{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T02:39:03Z","timestamp":1787020743144,"version":"build-2736575974"},"reference-count":18,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2020,10,20]],"date-time":"2020-10-20T00:00:00Z","timestamp":1603152000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2020,10,20]],"date-time":"2020-10-20T00:00:00Z","timestamp":1603152000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100013995","name":"Sanofi Genzyme","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013995","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001659","name":"Deutsche Forschungsgemeinschaft","doi-asserted-by":"crossref","award":["UE171\/5-1"],"award-info":[{"award-number":["UE171\/5-1"]}],"id":[{"id":"10.13039\/501100001659","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Projekt DEAL"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Orphanet J Rare Dis"],"published-print":{"date-parts":[[2020,12]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Pain is an early symptom of Fabry disease (FD) and is characterized by a unique phenotype with mainly episodic acral and triggerable burning pain. Recently, we designed and validated the first pain questionnaire for adult FD patients in an interview and a self-administered version in German: the W\u00fcrzburg Fabry Pain Questionnaire (FPQ). We now report the validation of the English version of the self-administered FPQ (enFPQ).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>After two forward\u2013backward translations of the FPQ by native German and native English speakers, the enFPQ was applied at The Mark Holland Metabolic Unit, Manchester, UK for validation. Consecutive patients with genetically ascertained FD and current or previous FD pain underwent a face-to-face interview using the enFPQ. Two weeks later, patients filled in the self-administered enFPQ at home. The agreement between entries collected by supervised administration and self-administration of the enFPQ was assessed via Gwet\u2019s AC1-statistics (AC1) for nominal-scaled scores and intraclass correlation coefficient (ICC) for interval-scaled elements.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Eighty-three FD patients underwent the face-to-face interview and 54 patients sent back a completed self-administered version of the enFPQ 2\u00a0weeks later. We found high agreement with a mean AC1-statistics of 0.725 for 55 items, and very high agreement with a mean ICC of 0.811 for 9 items.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>We provide the validated English version of the FPQ for self-administration in adult FD patients. The enFPQ collects detailed information on the individual FD pain phenotype and thus builds a solid basis for better pain classification and treatment in patients with FD.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1186\/s13023-020-01580-9","type":"journal-article","created":{"date-parts":[[2020,10,20]],"date-time":"2020-10-20T09:02:56Z","timestamp":1603184576000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["English version of the self-administered Fabry Pain Questionnaire for adult patients"],"prefix":"10.1186","volume":"15","author":[{"given":"Ana","family":"Jovanovic","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philipp","family":"Klassen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Heuschmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Sommer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Roberts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nurcan","family":"\u00dc\u00e7eyler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2020,10,20]]},"reference":[{"key":"1580_CR1","doi-asserted-by":"publisher","first-page":"463","DOI":"10.1097\/WCO.0b013e32834a9433","volume":"24","author":"K Toyooka","year":"2011","unstructured":"Toyooka K. Fabry disease. Curr Opin Neurol. 2011;24:463\u20138.","journal-title":"Curr Opin Neurol"},{"key":"1580_CR2","first-page":"345","volume":"2001","author":"CM Eng","year":"2001","unstructured":"Eng CM, Guffon N, Wilcox WR, Germain DP, Lee P, Waldek S. Safety and efficacy of recombinant human alpha-galactosidase A\u2013replacement therapy in Fabry\u2019s disease. N Engl J Med. 2001;2001:345.","journal-title":"N Engl J Med"},{"key":"1580_CR3","first-page":"285","volume":"2001","author":"R Schiffmann","year":"2001","unstructured":"Schiffmann R, Kopp JB, Austin HAI, Sabnis S, Moore DF, Weibel T. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA. 2001;2001:285.","journal-title":"JAMA"},{"key":"1580_CR4","doi-asserted-by":"publisher","first-page":"545","DOI":"10.1056\/NEJMoa1510198","volume":"375","author":"DP Germain","year":"2016","unstructured":"Germain DP, Hughes DA, Nicholls K, Bichet DG, Giugliani R, Wilcox WR, et al. 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All study participants gave written consent before inclusion.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"No individual person\u2019s data is included in our manuscript.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"AJ: speaker honoraria, travel grants, and research grants: Sanofi Genzyme, Amicus and Takeda. PK: no competing interest. PH: reports research grants from German Ministry of Research and Education, German Research Foundation, European Union, Charit\u00e9\u2014Universit\u00e4tsmedizin Berlin, Berlin Chamber of Physicians, German Parkinson Society, University Hospital W\u00fcrzburg, Robert Koch Institute, German Heart Foundation, Federal Joint Committee (G-BA) within the Innovationfond, University Hospital Heidelberg (within RASUNOA-prime; supported by an unrestricted research grant to the University Hospital Heidelberg from Bayer, BMS, Boehringer-Ingelheim, Daiichi Sankyo), Charit\u00e9\u2014Universit\u00e4tsmedizin Berlin (within Mondafis; supported by an unrestricted research grant to the Charit\u00e9 from Bayer), University G\u00f6ttingen (within FIND-AF randomized; supported by an unrestricted research grant to the University G\u00f6ttingen from Boehringer-Ingelheim), outside the submitted work. CS: speaker honoraria from Takeda and Sanofi. MR: speaker honoraria, travel grants,: Sanofi Genzyme, Amicus, and Takeda. N\u00dc: speaker honoraria, travel grants, and research grants: Sanofi Genzyme, Takeda, Idorsia.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"296"}}