Evidence map›Paper›PMID 36454436›Full record

ArticleOncology and therapy2023

Clinical Characteristics and Management of Children and Adults with Neurofibromatosis Type 1 and Plexiform Neurofibromas in Denmark: A Nationwide Study.

Cecilie Ejerskov, Stense Farholt, Flemming Secher Kromann Nielsen, Ingunn Berg, Stine Bogetofte Thomasen, Aparna Udupi, Trude Ågesen, Sofie de Fine Licht, Mette Møller Handrup

Open access · goldAbstract read
In one paragraph

Article in Oncology and therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 1 pooled it
1.6field-weighted citation impact, top 15% 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

18 citing papers in PubMed, 1 synthesis or guideline pooled it, 17 citations in OpenAlex.

  1. Pooled it
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  4. Management of plexiform neurofibromas in neurofibromatosis type 1: An Italian Delphi consensus.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026
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  6. [Interpretation of the Expert Consensus for Full Course Management of Plexiform Neurofibroma (2025 Edition)].Zhongguo xiu fu chong jian wai ke za zhi = Zhongguo xiufu chongjian waike zazhi = Chinese journal of reparative and reconstructive surgery · 2026
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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 4 institutions in 2 countries.

Cecilie EjerskovCentre for Rare Diseases, Paediatric and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark. cecilie@ejerskov.dk.ORCID http://orcid.org/0000-0003-3815-0613
Stense FarholtCentre for Rare Diseases, Paediatric and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark.
Flemming Secher Kromann NielsenDepartment of Radiology, Aarhus University Hospital, Aarhus, Denmark.
Ingunn BergCentre for Rare Diseases, Paediatric and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark.
Stine Bogetofte ThomasenCentre for Rare Diseases, Paediatric and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark.ORCID http://orcid.org/0000-0002-7150-4332
Aparna UdupiBiostatistical Advisory Service (BIAS), Faculty of Health, Aarhus University, Aarhus, Denmark.
Trude ÅgesenAstraZeneca Nordic, Södertälje, Sweden.
Sofie de Fine LichtAstraZeneca Nordic, Södertälje, Sweden.ORCID http://orcid.org/0000-0001-7855-4706
Mette Møller HandrupCentre for Rare Diseases, Paediatric and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark.
Aarhus University Hospital · DKAarhus University · DKAstraZeneca (Sweden) · SERigshospitalet · DK

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionPlexiform neurofibromas (PN) are benign nerve sheath tumours that are a frequent and potentially debilitating complication in patients with neurofibromatosis type 1 (NF1). The objective of this study was to describe the natural history of PN in children, adolescents and adults with NF1.

methodsThis was a nationwide, longitudinal cohort study of patients with NF1 under observation at the two national centres of NF1 expertise in Denmark between 2000 and 2020. Patient and clinical characteristics were documented from individual medical records.

resultsA total of 1099 patients with NF1 were included. Overall, 12% (35/296) of paediatric patients and 21% (172/803) of adult patients had ≥ 1 large PN (≥ 3 cm). Approximately half of patients with a large PN had ≥ 1 symptomatic PN. The most frequent symptoms were pain, neurological deficits, cosmetic issues, disfigurement, compression, increased psychosocial burden and vision loss. Clinical evaluations of PN size were available for 40 PN in 34 paediatric patients and 191 PN in 159 adult patients with large PN. Surgery (complete resection or debulking) was performed in 38% (15/40) of PN in paediatric patients and 45% (86/191) in adult patients. In addition, 35% of PN in paediatric patients and 33% in adult patients were inoperable. In a subgroup analysis, the overall PN size increased 1.06-fold per year. Malignant peripheral nerve sheath tumours (MPNST) were diagnosed in 21 patients (two paediatric and 19 adult patients).

conclusionsThis study shows that PN are common, their size and prevalence increase with age, many are often inoperable and pain and other symptoms are frequently associated. The results highlight the severe sequelae and unmet need for alternatives to analgesia and surgery in patients with PN.

Indexed as

EpidemiologyMalignant peripheral nerve sheath tumourNeurofibromatosis type 1Plexiform neurofibromas

Identifiers

PMID36454436
PMCPMC9935791
OpenAlexW4310524613

What OpenQuestion holds

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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.