{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T05:27:23Z","timestamp":1793251643915,"version":"4.5.0"},"reference-count":29,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2016,11,10]],"date-time":"2016-11-10T00:00:00Z","timestamp":1478736000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2016,11,10]],"date-time":"2016-11-10T00:00:00Z","timestamp":1478736000000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100010036","name":"China-Japan Friendship Hospital Youth Science and Technology Excellence Project","doi-asserted-by":"crossref","award":["2014-QNYC-A-07"],"award-info":[{"award-number":["2014-QNYC-A-07"]}],"id":[{"id":"10.13039\/501100010036","id-type":"DOI","asserted-by":"crossref"}]},{"name":"China-Japan Friendship Hospital Project","award":["2013-MS-25"],"award-info":[{"award-number":["2013-MS-25"]}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2017,5]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Phenylketonuria (PKU) is a rare inborn disease, which, untreated, leading to severe neurobehavioral dysfunction. Considering its complexity, the management of PKU may bring a formidable economic burden to parents and caregivers. It is still unknown what the out\u2010of\u2010pocket expenses are for a patient with PKU in China. This paper explores the household financial burden of classical PKU and its impact on Chinese families in a quantitative manner for the first time.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A non\u2010interventional and observational study was conducted at the China\u2010Japan Friendship Hospital, one of the national centers for inherited metabolic disorders in China. The medical and non\u2010medical household financial burdens were consolidated into a questionnaire to evaluate the out\u2010of\u2010pocket costs (OOPCs) of PKU treatment and follow\u2010up.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Findings<\/jats:title>\n                    <jats:p>\n                      The total OOPCs were USD$3766.1 (0y), USD$3795.2 (1\u20132 ys), USD$4657.7 (3\u20134 ys), USD$5979.9 (5\u20138 ys), and USD$5588.7 (9 ys and older) for PKU patients of different age groups. The median economic burden of classical PKU was 75.0 % of total annual family income (range 1.0\u2013779.1 %), and 94.4 % of the families exceeding the threshold considered as catastrophic expenditure. There was a negative correlation between the financial burden and the proportion of time when Phe concentrations were in the desired target range (120\u2013250 \u03bcmol\/L) in 0\u20134\u2010ys group (\n                      <jats:italic>r<\/jats:italic>\n                      \u2009=\u2009\u20100.474,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.026).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The management of PKU is associated with a severe financial burden on patients\u2019 families, which may lead to insufficient treatment or variation of blood Phe concentration. The current reimbursement policies are as yet inadequate. A national reimbursement system targeting treatment practices for PKU patients and other rare diseases across China is imperative.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1007\/s10545-016-9995-0","type":"journal-article","created":{"date-parts":[[2016,11,10]],"date-time":"2016-11-10T04:00:45Z","timestamp":1478750445000},"page":"369-376","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":39,"title":["Household financial burden of phenylketonuria and its impact on treatment in China: a cross\u2010sectional study"],"prefix":"10.1002","volume":"40","author":[{"given":"Lin","family":"Wang","sequence":"first","affiliation":[{"name":"Department of Preventive Health Care China\u2010Japan Friendship Hospital Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hui","family":"Zou","sequence":"additional","affiliation":[{"name":"Newborn Screening Center Jinan Maternity and Child Care Hospital Jinan Shandong Province China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fang","family":"Ye","sequence":"additional","affiliation":[{"name":"Department of Preventive Health Care China\u2010Japan Friendship Hospital Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kundi","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics China\u2010Japan Friendship Hospital No. 2, Yinghua East Street, Chaoyang District Beijing 100029 China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaowen","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Nutrition China\u2010Japan Friendship Hospital Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhihua","family":"Chen","sequence":"additional","affiliation":[{"name":"Clinical Research Institute China\u2010Japan Friendship Hospital Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jie","family":"Chen","sequence":"additional","affiliation":[{"name":"Department of Preventive Health Care China\u2010Japan Friendship Hospital Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bingjuan","family":"Han","sequence":"additional","affiliation":[{"name":"Newborn Screening Center Jinan Maternity and Child Care Hospital Jinan Shandong Province China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Weimin","family":"Yu","sequence":"additional","affiliation":[{"name":"Clinical Research Institute China\u2010Japan Friendship Hospital Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chun","family":"He","sequence":"additional","affiliation":[{"name":"Clinical Research Institute China\u2010Japan Friendship Hospital Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ming","family":"Shen","sequence":"additional","affiliation":[{"name":"Department of Pediatrics China\u2010Japan Friendship Hospital No. 2, Yinghua East Street, Chaoyang