PMID- 34624609 OWN - NLM STAT- MEDLINE DCOM- 20220125 LR - 20221202 IS - 1873-5150 (Electronic) IS - 0887-8994 (Print) IS - 0887-8994 (Linking) VI - 125 DP - 2021 Dec TI - Reliability of the Telemedicine Application of the Gross Motor Function Measure-88 in Patients With Leukodystrophy. PG - 34-39 LID - S0887-8994(21)00205-8 [pii] LID - 10.1016/j.pediatrneurol.2021.09.012 [doi] AB - BACKGROUND: Leukodystrophies are a rare class of disorders characterized by severe neuromotor disability. There is a strong need for research regarding the functional status of people with leukodystrophy which is limited by the need for in-person assessments of mobility. The purpose of this study is to assess the reliability of the Gross Motor Function Measure-88 (GMFM-88) using telemedicine compared with standard in-person assessments in patients with leukodystrophy. METHODS: A total of 21 subjects with a diagnosis of leukodystrophy (age range = 1.79-52.82 years) were evaluated by in-person and by telemedicine evaluations with the GMFM-88 by physical therapists. Inter-rater reliability was assessed through evaluation of the same subject by two independent raters within a three-week period (n = 10 encounters), and intrarater reliability was assessed through blinded rescoring of video-recorded assessments after a one-week time interval (n = 6 encounters). RESULTS: Remote assessments were performed by caregivers in all 21 subjects using resources found in the home with remote guidance. There was agreement between all paired in-person and remote measurements (Lin's concordance correlation >/=0.995). The Bland-Altman analysis indicated that the paired differences were within +/-5%. Intrarater and inter-rater reliability demonstrated an intraclass correlation coefficient of >0.90. CONCLUSIONS: These results support that remote application of the GMFM-88 is a feasible and reliable approach to assess individuals with leukodystrophy. Telemedicine application of outcome measures may be of particular value in rare diseases and those with severe neurologic disability that impacts the ability to travel. CI - Copyright (c) 2021 Elsevier Inc. All rights reserved. FAU - Gavazzi, Francesco AU - Gavazzi F AD - Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Department of Molecular and Translational Medicine, University of Brescia, Brescia, Italy. Electronic address: gavazzif@chop.edu. FAU - Adang, Laura AU - Adang L AD - Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania. FAU - Waldman, Amy AU - Waldman A AD - Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Jan, Amanda K AU - Jan AK AD - Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Liu, Geraldine AU - Liu G AD - Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Lorch, Scott A AU - Lorch SA AD - Department of Neonatology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - DeMauro, Sara B AU - DeMauro SB AD - Department of Neonatology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Shults, Justine AU - Shults J AD - Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Department of Biostatistics, Epidemiology and Informatics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania. FAU - Pierce, Samuel R AU - Pierce SR AD - Departmen of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Ballance, Elizabeth AU - Ballance E AD - Departmen of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Kornafel, Tracy AU - Kornafel T AD - Departmen of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Harrington, Ann AU - Harrington A AD - Departmen of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Glanzman, Allan M AU - Glanzman AM AD - Departmen of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. FAU - Vanderver, Adeline AU - Vanderver A AD - Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania. LA - eng GR - K23 NS114113/NS/NINDS NIH HHS/United States GR - U01 NS106845/NS/NINDS NIH HHS/United States GR - U54 NS115052/NS/NINDS NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PT - Research Support, Non-U.S. Gov't DEP - 20210924 PL - United States TA - Pediatr Neurol JT - Pediatric neurology JID - 8508183 SB - IM MH - Adolescent MH - Adult MH - Child MH - Child, Preschool MH - Feasibility Studies MH - Female MH - Humans MH - Infant MH - Leukoencephalopathies/complications/*diagnosis MH - Male MH - Middle Aged MH - Movement Disorders/*diagnosis/etiology MH - Patient Acuity MH - *Psychometrics/instrumentation/methods/standards MH - Reproducibility of Results MH - *Telemedicine/methods/standards MH - Young Adult PMC - PMC8629609 MID - NIHMS1742910 OTO - NOTNLM OT - Child OT - Child development/physiology OT - Leukodystrophy OT - Motor skills/physiology OT - Outcome assessment (health care) OT - Physical therapy/methods OT - Psychometrics/methods OT - Reliability EDAT- 2021/10/09 06:00 MHDA- 2022/01/27 06:00 PMCR- 2022/12/01 CRDT- 2021/10/08 20:20 PHST- 2021/06/01 00:00 [received] PHST- 2021/09/16 00:00 [revised] PHST- 2021/09/18 00:00 [accepted] PHST- 2021/10/09 06:00 [pubmed] PHST- 2022/01/27 06:00 [medline] PHST- 2021/10/08 20:20 [entrez] PHST- 2022/12/01 00:00 [pmc-release] AID - S0887-8994(21)00205-8 [pii] AID - 10.1016/j.pediatrneurol.2021.09.012 [doi] PST - ppublish SO - Pediatr Neurol. 2021 Dec;125:34-39. doi: 10.1016/j.pediatrneurol.2021.09.012. Epub 2021 Sep 24.