AnatomyOrganismsDiseasesChemicals and DrugsAnalytical, Diagnostic and Therapeutic Techniques and EquipmentPhenomena and Processes
Polycythemia VeraPrimary MyelofibrosisPolycythemiaThrombocythemia, EssentialJanus Kinase 2Myeloproliferative DisordersThrombocytosisPipobromanErythroid Precursor CellsAloeReceptors, ThrombopoietinErythropoietinPhlebotomyErythropoiesisHematopoiesis, ExtramedullaryGranulocytesSplenomegalyHydroxyureaThrombosisMegakaryocytesReceptors, ErythropoietinBloodlettingHematocritBone MarrowMutationMutation, MissensePlatelet CountAmino Acid SubstitutionErythroid CellsThrombopoietinGPI-Linked ProteinsLeukemia, Myeloid, Chronic, Atypical, BCR-ABL NegativeColony-Forming Units AssayHematopoietic Stem CellsBudd-Chiari SyndromeIsoantigensHomozygoteLeukocytosisChromosomes, Human, 19-20Bone Marrow Neoplasms
Graft-versus-RuxolitinibHigh-risk myelofibrosisNeoplasmsEssential thrombocytosisPatientsChronicSplenomegalySecondary myelofibrosisMyeloproliferative neoplasm characterizedClonalAlfa-2b-njftJAK2Hematopoietic stemExtramedullaryJakafi2022Primary or secondaryPlateletsTherapeuticsSplenectomyTeardropTreatmentAllogeneicAnemiaAbnormalPeripheralRespiratoryMPNsClinical trialPhlebotomyTargeted therapyDiseaseDisorder
Graft-versus-4
  • Total patients grew across myelofibrosis (MF), polycythemia vera (PV) and graft-versus-host disease (GVHD). (businesswire.com)
  • Jakafi treats a pair of bone-marrow diseases, myelofibrosis and polycythemia vera, as well as steroid-refractory acute graft-versus-host disease (GVHD), a complication of stem cell transplants. (thickmarkets.com)
  • Jakafi's (a first-in-class JAK1/JAK2 inhibitor approved for polycythemiavera, myelofibrosis and refractory acute graft-versus-host disease) revenues came in at almost $580 million, up 7% from the year-ago quarter's number. (yahoo.com)
  • Earlier in March, the FDA issued a complete response letter for ruxolitinib extended-release (XR) tablets for once-daily use in the treatment of certain types of myelofibrosis, polycythemia vera and graft-versus-host disease. (yahoo.com)
Ruxolitinib13
  • Phase 1/2 Study of the Activin Receptor-Like Kinase (ALK)-2 Inhibitor Zilurgisertib (INCB000928, LIMBER-104) as Monotherapy or with Ruxolitinib (RUX) in Patients (pts) with Anemia due to Myelofibrosis (MF) (Abstract #7017. (businesswire.com)
  • Phase 1b, Open-Label Study of Add-On Therapy with CK0804 in Participants with Myelofibrosis, with Suboptimal Response to Ruxolitinib (Abstract #TPS7087. (businesswire.com)
  • In the SIMPLIFY-1 multicenter, randomized, double-blind, phase III study, safety and efficacy of momelotinib was compared with ruxolitinib in 432 patients with myelofibrosis who had not received prior treatment with a JAK inhibitor. (medscape.com)
  • Ruxolitinib was the first drug approved (2011) to specifically treat patients with the intermediate- or high-risk myelofibrosis, including primary myelofibrosis, postpolycythemia vera myelofibrosis, and postessential thrombocythemia myelofibrosis. (org.in)
  • This approval was based on the results of 2 large randomized phase III trials that enrolled patients with intermediate-2 or high-risk myelofibrosis and compared ruxolitinib with placebo (COMFORT-I) 1 or best available therapy (COMFORT-II) 2 in JAK inhibitor-naïve patients with myelofibrosis. (org.in)
  • A greater proportion of patients in study 1 experienced a ≥ 50% reduction in the myelofibrosis total symptom score during treatment with ruxolitinib than with placebo (46% vs. 5%, P (org.in)
  • Long-term survival in patients treated with ruxolitinib for myelofibrosis: COMFORT-I and -II pooled analyses 3 showed OS advantage also in ruxolitinib treated patient. (org.in)
  • Ruxolitinib was approved by FDA (2014) to treat polycythemia vera patients who have an inadequate response to or cannot tolerate hydroxyurea. (org.in)
  • This was a phase 3 open-label study to evaluate the efficacy and safety of ruxolitinib versus standard therapy in patients with polycythemia vera who had an inadequate response to or had unacceptable side effects from hydroxyurea. (org.in)
