Hart, Blaine
Mayo Clinic
Department of Radiology (R.S., B.H., M.M.), University of New Mexico, Albuquerque.
Citations 1
Trial Readiness of Cavernous Malformations With Symptomatic Hemorrhage, Part I: Event Rates and Clinical Outcome
Flemming, Kelly D. , Kim, Helen , Hage, Stephanie , Mandrekar, Jay , Kinkade, Serena , Torbey, Michel , Huang, Judy , Huston, John , Shu, Yunhong , Selwyn, Reed , Hart, Blaine L. , Feghali, James , Sair, Haris I. , Narvid, Jared , Lupo, Janine , Stadnik, Agnieszka , Lane, Karen , McBee, Nichole, Treine, Kevin, Ostapkovich, Noeleen , Wang, Ying , Thompson, Richard E. , Awad, Issam A. , Huston, John , Torbey, Michel , Huang, Judy , Shu, Yunhong , Lanzino, Giuseppe , Selwyn, Reed , Hart, Blaine , Mabray, Marc C , Feghali, James , Narvid, Jared , Alcazar-Felix, Roberto J , Shenkar, Robert , Lane, Karen , Ostapkovich, Noeleen , Koenig, James I , Carroll, Timothy , Hanley, Daniel F , Awad, Issam , Flemming, Kelly D , Kim, Helen , Girard, Romuald , Lanzino, Giuseppe , Mabray, Marc C. , Lee, Justine C. , Alcazar‐Félix, Roberto J. , Shenkar, Robert , Koenig, James I. , Hanley, Daniel F.
Stroke | December 22, 2023 | Volume: 55 | Issue: 1 | Pages: 22-30
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.