TY - JOUR
T1 - The PedsQL™ in pediatric patients with Spinal Muscular Atrophy
T2 - Feasibility, reliability, and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales and Neuromuscular Module
AU - Iannaccone, Susan T.
AU - Hynan, Linda S.
AU - Morton, Anne
AU - Buchanan, Renee
AU - Limbers, Christine A.
AU - Varni, James W.
N1 - Funding Information:
This research was supported by National Institutes of Health RO1 NS 39327 (Principal Investigator: STI).
PY - 2009/12
Y1 - 2009/12
N2 - For Phase II and III clinical trials in children with Spinal Muscular Atrophy (SMA), reliable and valid outcome measures are necessary. Since 2000, the American Spinal Muscular Atrophy Randomized Trials (AmSMART) group has established reliability and validity for measures of strength, lung function, and motor function in the population from age 2 years to 18 years. The PedsQL™ (Pediatric Quality of Life Inventory™) Measurement Model was designed to integrate the relative merits of generic and disease-specific approaches, with disease-specific modules. The PedsQL™ 3.0 Neuromuscular Module was designed to measure HRQOL dimensions specific to children ages 2-18 years with neuromuscular disorders, including SMA. One hundred seventy-six children with SMA and their parents completed the PedsQL™ 4.0 Generic Core Scales and PedsQL™ 3.0 Neuromuscular Module. The PedsQL™ demonstrated feasibility, reliability, and validity in the SMA population. Consistent with the conceptualization of disease-specific symptoms as causal indicators of generic HRQOL, the majority of intercorrelations among the Neuromuscular Module Scales and the Generic Core Scales were in the medium to large range, supporting construct validity. For the purposes of a clinical trial, the PedsQL™ Neuromuscular Module and Generic Core Scales provide an integrated measurement model with the advantages of both generic and condition-specific instruments.
AB - For Phase II and III clinical trials in children with Spinal Muscular Atrophy (SMA), reliable and valid outcome measures are necessary. Since 2000, the American Spinal Muscular Atrophy Randomized Trials (AmSMART) group has established reliability and validity for measures of strength, lung function, and motor function in the population from age 2 years to 18 years. The PedsQL™ (Pediatric Quality of Life Inventory™) Measurement Model was designed to integrate the relative merits of generic and disease-specific approaches, with disease-specific modules. The PedsQL™ 3.0 Neuromuscular Module was designed to measure HRQOL dimensions specific to children ages 2-18 years with neuromuscular disorders, including SMA. One hundred seventy-six children with SMA and their parents completed the PedsQL™ 4.0 Generic Core Scales and PedsQL™ 3.0 Neuromuscular Module. The PedsQL™ demonstrated feasibility, reliability, and validity in the SMA population. Consistent with the conceptualization of disease-specific symptoms as causal indicators of generic HRQOL, the majority of intercorrelations among the Neuromuscular Module Scales and the Generic Core Scales were in the medium to large range, supporting construct validity. For the purposes of a clinical trial, the PedsQL™ Neuromuscular Module and Generic Core Scales provide an integrated measurement model with the advantages of both generic and condition-specific instruments.
KW - Health-related quality of life
KW - Neuromuscular Module
KW - Outcome measures
KW - PedsQL™ (Pediatric Quality of Life Inventory™)
KW - Spinal Muscular Atrophy
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UR - http://www.scopus.com/inward/citedby.url?scp=70449525150&partnerID=8YFLogxK
U2 - 10.1016/j.nmd.2009.09.009
DO - 10.1016/j.nmd.2009.09.009
M3 - Article
C2 - 19846309
AN - SCOPUS:70449525150
SN - 0960-8966
VL - 19
SP - 805
EP - 812
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - 12
ER -