II investigators, et al. Long-term findings from COMFORT-II, a phase 3 study of ruxolitinib vs best available therapy for myelofibrosis. Leukemia. 2016;30(8):1701–1707.
   PubMed Web of Science ®Google Scholar
• Vannucchi AM, Kantarjian HM, Kiladjian JJ, on behalf of the COMFORT investigators, 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(9):1139–1145.
   PubMed Web of Science ®Google Scholar
• Gupta V, Griesshammer M, Martino B, et al. Analysis of predictors of response to ruxolitinib in patients with myelofibrosis in the phase 3b expanded-access JUMP study. Leuk Lymphoma. 2021;62(4):918–926.
   PubMed Web of Science ®Google Scholar
• Maffioli M, Mora B, Ball S, et al. A prognostic model to predict survival after 6 months of ruxolitinib in patients with myelofibrosis. Blood Adv. 2022;6(6):1855–1864.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Gotlib J, Mesa RA, et al. Long-term survival in patients treated with ruxolitinib for myelofibrosis: COMFORT-I and -II pooled analyses. Verstovsek J Hematol Oncol 2017;10(1):156.
   Web of Science ®Google Scholar
• Verstovsek S, Parasuraman S, Yu J, et al. Real-world survival of US patients with intermediate- to high-risk myelofibrosis: impact of ruxolitinib approval. Ann Hematol. 2022;101(1):131–137.
   PubMed Web of Science ®Google Scholar
• Masarova L, Bose P, Pemmaraju N, et al. Improved survival of patients with myelofibrosis in the last decade: single-center experience. Cancer. 2022;128(8):1658–1665.
   PubMed Web of Science ®Google Scholar
• Guglielmelli P, Ghirardi A, Carobbio A, et al. Impact of ruxolitinib on survival of patients with myelofibrosis in the real world: update of the ERNEST study. Blood Adv. 2022;6(2):373–375.
   PubMed Web of Science ®Google Scholar
• Al-Ali HK, Stalbovskaya V, Gopalakrishna P, et al. Impact of ruxolitinib treatment on the hemoglobin dynamics and the negative prognosis of anemia in patients with myelofibrosis. Leuk Lymphoma. 2016;57(10):2464–2467.
   PubMed Web of Science ®Google Scholar
• Gupta V, Harrison C, Hexner EO, et al. The impact of anemia on overall survival in patients with myelofibrosis treated with ruxolitinib in the COMFORT studies. Haematologica. 2016;101(12):e482–e484.
   PubMed Web of Science ®Google Scholar
• Marti-Carvajal AJ, Anand V, Sola I. Janus kinase-1 and Janus kinase-2 inhibitors for treating myelofibrosis. Cochrane Database Syst Rev. 2015;(4):CD010298.
   Web of Science ®Google Scholar
• Pemmaraju N, Verstovsek S, Mesa R, et al. Defining disease modification in myelofibrosis in the era of targeted therapy. Cancer. 2022;128(13):2420–2432.
   PubMed Web of Science ®Google Scholar
• Vachhani P, Verstovsek S, Bose P. Disease modification in myelofibrosis: an elusive goal? J Clin Oncol. 2022;40(11):1147–1154.
   PubMed Web of Science ®Google Scholar
• Maffioli M. Making a case for disease-modifying agents in myelofibrosis. Lancet Haematol. 2022;9(6):e391–e392.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Hoffman R. A comprehensive review and analysis of the effect of ruxolitinib therapy on the survival of patients with myelofibrosis. Blood. 2013;121(24):4832–4837.
   PubMed Web of Science ®Google Scholar
• Oh S, Verstovsek S, Gotlib J, et al. Bone marrow fibrosis changes do not correlate with efficacy outcomes in myelofibrosis: analysis of more than 300 JAK inhibitor-naive patients treated with momelotinib or ruxolitinib. Presented at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, New Orleans, LA. 2022.
   Google Scholar
• Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med. 2010;363(12):1117–1127.
   PubMed Web of Science ®Google Scholar
• Fisher DAC, Miner CA, Engle EK, et al. Cytokine production in myelofibrosis exhibits differential responsiveness to JAK-STAT, MAP kinase, and NFkappaB signaling. Leukemia. 2019;33(8):1978–1995.
