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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…
LCFAOD patient education

Asymptomatic Diagnosis May Decrease Complications in LCFAOD

The prevalence of carnitine defects and mitochondrial long chain fatty acid oxidation disorder (LCFAOD) in 1 clinic was 4.75%, a retrospective cohort according to a recent study. The researchers set out to explore the genotypic and phenotypic spectrum of carnitine metabolism defects and mitochondrial long-chain fatty acid oxidation along with the corresponding treatment options. There…
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…
Photodynamic therapy research in lab

Photodynamic Therapy Linked With Increased Survival in Cholangiocarcinoma

Percutaneous transhepatic cholangioscopy (PTCS)-guided photodynamic therapy (PDT) appears to increase survival and improve overall quality of life in patients with postoperative cholangiocarcinoma (CCA) recurrence, according to an article recently published in Photodiagnosis and Photodynamic Therapy. Although surgical resection is the preferred treatment for CCA, the majority of patients have advanced disease at the time of diagnosis…
Pathologist

Treatment of Ruptured GIST Outside of Gastrointestinal Tract: A Case Study

Laparoscopic surgery is a minimally invasive and potentially useful way to remove gastrointestinal stromal tumors (GISTs) arising outside of the gastrointestinal tract, according to a case study published in the International Journal of Surgery Case Reports. “Ruptured extragastrointestinal stromal [tumors] (EGIST) are rare; therefore, there are no standard guidelines for its treatment. Herein, we report…
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