Trial Outcomes & Findings for CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness (NCT NCT03652181)

NCT ID: NCT03652181

Last Updated: 2024-06-27

Results Overview

Number of new bleeds reported during the two year follow-up period in the cohort with CASH. We will assess the change in the number of new symptomatic hemorrhage from baseline to year 1 and year 1 to year 2 follow-up.

• Recruitment status: COMPLETED
• Target enrollment: 123 participants
• Primary outcome timeframe: 2 year
• Results posted on: 2024-06-27

Participant Flow

123 participants were enrolled and followed up for 2 years and all results are provided only for these participants.

Participant milestones

Measure	CASH (Cavernous Angiomas With Symptomatic Hemorrhage) The adjudicated definition of CASH (Cavernous Angiomas with Symptomatic Hemorrhage) requires diagnostic evidence of new lesional bleeding or hemorrhagic growth, in association with directly attributable symptoms.
Overall Study STARTED	123
Overall Study Completed Year 1	102
Overall Study Completed Year 2	69
Overall Study COMPLETED	123
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Data were missing for 2 participants

Baseline characteristics by cohort

Measure	CASH (Cavernous Angiomas With Symptomatic Hemorrhage) n=123 Participants The adjudicated definition of CASH (Cavernous Angiomas with Symptomatic Hemorrhage) requires diagnostic evidence of new lesional bleeding or hemorrhagic growth, in association with directly attributable symptoms.
Age, Continuous	43.9 Years STANDARD_DEVIATION 15.3 • n=123 Participants
Sex: Female, Male Female	75 Participants n=123 Participants
Sex: Female, Male Male	48 Participants n=123 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	28 Participants n=123 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	95 Participants n=123 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=123 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=123 Participants
Race (NIH/OMB) Asian	5 Participants n=123 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=123 Participants
Race (NIH/OMB) Black or African American	10 Participants n=123 Participants
Race (NIH/OMB) White	99 Participants n=123 Participants
Race (NIH/OMB) More than one race	5 Participants n=123 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=123 Participants
Familial cavernous malformation syndrome	48 Participants n=121 Participants • Data were missing for 2 participants
History of hypertension	35 Participants n=123 Participants
History of diabetes	6 Participants n=123 Participants
Tobacco use	11 Participants n=123 Participants
Obstructive sleep apnea	10 Participants n=111 Participants • Data were missing for 12 participants
Alcohol use	56 Participants n=123 Participants
Statin use	25 Participants n=123 Participants
Vitamin D supplementation	88 Participants n=123 Participants
Propranolol	7 Participants n=123 Participants
Birth control	2 Participants n=75 Participants • 75 participants were females for whom birth control could be assessed
Systolic blood pressure, mm Hg	122.7 mmHg STANDARD_DEVIATION 14.7 • n=115 Participants • Data were missing for 8 participants
Diastolic blood pressure, mm Hg	78.3 mmHg STANDARD_DEVIATION 10.0 • n=115 Participants • Data were missing for 8 participants
Body mass index	26.7 kg/m^2 STANDARD_DEVIATION 5.1 • n=123 Participants
Qualifying Symptomatic Hemorrhage (SH) location: Brainstem Brainstem	50 Participants n=123 Participants
Qualifying Symptomatic Hemorrhage (SH) location: Brainstem Thalamus	13 Participants n=123 Participants
Qualifying Symptomatic Hemorrhage (SH) location: Brainstem Lobar	35 Participants n=123 Participants
Qualifying Symptomatic Hemorrhage (SH) location: Brainstem Cerebellum	12 Participants n=123 Participants
Qualifying Symptomatic Hemorrhage (SH) location: Brainstem Other	13 Participants n=123 Participants

PRIMARY outcome

Timeframe: 2 year

Number of new bleeds reported during the two year follow-up period in the cohort with CASH. We will assess the change in the number of new symptomatic hemorrhage from baseline to year 1 and year 1 to year 2 follow-up.

Outcome measures

Measure	Baseline to Year 1 n=102 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=69 Participants Change assessed from year 1 to year 2	Year 2 Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Rate of Recurrent Symptomatic Hemorrhage During the Two Year Follow-up Period in Patients With CASH.	10 Number of new bleeds	11 Number of new bleeds	—

SECONDARY outcome

Timeframe: 2 years of follow-up

Change in QSM value (lesional iron content) will be presented as a mean value across all participants from baseline to year 1 and year 1 to year 2 follow-up MRIs

Outcome measures

Measure	Baseline to Year 1 n=93 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=56 Participants Change assessed from year 1 to year 2	Year 2 Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Percent Change in QSM Value (Lesional Iron Content)	16.33 percent change Standard Deviation 74.93	19.61 percent change Standard Deviation 70.96	—

SECONDARY outcome

Timeframe: 2 years of follow-up

Change in DCEQP value (vascular permeability) will be presented as a mean value across all participants from baseline to year 1 and year 1 to year 2 follow-up MRIs

Outcome measures

Measure	Baseline to Year 1 n=85 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=55 Participants Change assessed from year 1 to year 2	Year 2 Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Percent Change in Contrast-enhanced Quantitative Perfusion (DCEQP) Value (Vascular Permeability)	61.62 percent change Standard Deviation 248.8	52.45 percent change Standard Deviation 206.4	—

SECONDARY outcome

Timeframe: 2 years of follow-up

Population: At year 1, 5 participants with symptomatic hemorrhage were analyzed, and 97 without symptomatic hemorrhage totaling, 102 participants analyzed. At year 2, 7 participants with symptomatic hemorrhage were analyzed, and 62 without symptomatic hemorrhage, totaling 69 participants analyzed.

Compare the number of patients with MRS 2 or higher who had prospective symptomatic hemorrhage to those with MRS 2 or higher without prospective symptomatic hemorrhage from baseline to year 1 and year 1 to year 2 follow-up periods.

