Evidence map›Paper›PMID 34538258›Full record

ArticleOrphanet journal of rare diseases2021

Utility of modified Rankin Scale for brain vascular malformations in hereditary hemorrhagic telangiectasia.

K P Thompson, J Nelson, H Kim, S M Weinsheimer, D A Marchuk, M T Lawton, T Krings, M E Faughnan, Brain Vascular Malformation Consortium HHT Investigator Group

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

Article in Orphanet journal of rare diseases, 2021. 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
0.1field-weighted citation impact, top 50% 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, 1 citations in OpenAlex.

  1. 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 at 5 institutions in 2 countries.

K P ThompsonToronto HHT Centre, St. Michael's Hospital and Li Ka Shing Knowledge Institute, Toronto, Canada.
J NelsonCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, San Francisco, CA, USA.
H KimCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, San Francisco, CA, USA.
S M WeinsheimerCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, San Francisco, CA, USA.
D A MarchukDepartment of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC, USA.
M T LawtonDepartment of Neurosurgery, Barrow Neurological Institute, Phoenix, AZ, USA.
T KringsDivision of Neurosurgery, Department of Medical Imaging, Department of Surgery, University of Toronto, Toronto, Canada.
M E FaughnanToronto HHT Centre, St. Michael's Hospital and Li Ka Shing Knowledge Institute, Toronto, Canada. marie.faughnan@unityhealth.to.ORCID 0000-0002-3746-383X
Brain Vascular Malformation Consortium HHT Investigator Group
University of California, San Francisco · USSt. Michael's Hospital · CABarrow Neurological Institute · USDuke Medical Center · USUniversity of Toronto · CA

Funding

The Impact of COVID-19 on People Living with Rare Diseases and Their FamiliesU2CTR002818 · NCATS · CINCINNATI CHILDRENS HOSP MED CTR · PI Michal Kouril · 2019 to 2026
$51.2M
VCRC Administration UnitU54NS065705 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI KIM, HELEN · 2009 to 2023
$20.8M
NCATS NIH HHS U2C TR002818NINDS NIH HHS U54 NS065705
6 · The paper itself

Abstract

backgroundApproximately 10% of hereditary hemorrhagic telangiectasia (HHT) patients harbour brain vascular malformations (VMs). Intracranial hemorrhage (ICH) from brain VMs can lead to death or morbidity, while treatment options for brain VMs also have associated morbidity. The modified Rankin Scale (mRS) may provide an approach to identifying HHT-brain VM patients with poor outcomes, and their predictors. We aimed to measure the relationship between mRS score and brain VM, brain VM number, as well as other aspects of HHT, at enrollment and during prospective follow-up.

methods1637 HHT patients (342 with brain VMs) were recruited from 14 HHT centres of the Brain Vascular Malformation Consortium since 2010 and followed prospectively (mean = 3.4 years). We tested whether the presence of brain VM, other HHT organ involvement, and HHT mutation genotype were associated with worse mRS scores at baseline and during follow-up, using linear mixed models, adjusting for age, sex, and year of visit.

resultsPresence of brain VMs was not associated with worse mRS score at baseline and there was no significant worsening of mRS with prospective follow-up in these patients; 92% had baseline mRS of 0-2. HHT-related gastrointestinal (GI) bleeding was associated with worse mRS scores at baseline (0.37, 95% CI 0.26-0.47, p < 0.001), as were history of anemia (0.35, 95% CI 0.27-0.43, p < 0.001) and liver VMs (0.19, 95% CI 0.09-0.30, p < 0.001). Presence of pulmonary arteriovenous malformations (AVMs) was not associated with worse mRS scores at baseline. mRS score was not associated with either HHT genotype (Endoglin vs ACVRL1). Only GI bleeding was associated with a significantly worsening mRS during prospective follow-up (0.64, 95% CI 0.21-1.08, p = 0.004).

conclusionMost HHT-brain VM patients had good functional capacity (mRS scores 0-2) at baseline that did not change significantly over 3.4 mean years of follow-up, suggesting that mRS may not be useful for predicting or measuring outcomes in these patients. However, HHT patients with GI bleeding, anemia history or liver VMs had worse mRS scores, suggesting significant impact of these manifestations on functional capacity. Our study demonstrates the insensitivity of the mRS as an outcomes measure in HHT brain VM patients and reinforces the continued need to develop outcomes measures, and their predictors, in this group.

Indexed as

Arteriovenous FistulaCentral Nervous System Vascular MalformationsIntracranial Arteriovenous MalformationsTelangiectasia, Hereditary HemorrhagicActivin Receptors, Type IIEndoglinHumansProspective StudiesActivin Receptors, Type IIACVRL1 protein, humanEndoglin

Identifiers

PMID34538258
PMCPMC8451134
OpenAlexW3199508671

What OpenQuestion holds

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LicenceCC BY
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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.