Articles related to HEMOPHILIA

Hemarthrosis affects an estimated 70–80% of people with hemophilia and can progress to permanent joint damage, with pain management presenting a specific medication safety intersection point for pharmacists at the OTC counter and bedside.

Valoctocogene roxaparvovec gene therapy demonstrates long-term efficacy in reducing bleeding episodes and factor VIII use in patients with severe hemophilia A, potentially changing the treatment landscape for this condition.

Roctavian represents a turning point in hemophilia A management, offering patients like Maxwell a potential lifetime of relief from traditional therapies. This gene therapy directly targets the genetic cause, paving the way for innovative treatments in genetic disorders.

This UK study highlights the urgent need for targeted interventions and close monitoring to improve the quality of life for hemophilia patients suffering from ankle arthropathy.

The business launched a precautionary global recall of desmopressin formulations used to treat mild-to-moderate hemophilia A and von Willebrand disease after higher-than-specified amounts of desmopressin were discovered in various formulations. Stimate, a nasal spray containing a synthetic form of desmopressin acetate used to control bleeding in hemophilia patients, is among the recalled products, as is Octostim Nasal Spray 1.5 mg/ml, DDAVP 10 micrograms (mcg)/0.1 ml, Minirin Nasal Spray 0.1 mg/ml, and Generic Desmopressin Acetate Nasal Spray 10 mcg/0.1 ml. Products containing higher-than-specified amounts of desmopressin were recalled due to concerns that increased desmopressin exposure can result in water retention, low blood pressure, and low blood salt levels in mild cases, as well as seizure, coma, and death.

These evidence-based guidelines from the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) are intended to support patients, clinicians, and health care professionals in their decisions about management of von Willebrand Disease (VWD), a common inherited bleeding disorder.