Philip Rosenthal
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View article: Biannual Mass Azithromycin Distributions for Preschool Children and Malaria Parasitemia
Biannual Mass Azithromycin Distributions for Preschool Children and Malaria Parasitemia Open
Importance Mass azithromycin distributions may reduce malaria parasitemia in the short term, but longer-term effectiveness is unclear. Objective To examine whether biannual mass azithromycin distributions are associated with lower rates of…
View article: Weight gain in infancy and metabolic dysfunction-associated steatotic liver disease (MASLD) in a prospective birth cohort of Latino children
Weight gain in infancy and metabolic dysfunction-associated steatotic liver disease (MASLD) in a prospective birth cohort of Latino children Open
Background Metabolic dysfunction-associated steatotic liver disease (MASLD) is the most common chronic liver disease among U.S. children. Early weight trajectories correlate with obesity, cardiometabolic syndrome, and MASLD in children bor…
View article: Highly multiplexed molecular inversion probe panel in Plasmodium falciparum targeting common SNPs approximates whole-genome sequencing assessments for selection and relatedness
Highly multiplexed molecular inversion probe panel in Plasmodium falciparum targeting common SNPs approximates whole-genome sequencing assessments for selection and relatedness Open
Introduction The use of next-generation sequencing technologies (NGS) to study parasite populations and their response and evolution to interventions is important to support malaria control and elimination efforts. While whole-genome seque…
View article: Highly multiplex molecular inversion probe panel in Plasmodium falciparum targeting common SNPs approximates whole genome sequencing assessments for selection and relatedness
Highly multiplex molecular inversion probe panel in Plasmodium falciparum targeting common SNPs approximates whole genome sequencing assessments for selection and relatedness Open
Introduction The use of next-generation sequencing technologies (NGS) to study parasite populations and their response and evolution to interventions is important to support malaria control and elimination efforts. While whole genome seque…
View article: Increased serum GM-CSF at diagnosis of biliary atresia is associated with improved biliary drainage
Increased serum GM-CSF at diagnosis of biliary atresia is associated with improved biliary drainage Open
View article: Prospective Multicenter Longitudinal Measurement of Liver Stiffness in School-Age Children With Cholestatic Liver Disease
Prospective Multicenter Longitudinal Measurement of Liver Stiffness in School-Age Children With Cholestatic Liver Disease Open
View article: Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial
Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial Open
View article: Sofosbuvir–velpatasvir in children 3–17 years old with hepatitis C virus infection
Sofosbuvir–velpatasvir in children 3–17 years old with hepatitis C virus infection Open
Background The safety and efficacy of sofosbuvir–velpatasvir in children aged 3–17 years with chronic hepatitis C virus (HCV) infection of any genotype were evaluated. Methods In this Phase 2, multicenter, open‐label study, patients receiv…
View article: Development of Novel Peptidyl Nitriles Targeting Rhodesain and Falcipain-2 for the Treatment of Sleeping Sickness and Malaria
Development of Novel Peptidyl Nitriles Targeting Rhodesain and Falcipain-2 for the Treatment of Sleeping Sickness and Malaria Open
In recent decades, neglected tropical diseases and poverty-related diseases have become a serious health problem worldwide. Among these pathologies, human African trypanosomiasis, and malaria present therapeutic problems due to the onset o…
View article: Food insecurity is a risk factor for metabolic dysfunction‐associated steatotic liver disease in Latinx children
Food insecurity is a risk factor for metabolic dysfunction‐associated steatotic liver disease in Latinx children Open
Summary Introduction Metabolic dysfunction‐associated steatotic liver disease (MASLD) is the most common chronic liver disease among US children. Studies have associated food insecurity with MASLD in adults, but there are few studies of pe…
View article: Diagnostic accuracy of serum matrix metalloproteinase-7 as a biomarker of biliary atresia in a large North American cohort
Diagnostic accuracy of serum matrix metalloproteinase-7 as a biomarker of biliary atresia in a large North American cohort Open
Background and Aims: High levels of serum matrix metalloproteinase-7 (MMP-7) have been linked to biliary atresia (BA), with wide variation in concentration cutoffs. We investigated the accuracy of serum MMP-7 as a diagnostic biomarker in a…
View article: Protein biomarkers GDF15 and FGF21 to differentiate mitochondrial hepatopathies from other pediatric liver diseases.
