ASH 2019 HIGHLIGHTS

    • admin | March 28, 2021

    The American Society of Hematology is always a mad dash, a chaotic rush of activity, all focused on blood. MPNRF goes each year to hear the latest and greatest from researchers and companies focused on PV, ET, and MF, both basic science and results of clinical trials. We also use this opportunity to connect with clinicians who treat patients globally, providing our free materials for them to hand out at their clinic and letting them know about initiatives like the Stem Cell Transplant Timing Tool and our participation in the MPN Advocates Network. 

    It’s always an inspiration to hear of the breadth of work being done to better understand how and why people develop PV, ET, and MF, and how we can find better management and eventually a cure or cures. We used the opportunity of the community gathering to share what we at the Foundation have learned along the way in partnering with other organizations and stakeholders in the community at Novartis’ patient advocacy dinner as well as host our own steering committee meetings for the MPN Interferon Initiative and myMPN.

    We will have an in-depth analysis of the research reports from ASH soon, but in the meantime here are some highlights to get you started:

    • Drs. Ruben Mesa, Robyn Scherber and Gareth Morgan gave an excellent interview on Patient Power, focused on both myeloma and MPN. – VIDEO
    • Another video, this one by MPN Advocacy and Education, International, also featured Dr. Mesa. – VIDEO
    • Constellation reported that over 80% of the MF patients on trial had a response on all outcome measures, even exceeding the 35% svr used as a clinically validated outcome measure in MPN drug trials now. – VIDEO
    • Dr. Mesa reported live on Twitter from the MPN Clinical Trial Session. He caught Jean-Jacques Kiladjian’s report that patients on long term therapy with Ropeginterferon had decreased thrombosis and higher rates of molecular response. Dr. Mesa also shared that a Phase III trial looking at Ropeginterferon in Essential Thrombocythemia is opening soon including at the Mays Cancer Center where he is Director. – VIDEO
    • On Luspatercept, Dr. Aaron Gerds observed the need for medications to help with transfusion-dependent MF. “Many of these patients’ anemia progresses to the point of requiring transfusions, which have many downstream effects such as transfusion reactions, development of allo-antibodies or iron overload. This is not to mention the reduction in quality of life that is associated with transfusion dependence,” says the study’s lead author, Aaron T. Gerds, MD, of Cleveland Clinic Cancer Center’s Department of Hematology and Medical Oncology. “It’s a widespread issue for these patients, and in the past, treatment options have been severely limited at best. We are working to change this.” – VIDEO

    The following are a selection of publications from ASH. We will have an analysis of the significance reports from ASH in early 2020. 