  • In this RESPONSE trial it was shown that ruxolitinib was superior to standard therapy in controlling the hematocrit, reducing the spleen volume, and improving symptoms associated with polycythemia vera and hence it was approved for this indication. (org.in)
  • RESPONSE-2 assessed the efficacy and safety of ruxolitinib in controlling disease in patients with polycythaemia vera without splenomegaly (no palpable spleen at enrollment) who need second-line therapy. (org.in)
  • Ruxolitinib is the first and only FDA-approved treatment for this indication. (org.in)
  • In this article, we provide updated data on ruxolitinib therapy for patients with myelofibrosis and offer expert opinion on the appropriate use of this agent in the community practice. (cancernetwork.com)
High-risk myelofibrosis1
  • Momelotinib is indicated for intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF (post polycythemia vera [PV] and post essential thrombocythemia [ET]), in adults with anemia. (medscape.com)
Neoplasms4
  • These mutations are not specific to myelofibrosis, but are observed in other myeloproliferative neoplasms, specifically polycythemia vera and essential thrombocythemia. (wikipedia.org)
  • Bromodomain and Extra-Terminal (BET) Inhibitor INCB057643 (LIMBER-103) in Patients (pts) with Relapsed or Refractory Myelofibrosis (R/R MF) and Other Advanced Myeloid Neoplasms: A Phase 1 Study (Abstract #7069. (businesswire.com)
  • Statin- and bisphosphonate treatment in patients with the Philadelphia-negative chronic myeloproliferative neoplasms - essential thrombocytosis, polycythemia vera and hypercellular myelofibrosis. (clinicaltrialsregister.eu)
  • New York, NY-Myeloproliferative neoplasms (MPNs) include myelofibrosis, polycythemia vera, and essential thrombocytopenia, and affect an estimated 295,00 individuals in the United States. (valuebasedcancer.com)
Essential thrombocytosis1
Patients21
  • Patients with myelofibrosis have an increased risk of acute meyloid leukemia and frank bone marrow failure. (wikipedia.org)
  • In 2016, prefibrotic primary myelofibrosis was formally classified as a distinct condition that progresses to overt PMF in many patients, the primary diagnostic difference being the grade of fibrosis. (wikipedia.org)
  • During the meeting several analyses will be presented in patients with haemophilia A, paroxysmal nocturnal hemoglobinuria (PNH), immune thrombocytopenia (ITP), relapsed or refractory diffuse large b-cell lymphoma, myelofibrosis, and haemophagocytic lymphohistiocytosis. (centralillinoisproud.com)
  • VONJO is an oral, small-molecule JAK2 inhibitor currently marketed by Swedish Orphan Biovitrum AB ("Sobi"), used for the treatment myelofibrosis ("MF") patients with severe thrombocytopenia. (drihealthcare.com)
  • Portal hypertension occurs in approximately 7% of patients with primary myelofibrosis and may be related to increased portal flow resulting from marked splenomegaly and to intrahepatic obstruction resulting from thrombotic obliteration of small portal veins. (medscape.com)
  • Patients with primary myelofibrosis are also prone to developing infectious complications because of defects in humoral immunity. (medscape.com)
  • In patients with primary myelofibrosis, the hematopoietic system is most affected. (medscape.com)
  • In patients with myelofibrosis (MF) and polycythemia vera (PV) treated with Jakafi in clinical trials, the rates of thromboembolic events were similar in Jakafi and control treated patients. (jakafi.com)
  • The key secondary endpoint in COMFORT-I was the proportion of patients who experienced a ≥ 50% improvement from baseline in myelofibrosis total symptom score at 24 weeks. (org.in)
  • In addition, we are taking steps to manage the anticipated increase in COVID-19 related requests for Jakavi without interrupting access for patients taking the drug for its licensed indications. (lifepronow.com)
  • Jakavi is approved by the European Commission for the treatment of adult patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea and for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (MF) (also known as chronic idiopathic MF), post-polycythemia vera MF or post-essential thrombocythemia MF. (lifepronow.com)
  • So, some patients where if their risk score is higher and their risk for thrombosis, that may be an appropriate indication. (powerfulpatients.org)
