Evidence map›Paper›PMID 31583187›Full record

ArticleNeurology. Clinical practice2019

Association between patient-reported outcomes and objective disease indices in people with NF2.

Aishwarya Shukla, Fang-Chi Hsu, Bronwyn Slobogean, Shannon Langmead, Yao Lu, Jaishri O Blakeley, Roy E Strowd

Open access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Neurology. Clinical practice, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
0.6field-weighted citation impact, top 31% 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

10 citing papers in PubMed, 11 citations in OpenAlex.

  1. Article
  2. Article
  3. Clinical severity grading of NF2-related schwannomatosis.Orphanet journal of rare diseases · 2025
    Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. 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

7 authors at 2 institutions in 1 country.

Aishwarya ShuklaJohns Hopkins School of Medicine (AS, BS, SL, JOB), the Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD; Department of Biostatistics and Data Science (F-CH), Wake Forest School of Medicine, Winston-Salem, NC; Division of Biostatistics and Epidemiology (YL), Weill Cornell Medical College, New York; Department of Neurology (RES), Wake Forest School of Medicine, Winston-Salem, NC; and Department of Neurology (RES), Johns Hopkins School of Medicine, Baltimore, MD.
Fang-Chi HsuJohns Hopkins School of Medicine (AS, BS, SL, JOB), the Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD; Department of Biostatistics and Data Science (F-CH), Wake Forest School of Medicine, Winston-Salem, NC; Division of Biostatistics and Epidemiology (YL), Weill Cornell Medical College, New York; Department of Neurology (RES), Wake Forest School of Medicine, Winston-Salem, NC; and Department of Neurology (RES), Johns Hopkins School of Medicine, Baltimore, MD.
Bronwyn SlobogeanJohns Hopkins School of Medicine (AS, BS, SL, JOB), the Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD; Department of Biostatistics and Data Science (F-CH), Wake Forest School of Medicine, Winston-Salem, NC; Division of Biostatistics and Epidemiology (YL), Weill Cornell Medical College, New York; Department of Neurology (RES), Wake Forest School of Medicine, Winston-Salem, NC; and Department of Neurology (RES), Johns Hopkins School of Medicine, Baltimore, MD.
Shannon LangmeadJohns Hopkins School of Medicine (AS, BS, SL, JOB), the Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD; Department of Biostatistics and Data Science (F-CH), Wake Forest School of Medicine, Winston-Salem, NC; Division of Biostatistics and Epidemiology (YL), Weill Cornell Medical College, New York; Department of Neurology (RES), Wake Forest School of Medicine, Winston-Salem, NC; and Department of Neurology (RES), Johns Hopkins School of Medicine, Baltimore, MD.
Yao LuJohns Hopkins School of Medicine (AS, BS, SL, JOB), the Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD; Department of Biostatistics and Data Science (F-CH), Wake Forest School of Medicine, Winston-Salem, NC; Division of Biostatistics and Epidemiology (YL), Weill Cornell Medical College, New York; Department of Neurology (RES), Wake Forest School of Medicine, Winston-Salem, NC; and Department of Neurology (RES), Johns Hopkins School of Medicine, Baltimore, MD.
Jaishri O BlakeleyJohns Hopkins School of Medicine (AS, BS, SL, JOB), the Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD; Department of Biostatistics and Data Science (F-CH), Wake Forest School of Medicine, Winston-Salem, NC; Division of Biostatistics and Epidemiology (YL), Weill Cornell Medical College, New York; Department of Neurology (RES), Wake Forest School of Medicine, Winston-Salem, NC; and Department of Neurology (RES), Johns Hopkins School of Medicine, Baltimore, MD.
Roy E StrowdJohns Hopkins School of Medicine (AS, BS, SL, JOB), the Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD; Department of Biostatistics and Data Science (F-CH), Wake Forest School of Medicine, Winston-Salem, NC; Division of Biostatistics and Epidemiology (YL), Weill Cornell Medical College, New York; Department of Neurology (RES), Wake Forest School of Medicine, Winston-Salem, NC; and Department of Neurology (RES), Johns Hopkins School of Medicine, Baltimore, MD.
Wake Forest University · USCornell University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveThe association between patient-reported outcomes and currently used clinical trial endpoints, including total vestibular schwannoma (VS) volume and word recognition score (WRS) in neurofibromatosis type 2 (NF2), is not known.

methodsA prospective observational study enrolling adult patients with NF2 was conducted at a single specialty center. Measures included: NF2 impact on quality of life (NFTI-QOL), short form (SF)-36; total VS volume, WRS; provider- and patient-reported disease severity (ProvSev, PatSev) measured with an institutionally derived multi-item (e.g., symptom burden, age-of-onset, and fatality-risk) and single-item Likert (mild, moderate, severe) scale.

resultsFifty-one patients were enrolled between June 2014 and August 2017. Mean age was 42.1 ± 18.2 years and 37.3% patients were male. Mean WRS was 74.4 ± 37.3%; mean total VS volume was 4.2 ± 5.2 cc. Additional lesions were common including meningioma (79.2%) and spinal ependymoma (39.6%). Mean NFTI-QOL score was 7.6 ± 4.9 and correlated with responses on the SF-36. NFTI-QOL also correlated well with PatSev (

conclusionsThe NFTI-QOL correlated well with multiple measures of disease severity but not commonly accepted endpoints for NF2 clinical trials including total VS volume in this US cohort of patients with NF2. This suggests that the NFTI-QOL captures components of the patient experience not sufficiently represented by objective measures of disease and underscores the important and complementary role of patient-focused measures in therapeutic outcome assessment in people with NF2.

Identifiers

PMID31583187
PMCPMC6745746
OpenAlexW2943204724

What OpenQuestion holds

Textmetadata
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.