   PubMed Web of Science ®Google Scholar
• Kvasnicka HM, Thiele J, Bueso-Ramos CE, et al. Long-term effects of ruxolitinib versus best available therapy on bone marrow fibrosis in patients with myelofibrosis. J Hematol Oncol. 2018;11(1):42.
   PubMedGoogle Scholar
• Kvasnicka HM, Thiele J, Bueso-Ramos CE, et al. Ruxolitinib (RUX) induced meaningful and directional changes in the bone marrow microenvironment of patients with myelofibrosis enrolled in the COMFORT-I study. Presented at the 61st American Society of Hematology Annual Meeting & Exposition, Orlando, FL. 2019.
   Google Scholar
• Kuykendall AT, Talati C, Al Ali N, et al. The treatment landscape of myelofibrosis before and after ruxolitinib approval. Clin Lymphoma Myeloma Leuk. 2017;17(12):e45–e53.
   PubMed Web of Science ®Google Scholar
• Bose P, Verstovsek S. JAK inhibition for the treatment of myelofibrosis: limitations and future perspectives. HemaSphere. 2020;4(4):e424–e424.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Gerds AT, Vannucchi AM, et al. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study. Lancet. 2023;401(10373):269–280.
   PubMed Web of Science ®Google Scholar
• Palandri F, Palumbo GA, Bonifacio M, et al. Baseline factors associated with response to ruxolitinib: an independent study on 408 patients with myelofibrosis. Oncotarget. 2017;8(45):79073–79086.
   PubMedGoogle Scholar
• Mead AJ, Milojkovic D, Knapper S, 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(1):29–39.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Ross DM, Radinoff A, et al. Efficacy and safety of a novel dosing strategy for ruxolitinib in the treatment of patients with myelofibrosis and anemia: the REALISE phase 2 study. Leukemia. 2021;35(12):3455–3465.
   PubMed Web of Science ®Google Scholar
• Al-Ahi HK, Griesshammer M, Le Coutre M, 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.
   PubMed Web of Science ®Google Scholar
• Vannucchi AM, Te Boekhorst PAW, Harrison CN, et al. EXPAND, a dose-finding study of ruxolitinib in patients with myelofibrosis and low platelet counts: 48-week follow-up analysis. Haematologica. 2019;104(5):947–954.
   PubMed Web of Science ®Google Scholar
• Gerds A, Su D, Martynova A, et al. Ruxolitinib rechallenge can improve constitutional symptoms and splenomegaly in patients with myelofibrosis: a case series. Clin Lymphoma Myeloma Leuk. 2018;18(11):e463–e468.
   PubMed Web of Science ®Google Scholar
• Gerds AT, Yu J, Scherber RM, et al. Ruxolitinib re-treatment in patients with myelofibrosis: real-world evidence on patient characteristics and outcomes. Acta Haematol. 2022;145(4):448–453.
   PubMed Web of Science ®Google Scholar
• National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Myeloproliferative Neoplasms, version 3; 2022. https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1477.
   Google Scholar
• Robin M, Porcher R, Orvain C, et al. Ruxolitinib before allogeneic hematopoietic transplantation in patients with myelofibrosis on behalf SFGM-TC and FIM groups. Bone Marrow Transplant. 2021;56(8):1888–1899.
   PubMed Web of Science ®Google Scholar
• Talpaz M, Paquette R, Afrin L, et al. Interim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts. J Hematol Oncol. 2013;6(1):81.
   PubMed Web of Science ®Google Scholar
• Talpaz M, Prchal J, Afrin L, et al. Safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts (50 − 100 × 109/L): final analysis of an open-label phase 2 study. Clin Lymphoma Myeloma Leuk. 2022;22(5):336–346.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Gotlib J, Gupta V, et al. Management of cytopenias in patients with myelofibrosis treated with ruxolitinib and effect of dose modifications on efficacy outcomes. Onco Targets Ther. 2013;7:13–21.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Cortes J. Optimizing management of ruxolitinib in patients with myelofibrosis: the need for individualized dosing. J Hematol Oncol. 2013;6(1):79.
