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
Literature Review Suggests Genetic Skeletal Disorder Therapies Are Continuously Being Developed
January 20th 2020Despite challenges of rare disease drug development, genetic skeletal disorder (GSD) therapies continue to be developed through novel approaches such as drug repurposing and in-utero stem cell transplants, according to a recent review.
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Steroids Ineffective in Treating One-Third of Acute GVHD Pediatric Patients, Study Finds
January 16th 2020A study investigating the clinical phenotype of acute graft-versus-host disease (GVHD) in children reveals that one-third of pediatric patients with the disease are not successfully treated with steroids as a primary GVHD therapy.
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FDA Drug Approvals Report for 2019 Highlights New Treatments for Rare Diseases
January 9th 2020This week, the FDA’s Center for Drug Evaluation and Research (CDER) released its annual report, Advancing Health Through Innovation: New Drug Therapy Approvals, outlining new drugs approved or marketed in America for the first time.
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In the first clinical trial of its kind, Danish researchers investigated the efficacy of a combination treatment of ruxolitinib and low-dose pegylated interferon-α2 (PEG-INF α2) in patients with polycythemia vera and myelofibrosis, according to a study published in Haematologica.
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Increased WBC Count Linked With Thrombotic Events in Patients With PV
January 5th 2020Thrombotic events are a significant cause of morbidity and mortality for patients with polycythemia vera (PV), and previous reports have shown a significant correlation between elevated white blood cell (WBC) count and thrombotic events. A recently published study among patients treated in the US Veterans Health Administration (VHA) has now substantiated those findings, suggesting that controlling WBC count should be an important facet of managing PV.
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Splenomegaly Linked With Thrombosis, Cardiovascular Risk for Patients With ET and PV
January 2nd 2020Splenomegaly, or enlargement of the spleen, is common in patients with Philadelphia-negative myeloproliferative neoplasms, and it is associated with symptoms like early satiety and abdominal pain. The role that splenomegaly has in terms of quality of life and prognosis in primary myelofibrosis is fairly well understood, but it has been less frequently investigated among patients with essential thrombocythemia (ET) and polycythemia vera (PV).
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What We're Reading: Novartis Therapy Lottery; Merck Ebola Vaccine Approval; California Cannabis Use
December 20th 2019Novartis hopes to distribute Zolgensma, its pricey spinal muscular atrophy drug, via lottery; Merck receives FDA approval for its Ebola vaccine, Ervebo; a new law in California hopes to resurrect compassionate use cannabis programs.
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Dr Michael Wang on Finding Chemo-Free Therapies for Mantle Cell Lymphoma
December 17th 2019To avoid the toxicities associated with use of chemotherapy, there has been progress in developing and utilizing chemotherapy-free therapies to treat mantle cell lymphoma, said Michael Wang, MD, professor in the Department of Lymphoma and Myeloma at MD Anderson.
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Disease Burden and Adherence for Patients With Myelofibrosis
December 17th 2019Disease burden is substantial for patients with myelofibrosis, even those with intermediate risk, and a not insubstantial percentage of patients have low or intermediate adherence during treatment, according to 2 abstracts from an Italian clinical trial presented at the 61st American Society of Hematology Annual Meeting & Exposition.
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What We're Reading: Pelosi Drug Bill; Sarepta's Second Chance; Mental Health Care
December 13th 2019Speaker Nancy Pelosi’s, D-California, drug bill passes in the House; the FDA approves Vyondys 53, from Sarepta Therapeutics, to treat Duchenne muscular dystrophy; San Francisco tackles homelessness, mental illness, and substance abuse.
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Genetic Variant Causing Heart Failure in Patients of African Descent Largely Goes Undiagnosed
December 12th 2019Hereditary transthyretin amyloid cardiomyopathy, which is caused by a genetic variant significantly associated with heart failure in individuals of African descent, is underdiagnosed, according to a new study published in JAMA.
