Background: Chronic red blood cell (RBC) transfusion is an established therapy to prevent stroke in patients with sickle cell anemia (SCA). It is unclear if adding daily hydroxyurea treatment to chronic transfusion is beneficial.
Study Design And Methods: We conducted a phase 2 clinical trial (NCT03644953) investigating the addition of dose-escalated hydroxyurea to chronic transfusion for patients with SCA receiving simple chronic transfusion for stroke prevention.
Background: The aim of the study was to evaluate the effectiveness of a novel e-learning module in teaching the physical exam of the temporomandibular joint (TMJ) in Juvenile idiopathic arthritis (JIA.).
Methods: An e-learning module was developed to convey the TMJ physical examination maneuvers that are considered to be best practice in JIA.
Introduction: Quality of life (QOL) is critical in screening and management of chronic medical conditions, including celiac disease (CD). The aim of this project was to develop a CD-specific pediatric QOL measure (Celiac Disease Life Inventory of Family Experiences [CDLIFE]) with parallel self-report and parent-report forms by generating items through concept elicitation interviews, iterative refinement using cognitive debriefing interviews, and evaluating its psychometric properties and validity.
Methods: Concept elicitation interviews were conducted to develop items (9 youth ages 8-19 years with CD; 10 parents of youth with CD), followed by cognitive interviews with additional stakeholders (3 youth with CD, 3 parents, and 8 clinicians) and item administration (parent/youth reports: n = 103/102).