  • The long-term risks of polycythemia vera (PV) include leukemic and fibrotic transformation, which occur in fewer than 5% and 10% of patients, respectively, at 10 years. (medscape.com)
  • San Francisco, CA-As a result of recent advances in therapy, indications for stem-cell transplant are expanding, along with the number of patients eligible for the procedure. (valuebasedcancer.com)
  • With the availability of an FDA-approved, next-generation interferon for this indication, it's time that we focus on preserving the long-term health of patients with polycythemia vera. (cliniexpert.com)
  • We are incredibly proud to deliver on our goal of bringing treatments like BESREMi to the polycythemia vera community where there is clear unmet need for more effective, tolerable and durable treatments to preserve patients' health and well-being," said Ko-Chung Lin, Ph.D., Co-Founder and Chief Executive Officer for PharmaEssentia and inventor of ropeginterferon alfa-2b-njft. (cliniexpert.com)
  • As the approved indication is identical to the patients enrolling on PACIFICA, enrollment of additional patients in the U.S. is closed effective March 2022. (pacifica-trial.com)
  • Although AML is the most common indication for allogeneic HCT, the relatively small number of patients and the highly complex nature of the procedure often render prospective studies on HCT unfeasible. (apbmt.org)
  • Food and Drug Administration expanded the indication of pomalidomide (POMALYST, Celgene Corporation) to include treating adult patients with AIDS-related Kaposi sarcoma after failure of highly active antiretroviral therapy and Kaposi sarcoma in adult patients who are HIV-negative. (blogspot.com)
  • Shanghai and Hong Kong, PRC, August24, 2021 - Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, today announced that China's National Medical Products Administration has approved a Phase II study of selinexor (XPOVIO®) for the treatment of patients with myelofibrosis (MF). (antengene.com)
  • Dr. Jay Mei, Founder, Chairman and CEO of Antengene , commented: "The NMPA's approval for this clinical trial of selinexor in patients with MF marks another major milestone in our effort in developing selinexor in a broad range of diseases, and a big step towards expanding potential indications for this candidate drug. (antengene.com)
Chronic4
  • Polycythemia vera is a chronic myeloproliferative neoplasm characterized by an increase in morphologically normal red cells (its hallmark), but also white cells and platelets. (msdmanuals.com)
  • The reality of living with a rare and chronic cancer like polycythemia vera is that it is often underrecognized and the limited treatments available cannot properly address the disease beyond the symptoms. (cliniexpert.com)
  • Polycythemia Vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. (cliniexpert.com)
  • intended to alter the natural history of this chronic and progressive myeloid malignancy, preventing its progression, for instance, to myelofibrosis (MF) and acute leukemia (AL). (targetedonc.com)
Splenomegaly2
Secondary myelofibrosis1
  • This condition may result in cardiovascular complications such as thrombosis and embolism, as well as transformation to secondary myelofibrosis or leukemia. (cliniexpert.com)
Myeloproliferative neoplasm characterized1
  • Polycythemia vera is a myeloproliferative neoplasm characterized by clonal hematopoiesis and an absolute increase in the red blood cell mass, with an associated leukocytosis and thrombocytosis. (targetedonc.com)
Clonal3
  • Myelofibrosis is a clonal neoplastic disorder of hematopoiesis, the formation of blood cellular components. (wikipedia.org)
  • Primary myelofibrosis is a clonal disorder arising from the neoplastic transformation of early hematopoietic stem cells. (medscape.com)
  • MF is a clonal bone narrow neoplasm which can emerge either as primary MF (PMF), polycythemia vera (PV) or essential thrombocythemia (ET). (antengene.com)
Alfa-2b-njft1
  • BURLINGTON, Mass.--(BUSINESS WIRE) November 12, 2021 --PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that the U.S. Food and Drug Administration (FDA) has approved BESREMi® (ropeginterferon alfa-2b-njft) for the treatment of adults with polycythemia vera (PV). (cliniexpert.com)
