Novel Concepts of Treatment for Patients with Myelofibrosis and Related Neoplasms

Final Results of Phase 2 Clinical Trial of LCL161, a Novel Oral SMAC Mimetic/IAP Antagonist, for Patients with Intermediate to High Risk Myelofibrosis

(2020) Naveen Pemmaraju et al.   BLOOD

Results from a Phase 2 Study of Navitoclax in Combination with Ruxolitinib in Patients with Primary or Secondary Myelofibrosis

(2020) Claire N Harrison et al.   BLOOD

A Phase 2 Study of Luspatercept in Patients with Myelofibrosis-Associated Anemia

(2020) Aaron T. Gerds et al.   BLOOD

Results of PAC203: A Randomized Phase 2 Dose-Finding Study and Determination of the Recommended Dose of Pacritinib

(2020) Aaron T. Gerds et al.   BLOOD

Thromboembolic Risk Reduction and High Rate of Complete Molecular Response with Long-Term Use of Ropeginterferon Alpha-2b in Polycythemia Vera: Results from a Randomized Controlled Study

(2020) Jean-Jacques Kiladjian et al.   BLOOD

Mutation‐enhanced international prognostic systems for essential thrombocythaemia and polycythaemia vera

(2020) Ayalew Tefferi et al.   BRITISH JOURNAL OF HAEMATOLOGY

Safety and efficacy of the maximum tolerated dose of givinostat in polycythemia vera: a two-part Phase Ib/II study

(2020) Alessandro Rambaldi et al.   LEUKEMIA

Long-term efficacy and safety of ruxolitinib versus best available therapy in polycythaemia vera (RESPONSE): 5-year follow up of a phase 3 study

(2020) Jean-Jacques Kiladjian et al.   Lancet Haematology

Ropeginterferon alfa-2b versus standard therapy for polycythaemia vera (PROUD-PV and CONTINUATION-PV): a randomised, non-inferiority, phase 3 trial and its extension study

(2020) Heinz Gisslinger et al.   Lancet Haematology

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

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

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

(2020) Yoshinori Ozono et al.   LEUKEMIA

Givinostat: an emerging treatment for polycythemia vera

(2020) Helen T. Chifotides et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

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

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

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

(2020) Friederike Pastore et al.   Cancer Discovery

Finding a Jill for JAK: Assessing Past, Present, and Future JAK Inhibitor Combination Approaches in Myelofibrosis

(2020) Andrew T. Kuykendall et al.   Cancers

Aurora kinase A inhibition provides clinical benefit, normalizes megakaryocytes and reduces bone marrow fibrosis in patients with myelofibrosis

(2019) Naseema Gangat et al.   CLINICAL CANCER RESEARCH

Programmed Cell Death Receptor (PD-1) Ligand (PD-L1) expression in Philadelphia chromosome-negative myeloproliferative neoplasms

(2019) Jen-Chin Wang et al.   LEUKEMIA RESEARCH

Oral Idasanutlin in Patients with Polycythemia Vera

(2019) John Mascarenhas et al.   BLOOD

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

Pegylated Interferon Alfa-2a for Polycythemia Vera or Essential Thrombocythemia Resistant or Intolerant to Hydroxyurea

(2019) Abdulraheem Yacoub et al.   BLOOD

Essential Thrombocythemia

(2019) Ayalew Tefferi et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2019) Francesca Palandri et al.   CANCER

The role of JAK2 inhibitors in MPNs 7 years after approval

(2018) Francesco Passamonti et al.   BLOOD

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

(2018) Maria Kleppe et al.   CANCER CELL

MIPSS70+ Version 2.0: Mutation and Karyotype-Enhanced International Prognostic Scoring System for Primary Myelofibrosis

(2018) Ayalew Tefferi et al.   JOURNAL OF CLINICAL ONCOLOGY

MIPSS70: Mutation-Enhanced International Prognostic Score System for Transplantation-Age Patients With Primary Myelofibrosis

(2018) Paola Guglielmelli et al.   JOURNAL OF CLINICAL ONCOLOGY

Autocrine Tnf signaling favors malignant cells in myelofibrosis in a Tnfr2-dependent fashion

