Mostrar el registro sencillo del ítem

Safety, efficacy, and acceptability of ADV7103 during 24 months of treatment: an open-label study in pediatric and adult patients with distal renal tubular acidosis

dc.rights.licenseopenen_US
dc.contributor.authorBERTHOLET-THOMAS, Aurelia
dc.contributor.authorGUITTET, Catherine
dc.contributor.authorMANSO-SILVAN, Maria A.
dc.contributor.authorJOUKOFF, Sophie
dc.contributor.authorNAVAS-SERRANO, Victor
dc.contributor.authorBAUDOUIN, Veronique
dc.contributor.authorCAILLIEZ, Mathilde
dc.contributor.authorDI MAIO, Massimo
dc.contributor.authorGILLION-BOYER, Olivia
dc.contributor.authorGOLUBOVIC, Emilija
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorHARAMBAT, Jerome
dc.contributor.authorKNEBELMANN, Bertrand
dc.contributor.authorNOBILI, Francois
dc.contributor.authorNOVO, Robert
dc.contributor.authorPODRACKA, Ludmila
dc.contributor.authorROUSSEY-KESLER, Gwenaelle
dc.contributor.authorGRANIER, Luc-Andre
dc.date.accessioned2021-04-02T07:51:00Z
dc.date.available2021-04-02T07:51:00Z
dc.date.issued2021-02
dc.identifier.issn0931-041xen_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/26850
dc.description.abstractEnBACKGROUND: A new prolonged-release formulation of potassium citrate and potassium bicarbonate, ADV7103, has been shown to improve metabolic control, palatability, and gastrointestinal safety in patients with distal renal tubular acidosis (dRTA) when compared to standard of care (SoC) treatments. The present work evaluates safety and efficacy of ADV7103 during 24 months. METHODS: Thirty pediatric and adult patients were included in an open-label extension study after a phase II/III trial. Safety and tolerability were assessed. Plasma bicarbonate and potassium levels, as well as urine parameters, were evaluated over time. Acceptability, adherence, and quality of life were also assessed. The evolution of clinical consequences of dRTA in the cohort was explored. RESULTS: There were 104 adverse events (AEs) reported, but only 9 gastrointestinal events observed in five patients (17%) were considered to be related to ADV7103 treatment. There were no AEs leading to treatment discontinuation. Plasma bicarbonate and potassium levels were in the normal ranges at the different visits, respectively, in 69-86% and 83-93% of patients. Overall adherence rates were ≥ 75% throughout the whole study in 79% patients. An average improvement of quality of life of 89% was reported at 24 months of study. CONCLUSIONS: Common AEs concerned metabolism and gastrointestinal disorders; the former being related to the disease. Less than half of the gastrointestinal AEs were related to ADV7103 treatment and they were mostly mild in severity. Metabolic parameters were maintained in the normal ranges in most patients. Patient satisfaction was high and adherence to treatment was good and remained stable. TRIAL REGISTRATION NUMBER: Registered as EudraCT 2013-003828-36 on the 3rd of September 2013.
dc.language.isoENen_US
dc.rightsAttribution 3.0 United States*
dc.rights.urihttp://creativecommons.org/licenses/by/3.0/us/*
dc.subject.endRTA
dc.subject.enSafety
dc.subject.enPlasma bicarbonate
dc.subject.enAcceptability
dc.subject.enAdherence to treatment
dc.title.enSafety, efficacy, and acceptability of ADV7103 during 24 months of treatment: an open-label study in pediatric and adult patients with distal renal tubular acidosis
dc.title.alternativePediatr Nephrolen_US
dc.typeArticle de revueen_US
dc.identifier.doi10.1007/s00467-020-04873-0en_US
dc.subject.halSciences du Vivant [q-bio]/Santé publique et épidémiologieen_US
dc.identifier.pubmed33635379en_US
bordeaux.journalPediatric Nephrologyen_US
bordeaux.hal.laboratoriesBordeaux Population Health Research Center (BPH) - U1219en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.teamLEHA_BPH
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
hal.identifierhal-03188475
hal.version1
hal.date.transferred2021-04-02T07:51:05Z
hal.audienceInternationale
hal.exporttrue
bordeaux.COinSctx_ver=Z39.88-2004&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.jtitle=Pediatric%20Nephrology&rft.date=2021-02&rft.eissn=0931-041x&rft.issn=0931-041x&rft.au=BERTHOLET-THOMAS,%20Aurelia&GUITTET,%20Catherine&MANSO-SILVAN,%20Maria%20A.&JOUKOFF,%20Sophie&NAVAS-SERRANO,%20Victor&rft.genre=article


Archivos en el ítem

Thumbnail
Nombre:
BPH_PN_2021_Bertholet-Thomas.pdf
Tamaño:
1.240Mb
Formato:
PDF
Ver/Abrir
Thumbnail
Nombre:
license_rdf
Tamaño:
914bytes
Formato:
application/rdf+xml
Ver/Abrir

Este ítem aparece en la(s) siguiente(s) colección(ones)

Mostrar el registro sencillo del ítem