Trial Readiness of Cavernous Malformations With Symptomatic Hemorrhage, Part I: Event Rates and Clinical Outcome-Reference-Cited by-同舟云学术

Trial Readiness of Cavernous Malformations With Symptomatic Hemorrhage, Part I: Event Rates and Clinical Outcome

Published:2024-01 Issue:1 Volume:55 Page:22-30
ISSN:0039-2499
Container-title:Stroke
language:en
Short-container-title:Stroke

Author:

Flemming Kelly D.¹^ORCID,Kim Helen²^ORCID,Hage Stephanie³^ORCID,Mandrekar Jay⁴,Kinkade Serena³,Girard Romuald³,Torbey Michel⁵^ORCID,Huang Judy⁶^ORCID,Huston John⁷^ORCID,Shu Yunhong⁷^ORCID,Lanzino Giuseppe⁸^ORCID,Selwyn Reed³⁹^ORCID,Hart Blaine⁹^ORCID,Mabray Marc⁹^ORCID,Feghali James⁶^ORCID,Sair Haris I.¹⁰,Narvid Jared¹¹^ORCID,Lupo Janine M.¹¹,Lee Justine³,Stadnik Agnieszka³,Alcazar-Felix Roberto J.³^ORCID,Shenkar Robert^ORCID,Lane Karen¹²^ORCID,McBee Nichole¹²,Treine Kevin¹²,Ostapkovich Noeleen¹²^ORCID,Wang Ying¹²,Thompson Richard¹²,Koenig James I.¹³^ORCID,Carroll Timothy¹⁴^ORCID,Hanley Daniel¹²^ORCID,Awad Issam³^ORCID

Affiliation:

1. Department of Neurology (K.D.F.), Mayo Clinic, Rochester, MN.

2. Center for Cerebrovascular Research, Department of Anesthesiology and Perioperative Care (H.K.), University of California San Francisco.

3. Neurovascular Surgery Program, Department of Neurological Surgery, University of Chicago Medicine and Biological Sciences, IL (S.H., S.K., R.G., J.L., A.S., R.J.A.-F., R.S., I.A.).

4. Department of Biostatistics (J.M.), Mayo Clinic, Rochester, MN.

5. Department of Neurology (M.T.), University of New Mexico, Albuquerque.

6. Department of Neurosurgery (J. Huang, J.F.), Johns Hopkins University Medical Institutions, Baltimore, MD.

7. Department of Radiology (J. Huston, Y.S.), Mayo Clinic, Rochester, MN.

8. Department of Neurosurgery (G.L.), Mayo Clinic, Rochester, MN.

9. Department of Radiology (R.S., B.H., M.M.), University of New Mexico, Albuquerque.

10. Department of Radiology, Johns Hopkins University, Baltimore, MD (H.I.S.).

11. Department of Radiology and Biomedical Imaging (J.N., J.M.L.), University of California San Francisco.

12. Brain Injury Outcomes Unit, Department of Neurology (K.L., N.M., K.T., N.O., Y.W., R.T., D.H.), Johns Hopkins University Medical Institutions, Baltimore, MD.

13. National Institute of Neurological Disorders and Stroke, Bethesda, MD (J.I.K.).

14. Department of Diagnostic Radiology, The University of Chicago Medicine and Biological Sciences, IL (T.C.).

Abstract

BACKGROUND: Cerebral cavernous malformation with symptomatic hemorrhage (SH) are targets for novel therapies. A multisite trial-readiness project ( https://www.clinicaltrials.gov ; Unique identifier: NCT03652181) aimed to identify clinical, imaging, and functional changes in these patients. METHODS: We enrolled adult cerebral cavernous malformation patients from 5 high-volume centers with SH within the prior year and no planned surgery. In addition to clinical and imaging review, we assessed baseline, 1- and 2-year National Institutes of Health Stroke Scale, modified Rankin Scale, European Quality of Life 5D-3 L, and patient-reported outcome-measurement information system, Version 2.0. SH and asymptomatic change rates were adjudicated. Changes in functional scores were assessed as a marker for hemorrhage. RESULTS: One hundred twenty-three, 102, and 69 patients completed baseline, 1- and 2-year clinical assessments, respectively. There were 21 SH during 178.3 patient years of follow-up (11.8% per patient year). At baseline, 62.6% and 95.1% of patients had a modified Rankin Scale score of 1 and National Institutes of Health Stroke Scale score of 0 to 4, respectively, which improved to 75.4% ( P =0.03) and 100% ( P =0.06) at 2 years. At baseline, 74.8% had at least one abnormal patient-reported outcome-measurement information system, Version 2.0 domain compared with 61.2% at 2 years ( P =0.004). The most common abnormal European Quality of Life 5D-3 L domains were pain (48.7%), anxiety (41.5%), and participation in usual activities (41.4%). Patients with prospective SH were more likely than those without SH to display functional decline in sleep, fatigue, and social function patient-reported outcome-measurement information system, Version 2.0 domains at 2 years. Other score changes did not differ significantly between groups at 2 years. The sensitivity of scores as an SH marker remained poor at the time interval assessed. CONCLUSIONS: We report SH rate, functional, and patient-reported outcomes in trial-eligible cerebral cavernous malformation with SH patients. Functional outcomes and patient-reported outcomes generally improved over 2 years. No score change was highly sensitive or specific for SH and could not be used as a primary end point in a trial.

Publisher

Ovid Technologies (Wolters Kluwer Health)

Subject

Advanced and Specialized Nursing,Cardiology and Cardiovascular Medicine,Neurology (clinical)

Reference20 articles.

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