Protein biomarkers GDF15 and FGF21 to differentiate mitochondrial hepatopathies from other pediatric liver diseases. Open
GDF15 and FGF21 are significantly higher in children with MH compared to other childhood liver diseases and controls and, when combined, were predictive of MH and had prognostic implications.
View article: Neonatal cholestasis in children with Alpha-1-AT deficiency is a risk for earlier severe liver disease with male predominance
Neonatal cholestasis in children with Alpha-1-AT deficiency is a risk for earlier severe liver disease with male predominance Open
Background: Our objective was to better understand the natural history and disease modifiers of Alpha-1-antitrypsin deficiency (AATD), a common genetic liver disease causing hepatitis and cirrhosis in adults and children. The clinical cour…
View article: Diagnosis and management of Alagille and progressive familial intrahepatic cholestasis
Diagnosis and management of Alagille and progressive familial intrahepatic cholestasis Open
Alagille syndrome and progressive familial intrahepatic cholestasis are conditions that can affect multiple organs. Advancements in molecular testing have aided in the diagnosis of both. The impairment of normal bile flow and secretion lea…
View article: Nutrition assessment and MASH severity in children using the Healthy Eating Index
Nutrition assessment and MASH severity in children using the Healthy Eating Index Open
Background: Pediatric metabolic-associated fatty liver disease (MAFLD) is a global health problem, with lifestyle modification as its major therapeutic strategy. Rigorous characterization of dietary content on MAFLD in children is lacking.…
View article: Sarcopenia is associated with osteopenia and impaired quality of life in children with genetic intrahepatic cholestatic liver disease
Sarcopenia is associated with osteopenia and impaired quality of life in children with genetic intrahepatic cholestatic liver disease Open
Background: Sarcopenia occurs in pediatric chronic liver disease, although the prevalence and contributing factors in genetic intrahepatic cholestasis are not well-described. The objective of this study was to measure muscle mass in school…
View article: Clinical spectrum and genetic causes of mitochondrial hepatopathy phenotype in children
Clinical spectrum and genetic causes of mitochondrial hepatopathy phenotype in children Open
Background: Alterations in both mitochondrial DNA (mtDNA) and nuclear DNA genes affect mitochondria function, causing a range of liver-based conditions termed mitochondrial hepatopathies (MH), which are subcategorized as mtDNA depletion, R…
View article: Shorter leukocyte telomere length protects against NAFLD progression in children
Shorter leukocyte telomere length protects against NAFLD progression in children Open
Leukocyte telomere length (LTL) gets shorter with each cell division and is also sensitive to reactive oxygen species damage and inflammatory processes. Studies in adults with non-alcoholic fatty liver disease (NAFLD) have found that incre…
View article: Elbasvir/grazoprevir in children aged 3–18 years with chronic HCV genotype 1 or 4 infection: a pharmacokinetic modeling study
Elbasvir/grazoprevir in children aged 3–18 years with chronic HCV genotype 1 or 4 infection: a pharmacokinetic modeling study Open
Background: Approximately 3.5 million children and adolescents worldwide are chronically infected with HCV. This study uses pharmacokinetic modeling to identify pediatric doses of elbasvir/grazoprevir (EBR/GZR) that achieve plasma concentr…
View article: Costs of pediatric liver transplantation among commercially insured and Medicaid-insured patients with cholestasis in the US
Costs of pediatric liver transplantation among commercially insured and Medicaid-insured patients with cholestasis in the US Open
Children with rare cholestatic liver diseases, such as Alagille syndrome, progressive familial intrahepatic cholestasis, and biliary atresia typically require liver transplantation (LT). The objective of this analysis was to assess the eco…
