Evidence map›Paper›PMID 39041931›Full record

ArticlePediatric pulmonology2024

Cyclosporine A in children with ABCA3 deficiency.

Xiaohua Yang, Maria E Forstner, Ina Rothenaigner, Marina Bullo, Tugba E Şismanlar, Ayse T Aslan, Philipp Latzin, Kamyar Hadian, Matthias Griese

Abstract readCase Reports
PubMed Full text at PMC Publisher
In one paragraph

Article in Pediatric pulmonology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
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

9 authors.

Xiaohua YangDr. von Haunersches Kinderspital, University of Munich, German Center for Lung Research (DZL), Munich, Germany.ORCID 0009-0001-4416-9962
Maria E ForstnerDr. von Haunersches Kinderspital, University of Munich, German Center for Lung Research (DZL), Munich, Germany.
Ina RothenaignerResearch Unit Signaling and Translation, Helmholtz Zentrum München, Neuherberg, Germany.
Marina BulloPediatric Pneumology and Allergology, University Children's Inselspital Bern, University of Bern, Bern, Switzerland.
Tugba E ŞismanlarDepartment of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey.ORCID 0000-0001-7284-4999
Ayse T AslanDepartment of Pediatric Pulmonology, Faculty of Medicine, Gazi University, Ankara, Turkey.
Philipp LatzinPediatric Pneumology and Allergology, University Children's Inselspital Bern, University of Bern, Bern, Switzerland.
Kamyar HadianResearch Unit Signaling and Translation, Helmholtz Zentrum München, Neuherberg, Germany.
Matthias GrieseDr. von Haunersches Kinderspital, University of Munich, German Center for Lung Research (DZL), Munich, Germany.ORCID 0000-0003-0113-912X

Funding

Deutsche Forschungsgemeinsachft (DFG Gr 970/9-2)German center for Lung Research, CPC Munich (82DZL053B2)UK child lung foundation
6 · The paper itself

Abstract

backgroundBiallelic ATP-binding cassette subfamily A member 3 (ABCA3) variants can cause interstitial lung disease in children and adults, for which no proven treatments exist. Recent in vitro evidence suggested that cyclosporine A (CsA) could correct some ABCA3 variants, however for other variants this is unknown and no data in patients exist.

methodsWe retrieved the clinical data of two children aged 2 and 4 years carrying homozygous ABCA3 variants (G210C and Q1045R, respectively) and empiric CsA treatment from the Kids Lung Register database. In vitro experiments functionally characterized the two variants and explored the effects of CsA alone or combined with hydroxychloroquine (HCQ) in a human alveolar epithelial cell line (A549) derived from adenocarcinoma cells.

resultsSix weeks following the introduction of CsA, both children required a reduced O

conclusionsCsA treatment might be helpful for certain patients with ABCA3 deficiency, however, currently strong clinical supporting evidence is lacking. Appropriate trials are necessary to overcome this unmet need.

Indexed as

ATP-Binding Cassette TransportersCyclosporineHydroxychloroquineA549 CellsAzithromycinChild, PreschoolHumansLung Diseases, InterstitialPrednisoloneABCA3 protein, humanATP-Binding Cassette TransportersAzithromycinCyclosporineHydroxychloroquinePrednisoloneABCA3ATP‐binding cassette subfamily A member 3childhoodcyclosporine Ahydroxychloroquineinterstitial lung disease

Identifiers

PMID39041931
PMCPMC11600998

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Registered trials

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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.