{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T17:35:43Z","timestamp":1793727343278,"version":"4.6.1"},"reference-count":23,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2016,6,9]],"date-time":"2016-06-09T00:00:00Z","timestamp":1465430400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2016,6,9]],"date-time":"2016-06-09T00:00:00Z","timestamp":1465430400000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"name":"ZonMw (NL)","award":["92003547"],"award-info":[{"award-number":["92003547"]}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2016,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>There is an urgent need for reliable and universally applicable outcome measures for children with mitochondrial diseases. In this study, we aimed to adapt the currently available Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) to the International Paediatric Mitochondrial Disease Scale (IPMDS) during a Delphi\u2010based process with input from international collaborators, patients and caretakers, as well as a pilot reliability study in eight patients. Subsequently, we aimed to test the feasibility, construct validity and reliability of the IPMDS in a multicentre study.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A clinically, biochemically and genetically heterogeneous group of 17 patients (age 1.6\u201316 years) from five different expert centres from four different continents were evaluated in this study.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The feasibility of the IPMDS was good, as indicated by a low number of missing items (4 %) and the positive evaluation of patients, parents and users. Principal component analysis of our small sample identified three factors, which explained 57.9 % of the variance. Good construct validity was found using hypothesis testing. The overall interrater reliability was good [median intraclass correlation coefficient for agreement between raters (ICC<jats:sub>agreement<\/jats:sub>) 0.85; range 0.23\u20130.99).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>In conclusion, we suggest using the IPMDS for assessing natural history in children with mitochondrial diseases. These data should be used to further explore construct validity of the IPMDS and to set age limits. In parallel, responsiveness and the minimal clinically important difference should be studied to facilitate sample size calculations in future clinical trials.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-016-9948-7","type":"journal-article","created":{"date-parts":[[2016,6,9]],"date-time":"2016-06-09T09:13:12Z","timestamp":1465463592000},"page":"705-712","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["International Paediatric Mitochondrial Disease Scale"],"prefix":"10.1002","volume":"39","author":[{"given":"Saskia","family":"Koene","sequence":"first","affiliation":[{"name":"Radboudn Center for Mitochondrial Medicine at the Department of Paediatrics Radboudumc PO BOX 9101 Geert Grooteplein 10. 6500 HB Nijmegen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan C. M.","family":"Hendriks","sequence":"additional","affiliation":[{"name":"Department of Health Evidence Radboudumc Nijmegen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilse","family":"Dirks","sequence":"additional","affiliation":[{"name":"Radboudn Center for Mitochondrial Medicine at the Department of Paediatrics Radboudumc PO BOX 9101 Geert Grooteplein 10. 6500 HB Nijmegen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lonneke","family":"de Boer","sequence":"additional","affiliation":[{"name":"Radboudn Center for Mitochondrial Medicine at the Department of Paediatrics Radboudumc PO BOX 9101 Geert Grooteplein 10. 6500 HB Nijmegen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maaike C.","family":"de Vries","sequence":"additional","affiliation":[{"name":"Radboudn Center for Mitochondrial Medicine at the Department of Paediatrics Radboudumc PO BOX 9101 Geert Grooteplein 10. 6500 HB Nijmegen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mirian C. H.","family":"Janssen","sequence":"additional","affiliation":[{"name":"Radboudn Center for Mitochondrial Medicine at the Department of Paediatrics Radboudumc PO BOX 9101 Geert Grooteplein 10. 6500 HB Nijmegen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Izelle","family":"Smuts","sequence":"additional","affiliation":[{"name":"Steve Biko Academic Hospital Ludwig\u2010Maximilians\u2010of Pretoria Pretoria South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cheuk\u2010Wing","family":"Fung","sequence":"additional","affiliation":[{"name":"Department of Paediatrics &amp; Adolescent Medicine University of Hong Kong Hong Kong Hong Kong"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Virginia C. N.","family":"Wong","sequence":"additional","affiliation":[{"name":"Department of Paediatrics &amp; Adolescent Medicine University of Hong Kong Hong Kong Hong Kong"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"I. Ren\u00e9 F. M.","family":"de Coo","sequence":"additional","affiliation":[{"name":"Department of Neurology ErasmusMC Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katharina","family":"Vill","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology and Developmental Medicine, Dr. v. 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