PMID- 30801923
OWN - NLM
STAT- MEDLINE
DCOM- 20200601
LR  - 20200601
IS  - 1464-410X (Electronic)
IS  - 1464-4096 (Linking)
VI  - 124
IP  - 5
DP  - 2019 Nov
TI  - Adverse events associated with currently used medical treatments for cystinuria 
      and treatment goals: results from a series of 442 patients in France.
PG  - 849-861
LID - 10.1111/bju.14721 [doi]
AB  - 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 
      <pH </=7.5, P = 0.03; OR 0.26 [95% CI 0.13-0.53] for 7.5 < pH </=8.0, P <0.001; OR 1 
      for specific gravity </=1.005 OR 5.76 [95% CI 1.45-22.85] for 1.006 </= specific 
      gravity </=1.010, P = 0.01; and OR 11.06 [95% CI 2.76-44.26] for 1.011 </= specific 
      gravity </= 1.014, P < 0.001). Increased urine pH significantly increased the risk 
      of calcium phosphate crystalluria (OR 1 for pH</= 6.5; OR 6.09 [95% CI 2.15-17.25] 
      for pH >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.
CI  - (c) 2019 The Authors BJU International (c) 2019 BJU International Published by John 
      Wiley & Sons Ltd.
FAU - Prot-Bertoye, Caroline
AU  - Prot-Bertoye C
AUID- ORCID: 0000-0003-1373-1323
AD  - Department of Physiology, Functional Renal Explorations Department, AP-HP (Public 
      Assistance Hospitals of Paris), Georges Pompidou European Hospital, Paris 
      Descartes University, Paris, France.
AD  - INSERM UMRS 1138, Paris, France.
FAU - Lebbah, Said
AU  - Lebbah S
AD  - Department of Biostatistics, AP-HP, Necker Hospital for Sick Children, Paris 
      Descartes University, Medicine, Paris, France.
FAU - Daudon, Michel
AU  - Daudon M
AD  - Department of Physiology, Functional Renal Explorations Department, AP-HP, Tenon 
      Hospital Pierre and Marie Curie University, INSERM UMR S 1155, Paris, France.
FAU - Tostivint, Isabelle
AU  - Tostivint I
AD  - Department of Nephrology, AP-HP, Pitie-Salpetriere Hospital, Paris, France.
FAU - Jais, Jean-Philippe
AU  - Jais JP
AD  - Department of Biostatistics, AP-HP, Necker Hospital for Sick Children, Paris 
      Descartes University, Paris, France.
AD  - Inserm UMRS 1138 team 22, Paris, France.
FAU - Lillo-Le Louet, Agnes
AU  - Lillo-Le Louet A
AD  - Department of Pharmacovigilance, AP-HP, Georges Pompidou European Hospital, 
      Paris, France.
FAU - Pontoizeau, Clement
AU  - Pontoizeau C
AD  - Functional Unit of Metabolomics, Functional Explorations Department, APHP, Necker 
      Hospital for Sick Children, Paris Descartes University, Paris, France.
FAU - Cochat, Pierre
AU  - Cochat P
AD  - Department of Pediatrics, Hospices Civils de Lyon, Centre de Reference des 
      Maladies Renales Rares Nephrogones, Lyon, France.
FAU - Bataille, Pierre
AU  - Bataille P
AD  - Department of Nephrology, Boulogne-sur-Mer Hospital, Boulogne sur Mer, France.
FAU - Bridoux, Franck
AU  - Bridoux F
AD  - Department of Neprhology, Poitiers University Hospital, Poitiers University, 
      Poitiers, France.
FAU - Brignon, Pierre
AU  - Brignon P
AD  - Department of Nephrology, Pasteur Hospital, Colmar, France.
FAU - Choquenet, Christian
AU  - Choquenet C
AD  - Department of Urology, Princesse Grace Hospital, Monaco, France.
FAU - Combe, Christian
AU  - Combe C
AD  - Department of Nephrology, Bordeaux University Hospital, Bordeaux University, 
      Bordeaux, France.
FAU - Conort, Pierre
AU  - Conort P
AD  - Department of Urology, AP-HP, Pitie-Salpetriere Hospital, Paris, France.
FAU - Decramer, Stephane
AU  - Decramer S
AD  - Department of Nephrology and Internal Medicine, Toulouse University Children 
      Hospital, Toulouse, France.
AD  - INSERM U1048, Toulouse, France.
FAU - Dore, Bertrand
AU  - Dore B
AD  - Department of Urology, Poitiers University Hospital, Poitiers University, 
      Poitiers, France.
FAU - Dussol, Bertrand
AU  - Dussol B
AD  - Department of Nephrology, AP-HM (Public Assistance Hospitals of Marseille), 
      Conception Hospital, Aix-Marseille University, Marseille, France.
FAU - Essig, Marie
AU  - Essig M
AD  - Department of Nephrology, Dialysis and Transplantation, Limoges University 
      Hospital, Limoges University, Limoges, France.
AD  - INSERM UMR-S850, Limoges, France.
