SOHO State of the Art Updates and Next Questions: Novel Therapies in Development for Myelofibrosis

Improved survival of patients with myelofibrosis in the last decade: Single‐center experience

(2022) Lucia Masarova et al.   CANCER

Disease Modification in Myelofibrosis: An Elusive Goal?

(2022) Pankit Vachhani et al.   JOURNAL OF CLINICAL ONCOLOGY

Momelotinib: an emerging treatment for myelofibrosis patients with anemia

(2022) Helen T. Chifotides et al.   Journal of Hematology & Oncology

Clinical Significance of Bone Marrow Blast Percentage in Patients with Myelofibrosis and the Effect of Ruxolitinib Therapy

(2021) Lucia Masarova et al.   Clinical Lymphoma Myeloma & Leukemia

MOMENTUM: momelotinib vs danazol in patients with myelofibrosis previously treated with JAKi who are symptomatic and anemic

(2021) Srdan Verstovsek et al.   Future Oncology

Therapeutic Cancer Vaccination With a Peptide Derived From the Calreticulin Exon 9 Mutations Induces Strong Cellular Immune Responses in Patients With CALR-Mutant Chronic Myeloproliferative Neoplasms

(2021) Jacob Handlos Grauslund et al.   Frontiers in Oncology

Accelerated Phase of Myeloproliferative Neoplasms

(2021) Omar A. Shahin et al.   ACTA HAEMATOLOGICA

Ruxolitinib rechallenge in resistant or intolerant patients with myelofibrosis: Frequency, therapeutic effects, and impact on outcome

(2021) Francesca Palandri et al.   CANCER

Immunotherapy and Immunomodulation in Myeloproliferative Neoplasms

(2021) Naveen Pemmaraju et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Epigenetic Dysregulation of Myeloproliferative Neoplasms

(2021) Andrew Dunbar et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Targeting CD123 in hematologic malignancies: identifying suitable patients for targeted therapy

(2021) Mrinal M. Patnaik et al.   LEUKEMIA & LYMPHOMA

SOHO State of the Art Updates and Next Questions: Identifying and Treating “Progression” in Myelofibrosis

(2021) Prithviraj Bose et al.   Clinical Lymphoma Myeloma & Leukemia

MPN-038: PACIFICA: A Randomized, Controlled Phase 3 Study of Pacritinib Versus Physician’s Choice in Patients with Primary or Secondary Myelofibrosis and Severe Thrombocytopenia

(2021) John Mascarenhas et al.   Clinical Lymphoma Myeloma & Leukemia

MPN-303: Longitudinal and Individual Symptom Analyses from the SIMPLIFY-1 Study Demonstrate Clinically Comparable Symptomatic Benefit of Momelotinib to Ruxolitinib in JAK Inhibitor-Naive Myelofibrosis Patients

(2021) Ruben Mesa et al.   Clinical Lymphoma Myeloma & Leukemia

Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia

(2021) Srdan Verstovsek et al.   HAEMATOLOGICA

Randomized, Single-Blind, Multicenter Phase II Study of Two Doses of Imetelstat in Relapsed or Refractory Myelofibrosis

(2021) John Mascarenhas et al.   JOURNAL OF CLINICAL ONCOLOGY

BOREAS: A global phase 3 study of KRT-232, a first-in-class murine double minute 2 (MDM2) inhibitor in TP53WT relapsed/refractory (R/R) myelofibrosis (MF).

(2021) Srdan Verstovsek et al.   JOURNAL OF CLINICAL ONCOLOGY

Paradigm shift: combination BET and JAK inhibition in myelofibrosis

(2021) John Mascarenhas et al.   LEUKEMIA

Efficacy and tolerability of Janus kinase inhibitors in myelofibrosis: a systematic review and network meta-analysis

(2021) Léa Sureau et al.   Blood Cancer Journal

TGF-β1 protein trap AVID200 beneficially affects hematopoiesis and bone marrow fibrosis in myelofibrosis

(2021) Lilian Varricchio et al.   JCI Insight

Final results of a phase 2 clinical trial of LCL161, an oral SMAC mimetic for patients with myelofibrosis

(2021) Naveen Pemmaraju et al.   Blood Advances

Favorable overall survival with imetelstat in relapsed/refractory myelofibrosis patients compared with real-world data

(2021) Andrew T. Kuykendall et al.   ANNALS OF HEMATOLOGY

Real-world survival of US patients with intermediate- to high-risk myelofibrosis: impact of ruxolitinib approval