   PubMedGoogle Scholar
• Palandri F, Palumbo GA, Elli EM, et al. Ruxolitinib discontinuation syndrome: incidence, risk factors, and management in 251 patients with myelofibrosis. Blood Cancer J. 2021;11:4.
   PubMed Web of Science ®Google Scholar
• Kuykendall AT, Shah S, Talati C, et al. Between a rux and a hard place: evaluating salvage treatment and outcomes in myelofibrosis after ruxolitinib discontinuation. Ann Hematol. 2018;97(3):435–441.
   PubMed Web of Science ®Google Scholar
• Palandri F, Breccia M, Stefano D, et al. F. Philadelphia-negative chronic myeloproliferative neoplasms during the COVID-19 pandemic: challenges and future scenarios. Cancers. 2021;13(19):4750.
   PubMed Web of Science ®Google Scholar
• Barbui T, Vannucchi AM, Alvarez-Larran A, et al. High mortality rate in COVID-19 patients with myeloproliferative neoplasms after abrupt withdrawal of ruxolitinib. Leukemia. 2021;35(2):485–493.
   PubMed Web of Science ®Google Scholar
• Azanza JR, Mensa J, González Del Castillo J, et al. Interactions listed in the Paxlovid fact sheet, classified according to risks, pharmacological groups, and consequences. Rev Esp Quimioter. 2022;35(4):357–361.
   PubMed Web of Science ®Google Scholar
• Palandri F, Breccia M, Bonifacio M, et al. Life after ruxolitinib: reasons for discontinuation, impact of disease phase, and outcomes in 218 patients with myelofibrosis. Cancer. 2020;126(6):1243–1252.
   PubMed Web of Science ®Google Scholar
• Inrebic® (fedratinib). Full prescribing information. Summit (NJ): Celgene Corporation; 2019.
   Google Scholar
• Harrison CN, Schaap N, Vannucchi AM, et al. Fedratinib in patients with myelofibrosis previously treated with ruxolitinib: an updated analysis of the JAKARTA2 study using stringent criteria for ruxolitinib failure. Am J Hematol. 2020;95(6):594–603.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Vannucchi AM, Mead A, et al. 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;4(5):e225–e236.
   PubMed Web of Science ®Google Scholar
• VONJO™ (pacritinib). Full prescribing information. Seattle (WA): CTI BioPharma; 2022.
   Google Scholar
• Bose P, Verstovsek S. Management of myelofibrosis after ruxolitinib failure. Leuk Lymphoma. 2020;61(8):1797–1809.
   PubMed Web of Science ®Google Scholar
• National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Hematopoietic Cell Transplantation (HCT), version 2; 2022. https://www.nccn.org/guidelines/guidelines-detail?category=3&id=1501
   Google Scholar
• Banerjee S, Biehl A, Gadina M, et al. JAK–STAT signaling as a target for inflammatory and autoimmune diseases: current and future prospects. Drugs. 2017;77(5):521–546.
   PubMed Web of Science ®Google Scholar
• Wernig G, Kharas MG, Okabe R, et al. Efficacy of TG101348, a selective JAK2 inhibitor, in treatment of a murine model of JAK2V617F-induced polycythemia vera. Cancer Cell. 2008;13(4):311–320.
   PubMed Web of Science ®Google Scholar
• Arana Yi C, Tam CS, Verstovsek S. Efficacy and safety of ruxolitinib in the treatment of patients with myelofibrosis. Future Oncol. 2015;11(5):719–733.
   PubMed Web of Science ®Google Scholar
• Zhang M, Xu CR, Shamiyeh E, et al. A randomized, placebo-controlled study of the pharmacokinetics, pharmacodynamics, and tolerability of the oral JAK2 inhibitor fedratinib (SAR302503) in healthy volunteers. J Clin Pharmacol. 2014;54(4):415–421.
   PubMed Web of Science ®Google Scholar
• Hart S, Goh KC, Novotny-Diermayr V, et al. SB1518, a novel macrocyclic pyrimidine-based JAK2 inhibitor for the treatment of myeloid and lymphoid malignancies. Leukemia. 2011;25(11):1751–1759.