(2018) William L. Heaton et al.   LEUKEMIA

GIPSS: genetically inspired prognostic scoring system for primary myelofibrosis

(2018) Ayalew Tefferi et al.   LEUKEMIA

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

Dysregulated iron metabolism in polycythemia vera: etiology and consequences

(2018) Yelena Z. Ginzburg et al.   LEUKEMIA

Nuclear-cytoplasmic transport is a therapeutic target in myelofibrosis

(2018) Dongqing Yan et al.   CLINICAL CANCER RESEARCH

Hydroxyurea prevents arterial and late venous thrombotic recurrences in patients with myeloproliferative neoplasms but fails in the splanchnic venous district. Pooled analysis of 1500 cases

(2018) Valerio De Stefano et al.   Blood Cancer Journal

Downregulation of GATA1 drives impaired hematopoiesis in primary myelofibrosis

(2017) Laure Gilles et al.   JOURNAL OF CLINICAL INVESTIGATION

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

Ruxolitinib for the treatment of inadequately controlled polycythaemia vera without splenomegaly (RESPONSE-2): a randomised, open-label, phase 3b study

(2017) Francesco Passamonti et al.   LANCET ONCOLOGY

Myeloproliferative leukemia protein activation directly induces fibrocyte differentiation to cause myelofibrosis

(2017) T Maekawa et al.   LEUKEMIA

A phase II study of panobinostat in patients with primary myelofibrosis (PMF) and post-polycythemia vera/essential thrombocythemia myelofibrosis (post-PV/ET MF)

(2017) John Mascarenhas et al.   LEUKEMIA RESEARCH

The thrombopoietin/MPL axis is activated in the Gata1low mouse model of myelofibrosis and is associated with a defective RPS14 signature

(2017) M Zingariello et al.   Blood Cancer Journal

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

Investigational histone deacetylase inhibitors (HDACi) in myeloproliferative neoplasms

(2016) Prithviraj Bose et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Role of neoplastic monocyte-derived fibrocytes in primary myelofibrosis

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

Long-term findings from COMFORT-II, a phase 3 study of ruxolitinib vs best available therapy for myelofibrosis

(2016) C N Harrison et al.   LEUKEMIA

Correlation of mutation profile and response in patients with myelofibrosis treated with ruxolitinib

(2015) K. P. Patel et al.   BLOOD

Ropeginterferon alfa-2b, a novel IFN -2b, induces high response rates with low toxicity in patients with polycythemia vera

(2015) H. Gisslinger et al.   BLOOD

The effect of long-term ruxolitinib treatment on JAK2p.V617F allele burden in patients with myelofibrosis

(2015) M. Deininger et al.   BLOOD

Targeting megakaryocytic-induced fibrosis in myeloproliferative neoplasms by AURKA inhibition

(2015) Qiang Jeremy Wen et al.   NATURE MEDICINE

A Pilot Study of the Telomerase Inhibitor Imetelstat for Myelofibrosis

(2015) Ayalew Tefferi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Telomerase Inhibitor Imetelstat in Patients with Essential Thrombocythemia

(2015) Gabriela M. Baerlocher et al.   NEW ENGLAND JOURNAL OF MEDICINE

Ruxolitinib versus Standard Therapy for the Treatment of Polycythemia Vera

(2015) Alessandro M. Vannucchi et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Integrated genomic analysis illustrates the central role of JAK-STAT pathway activation in myeloproliferative neoplasm pathogenesis

(2014) R. Rampal et al.   BLOOD

The orally bioavailable MDM2 antagonist RG7112 and pegylated interferon   2a target JAK2V617F-positive progenitor and stem cells

(2014) M. Lu et al.   BLOOD

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

(2014) A Tefferi et al.   LEUKEMIA

Genomic and functional analysis of leukemic transformation of myeloproliferative neoplasms

(2014) Raajit Rampal et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Characterization of the TGF- 1 signaling abnormalities in the Gata1low mouse model of myelofibrosis

(2013) M. Zingariello et al.   BLOOD

Revised response criteria for myelofibrosis: International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus report

