Objective: To develop a grading scale to predict the risk of intracerebral hemorrhage (ICH) and prognosis after treatment with IV tissue-plasminogen activator (t-PA) in patients with ischemic stroke.
Methods:We constructed a five-point scale based on NIH Stroke Scale score, extent of hypodensity on CT scan, serum glucose at baseline, and history of diabetes to predict the risk of hemorrhage after thrombolysis (HAT score). We evaluated the predictive ability of this scale, using c-statistics, in two independent cohorts: the t-PA treated group in the National Institute of Neurological Disorders and Stroke study, and consecutive patients treated with IV t-PA at our institution.
Results:The percentage of patients who developed any ICH after t-PA increased with higher scores in both cohorts. Collectively, the rate of any symptomatic ICH was 2% (0 point), 5% (1 point), 10% (2 points), 15% (3 points
Primary hyperoxaluria type 1 (PH1) is a rare, progressive, genetic disease with limited treatment options. We report the efficacy and safety of lumasiran, an RNA interference therapeutic, in infants and young children with PH1. Methods: This single-arm, open-label, phase 3 study evaluated lumasiran in patients aged <6 years with PH1 and an estimated glomerular filtration rate >45 mL/min/1.73 m 2 , if aged ≥12 months, or normal serum creatinine, if aged <12 months. The primary end point was percent change in spot urinary oxalate to creatinine ratio (UOx:Cr) from baseline to month 6. Secondary end points included proportion of patients with urinary oxalate ≤1.5× upper limit of normal and change in plasma oxalate. Results: All patients (N = 18) completed the 6-month primary analysis period. Median age at consent was 50.1 months. Least-squares mean percent reduction in spot UOx:Cr was 72.0%. At month 6, 50% of patients (9/18) achieved spot UOx:Cr ≤1.5× upper limit of normal. Least-squares mean percent reduction in plasma oxalate was 31.7%. The most common treatment-related adverse events were transient, mild, injection-site reactions. Conclusion: Lumasiran showed rapid, sustained reduction in spot UOx:Cr and plasma oxalate and acceptable safety in patients aged <6 years with PH1, establishing RNA interference therapies as safe, effective treatment options for infants and young children.
A small proportion of patients who present within 3 hours of symptom onset receive thrombolytic therapy. The observation that patients with more severe neurologic deficits and subsequently worse in-hospital outcomes appear to present early after symptom onset to the hospital may have implications for clinical studies.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.