Understanding the Association between Red Blood Cell Transfusion Utilization and Humanistic and Economic Burden in Patients with β-Thalassemia from the Patients' Perspective.

We assessed the humanistic and economic burden of chronic red blood cell (RBC) transfusions on patients with β-thalassemia. This cross-sectional, US-based study included adults (≥18 years) who self-reported a β-thalassemia physician diagnosis and had received ≥1 RBC transfusion in the past 6 months. The outcomes included the Functional Assessment of Cancer Therapy-Anemia (FACT-An), Patient Health Questionnaire-9, Generalized Anxiety Disorder-7, and ad hoc questions about treatment experience, side effects, direct/indirect costs, and psychological burden.

Disconnect in Assessments of Autosomal Dominant Polycystic Kidney Disease Burden Between Patients and Physicians: A Survey Study.

Background: Autosomal dominant polycystic kidney disease (ADPKD) is a rare inherited kidney disorder with considerable symptom burden and negative effects even in early-stage disease. Patients' reporting of ADPKD symptom burden may differ from physicians' impressions. In this quantitative, cross-section survey study, we evaluated patient and physician assessments of symptom burden at early- and late-stage ADPKD.