Evidence map›Paper›PMID 40804680›Full record

ArticleOrphanet journal of rare diseases2025

6-year treatment follow-up with an extended-release alkaline formulation (Sibnayal

Aurélia Bertholet-Thomas, Aurélie De Mul, Julie Bernardor, Gwenaëlle Roussey-Kesler, Ludmila Podracka, Robert Novo, François Nobili, Bertrand Knebelmann, Jérôme Harambat, Emilija Golubovic and 7 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

17 authors.

Aurélia Bertholet-ThomasCentre de Référence des Maladies Rénales Rares- MAREGE- Hôpital Femme Mère Enfant, Hospices Civils de Lyon- Filière ORKID (Orphan Kidney Diseases), ERK-Net (The European Rare Kidney Disease Network), Lyon, France. aurelia.bertholet-thomas@chu-lyon.fr.
Aurélie De MulCentre de Référence des Maladies Rénales Rares- MAREGE- Hôpital Femme Mère Enfant, Hospices Civils de Lyon- Filière ORKID (Orphan Kidney Diseases), ERK-Net (The European Rare Kidney Disease Network), Lyon, France.
Julie BernardorHôpital l'Archet, Service de Rhumatologie Pédiatrique et Médecine Interne de l'enfant, CHU de Nice, Nice, France.
Gwenaëlle Roussey-KeslerHôpital Mère-Enfant, Clinique Médicale Pédiatrique, CHU de Nantes, Unité de Néphrologie et Hémodialyse Pédiatrique, Nantes, France.
Ludmila PodrackaDepartment of Pediatrics, National Institute of Children's, Bratislava, Slovakia.
Robert NovoService de Néphrologie Pédiatrique, Hôpital Jeanne de Flandre, CHRU de Lille, Lille, France.
François NobiliHôpital Jean Minjoz, CHU de Besançon, Service de Pédiatrie 2, Besançon, France.
Bertrand KnebelmannService de Néphrologie adultes, Hôpital Necker, APHP, Paris, France.
Jérôme HarambatService de Pédiatrie, Hôpital Pellegrin- Enfants, CHU de Bordeaux, Bordeaux, France.
Emilija GolubovicKlinički Centar Niš, Niš, Serbia.
Olivia BoyerAPHP, Service de Néphrologie Pédiatrique, Hôpital Necker-Enfants Malades, Paris, France.
Massimo Di MaioService de Réanimation Néonatale et Néonatologie, CHU de Nîmes, Nîmes, France.
Mathilde CailliezAP-HM, Service de Pédiatrie Multidisciplinaire, Hôpital de la Timone, Marseille, France.
Véronique BaudouinService de Néphrologie Pédiatrique, Hôpital Universitaire Robert Debré-APHP, Paris, France.
Laure ChidlerADVICENNE, Paris, France.
Véronique LeblancADVICENNE, Paris, France.
Justine BacchettaCentre de référence des maladies rares du métabolisme du calcium et du phosphate-Hospices, Civils de Lyon-OSCAR-ERN BOND, Lyon, France.

Funding

Advicenne
6 · The paper itself

Abstract

backgroundDistal renal tubular acidosis (dRTA) is a rare disease characterized by hyperchloremic metabolic acidosis affecting growth, bone and kidney health.

methodsThe aim of B22CS study was to evaluate long-term safety and efficacy (anthropometric/pubertal, tubular damages/kidney function, bone biomarkers, compliance assessments) of Sibnayal

resultsA total of 30 patients with primary dRTA (mean age:10.6 ± 6.0 years) entered this long-term study (average of 6 years). At inclusion, most patients had adequate metabolic control, normal kidney function and height. Sibnayal

conclusionLong-term data support the good safety and efficacy profile of Sibnayal

Indexed as

Acidosis, Renal TubularAdolescentAdultChildChild, PreschoolDelayed-Action PreparationsFemaleFollow-Up StudiesHumansMaleYoung AdultDelayed-Action Preparations

Identifiers

PMID40804680
PMCPMC12351860

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.