View article: Advancing detection and response capacities for emerging and re-emerging pathogens in Africa
Advancing detection and response capacities for emerging and re-emerging pathogens in Africa Open
View article: Genotype-phenotype relationships of truncating mutations, p.E297G and p.D482G in bile salt export pump deficiency
Genotype-phenotype relationships of truncating mutations, p.E297G and p.D482G in bile salt export pump deficiency Open
View article: Health Care Resource Utilization by Patients with Alagille Syndrome
Health Care Resource Utilization by Patients with Alagille Syndrome Open
Patients with Alagille syndrome have a substantial HRU burden driven largely by numerous outpatient visits and costly inpatient stays. Given the complexity and variability of Alagille syndrome presentation, patients may benefit from multid…
View article: Serum biomarkers correlated with liver stiffness assessed in a multicenter study of pediatric cholestatic liver disease
Serum biomarkers correlated with liver stiffness assessed in a multicenter study of pediatric cholestatic liver disease Open
Background and Aims: Detailed investigation of the biological pathways leading to hepatic fibrosis and identification of liver fibrosis biomarkers may facilitate early interventions for pediatric cholestasis. Approach and Results: A target…
View article: Impact of long‐term administration of maralixibat on children with cholestasis secondary to Alagille syndrome
Impact of long‐term administration of maralixibat on children with cholestasis secondary to Alagille syndrome Open
There is growing interest in, but limited data about, intestinal bile acid transport inhibitors as treatment for cholestatic liver disease. The current analyses combine two similar randomized placebo‐controlled trials with subsequent exten…
View article: Use of funded multicenter prospective longitudinal databases to inform clinical trials in rare diseases—Examination of cholestatic liver disease in Alagille syndrome
Use of funded multicenter prospective longitudinal databases to inform clinical trials in rare diseases—Examination of cholestatic liver disease in Alagille syndrome Open
The conduct of long‐term conventional randomized clinical trials in rare diseases is very difficult, making evidenced‐based drug development problematic. As a result, real‐world data/evidence are being used more frequently to assess new th…
View article: Field evaluation of the diagnostic performance of EasyScan GO: a digital malaria microscopy device based on machine-learning
Field evaluation of the diagnostic performance of EasyScan GO: a digital malaria microscopy device based on machine-learning Open
View article: Risk of variceal hemorrhage and pretransplant mortality in children with biliary atresia
Risk of variceal hemorrhage and pretransplant mortality in children with biliary atresia Open
Background and Aims The natural history of gastroesophageal variceal hemorrhage (VH) in biliary atresia (BA) is not well characterized. We analyzed risk factors, incidence, and outcomes of VH in a longitudinal multicenter study. Approach a…
View article: Reply to Li, Henry, and Nguyen
Reply to Li, Henry, and Nguyen Open
Khalili, Mandana MD, MAS; Kleiner, David E. MD; King, Wendy C. PhD; Sterling, Richard K. MD, MSc; Ghany, Marc G. MD; Chung, Raymond T. MD; Bhan, Atul K. MD; Rosenthal, Philip MD; Lisker-Melman, Mauricio MD; Ramachandran, Rageshree MD; Lok,…
View article: Incidence and prediction of HBsAg seroclearance in a prospective multi‐ethnic HBeAg‐negative chronic hepatitis B cohort
Incidence and prediction of HBsAg seroclearance in a prospective multi‐ethnic HBeAg‐negative chronic hepatitis B cohort Open
Background and Aims Achieving HBsAg loss is an important landmark in the natural history of chronic hepatitis B (CHB). A more personalized approach to prediction of HBsAg loss is relevant in counseling patients. This study sought to develo…