JAK21
  • There is an association between mutations to the JAK2, CALR, or MPL genes and myelofibrosis. (wikipedia.org)
Hematopoietic stem1
  • Myelofibrosis (MF) is a hematopoietic stem cell malignancy classified as a myeloproliferative neoplasm (MPN). (cancernetwork.com)
Extramedullary1
Jakafi2
  • Our first quarter results demonstrate continued year-over-year double-digit revenue growth driven by Jakafi, which grew across all indications, and Opzelura, which is on track to become one of the most successful dermatology launches in recent years. (businesswire.com)
  • The new regulatory nod marked the fourth for Jakafi, and it is the only FDA-approved treatment for two of its indications (acute GVHD and polycythemia vera). (thickmarkets.com)
20222
  • It was approved by the U.S. Food and Drug Administration ("FDA") in February 2022 and is the only approved treatment for the indication. (drihealthcare.com)
  • Pacritinib (commercially known as VONJO™) was approved by U.S. Food and Drug Administration (FDA) on February 28, 2022 for the treatment of adults with myelofibrosis with a platelet count below 50 x 10 9 /L. (pacifica-trial.com)
Primary or secondary2
  • Intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis. (unboundmedicine.com)
  • [ 20 ] The JAK inhibitor fedratinib is approved treatment of adults with intermediate-2 or high-risk primary or secondary (post-PV or post-essential thrombocythemia) myelofibrosis. (medscape.com)
Platelets1
  • Polycythemia vera involves increased production of red blood cells (RBCs), white blood cells (WBCs), and platelets. (msdmanuals.com)
Therapeutics2
  • targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. (researchandmarkets.com)
  • Navtemadlin is under clinical development by Kartos Therapeutics and currently in Phase III for Post-Polycythemia Vera Myelofibrosis (PPV-MF). (pharmaceutical-technology.com)
Splenectomy2
Teardrop1
Treatment3
  • approval was based on the results of the COMFORT (COntrolled MyeloFibrosis study with ORal JAK inhibitor Treatment) I and II studies. (cancernetwork.com)
  • Changing a treatment approach for your essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF) can be intimidating. (powerfulpatients.org)
  • Aetna considers therapeutic phlebotomy experimental and investigational for treatment of the following conditions because its effectiveness for these indications has not been established (not an all-inclusive list). (aetna.com)
Allogeneic1
  • Allogeneic HCT has been widely used, while autologous HCT, which is still useful, is less prevalent because of concerns around post-transplant relapse, as well as the recently extended application of allogeneic HCT. (apbmt.org)
Anemia2
Abnormal1
Peripheral1
Respiratory1
  • In 2019, sales of branded biologics for respiratory indications reached nearly $1.6 billion in the major pharmaceutical markets under study (United States, EU5, and Japan). (clarivate.com)
MPNs1
Clinical trial1
  • FDA concluded that, 'in light of the failed primary analysis and data deficiencies noted, the clinical trial does not by itself support the indication. (formularywatch.com)
Phlebotomy1
Targeted therapy1
  • Identify and understand the targeted therapy areas and indications for E3 Ubiquitin Protein Ligase Mdm2 (Double Minute 2 Protein or Hdm2 or Oncoprotein Mdm2 or p53 Binding Protein Mdm2 or MDM2 or EC 6.3.2. (researchandmarkets.com)
Disease1
  • 1] PV and ET are both capable of progressing to a fibrotic stage that clinically resembles PMF, and collectively these three disease entities are termed myelofibrosis (MF). (cancernetwork.com)
Disorder2
  • A new indication was approved for paliperidone palmitate ( Invega Sustenna , Janssen ), the once-monthly atypical long-acting antipsychotic to treat schizoaffective disorder as either monotherapy or adjunctive therapy. (formularywatch.com)
  • The disorder is often seen in association with other behavioral conditions such as depression, substance abuse and post-traumatic stress disorder, and it is often under-diagnosed and under-treated," said Antony Loebel, M.D., President and Chief Executive Officer at Sunovion. (med-chemist.com)