(2013) A. Tefferi et al.   BLOOD

Revised response criteria for polycythemia vera and essential thrombocythemia: an ELN and IWG-MRT consensus project

(2013) G. Barosi et al.   BLOOD

Anagrelide compared with hydroxyurea in WHO-classified essential thrombocythemia: the ANAHYDRET Study, a randomized controlled trial

(2013) H. Gisslinger et al.   BLOOD

A phase I study of panobinostat (LBH589) in patients with primary myelofibrosis (PMF) and post-polycythaemia vera/essential thrombocythaemia myelofibrosis (post-PV/ET MF)

(2013) John Mascarenhas et al.   BRITISH JOURNAL OF HAEMATOLOGY

A phase II study of Givinostat in combination with hydroxycarbamide in patients with polycythaemia vera unresponsive to hydroxycarbamide monotherapy

(2013) Guido Finazzi et al.   BRITISH JOURNAL OF HAEMATOLOGY

ABT-199, a potent and selective BCL-2 inhibitor, achieves antitumor activity while sparing platelets

(2013) Andrew J Souers et al.   NATURE MEDICINE

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

Correlation of blood counts with vascular complications in essential thrombocythemia: analysis of the prospective PT1 cohort

(2012) P. J. Campbell et al.   BLOOD

Phase II Study of Single-Agent Navitoclax (ABT-263) and Biomarker Correlates in Patients with Relapsed Small Cell Lung Cancer

(2012) C. M. Rudin et al.   CLINICAL CANCER RESEARCH

Givinostat and hydroxyurea synergize in vitro to induce apoptosis of cells from JAK2V617F myeloproliferative neoplasm patients

(2012) Ariel Amaru Calzada et al.   EXPERIMENTAL HEMATOLOGY

Therapy with the histone deacetylase inhibitor pracinostat for patients with myelofibrosis

(2012) Alfonso Quintás-Cardama et al.   LEUKEMIA RESEARCH

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

TNF  facilitates clonal expansion of JAK2V617F positive cells in myeloproliferative neoplasms

(2011) A. G. Fleischman et al.   BLOOD

Risk factors for arterial and venous thrombosis in WHO-defined essential thrombocythemia: an international study of 891 patients

(2011) A. Carobbio et al.   BLOOD

Phase I Study of Navitoclax (ABT-263), a Novel Bcl-2 Family Inhibitor, in Patients With Small-Cell Lung Cancer and Other Solid Tumors

(2011) Leena Gandhi et al.   JOURNAL OF CLINICAL ONCOLOGY

Substantial Susceptibility of Chronic Lymphocytic Leukemia to BCL2 Inhibition: Results of a Phase I Study of Navitoclax in Patients With Relapsed or Refractory Disease

(2011) Andrew W. Roberts et al.   JOURNAL OF CLINICAL ONCOLOGY

p53 Lesions in Leukemic Transformation

(2011) Ashot Harutyunyan et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2011) M Nakatake et al.   ONCOGENE

PRM-151 (recombinant human serum amyloid P/pentraxin 2) for the treatment of fibrosis

(2010) J.S. Duffield et al.   DRUG NEWS & PERSPECTIVES

Navitoclax, a targeted high-affinity inhibitor of BCL-2, in lymphoid malignancies: a phase 1 dose-escalation study of safety, pharmacokinetics, pharmacodynamics, and antitumour activity

(2010) Wyndham H Wilson et al.   LANCET ONCOLOGY

IDH1 and IDH2 mutation analysis in chronic- and blast-phase myeloproliferative neoplasms

(2010) A Pardanani et al.   LEUKEMIA

Somatic Mutations ofIDH1andIDH2in the Leukemic Transformation of Myeloproliferative Neoplasms

(2010) Anthony Green et al.   NEW ENGLAND JOURNAL OF MEDICINE

Leukocytosis and Risk Stratification Assessment in Essential Thrombocythemia

(2008) Alessandra Carobbio et al.   JOURNAL OF CLINICAL ONCOLOGY

A phase II study of 5-azacitidine for patients with primary and post-essential thrombocythemia/polycythemia vera myelofibrosis

(2008) A Quintás-Cardama et al.   LEUKEMIA