Patient Reported Outcomes by Location in Patients with Cavernous Malformation (P6-5.012)

Abstract

<h3>Objective:</h3> To describe differences in patient reported outcomes between patients with cavernous malformations (CM) of different locations. <h3>Background:</h3> Little is known about patient reported outcomes in patients with CM. <h3>Design/Methods:</h3> A database of adults with CM has been maintained since January 1, 2015. Annual surveys are sent to patients for five years after diagnosis, followed by biennial surveys indefinitely. PROMIS-29 profiles, which assess seven health domains by self-report, have been collected since 2018. T-scores standardize raw scores to the general population with a mean of 50 and standard deviation (SD) of 10. We assessed the T-scores and proportion of abnormal scores in each domain by CM location at the time of last follow up (LFU). <h3>Results:</h3> Of the 382 patients enrolled as of June 1, 2022, 267 had available PROMIS-29 data. The cohort was 61.8% female with an average age at the time of LFU of 54.0. Median time from diagnosis to LFU was 5.3 years (range, 1.0 to 38.1). 18.7% were familial and 81.3% sporadic. 40.1% were cortical, 19.5% subcortical, 25.1% brainstem, 6.4% cerebellum, and 7.9% spinal cord, with 1.1% in other locations. 43.9% of patients were abnormal in at least one of seven domains (&gt;1 SD). The most commonly affected domains were pain (18.4%), anxiety (18.1%), physical function (16.5%), and fatigue (15.8%). Patients with brainstem or spinal cord CM reported significantly worse physical functional than their counterparts with CM in other locations (p&lt;0.0001). Patients with brainstem or spinal cord CM had a median T-score of 48.0 (range, 22.9 to 56.9) compared to a median of 56.9 for CM in other locations (range, 29.1 to 56.9). Other domains did not differ significantly by location. <h3>Conclusions:</h3> Patients with brainstem or spinal cord CM report decreased physical functionality relative to patients with other CM locations but do not score significantly different in other PROMIS domains. <b>Disclosure:</b> Dr. Griffin has nothing to disclose. Dr. Lanzino has nothing to disclose. Dr. Flemming has nothing to disclose.