   PubMed Web of Science ®Google Scholar
• Luo Q, Xiao Z, Peng L. Effects of ruxolitinib on infection in patients with myeloproliferative neoplasm: a meta-analysis. Hematology. 2021;26(1):663–669.
   PubMed Web of Science ®Google Scholar
• Porpaczy E, Tripolt S, Hoelbl-Kovacic A, et al. Aggressive B-cell lymphomas in patients with myelofibrosis receiving JAK1/2 inhibitor therapy. Blood. 2018;132(7):694–706.
   PubMed Web of Science ®Google Scholar
• Pemmaraju N, Kantarjian H, Nastoupil L, et al. Characteristics of patients with myeloproliferative neoplasms with lymphoma, with or without JAK inhibitor therapy. Blood. 2019;133(21):2348–2351.
   PubMed Web of Science ®Google Scholar
• Lin JQ, Li SQ, Li S, et al. A 10-year retrospective cohort study of ruxolitinib and association with nonmelanoma skin cancer in patients with polycythemia vera and myelofibrosis. J Am Acad Dermatol. 2022;86(2):339–344.
   PubMed Web of Science ®Google Scholar
• Jalles C, Lepelley M, Mouret S, et al. Skin cancers under Janus kinase inhibitors: a world health organization drug safety database analysis. Therapie. 2022;77(6):649–656.
   PubMedGoogle Scholar
• Rampotas A, Carter-Brzezinski L, Somervaille TCP, et al. Disease characteristics and outcomes of non-melanoma skin cancers in myeloproliferative neoplasm (MPN) patients treated with ruxolitinib. Blood. 2022;140(Suppl. 1):9721–9723.
   Google Scholar
• Arikan F, Toptas T, Atagunduz IK, et al. Real-life ruxolitinib experience in intermediate-risk myelofibrosis. Blood Res. 2021;56(4):322–331.
   PubMed Web of Science ®Google Scholar
• Barraco F, Greil R, Herbrecht R, et al. Real-world non-interventional long-term post-authorisation safety study of ruxolitinib in myelofibrosis. Br J Haematol. 2020;191(5):764–774.
   Google Scholar
• Quintás-Cardama A, Vaddi K, Liu P, et al. Preclinical characterization of the selective JAK1/2 inhibitor INCB018424: therapeutic implications for the treatment of myeloproliferative neoplasms. Blood. 2010;115(15):3109–3117.
   PubMed Web of Science ®Google Scholar
• Shi JG, Chen X, McGee RF, et al. The pharmacokinetics, pharmacodynamics, and safety of orally dosed INCB018424 phosphate in healthy volunteers. J Clin Pharmacol. 2011;51(12):1644–1654.
   PubMed Web of Science ®Google Scholar
• Asshoff M, Petzer V, Warr MR, et al. Momelotinib inhibits ACVR1/ALK2, decreases hepcidin production, and ameliorates anemia of chronic disease in rodents. Blood. 2017;129(13):1823–1830.
   PubMed Web of Science ®Google Scholar
• Zheng J, Xin Y, Zhang J, et al. Pharmacokinetics and disposition of momelotinib revealed a disproportionate human metabolite—resolution for clinical development. Drug Metab Dispos. 2018;46(3):237–247.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Kiladjian JJ, Catalano JV, et al. SIMPLIFY-1: a phase III randomized trial of momelotinib versus ruxolitinib in Janus kinase inhibitor-naive patients with myelofibrosis. J Clin Oncol. 2017;35(34):3844–3850.
   PubMed Web of Science ®Google Scholar
• Harrison CN, Vannucchi AM, Platzbecker U, et al. 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;5(2):e73–e81.
   PubMed Web of Science ®Google Scholar
• Pardanani A, Harrison C, Cortes JE, et al. Safety and efficacy of fedratinib in patients with primary or secondary myelofibrosis: a randomized clinical trial. JAMA Oncol. 2015;1(5):643–651.