    • Cord Blood Transplantation Is an Effective Treatment Option in Patients with Myelodysplastic and Myeloproliferative Syndromes – VIDEO
    • Outcomes Following Second Allogeneic Haematopoietic Cell Transplantation in Patients with Myelofibrosis: A Retrospective Study on Behalf of the Chronic Malignancies Working Party of EBMT – VIDEO
    • Impact of Donor-Derived CD34+ Infused Cell Dose on Outcomes of Patients Undergoing Allo-HCT Following Reduced Intensity Regimen for Myelofibrosis: a Study from the Chronic Malignancies Working Party of the EBMT. – VIDEO
    • Significant Impact of the Molecular Profile on the Prognosis of Patients with Myeloproliferative Neoplasms and Splanchnic Vein Thromboses – VIDEO
    • Outcomes of Patients Receiving Direct Oral Anticoagulants for Myeloproliferative Neoplasm Associated Venous Thromboembolism – VIDEO
    • Validation of the International Prognostic Score for Thrombosis in Essential Thrombocythemia. – VIDEO
    • Germline Mutations Predispose to Familial Myeloproliferative Neoplasms. – VIDEO
    • Genomic Profiling of a Phase III Clinical Trial of Interferon Versus Hydroxyurea in MPN Patients Reveals Mutation-Specific & treatment-specific Patterns of Response- VIDEO
    • Predicting the Long-Term Efficacy of Ifnα in JAK2V617F and Calr-Mutated MPN Patients. – VIDEO
    • Fedratinib Induces Spleen Responses and Reduces Symptom Burden in Patients with Myeloproliferative Neoplasm (MPN)-Associated Myelofibrosis (MF) and Low Platelet Counts, who were Either Ruxolitinib-Naïve or were Previously Treated with Ruxolitinib – VIDEO
    • Identifying Cytokine Biomarkers of Response to Pegylated-Interferon Therapy in Polycythemia Vera and Essential Thrombocythemia: Correlative Analysis from the MPN-RC 111/112 Trials – VIDEO
    • Germline Genetic Factors Affecting Interferon Alpha Therapy in Polycythemia Vera – VIDEO
    • Significantly Upregulated Thrombo-Inflammatory Genes Are Normoregulated or Significantly Downregulated during Treatment with Interferon-Alpha2 in Patients with Philadelphia-Negative Chronic Myeloproliferative Neoplasms – VIDEO
    • Final Results of Prospective Treatment with Pegylated Interferon Alfa-2a for Patients with Polycythemia Vera and Essential Thrombocythemia in First and Second-Line Settings – VIDEO
    • Recombinant Interferon-α Reduces Thrombotic Events in Patients with Polycythemia Vera – VIDEO
    • Baseline Mutational Status of Patients with Myelofibrosis and Anemia in the Realise Trial and Impact on Outcome – VIDEO
    • Combination Treatment with Imetelstat, a Telomerase Inhibitor, and Ruxolitinib Depletes Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells – VIDEO
    • Ruxolitinib (RUX) Induced Meaningful and Directional Changes in the Bone Marrow Microenvironment of Patients with Myelofibrosis Enrolled in the COMFORT-I Study – VIDEO
    • Health-Related Quality of Life (HRQoL) in Patients with Myelofibrosis Treated with Fedratinib, an Oral, Selective Inhibitor of Janus Kinase 2 (JAK2), in the Randomized, Placebo-Controlled, Phase III JAKARTA Study – VIDEO
    • Fedratinib Induces Spleen Responses in Patients with Myeloproliferative Neoplasm-Associated Intermediate- or High-Risk Myelofibrosis (MF) Previously Exposed to Ruxolitinib (RUX), Regardless of Reason for Discontinuing RUX – VIDEO
    • Health-Related Quality of Life (HRQoL) with Fedratinib, a Selective, Oral Inhibitor of Janus Kinase 2 (JAK2), in the Phase II JAKARTA2 Study in Patients with Intermediate- or High-Risk Myelofibrosis Previously Treated with Ruxolitinib – VIDEO
    • Pacritinib Demonstrates Efficacy Versus Best Available Therapy in Myelofibrosis Patients with Severe Thrombocytopenia in Two Phase 3 Studies – VIDEO
    • The Oral JAK2/IRAK1 Inhibitor Pacritinib Demonstrates Spleen Volume Reduction in Myelofibrosis Patients Independent of JAK2V617F Allele Burden – VIDEO
    • Requirements for Janus Kinase Inhibitor–Naïve Myelofibrosis Patients Treated with Momelotinib Compared Head to Head with Ruxolitinib – VIDEO
    • Results from a Phase 1/2 Clinical Trial of Tagraxofusp (SL-401) in Patients with Intermediate, or High Risk, Relapsed/Refractory Myelofibrosis – VIDEO
    • Evaluation of Tagraxofusp (SL-401) Alone and in Combination with Ruxolitinib for the Treatment of Myeloproliferative Neoplasms – VIDEO
    • Analysis of Metformin Effects on Bone Marrow Fibrosis and Disease Progression in Primary Myelofibrosis Patients: Preliminary Results of an Open-Label Phase II Trial (FIBROMET) – VIDEO
    • Expansion of Prior Existing TP53 Mutated Clones in Polycythemia Vera Patients Treated with Idasanutlin – VIDEO
    • A Phase 2a Study of the LSD1 Inhibitor Img-7289 (bomedemstat) for the Treatment of Myelofibrosis – VIDEO
    • Safety and Efficacy of Combined Ruxolitinib and Thalidomide in Patients with Myelofibrosis: Phase II Study – VIDEO
    • MANIFEST, a Phase 2 Study of CPI-0610, a Bromodomain and Extraterminal Domain Inhibitor (BETi), As onotherapy or “Add-on” to Ruxolitinib, in Patients with Refractory or Intolerant Advanced Myelofibrosis – VIDEO
    • Preliminary Report of MANIFEST, a Phase 2 Study of CPI-0610, a Bromodomain and Extraterminal Domain Inhibitor (BETi), in Combination with Ruxolitinib, in JAK Inhibitor (JAKi) Treatment Naïve Myelofibrosis Patients – VIDEO
    • A Phase 2 Study of Luspatercept in Patients with Myelofibrosis-Associated Anemia – VIDEO
    • Altered Dynamics of Monocyte Subpopulations and Pro-Inflammatory Signaling Pathways in Polycythemia Vera Revealed By Mass Cytometry – VIDEO
    • TNFR1 Blockade Rather than TNFR2 Blockade Reduces Chronic Inflammation in JAK2+/V617F Mice – VIDEO
    • Dysregulated IL-6/GP130/JAK Signaling in Calreticulin Mutated Myeloproliferative Neoplasms (MPN) – VIDEO
    • Zinc-Dependent Homomultimerization of Mutant Calreticulin Is Required for MPN Pathogenesis – VIDEO
    • New Paradigms for the Mechanisms of Thrombopoietin Receptor Activation and Dysregulation By the JAK2V617F Mutation – VIDEO
    • Molecular, Epigenetic and Gene Expression Profiling of Triple Negative Essential Thrombocythaemia – VIDEO
    • Phase I Clinical Trial of CK0801 (cord blood regulatory T cells) in Patients with Bone Marrow Failure Syndrome (BMF) Including Aplastic Anemia, Myelodysplasia and Myelofibrosis – VIDEO
    • IL-1β Secreted from Mutant Cells Carrying JAK2-V617F favors Early Clonal Expansion and Promotes MPN Disease Initiation and Progression – VIDEO
    • The landscape of TP53 Mutations in MPN – VIDEO
    • Worse Outcomes in Males with MPN Are Associated with Qualitative and Quantitative Differences in Non-Driver Mutational Burden – VIDEO
    • ATM Germline Variant Increases the Risk of MPN Progression – VIDEO
    • High Mobility Group A1 Chromatin Regulators: Key Epigenetic Switches and Therapeutic Targets Required for Leukemic Transformation in JAK2 Mutant MPN – VIDEO
    • Exon-Level Genomic Abnormalities Are Frequent in Cytogenetically Normal Patients with Philadelphia-Chromosome Negative Myeloproliferative Neoplasms (MPNs) – VIDEO
    • HSCs Fated to Progress to Blast Phase Can be Detected in Myelofibrosis Patients Several Years Prior to Leukemic Transformation – VIDEO