1. Adverse events associated with currently used medical treatments for cystinuria and treatment goals: results from a series of 442 patients in France.
- Author
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Prot-Bertoye C, Lebbah S, Daudon M, Tostivint I, Jais JP, Lillo-Le Louët A, Pontoizeau C, Cochat P, Bataille P, Bridoux F, Brignon P, Choquenet C, Combe C, Conort P, Decramer S, Doré B, Dussol B, Essig M, Frimat M, Gaunez N, Joly D, Le Toquin-Bernard S, Méjean A, Meria P, Morin D, N'Guyen HV, Normand M, Pietak M, Ronco P, Saussine C, Tsimaratos M, Friedlander G, Traxer O, Knebelmann B, and Courbebaisse M
- Subjects
- Adolescent, Adult, Aged, Child, Child, Preschool, Drug-Related Side Effects and Adverse Reactions, Female, France, Humans, Hydrogen-Ion Concentration, Infant, Male, Middle Aged, Penicillamine adverse effects, Penicillamine therapeutic use, Retrospective Studies, Sodium Bicarbonate adverse effects, Sodium Bicarbonate therapeutic use, Tiopronin adverse effects, Tiopronin therapeutic use, Treatment Outcome, Urinalysis, Young Adult, Cystinuria drug therapy, Cystinuria prevention & control
- Abstract
Objective: To evaluate medical treatments, in terms of adverse events (AEs) and therapeutic goals, in a large series of patients with cystinuria., Patients and Methods: Data from 442 patients with cystinuria were recorded retrospectively. Crystalluria was studied in 89 patients. A mixed-effects logistic regression model was used to estimate how urine pH, specific gravity and cysteine-binding thiols (CBT) correlate with risk of cystine crystalluria., Results: Alkalizing agents and CBT agents were given to 88.8% (n = 381) and 55.3% (n = 238) of patients, respectively. Gastrointestinal AEs were reported in 12.3%, 10.4% and 2.6% of patients treated with potassium bicarbonate, potassium citrate and sodium bicarbonate, respectively (P = 0.008). The percentages of patients who experienced at least one AE with tiopronin (24.6%) and with D-penicillamine (29.5%) were similar (P = 0.45). Increasing urine pH and decreasing urine specific gravity significantly reduced the risk of cystine crystalluria, whereas D-penicillamine and tiopronin treatments did not reduce this risk (odds ratio [OR] 1 for pH ≤6.5; OR 0.52 [95% confidence interval {95% CI} 0.28-0.95] for 7.0
8.0, P <0.001)., Conclusion: Adverse events were frequent with D-penicillamine and tiopronin. Alkaline hyperdiuresis was well tolerated and reduced cystine crystalluria. Urine specific gravity ≤1.005 and urine pH >7.5, while warning about calcium-phosphate crystallization, should be the goals of medical therapy., (© 2019 The Authors BJU International © 2019 BJU International Published by John Wiley & Sons Ltd.) - Published
- 2019
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