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Efficacy and Safety of Ileal Bile Acid Transport Inhibitors in Inherited Cholestatic Liver Disorders: A Meta-analysis of Randomized Controlled Trials.
J Clin Exp Hepatol
November 2024
Faculty of Medicine, Tanta University, Tanta, Egypt.
Background: Inherited cholestatic liver disorders such as progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome result in significant pruritus and increased serum bile acids, necessitating liver transplantation. This study aims to evaluate the efficacy and safety of Ileal bile acid transport inhibitors (IBATIs) in children with PFIC and Alagille syndrome.
Methods: We conducted a comprehensive search across the databases to identify relevant randomized controlled trials (RCTs), and Covidence was used to screen eligible articles.
Preparation and Rationale for a Patient-Centered Clinical Outcome Assessment Set of Fluid Overload for Drug Development in Nephrotic Syndrome.
Glomerular Dis
August 2024
Department of Medical Social Sciences, Northwestern University, Chicago, IL, USA.
Introduction: Fluid overload is a source of substantial morbidity for adults and children with nephrotic syndrome (NS). Preparation and Rationale for a Fluid Overload in Nephrotic Syndrome Clinical Outcomes Assessment Set for Drug Development (Prepare-NS, 5UG3FD007308) was funded by the US Food and Drug Administration to develop a core set of patient-reported and observer-reported (for young children) outcome measures of fluid overload for use in pharmaceutical trials across the lifespan.
Methods: The Prepare-NS study team developed the proposed context of use with input from stakeholders.
Development and validation of the Child Hemophilia Treatment Experience Measure: A new observer-reported outcome measure.
Haemophilia
October 2024
Clinical Development & Project Leadership, Novo Nordisk A/S, Søborg, Denmark.
Introduction: The Child Hemophilia Treatment Experience Measure (Child Hemo-TEM) was developed to capture the treatment burden experience of children with haemophilia (CwH).
Aim: Describe the development of this novel haemophilia-specific measure.
Methods: Interviews were conducted with clinical experts, CwH and CwH's caregivers.
Development of a signs and symptoms outcome measure for caregivers of patients with methylmalonic acidemia and propionic acidemia (MMAPAQ).
Mol Genet Metab
October 2024
ICON plc, Raleigh, NC, USA. Electronic address:
Article Synopsis
- Methylmalonic acidemia (MMA) and propionic acidemia (PA) are rare illnesses that affect how the body processes certain substances, and they can make patients very sick.
- Researchers want to create a special questionnaire called the Methylmalonic Acidemia and Propionic Acidemia Questionnaire (MMAPAQ) that helps caregivers share important information about symptoms of these conditions.
- They interviewed caregivers and some patients to understand the main signs and symptoms of MMA and PA, and they found consistent feedback to help develop this new questionnaire.
The Association Between Physical Distancing Behaviors to Avoid COVID-19 and Health-Related Quality of Life in Immunocompromised and Nonimmunocompromised Individuals: Patient-Informed Protocol for the Observational, Cross-Sectional EAGLE Study.
JMIR Res Protoc
August 2024
Patient Centered Solutions, IQVIA, Amsterdam, Netherlands.
Background: Immunocompromised individuals are known to respond inadequately to SARS-CoV-2 vaccines, placing them at high risk of severe or fatal COVID-19. Thus, immunocompromised individuals and their caregivers may still practice varying degrees of social or physical distancing to avoid COVID-19. However, the association between physical distancing to avoid COVID-19 and quality of life has not been comprehensively evaluated in any study.
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