   PubMed Web of Science ®Google Scholar
• Singer JW, Al-Fayoumi S, Ma H, et al. Comprehensive kinase profile of pacritinib, a nonmyelosuppressive Janus kinase 2 inhibitor. J Exp Pharmacol. 2016;8:11–19.
   PubMedGoogle Scholar
• Oh S, Mesa R, Harrison C, et al. MPN-145 retrospective analysis of anemia benefit of pacritinib from the PERSIST-2 trial. Clin Lymphoma Myeloma Leuk. 2022;22(Suppl 2):s327.
   Google Scholar
• Younes A, Romaguera J, Fanale M, et al. Phase I study of a novel oral Janus kinase 2 inhibitor, SB1518, in patients with relapsed lymphoma: evidence of clinical and biologic activity in multiple lymphoma subtypes. J Clin Oncol. 2012;30(33):4161–4167.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Hoffman R, Talpaz M, et al. Pacritinib vs best available therapy, including ruxolitinib, in patients with myelofibrosis: a randomized clinical trial. JAMA Oncol. 2018;4(5):652–659.
   PubMed Web of Science ®Google Scholar
• Gerds AT, Savona MR, Scott BL, et al. Determining the recommended dose of pacritinib: results from the PAC203 dose-finding trial in advanced myelofibrosis. Blood Adv. 2020;4(22):5825–5835.
   PubMed Web of Science ®Google Scholar
• Singer JW, Fleischman A, Al-Fayoumi S, et al. Inhibition of interleukin-1 receptor-associated kinase 1 (IRAK1) as a therapeutic strategy. Oncotarget. 2018;9(70):33416–33439.
   PubMedGoogle Scholar
• Chifotides HT, Bose P, Verstovsek S. Momelotinib: an emerging treatment for myelofibrosis patients with anemia. J Hematol Oncol. 2022;15(1):7.
   PubMed Web of Science ®Google Scholar
• Oh ST, Talpaz M, Gerds AT, et al. ACVR1/JAK1/JAK2 inhibitor momelotinib reverses transfusion dependency and suppresses hepcidin in myelofibrosis phase 2 trial. Blood Adv. 2020;4(18):4282–4291.
   PubMed Web of Science ®Google Scholar
• Sureau L, Orvain C, Ianotto JC, et al. Efficacy and tolerability of Janus kinase inhibitors in myelofibrosis: a systematic review and network meta-analysis. Blood Cancer J. 2021;11:135.
   PubMed Web of Science ®Google Scholar
• Singer JW, Al-Fayoumi S, Taylor J, et al. O'Mahony A. Comparative phenotypic profiling of the JAK2 inhibitors ruxolitinib, fedratinib, momelotinib, and pacritinib reveals distinct mechanistic signatures. PLOS One. 2019;14(9):e0222944.
   PubMed Web of Science ®Google Scholar
• Chifotides HT, Bose P, Masarova L, et al. SOHO state of the art updates and next questions: novel therapies in development for myelofibrosis. Clin Lymphoma Myeloma Leuk. 2022;22(4):210–223.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Kantarjian HM, Estrov Z, et al. Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls. Blood. 2012;120(6):1202–1209.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Kantarjian H, Tefferi A, et al. Evaluating the serial use of the myelofibrosis symptom assessment form for measuring symptomatic improvement: performance in 87 myelofibrosis patients on a JAK1 and JAK2 inhibitor (INCB018424) clinical trial. Cancer. 2011;117(21):4869–4877.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Schwager S, Radia D, et al. The Myelofibrosis Symptom Assessment Form (MFSAF): an evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis. Leuk Res. 2009;33(9):1199–1203.
   PubMed Web of Science ®Google Scholar
• Guaraná M, Soares A, Daumas A, et al. Myeloproliferative Neoplasm Symptom Assessment Form - Total Symptom Score (MPN-SAF TSS) questionnaire: translation, cultural adaptation and validation to Brazilian Portuguese. Hematol Transfus Cell Ther. 2022;44(3):321–327.
