Abstract Details

Title Results from a 12-month Natural History Study of Patients with Cerebral Cavernous Malformations

Topic Cerebrovascular Disease and Interventional Neurology

Presentation(s) S41 - Sociodemographics of Stroke and Policy in Stroke Care (2:00 PM-2:12 PM)

Poster/Presentation
Number 006

Objective

To evaluate multifactorial disease progression in a diverse population of patients with cerebral cavernous malformations and to identify which factors are associated with a faster or slower progression of disease.

Background In order to facilitate therapeutic studies in CCM, it is necessary to: 1) understand the natural history of CCM; 2) predict which patients will have the fastest disease progression; and, 3) determine the performance metrics of outcome measures designed to measure disease burden overtime.

Design/Methods We conducted a 12-month prospective observational study of adults with CCM who were assessed at baseline, 6 months, and 12 months. Participants serially completed the regulatory-grade Cerebral Cavernous Malformation - Health Index (CCM-HI) and the SymptoMScreen at each time point. In addition, participants completed a survey preference questionnaire at baseline, and global impression of change questionnaires at 6 and 12 months. Minimal clinically important difference (MCID) values were assessed using anchor-based methodology. CCM-HI scores were analyzed by demographic subgroups and participants’ preferences for the CCM-HI versus the SymptoMScreen was determined.

Results Three-hundred and sixty-one adults with CCM representing 23 countries participated in this study. Participants with higher disability levels and younger age had a faster progression of disease in some CCM-HI subscales and males experienced a faster disease progression of headaches compared to females. The weighted average MCID of the CCM-HI total score (0-100) from baseline to 12 months was 2.5 points. Seventy-seven point five percent of our cohort preferred the CCM-HI over the SymptoMScreen as a clinical trial outcome measure.

Conclusions

Disease progression is slow in CCM but can be tracked using the CCM-HI. Select subgroups demonstrate a faster progression of disease as measured by CCM-HI subscales. CCM-HI performance metrics generated through this study can help facilitate the planning and interpretation of future therapeutic trials in CCM.

Authors/Disclosures
Chad R. Heatwole, MD, FAAN (University of Rochester Medical Center) PRESENTER	Dr. Heatwole has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Virginia Commonwealth University. Dr. Heatwole has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Legal Med. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurocrine. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Swan Bio. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Harmony. Dr. Heatwole has received personal compensation in the range of $0-$499 for serving as a Consultant for Iris. Dr. Heatwole has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Recursion. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity Biosciences. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lupin. Dr. Heatwole has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neurocrine. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for New York Central Mutual. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Penn Prop and Gas. The institution of Dr. Heatwole has received research support from Department of Defense. The institution of Dr. Heatwole has received research support from Novartis. The institution of Dr. Heatwole has received research support from MJFF. The institution of Dr. Heatwole has received research support from FARA. The institution of Dr. Heatwole has received research support from NIH. The institution of Dr. Heatwole has received research support from University of Miami. The institution of Dr. Heatwole has received research support from MDA. Dr. Heatwole has received intellectual property interests from a discovery or technology relating to health care.
Jamison Seabury (University of Rochester)	Jamison Seabury has nothing to disclose.
Anika Varma (Center for Health + Technology, University of Rochester)	Anika Varma has nothing to disclose.
Spencer Rosero (University of Rochester, Center for Health and Technology)	Spencer Rosero has nothing to disclose.
Charlotte Engebrecht (University of Rochester Center for Health + Technology)	Ms. Engebrecht has nothing to disclose.
Shaweta Khosa	Ms. Khosa has nothing to disclose.
Christina Shupe (Center for Health and Technology, University of Rochester)	Ms. Shupe has nothing to disclose.
Charlotte Irwin	Ms. Irwin has nothing to disclose.
John Heatwole (39465)	An immediate family member of John Heatwole has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for legal med. An immediate family member of John Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for neurocrine. An immediate family member of John Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for novartis. An immediate family member of John Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for swan bio. An immediate family member of John Heatwole has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for recursion. An immediate family member of John Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for sanofi. An immediate family member of John Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for lupin. An immediate family member of John Heatwole has received personal compensation in the range of $0-$499 for serving as a Consultant for vertex. The institution of an immediate family member of John Heatwole has received research support from dod. The institution of an immediate family member of John Heatwole has received research support from novartis. The institution of an immediate family member of John Heatwole has received research support from mjff. The institution of an immediate family member of John Heatwole has received research support from fara. The institution of an immediate family member of John Heatwole has received research support from nih. The institution of an immediate family member of John Heatwole has received research support from unviversity of miami. The institution of an immediate family member of John Heatwole has received research support from mda. An immediate family member of John Heatwole has received intellectual property interests from a discovery or technology relating to health care.
Christine Zizzi (University of Rochester Medical Center, Center for Health + Technology (CHeT))	No disclosure on file
Danae Alexandrou	Ms. Alexandrou has nothing to disclose.
Nuran Dilek (University of Rochester)	Nuran Dilek has nothing to disclose.
Roshawn Watson	No disclosure on file
Holly Blei	No disclosure on file
Cornelia Lee (Alliance to Cure Cavernous Malformation)	No disclosure on file
Jennifer Weinstein	Jennifer Weinstein has nothing to disclose.

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