Variant not associated with CLL, AIHA, or ITP in certain patients

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DNA helix
Image by Spencer Phillips
New research suggests there is no association between the PTPN22 R620W polymorphism and chronic lymphocytic leukemia (CLL) or autoimmune hematologic disorders in patients from the Republic of Macedonia. Past studies have shown an association between the PTPN22 R620W variant and both CLL and autoimmune diseases in patients from... [Read Article]
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Researchers develop genetics-based prognostic tool for MDS

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Micrograph showing MDS
Researchers have developed a new risk model for primary myelodysplastic syndromes (MDS) that integrates genetic and clinical information. The research team considered the current standard for prognostication—the revised International Prognostic Scoring System (IPSS-R)—to be too complex, limited to newly diagnosed cases, and missing information on mutations and age. So they devised a... [Read Article]
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CHMP backs proposed biosimilars of pegfilgrastim

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Neulasta Onpro Kit
Photo from Amgen
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval for three proposed biosimilars of pegfilgrastim—Ziextenzo, Pelmeg, and Fulphila. If approved by the European Commission (EC), these products would be used for the same indication as the reference medicine, Neulasta (pegfilgrastim). Neulasta has been... [Read Article]
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Sequencing informs prognosis after HSCT in MDS

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HSCT preparation
Photo by Chad McNeeley
Gene sequencing early after transplant may provide important prognostic information in patients with myelodysplastic syndromes (MDS), according to a new study. Patients who had disease-associated mutations in the bone marrow 30 days after hematopoietic stem cell transplant (HSCT) were significantly more likely to experience disease progression and have lower... [Read Article]
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HIF1A could be therapeutic target for MDS

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Lab mouse
The transcription factor HIF1A could be a therapeutic target for “a broad spectrum” of patients with myelodysplastic syndromes (MDS), according to researchers. Preclinical experiments indicated that HIF1A fuels the biological processes that cause different types of MDS. Researchers also found that inhibiting HIF1A reversed MDS symptoms and prolonged survival in mouse models of... [Read Article]
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AMP publishes report on DNA variants in CMNs

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Polycythemia vera
Image courtesy of AFIP
A new report addresses the clinical relevance of DNA variants in chronic myeloid neoplasms (CMNs). The report is intended to aid clinical laboratory professionals with the management of most CMNs and the development of high-throughput pan-myeloid sequencing testing panels. The authors list 34 genes they consider “critical” for sequencing... [Read Article]
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FDA grants priority review to drug for PNH

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Red blood cells
The US Food and Drug Administration (FDA) has accepted for priority review the biologics license application (BLA) for ALXN1210, a long-acting C5 complement inhibitor. With this BLA, Alexion Pharmaceuticals, Inc., is seeking approval for ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). The FDA grants priority review to applications... [Read Article]
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Familial risk of myeloid malignancies

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Three generations of
women in a family
A large study has revealed “the strongest evidence yet” supporting genetic susceptibility to myeloid malignancies, according to a researcher. The study showed that first-degree relatives of patients with myeloid malignancies had double the risk of developing a myeloid malignancy themselves, when compared to the general population. The researchers... [Read Article]
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FA pathway key to DNA repair after CRISPR cutting

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DNA repair
Image by Tom Ellenberger
New research suggests the Fanconi anemia (FA) pathway plays a key role in repairing double-strand breaks (DSBs) created by CRISPR-Cas9 genome editing. Researchers said they found that Cas9-induced single-strand template repair requires the FA pathway, and the protein FANCD2 localizes to Cas9-induced DSBs. The team said this research provides... [Read Article]
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CHMP recommends generic deferiprone

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Micrograph showing thalassemia
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval for Deferiprone Lipomed to treat iron overload in patients with thalassemia major. Deferiprone Lipomed is a generic version of the iron chelating agent Ferriprox, which has been authorized in the European Union since August 1999. According to... [Read Article]
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