Evidence map›Paper›PMID 35945193›Full record

Trial reportNature communications2022

An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich's ataxia.

Kevin C Kemp, Anastasia Georgievskaya, Kelly Hares, Juliana Redondo, Steven Bailey, Claire M Rice, Neil J Scolding, Chris Metcalfe, Alastair Wilkins

Open access · goldAbstract readClinical Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Nature communications, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.2field-weighted citation impact, top 59% of its field
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

1 citing paper in PubMed, 2 citations in OpenAlex.

  1. Review
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 at 2 institutions in 1 country.

Kevin C KempTranslational Health Sciences, Bristol Medical School, University of Bristol, Southmead Hospital, Bristol, UK. kevin.kemp@bristol.ac.uk.ORCID 0000-0003-1465-1309
Anastasia GeorgievskayaTranslational Health Sciences, Bristol Medical School, University of Bristol, Southmead Hospital, Bristol, UK.ORCID 0000-0002-1781-2174
Kelly HaresTranslational Health Sciences, Bristol Medical School, University of Bristol, Southmead Hospital, Bristol, UK.ORCID 0000-0002-4637-584X
Juliana RedondoTranslational Health Sciences, Bristol Medical School, University of Bristol, Southmead Hospital, Bristol, UK.
Steven BaileyTranslational Health Sciences, Bristol Medical School, University of Bristol, Southmead Hospital, Bristol, UK.ORCID 0000-0001-8390-9399
Claire M RiceTranslational Health Sciences, Bristol Medical School, University of Bristol, Southmead Hospital, Bristol, UK.ORCID 0000-0002-9851-4426
Neil J ScoldingTranslational Health Sciences, Bristol Medical School, University of Bristol, Southmead Hospital, Bristol, UK.ORCID 0000-0001-9177-1043
Chris MetcalfePopulation Health Sciences, Bristol Medical School, University of Bristol, Canynge Hall, Bristol, BS8 2PR, UK.ORCID 0000-0001-8318-8907
Alastair WilkinsTranslational Health Sciences, Bristol Medical School, University of Bristol, Southmead Hospital, Bristol, UK.ORCID 0000-0001-8176-8078
Southmead Hospital · GBUniversity of Bristol · GB

Funding

Medical Research Council MR/T02089X/1Wellcome TrustWellcome Trust 204813/Z/16/Z
6 · The paper itself

Abstract

Friedreich's ataxia (FA) is an inherited progressive neurodegenerative disease for which there is no proven disease-modifying treatment. Here we perform an open-label, pilot study of recombinant human granulocyte-colony stimulating factor (G-CSF) administration in seven people with FA (EudraCT: 2017-003084-34); each participant receiving a single course of G-CSF (Lenograstim; 1.28 million units per kg per day for 5 days). The primary outcome is peripheral blood mononuclear cell frataxin levels over a 19-day period. The secondary outcomes include safety, haematopoietic stem cell (HSC) mobilisation, antioxidant levels and mitochondrial enzyme activity. The trial meets pre-specified endpoints. We show that administration of G-CSF to people with FA is safe. Mobilisation of HSCs in response to G-CSF is comparable to that of healthy individuals. Notably, sustained increases in cellular frataxin concentrations and raised PGC-1α and Nrf2 expression are detected. Our findings show potential for G-CSF therapy to have a clinical impact in people with FA.

Indexed as

Friedreich AtaxiaGranulocyte Colony-Stimulating FactorRecombinant ProteinsGranulocytesHumansLeukocytes, MononuclearPilot ProjectsGranulocyte Colony-Stimulating FactorRecombinant Proteins

Identifiers

PMID35945193
PMCPMC9363409
OpenAlexW3111062374

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.