(2021) Srdan Verstovsek et al.   ANNALS OF HEMATOLOGY

Disease-Modifying Potential of BET Inhibitor Pelabresib (CPI-0610) As Demonstrated By Improvements in Bone Marrow Function and Clinical Activity in Patients with Myelofibrosis - Preliminary Data

(2021) Srdan Verstovsek et al.   BLOOD

Final Results of a Phase 2 Study of Sotatercept (ACE-011) for Anemia of MPN-Associated Myelofibrosis

(2021) Prithviraj Bose et al.   BLOOD

Pelabresib (CPI-0610) Monotherapy in Patients with Myelofibrosis - Update of Clinical and Translational Data from the Ongoing Manifest Trial

(2021) Marina Kremyanskaya et al.   BLOOD

Treatment of Myelofibrosis Patients with the TGF-β 1/3 Inhibitor AVID200 (MPN-RC 118) Induces a Profound Effect on Platelet Production

(2021) John Mascarenhas et al.   BLOOD

PD-1 inhibition in advanced myeloproliferative neoplasms

(2021) Gabriela Hobbs et al.   Blood Advances

The Myelodepletive Phenotype in Myelofibrosis: Clinical Relevance and Therapeutic Implication

(2020) Bridget K. Marcellino et al.   Clinical Lymphoma Myeloma & Leukemia

Management of myelofibrosis after ruxolitinib failure

(2020) Claire N Harrison et al.   ANNALS OF HEMATOLOGY

Secondary leukemia to myeloproliferative neoplasms

(2020) Andrew Dunbar et al.   BLOOD

Management of challenging myelofibrosis after JAK inhibitor failure and/or progression

(2020) Robyn M. Scherber et al.   BLOOD REVIEWS

Clinical outcomes of IDH2 ‐mutated advanced‐phase Ph‐negative myeloproliferative neoplasms treated with enasidenib

(2020) Anand A. Patel et al.   BRITISH JOURNAL OF HAEMATOLOGY

First-in-human study of palcitoclax (APG-1252), a novel dual Bcl-2/Bcl-xL inhibitor, demonstrated advantages in platelet safety while maintaining anticancer effect in U.S. patients with metastatic solid tumors.

(2020) Nehal J. Lakhani et al.   JOURNAL OF CLINICAL ONCOLOGY

Neoplastic fibrocytes play an essential role in bone marrow fibrosis in Jak2V617F-induced primary myelofibrosis mice

(2020) Yoshinori Ozono et al.   LEUKEMIA

Management of myelofibrosis after ruxolitinib failure

(2020) Prithviraj Bose et al.   LEUKEMIA & LYMPHOMA

Patient characteristics and outcomes after ruxolitinib discontinuation in patients with myelofibrosis

(2020) John Mascarenhas et al.   JOURNAL OF MEDICAL ECONOMICS

Prognostic value of blasts in peripheral blood in myelofibrosis in the ruxolitinib era

(2020) Lucia Masarova et al.   CANCER

Bcl‐xL represents a therapeutic target in Philadelphia negative myeloproliferative neoplasms

(2020) Jessica Petiti et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Fedratinib, a newly approved treatment for patients with myeloproliferative neoplasm-associated myelofibrosis

(2020) Moshe Talpaz et al.   LEUKEMIA

PRMT5 inhibition modulates E2F1 methylation and gene regulatory networks leading to therapeutic efficacy in JAK2V617F mutant MPN

(2020) Friederike Pastore et al.   Cancer Discovery

Fedratinib in myelofibrosis

(2020) Ann Mullally et al.   Blood Advances

Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms

(2020) Prithviraj Bose et al.   Cancers

Heat Shock Proteins and PD-1/PD-L1 as Potential Therapeutic Targets in Myeloproliferative Neoplasms

(2020) Steven De Almeida et al.   Cancers

ACVR1/JAK1/JAK2 inhibitor momelotinib reverses transfusion dependency and suppresses hepcidin in myelofibrosis phase 2 trial

(2020) Stephen T. Oh et al.   Blood Advances

Determining the recommended dose of pacritinib: results from the PAC203 dose-finding trial in advanced myelofibrosis

(2020) Aaron T. Gerds et al.   Blood Advances

Luspatercept for the treatment of anemia in myelodysplastic syndromes (MDS) and primary myelofibrosis (PMF)

(2019) Pierre Fenaux et al.   BLOOD

Tagraxofusp in Blastic Plasmacytoid Dendritic-Cell Neoplasm

(2019) Naveen Pemmaraju et al.   NEW ENGLAND JOURNAL OF MEDICINE

Increased SLAMF7high monocytes in myelofibrosis patients harboring JAK2V617F provide a therapeutic target of elotuzumab

