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Cytopenias

Model illustrates progression to MDS, AML

HT Staff Read Article
Published: 02/21/17

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Induced pluripotent stem cells
Image by James Thomson

Researchers say they have created a model that shows the step-by-step progression from normal blood cells to acute myeloid leukemia (AML). The team generated induced pluripotent stem cell (iPSC) lines capturing disease stages that included preleukemia, low-risk myelodysplastic syndrome (MDS), high-risk MDS, and AML. The researchers then used CRISPR/Cas9 genome editing to induce disease progression and reversal. [Read Article]

Understanding a rare hemoglobin mutation

HT Staff Read Article
Published: 02/18/17

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Woman smoking
Photo by Tiffany Dawn Nicholson

Smoking can prevent anemia in individuals with a rare hemoglobin mutation, according to research published in the Journal of Biological Chemistry. The so-called Kirklareli mutation was found to be the cause of mild anemia in a young woman in Germany. But a smoking habit protected the young woman’s father, who also carried the mutation, from developing anemia. The Kirklareli mutation is one of more than 1000 discovered so far in adult human hemoglobin. [Read Article]

Study reveals patterns of ED use in SCD patients

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Published: 02/15/17

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Doctor examines SCD patient
Photo courtesy of St. Jude
Children’s Research Hospital

Population-based surveillance data has revealed patterns of emergency department (ED) visits among Californians with sickle cell disease (SCD). Previous research suggested that between one-half and two-thirds of SCD patients’ ED visits end in a discharge from the ED, called a treat-and-release visit. The remainder result in admission to a hospital or other treatment facility. The purpose of the current study was to use data from the . . . [Read Article]

Genetic profiling can guide HSCT in MDS, team says

HT Staff Read Article
Published: 02/10/17

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Micrograph showing MDS

Genetic profiling can be used to determine which patients with myelodysplastic syndrome (MDS) are likely to benefit from allogeneic hematopoietic stem cell transplant (HSCT), according to research published in NEJM. Targeted sequencing of 129 genes revealed mutations that, after adjustment for clinical variables, were associated with shorter survival and/or relapse after HSCT.  Patients with mutations in TP53, JAK2, and the RAS pathway tended to . . . [Read Article]

FDA approves IVIG product for PI and chronic ITP

HT Staff Read Article
Published: 02/09/17

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Vials of product
Photo by Bill Branson

The US Food and Drug Administration (FDA) has approved an intravenous immunoglobulin (IVIG) product (Gammaplex® 10%) for the treatment of primary immunodeficiency (PI) and chronic immune thrombocytopenia (ITP) in adults. PI includes, but is not limited to, the humoral immune defect in common variable immunodeficiency, X-linked and congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. [Read Article]

 

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