{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,10]],"date-time":"2026-08-10T14:44:42Z","timestamp":1786373082287,"version":"build-2736575974"},"reference-count":30,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"7","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2012,7]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background and objectives<\/jats:title>\n                    <jats:p>Immediate-release cysteamine bitartrate (Cystagon; Mylan Pharmaceuticals, Canonsburg, PA) may prevent or delay kidney transplantation and other serious outcomes in patients with cystinosis, but has never been subjected to a prospective clinical trial. Cystagon efficacy requires strict lifelong dosing every 6 hours. Such a dosing schedule and Cystagon-associated side effects are often cited by patients as reasons for nonadherence.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design, setting, participants, &amp; measurements<\/jats:title>\n                    <jats:p>This open-label, randomized, controlled, crossover trial was powered to show that a new delayed-release formulation of cysteamine bitartrate, RP103, taken every 12 hours, was noninferior to Cystagon for maintenance of white blood cell (WBC) cystine at levels associated with optimal outcomes in the disease.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Forty-three patients were randomized. Using a mixed-effects statistical analysis model, the least-squares mean peak value of WBC cystine level was 0.62\u00b10.05 nmol 1\/2 cystine\/mg protein after 12 hours under RP103 and 0.54\u00b10.05 nmol 1\/2 cystine\/mg protein after 6 hours under Cystagon, a difference of 0.08\u00b10.04 nmol 1\/2 cystine\/mg protein (95.8% confidence interval, 0\u20130.16). The average steady-state total daily dose of RP103 was 82% of the incoming steady-state total daily dose of Cystagon. There were three-fold more gastrointestinal side effects compared with using Cystagon.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>A new delayed-release Q12H formulation of cysteamine bitartrate is not inferior to the Q6H formulation (Cystagon) in maintaining low WBC cystine levels in patients with cystinosis but at a lower total daily dose.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.2215\/cjn.12321211","type":"journal-article","created":{"date-parts":[[2012,5,4]],"date-time":"2012-05-04T02:02:31Z","timestamp":1336096951000},"page":"1112-1120","source":"Crossref","is-referenced-by-count":83,"title":["A Randomized Controlled Crossover Trial with Delayed-Release Cysteamine Bitartrate in Nephropathic Cystinosis"],"prefix":"10.2215","volume":"7","author":[{"given":"Craig B.","family":"Langman","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Larry A.","family":"Greenbaum","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Minnie","family":"Sarwal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Grimm","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Niaudet","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Georges","family":"Desch\u00eanes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elisabeth","family":"Cornelissen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Denis","family":"Morin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Cochat","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Debora","family":"Matossian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Segolene","family":"Gaillard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary Jo","family":"Bagger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrice","family":"Rioux","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","reference":[{"key":"R1-10-20230811","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1086\/302509","article-title":"Molecular characterization of CTNS deletions in nephropathic cystinosis: Development of a PCR-based detection assay.","volume":"65","author":"Forestier","year":"1999","journal-title":"Am J Hum Genet"},{"key":"R2-10-20230811","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1038\/ng0498-319","article-title":"A novel gene encoding an integral membrane protein is mutated in nephropathic cystinosis.","volume":"18","author":"Town","year":"1998","journal-title":"Nat Genet"},{"key":"R3-10-20230811","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1002\/humu.10141","article-title":"Identification of 14 novel CTNS mutations and characterization of seven splice site mutations associated with cystinosis.","volume":"20","author":"Kalatzis","year":"2002","journal-title":"Hum Mutat"},{"key":"R4-10-20230811","doi-asserted-by":"crossref","first-page":"F905","DOI":"10.1152\/ajprenal.00318.2010","article-title":"The pathogenesis of cystinosis: Mechanisms beyond cystine accumulation.","volume":"299","author":"Wilmer","year":"2010","journal-title":"Am J Physiol Renal Physiol"},{"key":"R5-10-20230811","doi-asserted-by":"crossref","first-page":"883","DOI":"10.7326\/0003-4819-68-4-883","article-title":"Cystinosis. 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