{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,25]],"date-time":"2026-10-25T11:52:00Z","timestamp":1792929120026,"version":"4.5.0"},"reference-count":47,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"12","funder":[{"DOI":"10.13039\/100000005","name":"U.S. Department of Defense","doi-asserted-by":"publisher","award":["W81XWH-15-1-0663"],"award-info":[{"award-number":["W81XWH-15-1-0663"]}],"id":[{"id":"10.13039\/100000005","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["UL1TR002544"],"award-info":[{"award-number":["UL1TR002544"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["1UL1TR003098"],"award-info":[{"award-number":["1UL1TR003098"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["P30 DK090868"],"award-info":[{"award-number":["P30 DK090868"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Kidney360"],"published-print":{"date-parts":[[2020,12]]},"abstract":"<jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>\n                      Autosomal dominant polycystic kidney disease (ADPKD) has been associated with metabolic disturbances characterized by downregulation of AMP-activated protein kinase (AMPK), a critical sensor of the cellular energy status. Therapeutic activation of AMPK by metformin could inhibit cyst enlargement by inhibition of both the mammalian target of rapamycin pathway and fluid secretion\n                      <jats:italic toggle=\"yes\">via<\/jats:italic>\n                      the CFTR chloride channel.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      We designed a phase-2, randomized, placebo-controlled, clinical trial to assess the safety, tolerability, and efficacy of metformin on total kidney volume in adults without diabetes (age 18\u201360 years) with ADPKD and eGFR of \u226550 ml\/min per 1.73 m\n                      <jats:sup>2<\/jats:sup>\n                      . There were no eligibility criteria relating to kidney volume. In addition to demographics and clinical\/family history, baseline parameters included eGFR, total kidney and liver volumes measured by MRI, and patient-reported outcomes were ascertained by the Medical Outcomes Study Short Form-36, the Gastrointestinal Safety Rating Scale, and the HALT-PKD pain questionnaire.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      We successfully randomized 97 participants recruited from two university-based clinical sites in Baltimore and Boston. The mean age of participants was 41.9 years, 72% were female, and 94% of participants were White. The majority of study participants had early stage disease, with a mean eGFR of 86.8\u00b119.0 ml\/min per 1.73 m\n                      <jats:sup>2<\/jats:sup>\n                      . Approximately half of the study participants (48%) were classified as high risk for progression (Mayo imaging classes 1C, 1D, or 1E). There was no correlation between kidney and\/or liver size and health-related quality of life (HRQoL) or gastrointestinal symptom severity.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>We report successful recruitment in this ongoing, novel, clinical trial of metformin in ADPKD, with a study sample comprising patients with early stage disease and nearly a half of participants considered at high estimated risk for progression. Participants reported a low gastrointestinal symptom burden at baseline, and HRQoL similar to that of the general population, with no differences in symptoms or HRQoL related to organomegaly.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Clinical Trial registry name and registration number<\/jats:title>\n                    <jats:p>Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease (TAME), NCT02656017<\/jats:p>\n                  <\/jats:sec>","DOI":"10.34067\/kid.0004002020","type":"journal-article","created":{"date-parts":[[2020,9,23]],"date-time":"2020-09-23T18:10:13Z","timestamp":1600884613000},"page":"1363-1372","source":"Crossref","is-referenced-by-count":16,"title":["Baseline Characteristics and Patient-Reported Outcomes of ADPKD Patients in the Multicenter TAME-PKD Clinical Trial"],"prefix":"10.34067","volume":"1","clinical-trial-number":[{"clinical-trial-number":"nct02656017","registry":"10.18810\/clinical-trials-gov"}],"author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1693-0734","authenticated-orcid":false,"given":"Stephen L.","family":"Seliger","sequence":"first","affiliation":[{"name":"Department of Medicine, Division of Nephrology, University of Maryland School of Medicine, Baltimore, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Terry","family":"Watnick","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Nephrology, University of Maryland School of Medicine, Baltimore, Maryland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew D.","family":"Althouse","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Internal Medicine, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ronald D.","family":"Perrone","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Nephrology, Tufts Medical Center, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kaleab Z.","family":"Abebe","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Internal Medicine, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenneth R.","family":"Hallows","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Nephrology and Hypertension, University of Southern California Keck School of Medicine, Los Angeles, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dana C.","family":"Miskulin","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Internal Medicine, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kyongtae T.","family":"Bae","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Nephrology, Tufts Medical Center, Boston, Massachusetts"},{"name":"Department of RadiologyDepartment of Radiology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","published-online":{"date-parts":[[2020,12,31]]},"reference":[{"key":"B1-20230821","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1146\/annurev.med.60.101707.125712","article-title":"Polycystic kidney disease","volume":"60","author":"Harris","year":"2009","journal-title":"Annu Rev Med"},{"key":"B2-20230821","doi-asserted-by":"crossref","first-page":"1477","DOI":"10.1056\/NEJMcp0804458","article-title":"Clinical practice. 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