hematologists

subcutaneous injection

Rondoraptivon Pegol Increases Factor VIII Half-Life and Levels in Hemophilia A Patients

A new study has found that the von Willebrand factor (VWF)-binding aptamer rondoraptivon pegol increased the half-life of substituted factor VIII (FVIII) as well as endogenous FVIII levels in patients with hemophilia A. The study, published in Blood, also found that the patients tolerated the subcutaneous treatment well. “Recently, rondoraptivon pegol (previously BT200), a VWF-binding…

Young Children Drawing

New Approach Could Improve Mental Health Screening in Hemophilia

A team from St. Jude Children’s Research Hospital in New York and Vanderbilt University Medical Center in Nashville, Tennessee, have developed an approach to help identify children with hemophilia at risk for attention-deficit/hyperactivity disorder (ADHD). “A reliable method of screening that leads to early identification of [inattention (IN)]/[hyperactivity/impulsivity (HI)] can facilitate early intervention in the…

Mastocytosis

Avapritinib Shows Potential for Treating KIT-Negative Advanced SM

A 53-year-old male diagnosed with KIT-negative advanced systemic mastocytosis (SM) had his tryptase level decreased and his symptoms relieved after starting treatment with avapritinib (Ayvakit®). “In our patient, the starting dose was 200 mg daily, which led to a rapid decline of tryptase level, with symptomatic improvement within months at a reduced dose of 100…

technician placing a sample on assay tube

Droplet Digital PCR Might Be a Useful Alternative for Detecting SM

A new study assessed droplet digital polymerase chain reaction (ddPCR) as an alternative diagnostic tool to allele-specific oligonucleotide real-time quantitative PCR (ASO-qPCR) to detect the D816V KIT mutation in patients with suspected systemic mastocytosis (SM). The study, published in Clinical Lymphoma, Myeloma & Leukemia, found that ddPCR could reliably detect the mutation in a noninvasive…

clinical research

Cancer Diagnosis in Hemophilia Trial Likely Unrelated to Experimental Therapy

BioMarin will proceed with phase 3 study of the investigational gene therapy BMN 270 (valoctocogene roxaparvovec) for the treatment of adults with severe hemophilia A despite a participant being diagnosed with B-cell acute lymphoblastic leukemia (B-ALL). The diagnosis was confirmed approximately 3 years after the participant received the dose of BMN 270. Early assessment of…

clinical trial

Phase 3 AFFINE Study of Novel Gene Therapy for Hemophilia Reopens Recruitment

The Pfizer and Sangamo Therapeutics phase 3 AFFINE study assessing the efficacy and safety of giroctocogene fitelparvovec, a novel gene therapy for patients with moderately severe to severe hemophilia A, has reopened recruitment. The reopening, announced by news release, will resume enrollment this month and dosing is expected to restart in October. Giroctocogene fitelparvovec is…

ECG block

ST-Segment Elevation Myocardial Infarction: A Potential Complication of SCD

Epidemiological studies reveal that cardiovascular disease is a leading cause of death around the world. Specifically, the prevalence of ST-segment elevation myocardial infarction (STEMI) has steadily increased in recent years. Acute coronary syndrome (ACS), which encompasses STEMI, is one of the potential complications of sickle cell disease. It is often fatal. Cardiovascular disease represents a…

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