title: Health-related quality of life in patients with hemoglobinopathies.
abstract: The use of patient-reported outcomes to measure the health and well-being of patients from their perspective has become an acceptable method to determine the impact of a disease and its treatment on patients. In patients with hemoglobinopathies, prior work has demonstrated that patients experience significant impairment in health-related quality of life (HRQL, a type of patient-reported outcome). This work has provided a better understanding of the burden that these patients experience and the factors that are associated with worse HRQL. The recent development of disease-specific HRQL instruments in sickle cell disease heralds new opportunities to explore the impact of the disease and its treatment on patients. The standards necessary to incorporate the measurement of HRQL into clinical trials are now well outlined by regulatory agencies. Measuring HRQL within a clinical practice setting and outside of the healthcare setting while the patient is at home are now possible and present new opportunities to understand the health and well-being of patients with hemoglobinopathies.
authors.affiliation: Department of Pediatrics, Section of Hematology/Oncology/Bone Marrow Transplantation, Children's Hospital of Wisconsin/Medical College of Wisconsin, Milwaukee, WI 53226, USA. jpanepin@mcw.edu
authors.forename: Julie A
authors.initials: JA
authors.lastname: Panepinto
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000523:psychology* / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D006304:Health Status; D006453:Hemoglobinopathies / Q000523:psychology* / Q000628:therapy*; D006801:Humans; D007223:Infant; D011594:Psychometrics / Q000379:methods; D011788:Quality of Life; D018434:Sickness Impact Profile; D011795:Surveys and Questionnaires; D016896:Treatment Outcome