Publications by authors named "W Garner"
Stevens-Johnson syndrome and toxic epidermal necrolysis (SJS/TEN) is an autoimmune process resulting in painful epidermal sloughing that can involve the vulva and vagina. Current guideline recommendations are based on expert opinion and may not reflect modern management of SJS/TEN in burn centers. We performed a retrospective chart review of 34 female patients treated for SJS/TEN at our burn center from 2015 to 2023.
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- Progressive familial intrahepatic cholestasis (PFIC) is an autosomal recessive disorder mainly caused by BSEP deficiency, leading to issues like bile formation disruption and severe itching (pruritus).!* -
- The MARCH-PFIC study was a phase 3, double-blind, placebo-controlled trial involving participants aged 1-17 from 29 centers across 16 countries, focusing on the effects of the drug maralixibat on different types of PFIC.!* -
- The study aimed to measure changes in pruritus severity and serum bile acid levels over 26 weeks, specifically looking at outcomes in cohorts with varying forms of PFIC, comparing those treated with maralixibat
Background And Aims: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study.
View Article and Find Full Text PDFIn the age of progressive antimicrobial resistance and increased difficulty combating infections in immunocompromised hosts, there has been renewed interest in the use of nontraditional therapeutics for infections. Herein, we review the use of investigational non-pharmaceutical anti-infective agents targeting fungal, bacterial, and viral infections in patients with hematologic malignancies, focusing on those receiving hematopoietic cell transplantation or cellular therapies. We discuss immune checkpoint inhibitors, granulocyte transfusions, bone marrow colony-stimulating factors, bacteriophages, fecal microbiota transplantation, and virus specific T-cell therapy.
View Article and Find Full Text PDFBackground And Aims: Refractory pruritus and other complications of cholestasis are indications for liver transplantation (LT) in patients with Alagille syndrome (ALGS). We evaluated predictors of event-free survival and transplant-free survival in patients with ALGS treated with maralixibat (MRX), an ileal bile acid transporter inhibitor.
Approach And Results: We assessed patients with ALGS from 3 clinical trials of MRX with up to 6 years of follow-up.