title: Development of disease-specific health-related quality-of-life instruments for children with immune thrombocytopenic purpura and their parents.
abstract: OBJECTIVE
  Immune thrombopenic purpura (ITP) is an important childhood hematologic disorder that is often frightening to patients and their parents because of its acute onset and bleeding symptoms. There is no consensus on the management of ITP in children. Pediatric hematologists have differing management philosophies, yet most, explicitly or implicitly, incorporate into their management approach the potential impact on the child's and family's quality of life. There is no validated ITP-specific health-related quality-of-life instrument for use with children with ITP, nor is there one to evaluate the burden experienced by their parents. ITP is usually a self-limited disorder. With current controversy over management approaches, an evaluation of the disease burden experienced by the child and the family may assist with the assessment of alternative treatment approaches.
  
  
  METHODS
  Using standard clinimetric methodology, 88 children with acute or chronic ITP, along with their parents, participated in the development of the instruments.
  
  
  RESULTS
  The 26-item ITP-Child Quality-of-Life Questionnaire includes five domains: treatment side effect-related, intervention-related, disease-related, activity-related, and family-related. This instrument can be used as a self-completed instrument for most children older than 7 years or as a proxy-completed instrument by parents of children younger than 7 years. The 26-item ITP-Parental Burden Quality-of-Life Questionnaire includes six domains: concerns related to diagnosis/investigation, treatment/disease monitoring, monitoring of child's activities, interference with daily life, disease outcome, and emotional impacts.
  
  
  CONCLUSIONS
  The first steps of the development of these formally developed instruments are complete. The instruments are available for study to validate and test their responsiveness through use in clinical research studies. Such instruments are increasingly recognized as important for comprehensive measurement of patient outcomes in this and other areas of pediatric hematology/oncology practice.
authors.affiliation: IWK Health Center, Dalhousie University, 5850 University Avenue, Halifax, Nova Scotia B3J 3G9, Canada. dorothy.barnard@iwk.nshealth.ca
authors.forename: Dorothy
authors.initials: D
authors.lastname: Barnard
authors.forename: Michael
authors.initials: M
authors.lastname: Woloski
authors.forename: David
authors.initials: D
authors.lastname: Feeny
authors.forename: Patricia
authors.initials: P
authors.lastname: McCusker
authors.forename: John
authors.initials: J
authors.lastname: Wu
authors.forename: Michele
authors.initials: M
authors.lastname: David
authors.forename: James
authors.initials: J
authors.lastname: Bussel
authors.forename: Jean
authors.initials: J
authors.lastname: Lusher
authors.forename: Cindy
authors.initials: C
authors.lastname: Wakefield
authors.forename: Susan
authors.initials: S
authors.lastname: Henriques
authors.forename: Victor
authors.initials: V
authors.lastname: Blanchette
mesh_terms: D000293:Adolescent; D002170:Canada; D002648:Child; D002675:Child, Preschool; D005069:Evaluation Studies as Topic; D005260:Female; D006304:Health Status; D006305:Health Status Indicators; D006801:Humans; D007223:Infant; D008297:Male; D010290:Parents / Q000523:psychology; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000175:diagnosis* / Q000628:therapy*; D011788:Quality of Life; D015203:Reproducibility of Results; D012107:Research Design; D011795:Surveys and Questionnaires; D014481:United States