{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,18]],"date-time":"2026-01-18T00:22:28Z","timestamp":1768695748595,"version":"3.49.0"},"reference-count":21,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2021,6,3]],"date-time":"2021-06-03T00:00:00Z","timestamp":1622678400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2021,6,3]],"date-time":"2021-06-03T00:00:00Z","timestamp":1622678400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100002322","name":"Coordena\u00e7\u00e3o de Aperfei\u00e7oamento de Pessoal de N\u00edvel Superior","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002322","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003593","name":"Conselho Nacional de Desenvolvimento Cient\u00edfico e Tecnol\u00f3gico","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003593","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003810","name":"Hospital de Cl\u00ednicas de Porto Alegre","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003810","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004263","name":"Funda\u00e7\u00e3o de Amparo \u00e0 Pesquisa do Estado do Rio Grande do Sul","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004263","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Orphanet J Rare Dis"],"published-print":{"date-parts":[[2021,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec>\n                <jats:title>Background<\/jats:title>\n                <jats:p>Glycogen storage disease type 1a (GSD Ia) is characterized by severe fasting hypoglycemia. The clinical management includes the administration of uncooked cornstarch (UCCS). Although such a diet approach is effective in achieving euglycemia, its impact on the quality of life of patients should be considered. In vitro analyses suggest a longer release of glucose when using sweet manioc starch (SMS).<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>We compared the efficacy and safety of the administration of SMS and UCCS during a short-fasting challenge in patients with GSD Ia in a randomized, triple-blind, phase I\/II, cross-over study. GSD Ia patients aged \u2265\u200916 years and treated with UCCS were enrolled. Participants were hospitalized for two consecutive nights, receiving UCCS or SMS in each night. After the administration of the starches, glucose, lactate and insulin levels were measured in 1-h interval throughout the hospitalization period. The procedures were interrupted after 10\u00a0h of fasting or in a hypoglycemic episode (&lt;\u20093.88\u00a0mmol\/L).<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>Eleven individuals (mean age: 21.6\u2009\u00b1\u20094.3\u00a0years; all presenting body mass index &gt;\u200925\u00a0kg\/m<jats:sup>2<\/jats:sup>) participated in the study. The average fasting period was 8.2\u2009\u00b1\u20092.0\u00a0h for SMS and 7.7\u2009\u00b1\u20092.3\u00a0h for UCCS (<jats:italic>p<\/jats:italic>\u2009=\u20090.04). SMS maintained euglycemia for a greater period over UCCS. Increased lactate concentrations were detected even in absence of hypoglycemia, not being influenced by the different starches investigated (<jats:italic>p<\/jats:italic>\u2009=\u20090.17). No significant difference was found in total cholesterol, HDL, triglycerides and uric acid levels in both arms. None of the patients showed severe adverse events.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusions<\/jats:title>\n                <jats:p>SMS appears to be non-inferior to UCCS in the maintenance of euglycemia, thus emerging as a promising alternative to\u00a0the treatment of GSD Ia.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s13023-021-01877-3","type":"journal-article","created":{"date-parts":[[2021,6,3]],"date-time":"2021-06-03T11:09:23Z","timestamp":1622718563000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["A triple-blinded crossover study to evaluate the short-term safety of sweet manioc starch for the treatment of glycogen storage disease type Ia"],"prefix":"10.1186","volume":"16","author":[{"given":"Vaneisse C. L.","family":"Monteiro","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bibiana M.","family":"de Oliveira","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bruna B.","family":"dos Santos","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fernanda","family":"Sperb-Ludwig","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lilia F.","family":"Refosco","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tatiele","family":"Nalin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Terry G. J.","family":"Derks","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Carolina F.","family":"Moura de Souza","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7933-6687","authenticated-orcid":false,"given":"Ida V. D.","family":"Schwartz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2021,6,3]]},"reference":[{"issue":"1","key":"1877_CR1","doi-asserted-by":"publisher","first-page":"95","DOI":"10.1023\/a:1021831621210","volume":"4","author":"JI Wolfsdorf","year":"2003","unstructured":"Wolfsdorf JI, Weinstein DA. Glycogen storage diseases. 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Glycogen Storage Disease Type I. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews(\u00ae). University of Washington, Seattle. Copyright \u00a9 1993\u20132020, University of Washington, Seattle. GeneReviews is a registered trademark of the University of Washington, Seattle. All rights reserved.; 1993."},{"key":"1877_CR7","doi-asserted-by":"crossref","unstructured":"Lafor\u00eat P, Weinstein D, Smit P. Inborn metabolic diseases: diagnosis and treatment\u00a0In: Saudubray J, van den Berghe G, Walter J, eds. 2012:115\u2013139:chap The glycogen storage diseases and related disorders.","DOI":"10.1007\/978-3-642-15720-2_6"},{"issue":"11","key":"1877_CR8","doi-asserted-by":"publisher","first-page":"e1","DOI":"10.1038\/gim.2014.128","volume":"16","author":"PS Kishnani","year":"2014","unstructured":"Kishnani PS, Austin SL, Abdenur JE, et al. Diagnosis and management of glycogen storage disease type I: a practice guideline of the American College of Medical Genetics and Genomics. 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All participants or their legal guardians signed an informed consent form.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"\u201cNot applicable\u201d.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"TN is currently an employee of Ultragenyx Farmac\u00eautica Brasil LTDA and hold stock in Ultragenyx Pharmaceutical Inc. All other authors declare no conflicts of interest.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"254"}}