District Beijing 100029 China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2016,11,10]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"crossref","first-page":"860","DOI":"10.1542\/peds.93.5.860","article-title":"Reimbursement for medical foods for inborn errors of metabolism","volume":"93","author":"American Academy of Pediatrics Committee on Nutrition","year":"1994","journal-title":"Pediatrics"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2011.12.004"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(10)60961-0"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-007-9988-0"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1258\/jms.2009.009088"},{"key":"e_1_2_10_7_1","first-page":"344","article-title":"Preliminary report on neonatal screening for hypothyroidism, phenylketonuria and galactosemia","volume":"6","author":"Chen RG","year":"1983","journal-title":"Shanghai Medical Journal"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2013.05.003"},{"key":"e_1_2_10_9_1","unstructured":"European society for phenylketonuria and allied disorders treated as phenylketonuria.Reimbursement PKU products in Europe.http:\/\/www.espku.org\/images\/stories\/reimbursement_ESPKU_2011_vs3.pdf. Accessed 5 Nov2015."},{"key":"e_1_2_10_10_1","first-page":"126","article-title":"Rare disease patients in China anticipate the sunlight of legislation","volume":"7","author":"Gao JJ","year":"2013","journal-title":"Drug Discov Ther"},{"key":"e_1_2_10_11_1","first-page":"104","article-title":"Newborn screening in China: phenylketonuria, congenital hypothyroidism and expanded screening","volume":"37","author":"Gu X","year":"2008","journal-title":"Ann Acad Med Singapore"},{"key":"e_1_2_10_12_1","unstructured":"Guangdong Newborn Screening Network.Summary of Experiences with Newborn Screening for and Free Treatment of PKU in Guangzhou.http:\/\/www.gdnsn.com\/qiansengaikuan\/pkuzl.htm. Accessed 5 Nov 2015 (in Chinese)"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2788.2012.01568.x"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.32.3.338"},{"key":"e_1_2_10_15_1","unstructured":"Human Resources and Social Security Bureau of Hefei City.Further adjustment of urban residents' basic medical insurance range.http:\/\/www.ahhfld.gov.cn\/n1105\/n32819\/n169172\/n173056\/16683609.html. Accessed 5 Nov 2015 (in Chinese)"},{"issue":"1","key":"e_1_2_10_16_1","first-page":"8","article-title":"Newborn screening for inborn errors of metabolism in Japan. A history of the development of newborn screening","volume":"10","author":"Kitagawa T","year":"2012","journal-title":"Pediatr Endocrinol Rev"},{"key":"e_1_2_10_17_1","first-page":"180","volume-title":"PKU and BH4: advances in phenylketonuria and tetrahydrobiopterin","author":"Lindner M","year":"2006"},{"key":"e_1_2_10_18_1","first-page":"106","article-title":"Current situation and prospects of newborn screening and treatment for phenylketonuria in China\u2014compared with the current situation in the United States, UK and Japan.","volume":"2","author":"Mei L","year":"2013","journal-title":"Intractable Rare Dis Res"},{"key":"e_1_2_10_19_1","first-page":"72","article-title":"Newborn screening and related policy against Phenylketonuria in China","volume":"2","author":"Mei L","year":"2013","journal-title":"Intractable Rare Dis Res"},{"key":"e_1_2_10_20_1","unstructured":"National Institutes of Health.Who pays for newborn screening?http:\/\/www.nichd.nih.gov\/health\/topics\/newborn\/conditioninfo\/how\u2010used\/pages\/pays.aspx. Accessed 4 Sept2015."},{"key":"e_1_2_10_21_1","unstructured":"NHFPC.The determining of key points of health work in 2013.http:\/\/www.moh.gov.cn\/wsb\/pxwfb\/201301\/53164cc1472446a48c47734a005ff5da.shtml. Accessed 5 Nov 2015 (in Chinese)"},{"key":"e_1_2_10_22_1","unstructured":"NHFPC.The technical specification of newborn screening.http:\/\/www.moh.gov.cn\/cmsresources\/mohfybjysqwss\/cmsrsdocument\/doc10798.doc. Accessed 2 Nov 2015 (in Chinese)"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-007-0687-7"},{"key":"e_1_2_10_24_1","volume-title":"Conference on Social Protection and Poverty","author":"Prescott N","year":"1999"},{"key":"e_1_2_10_25_1","first-page":"613","article-title":"Reduction of catastrophic health care expenditures by a community\u2010based health insurance scheme in Gujarat, India: current experiences and challenges","volume":"80","author":"Ranson MK","year":"2002","journal-title":"Bull World Health Organ"},{"key":"e_1_2_10_26_1","first-page":"1015","volume-title":"The metabolic and molecular bases of inherited disease","author":"Scriver CR","year":"1995"},{"key":"e_1_2_10_27_1","doi-asserted-by":"publisher","DOI":"10.1007\/8904_2011_119"},{"key":"e_1_2_10_28_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(10)60240-1"},{"key":"e_1_2_10_29_1","doi-asserted-by":"publisher","DOI":"10.1258\/jms.2011.011133"},{"key":"e_1_2_10_30_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-012-9550-6"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s10545-016-9995-0.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s10545-016-9995-0\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s10545-016-9995-0.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/s10545-016-9995-0","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T03:20:24Z","timestamp":1694748024000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1007\/s10545-016-9995-0"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,11,10]]},"references-count":29,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2017,5]]}},"alternative-id":["10.1007\/s10545-016-9995-0"],"URL":"https:\/\/doi.org\/10.1007\/s10545-016-9995-0","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,11,10]]}}}