January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
Study Finds 74% Long-Term Survival Rate for Patients With Myelofibrosis
February 23rd 2019The only curative treatment for myelofibrosis (MF) continues to be allogeneic hematopoietic stem cell transplant (HSCT). Occasionally, adverse events posttransplantation can occur and usually present within the first 2 years after posttransplant. Researchers recently sought to analyze the outcome of 2-year disease-free survivors in a systematic review published in Haematologica.
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A Tale of Drug Company Collaboration on a Rare Disease
February 14th 2019The PML Consortium formed among several pharmaceutical companies to prevent and treat a rare disease that emerged among patients taking immunomodulatory drugs. It could offer a model for finding solutions for adverse drug reactions.
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FDA Approves the First Drug Specifically Indicated for aTTP
February 11th 2019On February 6, the FDA approved caplacizumab-yhdp (Cablivi), the first therapy specifically indicated for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults. The drug is approved for use in combination with plasma exchange and immunosuppressive therapy.
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Thymectomy Plus Prednisone Shows Benefit in Patients With Nonthymomatous Myasthenia Gravis
February 10th 2019Five-year results from the extended randomized MGTX trial demonstrated that the addition of thymectomy to prednisone leads to long-lasting lower quantitative myasthenia gravis (QMG) scores and prednisone dosing.
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ASCO Names "Progress in Treating Rare Cancers" as the Advance of the Year
January 31st 2019Reflecting on a year of breakthrough advancements in the treatment of rare, difficult to treat cancers, the American Society of Clinical Oncology (ASCO) has named “Progress in Treating Rare Cancers” as the Advance of the Year.
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Defective Toll-Like Receptor Regulation May Predispose Individuals to MPNs
January 28th 2019A recent study reports that monocytes from patients with myeloproliferative neoplasms (MPNs) have a defective negative regulation of Toll-like receptor (TLR) signaling that allows for the unrestrained production of tumor necrosis factor alpha after TLR activation. Additionally, the authors write that TLR signaling not only contributes to chronic inflammation in patients with MPN, but also may predispose individuals to acquire MPNs.
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Results From Phase 3 Trial Report Sorafenib Show Positive Results for Treating Desmoid Tumors
January 13th 2019Patients with progressive, symptomatic, or recurrent desmoid tumors who received sorafenib have significantly improved progression-free survival (PFS) rates at 1 and 2 years compared with patients who received placebo, according to the results of a recent phase 3 trial.
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Rethinking the Business of Cures
January 11th 2019One-time curative treatments provide a huge challenge to health systems that were not created with them in mind. Despite having no approved treatments, bluebird bio has proactively released a model to pay for these one-time cures in a way that provides value to patients and the health system.
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Investigators Seek to Better Understand the Molecular Basis of Polycythemia Vera
January 7th 2019While a common genetic mutation among patients with polycythemia vera (PV) is V617F in exon 14 of the Janus kinase 2 (JAK2) gene—which activates the tyrosine kinase—it has been reported that patients with V617F-negative PV have mutations in exon 12 of JAK2. Exon 12 mutations are involved in approximately 3% of patients with PV, and these patients often have reduced erythroblasts in the bone marrow and hypercellular bone marrow.
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5 Things About the Orphan Drug Act
January 4th 2019January 4 marks the anniversary of the Orphan Drug Act, which was enacted in 1983. Since the law was passed, it has successfully encouraged more orphan drug development, but some now say drug makers are manipulating the system and the incentives need to be revisited.
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Early Study Promising for Ruxolitinib, Decitabine for Myeloproliferative Neoplasms
January 3rd 2019A phase 1 trial by showed that decitabine with ruxolitinib was generally well tolerated and displayed promising clinical efficacy in patients with accelerated or blast-phase myeloproliferative neoplasms (MPN-AP/BP).