FAU - Frimat, Marie
AU  - Frimat M
AD  - Department of Nephrology, Claude Huriez University Hospital, Lille, France.
FAU - Gaunez, Nicolas
AU  - Gaunez N
AD  - Department of Urology, Niort Hospital, Niort, France.
FAU - Joly, Dominique
AU  - Joly D
AD  - Department of Nephrology, AP-HP, Necker Hospital for Sick Children, Paris 
      Descartes University, Medicine, Paris, France.
FAU - Le Toquin-Bernard, Sophie
AU  - Le Toquin-Bernard S
AD  - Department of Urology, Private Saint-Martin Hospital, Caen, France.
FAU - Mejean, Arnaud
AU  - Mejean A
AD  - Department of Urology, AP-HP, Georges Pompidou European Hospital, Paris Descartes 
      University, Medicine, Paris, France.
FAU - Meria, Paul
AU  - Meria P
AD  - Department of Urology, AP-HP, Saint-Louis Hospital, Paris, France.
FAU - Morin, Denis
AU  - Morin D
AD  - Department of Pediatrics (Pediatric Nephrology and Diabetology), Montpellier 
      University Hospital, Montpellier, France.
AD  - CNRS, UMR 5203-INSERM U661, Montpellier, France.
FAU - N'Guyen, Hung V
AU  - N'Guyen HV
AD  - Department of Urology, AP-HP, Saint-Louis Hospital, Paris, France.
FAU - Normand, Michel
AU  - Normand M
AD  - Department of Nephrology, Private Saint-Martin Hospital, Pessac, France.
FAU - Pietak, Michel
AU  - Pietak M
AD  - Department of Urology, AP-HP, Georges Pompidou European Hospital, Paris, France.
FAU - Ronco, Pierre
AU  - Ronco P
AD  - Department of Nephrology and Dialysis, AP-HP, Tenon Hospital, Pierre and Marie 
      Curie University, Paris, France.
AD  - INSERM Unit 702, Paris, France.
FAU - Saussine, Christian
AU  - Saussine C
AD  - Department of Urology, Strasbourg University Hospital, Strasbourg University, 
      Strasbourg, France.
FAU - Tsimaratos, Michel
AU  - Tsimaratos M
AD  - Department of Pediatrics, AP-HM, La Timone Hospital, Aix-Marseille University, 
      Marseille, France.
FAU - Friedlander, Gerard
AU  - Friedlander G
AD  - Department of Physiology, Functional Renal Explorations Department, AP-HP (Public 
      Assistance Hospitals of Paris), Georges Pompidou European Hospital, Paris 
      Descartes University, Paris, France.
AD  - INSERM U1151, Paris, France.
FAU - Traxer, Olivier
AU  - Traxer O
AD  - Department of Urology, AP-HP, Tenon Hospital, Pierre and Marie Curie University, 
      Paris, France.
FAU - Knebelmann, Bertrand
AU  - Knebelmann B
AD  - Department of Nephrology, AP-HP, Necker Hospital for Sick Children, Paris 
      Descartes University, Medicine, Paris, France.
FAU - Courbebaisse, Marie
AU  - Courbebaisse M
AD  - Department of Physiology, Functional Renal Explorations Department, AP-HP (Public 
      Assistance Hospitals of Paris), Georges Pompidou European Hospital, Paris 
      Descartes University, Paris, France.
AD  - INSERM U1151, Paris, France.
CN  - French Cystinuria Group
LA  - eng
PT  - Journal Article
DEP - 20190325
PL  - England
TA  - BJU Int
JT  - BJU international
JID - 100886721
RN  - 8MDF5V39QO (Sodium Bicarbonate)
RN  - C5W04GO61S (Tiopronin)
RN  - GNN1DV99GX (Penicillamine)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Child
MH  - Child, Preschool
MH  - *Cystinuria/drug therapy/prevention & control
MH  - Drug-Related Side Effects and Adverse Reactions
MH  - Female
MH  - France
MH  - Humans
MH  - Hydrogen-Ion Concentration
MH  - Infant
MH  - Male
MH  - Middle Aged
MH  - Penicillamine/adverse effects/therapeutic use
MH  - Retrospective Studies
MH  - Sodium Bicarbonate/adverse effects/therapeutic use
MH  - Tiopronin/adverse effects/therapeutic use
MH  - Treatment Outcome
MH  - Urinalysis
MH  - Young Adult
OTO - NOTNLM
OT  - D-penicillamine
OT  - cystinuria
OT  - potassium citrate
OT  - sodium bicarbonate
OT  - tiopronin
EDAT- 2019/02/26 06:00
MHDA- 2020/06/02 06:00
CRDT- 2019/02/26 06:00
PHST- 2019/02/26 06:00 [pubmed]
PHST- 2020/06/02 06:00 [medline]
PHST- 2019/02/26 06:00 [entrez]
AID - 10.1111/bju.14721 [doi]
PST - ppublish
SO  - BJU Int. 2019 Nov;124(5):849-861. doi: 10.1111/bju.14721. Epub 2019 Mar 25.