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Real-World Data Shows Substantial Burden of Cost, AEs for Patients With MCL
December 7th 2019Patients with mantle cell lymphoma (MCL) face a substantial economic burden and susceptibility to adverse events (AEs) in the real world, according to an abstract presented at the 61st American Society of Hematology Annual Meeting and Exposition.
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Improvements in Survival for Patients With Severe Acute or Severe Chronic GVHD
December 1st 2019Historically, patients with severe acute graft-versus-host disease (GVHD) and severe chronic GVHD as determined by the National Institutes of Health have poor survival. New research being presented at the 61st American Society of Hematology Annual Meeting & Exposition shows that earlier treatment with novel therapies can improve outcomes for these patients.
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First-Line Use of Ruxolitinib and Dosing Modifications in Patients With Polycythemia Vera
November 29th 2019There are no cures available to patients with polycythemia vera (PV), who are first treated with hydroxyurea (HU); ruxolitinib is approved as a second-line therapy in both Europe and the United States for patients who are intolerant of or resistant to HU. Two abstracts being presented at the 61st American Society of Hematology Annual Meeting & Exposition explore the use of ruxolitinib in patients with PV, either in patients who first tried HU or had ruxolitinib as a first-line therapy.
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Payers Slow to Adopt Real-World Evidence in Rare Diseases
November 26th 2019While there is an increased interest in using real-world evidence (RWE) to design, test, and review rare disease treatments, payers may not be as receptive to using RWE when making reimbursement and formulary decisions, according to a new report from Syneos Health.
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Dr David Snyder on When to Add Ruxolitinib to Treat GVHD
November 17th 2019Ruxolitinib should be added to treatment of patients with graft-versus-host disease (GVHD) are not improving on steroids or whose symptoms return after tapering, said David Snyder, MD, associate chair of the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope.
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FDA Approves First Treatment for Rare Blood Disorder, Beta Thalassemia
November 15th 2019Adult patients with beta thalassemia will now have an FDA-approved treatment available with luspatercept-aamt (Reblozyl). The therapy treats the rare inherited blood disorder, which requires patients to have regular red blood cell transfusions.
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Dr David Snyder on Responses to Ruxolitinib in Patients With Acute GVHD
November 7th 2019A majority of patients with acute graft-versus-host disease (GVHD), but not all, have significant responses to the addition of ruxolitinib, said David Snyder, MD, associate chair of the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope.
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Orphan Drug May Be Frequently Used Off-Label, Inflating Revenue, Letter Suggests
November 1st 2019Although eculizumab is only approved by the FDA to treat 3 rare indications, the high and increasing net sales for the drug have raised concerns about off-label use, according to a research letter in JAMA Internal Medicine.
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Dr Naveen Pemmaraju: PCPs Play an Important Role Treating Myelofibrosis
October 29th 2019The local community doctor has an important role in recognizing, diagnosing, and managing myelofibrosis, said Naveen Pemmaraju, MD, associate professor in the Department of Leukemia at MD Anderson Cancer Center.
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Dr David Snyder Discusses Treating GVHD, the Differences Between Chronic and Acute GVHD
October 25th 2019Ruxolitinib was recently approved to treat acute graft-versus-host disease (GVHD), and trials are ongoing to understand the best way to use the treatment with transplantation, said David Snyder, MD, associate chair of the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope.
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The 12 Democratic candidates for president spent a good deal of time at their debate Tuesday night discussing healthcare; a group of state attorneys general tried to convince a US District Court judge to give them more time to create a settlement in their opioid lawsuits but failed; Alexion Pharmaceuticals agreed to buy small biotech Achillion Pharmaceuticals in a deal initially valued at $930 million.
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Analysis Highlights How Rare Diseases Have Broader Fiscal Impact Than Health Costs
October 16th 2019Rare diseases may affect a small number of people, but they have fiscal impacts beyond just healthcare costs. A new study in Orphanet Journal of Rare Diseases used a public economic framework to identify how hereditary transthyretin-mediated amyloidosis has a public economic burden beyond just health costs in the Netherlands.
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