Mp26-18 Alpha Lipoic Acid Reduces Stone Growth for Cystinuria Patients: Results From a Randomized, Double-Blind, Placebo-Controlled Clinical Trial

You have accessJournal of UrologyStone Disease: Medical & Dietary Therapy (MP26)1 May 2024MP26-18 ALPHA LIPOIC ACID REDUCES STONE GROWTH FOR CYSTINURIA PATIENTS: RESULTS FROM A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL Thomas Chi, David Bayne, Justin Ahn, Krishna Ramaswamy, David T. Tzou, Scott V. Wiener, Jonathan D. Harper, Margaret S. Pearle, Noah E. Canvasser, Simon L. Conti, Roger L. Sur, Greg E. Tasian, Wilson Sui, Heiko Yang, Ukrit Rompsaithong, and Marshall Stoller Thomas ChiThomas Chi , David BayneDavid Bayne , Justin AhnJustin Ahn , Krishna RamaswamyKrishna Ramaswamy , David T. TzouDavid T. Tzou , Scott V. WienerScott V. Wiener , Jonathan D. HarperJonathan D. Harper , Margaret S. PearleMargaret S. Pearle , Noah E. CanvasserNoah E. Canvasser , Simon L. ContiSimon L. Conti , Roger L. SurRoger L. Sur , Greg E. TasianGreg E. Tasian , Wilson SuiWilson Sui , Heiko YangHeiko Yang , Ukrit RompsaithongUkrit Rompsaithong , and Marshall StollerMarshall Stoller View All Author Informationhttps://doi.org/10.1097/01.JU.0001009408.66023.77.18AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: Cystinuria is a rare disease that causes lifelong, recurrent nephrolithiasis. Thiol derivatives are the current standard of care, yet they are expensive and have severe side effect profiles leading to poor compliance. Alpha-lipoic acid (ALA) is a well-tolerated antioxidant compound. In a preclinical murine model, ALA dramatically decreased stone growth compared to controls. This randomized clinical trial aimed to assess the effect of ALA on stone recurrence in cystinuria patients. METHODS: In this randomized, double-blinded, placebo-controlled single institution trial, patients>18 years old with a diagnosis of cystinuria were recruited. Participants were randomized to twice daily 600mg ALA versus placebo for three years. The primary endpoint was stone recurrence, defined as symptomatic stones or interval stone growth measured by computed tomography at baseline and end of the trial. The secondary endpoint was quantitative 24-hour urinary cystine levels. Continuous variables were compared using Mann-Whitney U test or repeated measures of analysis of variance and categorical by chi-square analysis. RESULTS: Of 249 patients screened, 50 participants were randomized to either ALA (n=25) or placebo (n=25). Nine patients on ALA and two on placebo withdrew from the study. The mean stone events were 6±4 versus 5±4 in the ALA and placebo groups, respectively (p=0.47). Compared to only 14% on placebo, 40% percent of patients who received ALA experienced a decrease in stone burden (Figure 1, p=0.06). Overall, mean stone growth was lower in the ALA group (-0.4mm±14.9 vs 8.7mm±15.8, p=0.08). One patient in the ALA group had significantly more growth compared to the rest, in a sub-analysis excluding this patient, the growth rate in the ALA group was -3.0mm±11.5 (p=0.03). There were no differences in quantitative urinary cystine levels. The most common adverse reactions were gastroesophageal reflux (6%) and nausea (4%). CONCLUSIONS: In a randomized clinical trial, 40% of patients who received ALA demonstrated a decrease in their overall stone burden. This occurred without changes in urinary chemistry suggesting that the mechanism of ALA remains unknown. ALA represents a promising alternative medication for cystinuria that reduces stone growth and has minimal adverse reactions. Download PPT Source of Funding: NIH/FDA FD005716 © 2024 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 211Issue 5SMay 2024Page: e420 Advertisement Copyright & Permissions© 2024 by American Urological Association Education and Research, Inc.Metrics Author Information Thomas Chi More articles by this author David Bayne More articles by this author Justin Ahn More articles by this author Krishna Ramaswamy More articles by this author David T. Tzou More articles by this author Scott V. Wiener More articles by this author Jonathan D. Harper More articles by this author Margaret S. Pearle More articles by this author Noah E. Canvasser More articles by this author Simon L. Conti More articles by this author Roger L. Sur More articles by this author Greg E. Tasian More articles by this author Wilson Sui More articles by this author Heiko Yang More articles by this author Ukrit Rompsaithong More articles by this author Marshall Stoller More articles by this author Expand All Advertisement PDF downloadLoading ...