title: Reliability of telephone administration of the PedsQL Generic Quality of Life Inventory and Neuromuscular Module in spinal muscular atrophy (SMA).
abstract: Clinical research visits are challenging for people with SMA because of limited mobility and intercurrent illnesses. Missing data threaten the validity of research results. Obtaining outcomes remotely would represent a solution. To evaluate reliability of telephone administration of the PedsQL Pediatric Generic Core Quality of Life Inventory 4.0 (Generic) and Neuromuscular Module 3.0 (NM) in SMA, we recruited 21 participants of a Natural History Study for telephone administration of both modules no more than 7 days before or after an in-person study visit. We found excellent reliability between telephone and in-person administration of both modules with the NM slightly better than the Generic. Reliability of the child and parent forms was similar. We concluded that both modules can be administered reliably over the telephone to SMA patients and caregivers, expanding the utility of these tools in clinical trials. Notably, telephone administration is reliable in children as young as 8 years.
authors.affiliation: SMA Clinical Research Center, Columbia University Medical Center, New York, NY, USA.
authors.forename: Sally
authors.initials: S
authors.lastname: Dunaway
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Montes
authors.forename: Megan
authors.initials: M
authors.lastname: Montgomery
authors.forename: Vanessa
authors.initials: V
authors.lastname: Battista
authors.forename: Benjamin
authors.initials: B
authors.lastname: Koo
authors.forename: Jonathan
authors.initials: J
authors.lastname: Marra
authors.forename: Darryl C
authors.initials: DC
authors.lastname: De Vivo
authors.forename: Linda S
authors.initials: LS
authors.lastname: Hynan
authors.forename: Susan T
authors.initials: ST
authors.lastname: Iannaccone
authors.forename: Petra
authors.initials: P
authors.lastname: Kaufmann
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007398:Interpersonal Relations; D008297:Male; D017063:Outcome Assessment, Health Care / Q000379:methods*; D010290:Parents / Q000523:psychology; D011594:Psychometrics / Q000379:methods*; D011788:Quality of Life; D015203:Reproducibility of Results; D012649:Self Concept; D014897:Spinal Muscular Atrophies of Childhood / Q000175:diagnosis* / Q000523:psychology*; D013223:Statistics as Topic; D011795:Surveys and Questionnaires; D013689:Telephone