   PubMedGoogle Scholar
• Harrison CN, Schaap N, Vannucchi AM, et al. Safety and efficacy of fedratinib, a selective oral inhibitor of Janus kinase-2 (JAK2), in patients with myelofibrosis and low pretreatment platelet counts. Br J Haematol. 2022;198(2):317–327.
   PubMed Web of Science ®Google Scholar
• Newberry KJ, Patel K, Masarova L, et al. Clonal evolution and outcomes in myelofibrosis after ruxolitinib discontinuation. Blood. 2017;130(9):1125–1131.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Kremyanskaya M, Patriarca A, et al. BET inhibitor pelabresib (CPI-0610) combined with ruxolitinib in patients with myelofibrosis—JAK inhibitor-naive or with suboptimal response to ruxolitinib—preliminary data from the manifest study. Presented at the European Hematology Association 2022 Hybrid Congress, Vienna/Virtual; 2022.
   Google Scholar
• Yacoub A, Wang ES, Rampal RK, et al. Abstract CT162: addition of parsaclisib (INCB050465), a PI3Kδ inhibitor, in patients with suboptimal response to ruxolitinib: a phase 2 study in patients with myelofibrosis. Cancer Res. 2021;81(13 Suppl.):CT162–CT162.
   Web of Science ®Google Scholar
• Masarova L, Verstovsek S, Hidalgo-Lopez JE, et al. A phase 2 study of ruxolitinib in combination with azacitidine in patients with myelofibrosis. Blood. 2018;132(16):1664–1674.
   PubMed Web of Science ®Google Scholar
• Harrison CN, Garcia JS, Somervaille TCP, et al. Addition of navitoclax to ongoing ruxolitinib therapy for patients with myelofibrosis with progression or suboptimal response: phase II safety and efficacy. J Clin Oncol. 2022;40(15):1671–1680.
   PubMed Web of Science ®Google Scholar
• Gowin K, Kosiorek H, Dueck A, et al. Multicenter phase 2 study of combination therapy with ruxolitinib and danazol in patients with myelofibrosis. Leuk Res. 2017;60:31–35.
   PubMed Web of Science ®Google Scholar
• Gerds AT, Vannucchi AM, Passamonti F, et al. A phase 2 study of luspatercept in patients with myelofibrosis-associated anemia. Blood. 2019;134(Suppl._1):557–557.
   Google Scholar
• Gerds AT, Vannucchi AM, Passamonti F, et al. Duration of response to luspatercept in patients (pts) requiring red blood cell (RBC) transfusions with myelofibrosis (MF) – updated data from the phase 2 ACE-536-MF-001 study. Blood. 2020;136(Suppl. 1):47–48.
   Google Scholar
• Bose P, Daver N, Pemmaraju N, et al. Sotatercept (ACE-011) alone and in combination with ruxolitinib in patients (pts) with myeloproliferative neoplasm (MPN)-associated myelofibrosis (MF) and anemia. Blood. 2017;130(Suppl. 1):255.
   Google Scholar
• Rampal RK, Verstovsek S, Devlin SM, et al. Safety and efficacy of combined ruxolitinib and thalidomide in patients with myelofibrosis: a phase II study. Blood. 2019;134(Suppl. 1):4163–4163.
   Google Scholar
• Qu S, Xu Z, Qin T, et al. Ruxolitinib combined with prednisone, thalidomide and danazol in patients with myelofibrosis: results of a pilot study. Hematol Oncol. 2022;40(4):787–795.
   PubMed Web of Science ®Google Scholar
• Ali H, Kishtagari A, Maher KR, et al. A phase 1, open-label, dose-escalation study of selinexor plus ruxolitinib in patients with treatment-naïve myelofibrosis. J Clin Oncol. 2022;40(16 Suppl.):7060–7060.
   Web of Science ®Google Scholar
• Bankar A, Gupta V. Investigational non-JAK inhibitors for chronic phase myelofibrosis. Expert Opin Investig Drugs. 2020;29(5):461–474.
   PubMed Web of Science ®Google Scholar
• Waksal JA, Harrison CN, Mascarenhas JO. Novel therapeutics and targets in myelofibrosis. Leuk Lymphoma. 2022;63(5):1020–1033.
   PubMed Web of Science ®Google Scholar