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FDA Approves First Drug to Treat Blastic Plasmacytoid Dendritic Cell Neoplasm
January 3rd 2019The FDA has approved Stemline Therapeutics’ tagraxofusp-erzs (Elzonris), the first drug approved to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients aged 2 years or older.
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Bill Addressing Sickle Cell Disease/Heritable Blood Disorders Signed Into Law
December 29th 2018The Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2018, which reauthorizes a sickle cell disease prevention and treatment program and provides grants for research, surveillance, prevention, and treatment of heritable blood disorders, has passed Congress and been signed into law.
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Study Suggests Support Cells in the Brain Are Key Contributors to Huntington Disease
December 20th 2018Faulty glia cells in the brain may trigger Huntington disease (HD), a finding that may potentially create a path for new treatments and therapies, according to a recent study in Cell Stem Cell.
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Daily Bleed Diaries of Patients With Hemophilia A Show Surprisingly High Number of Untreated Bleeds
December 13th 2018The observational, noninterventional study was part of an emicizumab clinical development program in which a daily bleed and medication questionnaire developed by the sponsor on a handheld device was used to prospectively collect data on treatment with factor VIII or bypassing agents in adult and adolescent people with hemophilia A, with and without inhibitors to FVIII.
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Study Confirms Safe Use of Opioids for Pain Control in Sickle Cell Disease
December 11th 2018According to a study results presented at the 60th American Society of Hematology Annual Meeting & Exposition, the use of opioid medications for pain control in sickle cell disease (SCD) is relatively safe, and there has been no associated increase in hospital SCD mortality.
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A literatur survey concludes that the efficacy of recombinant activated factor VII administered by continuous infusion (rFVIIa CI) in patients with congenital hemophilia with inhibitors or congenital factor VII deficiency undergoing surgery and during bleeding episodes appears to be high and comparable to that of rFVIIa delivered by bolus injection.
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Hydroxyurea Better Than Phlebotomy at Preventing Nonfatal Arterial Thrombosis in Polycythemia Vera
December 4th 2018The researchers wrote that theirs was the first such study to document the greater antithrombotic protection of hydroxyurea over PHL against arterial thrombosis. They also found that the 2 treatments lent similar protection from venous thrombosis.
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Discovery of Subtype-Specific Transcriptional Regulators in AML Can Improve Personalized Treatment
November 29th 2018A new study published in Nature Genetics suggests there may be new ways for personalized and precise treatment of acute myeloid leukemia (AML) that could increase the chances of survival in patients with this aggressive cancer of the white blood cells.
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Prevalence of Pulmonary Hypertension in Patients With MPN Lower Than Previously Reported
November 27th 2018Pulmonary hypertension (PH) has been reported to be associated with myeloproliferative neoplasms (MPN) in 5% to 48% of MPN patients. Now, authors of the largest PH study in patients with Philadelphia chromosome-negative MPN have concluded that the prevalence of PH is lower than has been previously reported.
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New Guideline Released on Managing Complications in Polycythemia Vera
November 23rd 2018The British Society for Haematology has released a new updated guideline that offers practical guidance for the management of specific situations and complications of polycythemia vera and outlines management of the diverse types of secondary erythrocytosis.
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Early Data Suggests CC-486 Relatively Safe, Effective in Thrombocytopenic Patients With MDS
November 21st 2018There have been few therapeutic options for treating thrombocytopenia in MDS patients. Now, early phase data suggest that CC-486 (oral azacytidine, an investigational drug sponsored by Celgene) is a relatively safe and effective treatment for thrombocytopenic patients with MDS.
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Crizanlizumab Can Reduce or Delay Pain Crises Events in Patients With Sickle Cell Disease
November 14th 2018Treatment with the investigational drug crizanlizumab (SEG101) reduced pain in patients with sickle cell disease (SCD) who were experiencing vaso-occlusive crisis according to posthoc results of the phase II SUSTAIN study published online in The American Journal of Hematology. The study found that more than twice as many patients with SCD treated with crizanlizumab did not experience a VOC compared with those treated with placebo.
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