(2019) Takaaki Maekawa et al.   BLOOD

Immune Checkpoint Blockade Enhances Shared Neoantigen-Induced T Cell Immunity Directed against Mutated Calreticulin in Myeloproliferative Neoplasms

(2019) Cansu Cimen Bozkus et al.   Cancer Discovery

Mutant calreticulin in myeloproliferative neoplasms

(2019) Joan How et al.   BLOOD

Advances in potential treatment options for myeloproliferative neoplasm associated myelofibrosis

(2019) Prithviraj Bose   Expert Opinion on Orphan Drugs

Phase 1b Study of the Epichaperome Inhibitor PU-H71 Administered Orally with Ruxolitinib Continuation for the Treatment of Patients with Myelofibrosis

(2019) Naveen Pemmaraju et al.   BLOOD

The Oral JAK2/IRAK1 Inhibitor Pacritinib Demonstrates Spleen Volume Reduction in Myelofibrosis Patients Independent of JAK2V617F Allele Burden

(2019) Srdan Verstovsek et al.   BLOOD

Safety and Efficacy of Combined Ruxolitinib and Thalidomide in Patients with Myelofibrosis: A Phase II Study

(2019) Raajit K. Rampal et al.   BLOOD

Life after ruxolitinib: Reasons for discontinuation, impact of disease phase, and outcomes in 218 patients with myelofibrosis

(2019) Francesca Palandri et al.   CANCER

Dual Targeting of Oncogenic Activation and Inflammatory Signaling Increases Therapeutic Efficacy in Myeloproliferative Neoplasms

(2018) Maria Kleppe et al.   CANCER CELL

Significance of thrombocytopenia in patients with primary and postessential thrombocythemia/polycythemia vera myelofibrosis

(2018) Lucia Masarova et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

The development of Bruton's tyrosine kinase (BTK) inhibitors from 2012 to 2017: A mini-review

(2018) Chengyuan Liang et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

JAK2/IDH-mutant–driven myeloproliferative neoplasm is sensitive to combined targeted inhibition

(2018) Anna Sophia McKenney et al.   JOURNAL OF CLINICAL INVESTIGATION

Pacritinib to treat myelofibrosis patients with thrombocytopenia

(2018) Douglas Tremblay et al.   Expert Review of Hematology

Momelotinib therapy for myelofibrosis: a 7-year follow-up

(2018) Ayalew Tefferi et al.   Blood Cancer Journal

Management of Myelofibrosis-Related Cytopenias

(2018) Prithviraj Bose et al.   Current Hematologic Malignancy Reports

Momelotinib versus best available therapy in patients with myelofibrosis previously treated with ruxolitinib (SIMPLIFY 2): a randomised, open-label, phase 3 trial

(2018) Claire N Harrison et al.   Lancet Haematology

Pacritinib vs Best Available Therapy, Including Ruxolitinib, in Patients With Myelofibrosis

(2018) John Mascarenhas et al.   JAMA Oncology

A phase II study of ruxolitinib in combination with azacytidine in patients with myelofibrosis

(2018) Lucia Masarova et al.   BLOOD

Nuclear-cytoplasmic transport is a therapeutic target in myelofibrosis

(2018) Dongqing Yan et al.   CLINICAL CANCER RESEARCH

SIMPLIFY-1: A Phase III Randomized Trial of Momelotinib Versus Ruxolitinib in Janus Kinase Inhibitor–Naïve Patients With Myelofibrosis

(2017) Ruben A. Mesa et al.   JOURNAL OF CLINICAL ONCOLOGY

Long-term survival in patients treated with ruxolitinib for myelofibrosis: COMFORT-I and -II pooled analyses

(2017) Srdan Verstovsek et al.   Journal of Hematology & Oncology

Imetelstat, a telomerase inhibitor, differentially affects normal and malignant megakaryopoiesis

(2017) G Mosoyan et al.   LEUKEMIA

Immunotherapy based approaches in myelofibrosis

(2017) Lucia Masarova et al.   Expert Review of Hematology

Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): a single-arm, open-label, non-randomised, phase 2, multicentre study

(2017) Claire N Harrison et al.   Lancet Haematology

Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial

(2017) Ruben A Mesa et al.   Lancet Haematology

A phase 1/2, open-label study evaluating twice-daily administration of momelotinib in myelofibrosis

(2016) Vikas Gupta et al.   HAEMATOLOGICA

Role of neoplastic monocyte-derived fibrocytes in primary myelofibrosis

(2016) Srdan Verstovsek et al.   JOURNAL OF EXPERIMENTAL MEDICINE

A phase I, open-label, dose-escalation, multicenter study of the JAK2 inhibitor NS-018 in patients with myelofibrosis

