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
Only Modest Benefit Seen in Combining Ruxolitinib, Buparlisib
May 25th 2019A study combining ruxolitinib and buparlisib in myelofibrosis showed only a modest benefit compared with ruxolitinib alone, and further studies to see if there is a synergistic effect between the 2 agents will not continue.
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Ascending Dose Study Demonstrates Safety, Efficacy of Voxelotor in Sickle Cell Disease
May 15th 2019Voxelotor, a first-in-class oral therapy, is both safe and effective in sickle cell disease, according to a phase 1/2 randomized study assessing the drug. These findings were consistent across all doses, ranging from 500 to 1000 mg.
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Pfizer Gets FDA Approval for Tafamadis for ATTR-CM
May 7th 2019The FDA approved Pfizer’s transthyretin stabilizer tafamidis, the first treatment for cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), a rare, incurable disease. Pfizer will sell the oral drugs under the names Vyndaqel and Vyndamax.
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Ruxolitinib Resolves Sarcoidosis in Patient With PV
April 27th 2019Sarcoidosis has rarely been reported with polycythemia vera (PV), an acquired myeloproliferative neoplasm characterized by mutant Janus kinase 2 (JAK2) signaling leading to erythrocyte overproduction. In a recent article in the Journal of the American Academy of Dermatology, physicians report on the case of a female patient with PV and sarcoidosis.
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Tagraxofusp Elicits 90% Response Rate in Patients With Rare, Aggressive Blood Cancer
April 26th 2019Study results demonstrated that in adult patients with blastic plasmacytoid dendritic cell neoplasm, the targeted therapy resulted in high response rates, particularly among treatment-naïve patients.
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Splenectomy Before Stem Cell Transplant May Help Certain Patients With MF
April 20th 2019Splenectomy before allogeneic hematopoietic stem cell transplantation (alloHSCT) might be a promising option in patients with myelofibrosis (MF) who failed to achieve significant spleen response after ruxolitinib therapy, according to results from a recently published study.
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The PAN Foundation Opens New Carcinoid Syndrome Patient Assistance Fund
April 17th 2019The Patient Access Network (PAN) Foundation today opened a new patient assistance program for people living with carcinoid syndrome. Carcinoid syndrome occurs when an underlying carcinoid tumor produces excessive amounts of serotonin, often due to liver metastases.
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Oncologists, Hematologists Welcome CDC Clarification on Opioid Therapy for Chronic Pain
April 9th 2019Oncologists and hematologists are welcoming a clarification from the CDC about the use of opioid pain relief for patients with cancer, past cancer, or sickle cell disease, and they are hopeful that payers see the update so that patients in pain are not denied or delayed relief.
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The PAN Foundation Opens New Waldenstrom Macroglobulinemia Patient Assistance Fund
April 2nd 2019The Patient Access Network (PAN) Foundation today opened a new patient assistance program for people living with Waldenstrom macroglobulinemia, a rare type of blood cancer that originates in the lymphatic system and is characterized by an excess of abnormal white blood cells and proteins called immunoglobulins.
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Researchers Develop Targeted Next-Generation Sequencing Assay for Myeloid Neoplasms
March 30th 2019Researchers from South Korea said they have developed a next-generation sequencing (NGS) assay to detect somatic mutations, translocations, and germline mutations in a single assay for the purpose of supplementing or replacing conventional tests in patients with myeloid neoplasms.
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FDA Releases Draft Guidance Document on Natural History Studies for Rare Diseases
March 26th 2019Earlier this week, the FDA released a draft guidance document titled, "Rare Diseases: Natural History Studies for Drug Development” with the intention of informing both the design and implementation of natural history studies that can be used to support the development of safe and effective treatments for rare diseases.
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UK Patients, Physicians Feel More Burdened by Myeloproliferative Neoplasms
March 22nd 2019A recent study found that patients with myeloproliferative neoplasms living in the United Kingdom, as well as their doctors, felt more burdened by the disease than other patients and providers in other parts of the world.
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Continuing Systemic Treatment in Cutaneous T-Cell Lymphoma Associated With Lower Healthcare Costs
March 20th 2019The findings show that patients with cutaneous T-cell lymphoma who had continuous systemic treatment had average monthly emergency department costs that were $100 lower than those with interrupted treatment.
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Greater Symptom Burden Associated With Medical Disability Leave in Patients With MPNs
March 16th 2019In a recent survey-based study, researchers analyzed the relationship between symptom burden and incidence of medical disability leave (MDL) among patients with myeloproliferative neoplasms, including myelofibrosis, polycythemia vera, and essential thrombocythemia.
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Amphotericin to Fight Cystic Fibrosis? Researchers Hope Human Trials Prove Early Results
March 14th 2019The fungal drug amphotericin, which was discovered in 1953, could possibly help patients with cystic fibrosis fight chronic bacterial lung infections if results from an early study are validated in humans, researchers said Wednesday.
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Study Tracks Myotonic Dystrophy Progression in 5-Year Follow-up
March 9th 2019A longitudinal 5-year observational study to examine functional and structural cerebral changes in adult-onset myotonic dystrophies found some differences between the 2 types of this incurable genetic disorder and said that additional brain studies are needed in light of upcoming treatment trials for this rare disease.
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Fedratinib Earns Priority Review Status for Myelofibrosis From FDA
March 8th 2019Earlier this week, the FDA granted priority review to a new drug application for fedratinib for the treatment of myelofibrosis. The drug had previously been awarded an orphan drug designation for the treatment of primary and secondary myelofibrosis.
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CHMP Recommends First Treatment of FCS for Marketing Approval
March 2nd 2019The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion that an orphan drug, volanesorsen (Waylivra), receive a conditional marketing authorization. The drug would be the first medication approved for the treatment of the familial chylomicronemia syndrome (FCS).
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Liver Biopsies Can Assist With Determining Future Status of Ruxolitinib Therapies
February 23rd 2019A case series discusses how a liver biopsy may prove instrumental in determining if ruxolitinib therapy should be discontinued or continued in patients with myelofibrosis (MF) and polycythemia vera (PV) experiencing liver damage.
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