Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy

Article type: Research Article

Authors: Fragala-Pinkham, Maria^{a; b; *} | Pasternak, Amy^{b; c} | McDermott, Michael P.^d | Mirek, Elizabeth^{b; c} | Glanzman, Allan M.^e | Montes, Jacqueline^{f; g} | Dunaway Young, Sally^{f; g} | Salazar, Rachel^g | Quigley, Janet^{b; c} | Riley, Susan O.^h | Chiriboga, Claudia A.^g | Finkel, Richard S.ⁱ | Tennekoon, Gihan^j | Martens, William B.^k | De Vivo, Darryl C.^{g; 1} | Darras, Basil T.^{c; 1}

Affiliations: [a] Research Center, Franciscan Children’s Hospital, Brighton, MA, USA | [b] Department of Physical Therapy and Occupational Therapy Services, Boston Children’s Hospital, Boston, MA, USA | [c] Department of Neurology, Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA | [d] Department of Biostatistics and Computational Biology, University of Rochester, Rochester, NY, USA | [e] Department of Physical Therapy, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA | [f] Department of Rehabilitation and Regenerative Medicine, Columbia University Irving Medical Center, New York, NY, USA | [g] Department of Neurology, Columbia University Irving Medical Center, New York, NY, USA | [h] Department of Physical and Occupational Therapy, Massachusetts General Hospital, Boston, MA, USA | [i] Center for Experimental Neurotherapeutics, St. Jude Children’s Research Hospital, Memphis, TN, USA | [j] Departments of Neurology, Pediatrics, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA | [k] Department of Neurology, University of Rochester, Rochester, NY, USA

Correspondence: [*] Corresponding author: Maria Fragala-Pinkham Department of Physical and Occupational Therapy, Boston Children’s Hospital, Boston, MA, USA. E-mail: [email protected].

Note: [1] Both senior authors.

Abstract: PURPOSE:The purpose of this study was to examine the psychometric properties of the Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT) in children and youth with Spinal Muscular Atrophy (SMA). METHODS:In this prospective cross-sectional study, caregivers of children and youth with SMA completed the PEDI-CAT Daily Activities and Mobility domains. A subset of caregivers completed a questionnaire about the measure. RESULTS:Mean ranks of scaled scores for Daily Activities (n = 96) and Mobility (n = 95) domains were significantly different across the three SMA types and across the three motor classifications. Normative scores indicated that 85 participants (89.5%) had limitations in Mobility and 51 in Daily Activities (53.1%). Floor effects were observed in≤10.4% of the sample for Daily Activities and Mobility. On average, caregivers completed the Mobility domain in 5.4 minutes and the Daily Activities domain in 3.3 minutes. Most caregivers reported that they provided meaningful information (92.1%), were willing to use the PEDI-CAT format again (79%), and suggested adding content including power wheelchair mobility items. CONCLUSION:Convergent validity was demonstrated for the Daily Activities and Mobility domains. Normative scores detected limitations in Mobility and Daily Activity performance for most participants with SMA. The PEDI-CATwas feasible to administer and caregivers expressed willingness to complete the PEDI-CAT in the future.

Keywords: Spinal muscular atrophy, caregiver-reported outcome measure, Pediatric evaluation of Disability Inventory-Computer Adaptive Test, functional performance measure, psychometric properties

DOI: 10.3233/PRM-190664

Journal: Journal of Pediatric Rehabilitation Medicine, vol. 14, no. 3, pp. 451-461, 2021