(2016) S Verstovsek et al.   LEUKEMIA

BET protein bromodomain inhibitor-based combinations are highly active against post-myeloproliferative neoplasm secondary AML cells

(2016) D T Saenz et al.   LEUKEMIA

JAK2V617F allele burden ⩾50% is associated with response to ruxolitinib in persons with MPN-associated myelofibrosis and splenomegaly requiring therapy

(2016) G Barosi et al.   LEUKEMIA

A phase 1/2, open-label study evaluating twice-daily administration of momelotinib in myelofibrosis

(2016) Vikas Gupta et al.   HAEMATOLOGICA

A pooled analysis of overall survival in COMFORT-I and COMFORT-II, 2 randomized phase III trials of ruxolitinib for the treatment of myelofibrosis

(2015) A. M. Vannucchi et al.   HAEMATOLOGICA

A pooled analysis of overall survival in COMFORT-I and COMFORT-II, 2 randomized phase III trials of ruxolitinib for the treatment of myelofibrosis

(2015) A. M. Vannucchi et al.   HAEMATOLOGICA

Safety and Efficacy of Fedratinib in Patients With Primary or Secondary Myelofibrosis

(2015) Animesh Pardanani et al.   JAMA Oncology

Clinical effect of driver mutations of JAK2, CALR, or MPL in primary myelofibrosis

(2014) E. Rumi et al.   BLOOD

Improved targeting of JAK2 leads to increased therapeutic efficacy in myeloproliferative neoplasms

(2014) Neha Bhagwat et al.   BLOOD

CALR vs JAK2 vs MPL-mutated or triple-negative myelofibrosis: clinical, cytogenetic and molecular comparisons

(2014) A Tefferi et al.   LEUKEMIA

The expression of lysyl-oxidase gene family members in myeloproliferative neoplasms

(2013) T. Tadmor et al.   AMERICAN JOURNAL OF HEMATOLOGY

Rationale for Targeting the PI3K/Akt/mTOR Pathway in Myeloproliferative Neoplasms

(2013) Niccolò Bartalucci et al.   Clinical Lymphoma Myeloma & Leukemia

Anti-transforming growth factor-β therapy in patients with myelofibrosis

(2013) John Mascarenhas et al.   LEUKEMIA & LYMPHOMA

Dual PI3K/AKT/mTOR Inhibitor BEZ235 Synergistically Enhances the Activity of JAK2 Inhibitor against Cultured and Primary Human Myeloproliferative Neoplasm Cells

(2013) W. Fiskus et al.   MOLECULAR CANCER THERAPEUTICS

Combined Targeting of JAK2 and Bcl-2/Bcl-xL to Cure Mutant JAK2-Driven Malignancies and Overcome Acquired Resistance to JAK2 Inhibitors

(2013) Michaela Waibel et al.   Cell Reports

Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls

(2012) S. Verstovsek et al.   BLOOD

One Thousand Patients With Primary Myelofibrosis: The Mayo Clinic Experience

(2012) Ayalew Tefferi et al.   MAYO CLINIC PROCEEDINGS

A Double-Blind, Placebo-Controlled Trial of Ruxolitinib for Myelofibrosis

(2012) Srdan Verstovsek et al.   NEW ENGLAND JOURNAL OF MEDICINE

JAK Inhibition with Ruxolitinib versus Best Available Therapy for Myelofibrosis

(2012) Claire Harrison et al.   NEW ENGLAND JOURNAL OF MEDICINE

JAK2V617F-Mediated Phosphorylation of PRMT5 Downregulates Its Methyltransferase Activity and Promotes Myeloproliferation

(2011) Fan Liu et al.   CANCER CELL

Heat Shock Protein 90 Inhibitor Is Synergistic with JAK2 Inhibitor and Overcomes Resistance to JAK2-TKI in Human Myeloproliferative Neoplasm Cells

(2011) W. Fiskus et al.   CLINICAL CANCER RESEARCH

JAK2V617F negatively regulates p53 stabilization by enhancing MDM2 via La expression in myeloproliferative neoplasms

(2011) M Nakatake et al.   ONCOGENE

Red blood cell transfusion-dependency implies a poor survival in primary myelofibrosis irrespective of IPSS and DIPSS

(2010) C. Elena et al.   HAEMATOLOGICA

HSP90 is a therapeutic target in JAK2-dependent myeloproliferative neoplasms in mice and humans

(2010) Sachie Marubayashi et al.   JOURNAL OF CLINICAL INVESTIGATION

Low-dose thalidomide in myelofibrosis

(2008) R. Weinkove et al.   HAEMATOLOGICA