The mRS is a simple global measure of functional disability. Scores range from 0 (no symptoms) to 6 (death). An mRS score of 0 to 1 is considered a minimal clinical disability, and 0 to 2 is independent.

Outcome measures

Measure	Baseline to Year 1 n=102 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=69 Participants Change assessed from year 1 to year 2	Year 2 Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Compare the Number of Patients With MRS 2 or Higher Who Had Prospective Symptomatic Hemorrhage to Those With MRS 2 or Higher Without Prospective Symptomatic Hemorrhage During the 2 Year Follow-up Period. Symptomatic hemorrhage	1 Participants	2 Participants	—
Compare the Number of Patients With MRS 2 or Higher Who Had Prospective Symptomatic Hemorrhage to Those With MRS 2 or Higher Without Prospective Symptomatic Hemorrhage During the 2 Year Follow-up Period. No symptomatic hemorrhage	27 Participants	15 Participants	—

SECONDARY outcome

Timeframe: 2 years of follow-up

Percentage of patients with National Institutes of Health Stroke Scale (NIHSS) 0-4, 5-14 and 14+ from baseline to year 1 and year 1 to year 2 follow-up periods.

NIHSS values range from 0 to 42, with stroke severity categorized as mild (0 to 4), moderate (5 to 14), severe (15 to 24), and very severe (≥25). 4-point decline in ischemic stroke clinical trials typically measures functional decline.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=98 Participants Change assessed from year 1 to year 2	Year 2 n=61 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Percentage of Patients With National Institutes of Health Stroke Scale (NIHSS) 0-4, 5-14 and 14+ During the 2 Year Follow-up Period. NIHSS score 0-4	117 Participants	97 Participants	61 Participants
Percentage of Patients With National Institutes of Health Stroke Scale (NIHSS) 0-4, 5-14 and 14+ During the 2 Year Follow-up Period. NIHSS score 5-14	5 Participants	1 Participants	0 Participants
Percentage of Patients With National Institutes of Health Stroke Scale (NIHSS) 0-4, 5-14 and 14+ During the 2 Year Follow-up Period. NIHSS score 14+	1 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 2 years of follow-up

Median score of European Quality of Life Visual Analogue Scale (EQ-VAS) at baseline, year 1, year 2.

The EQ-VAS is a vertical visual analogue scale that takes values between 100 (best imaginable health) and 0 (worst imaginable health), on which patients provide a global assessment of their health.

Outcome measures

Measure	Baseline to Year 1 n=119 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=68 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Median Score of European Quality of Life Visual Analogue Scale (EQ-VAS) During the 2 Year Follow-up Period.	80 score on a scale Interval 9.0 to 100.0	80 score on a scale Interval 30.0 to 100.0	80 score on a scale Interval 35.0 to 100.0

SECONDARY outcome

Timeframe: 2 years of follow-up

Proportion of patients with no problems, mild problems, or severe problems in the "Mobility" domain of EQ-5D-3 L will be assessed at baseline, year 1, and year 2 follow-up periods.

EQ-5D-3 L is a generic tool for Patient Reported Outcomes (PRO) measurement that can assess patients' quality of life, irrespective of the disease and includes 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. There are 3 questions per domain where patients respond if they have no problems, mild, or severe problems.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=68 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Mobility" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. No problems	83 Participants	69 Participants	44 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Mobility" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Mild problems	40 Participants	33 Participants	24 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Mobility" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Severe Problems	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 2 years of follow-up

Proportion of patients with no problems, mild problems, or severe problems in the "Self-care" domain of EQ-5D-3 L will be assessed at baseline, year 1, and year 2 follow-up periods.

EQ-5D-3 L is a generic tool for Patient Reported Outcomes (PRO) measurement that can assess patients' quality of life, irrespective of the disease and includes 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. There are 3 questions per domain where patients respond if they have no problems, mild, or severe problems.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=68 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Self-care" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. No problems	111 Participants	95 Participants	63 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Self-care" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Mild problems	12 Participants	7 Participants	5 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Self-care" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Severe problems	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 2 years of follow-up

Proportion of patients with no problems, mild problems, or severe problems in the "Usual activities" domain of EQ-5D-3 L will be assessed at baseline, year 1, and year 2 follow-up periods.

EQ-5D-3 L is a generic tool for Patient Reported Outcomes (PRO) measurement that can assess patients' quality of life, irrespective of the disease and includes 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. There are 3 questions per domain where patients respond if they have no problems, mild, or severe problems.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=68 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Usual Activities" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. No problems	72 Participants	66 Participants	50 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Usual Activities" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Mild problems	50 Participants	34 Participants	17 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Usual Activities" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Severe problems	1 Participants	2 Participants	1 Participants

SECONDARY outcome

Timeframe: 2 years of follow-up

Proportion of patients with no problems, mild problems, or severe problems in the "Pain / Discomfort" domain of EQ-5D-3 L will be assessed at baseline, year 1, and year 2 follow-up periods.

EQ-5D-3 L is a generic tool for Patient Reported Outcomes (PRO) measurement that can assess patients' quality of life, irrespective of the disease and includes 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. There are 3 questions per domain where patients respond if they have no problems, mild, or severe problems.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=68 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Pain / Discomfort" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. No problems	63 Participants	63 Participants	48 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Pain / Discomfort" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Mild problems	51 Participants	36 Participants	19 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Pain / Discomfort" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Severe problems	9 Participants	3 Participants	1 Participants

SECONDARY outcome

Timeframe: 2 years of follow-up

Proportion of patients with no problems, mild problems, or severe problems in the "Anxiety / Depression" domain of EQ-5D-3 L will be assessed at baseline, year 1, and year 2 follow-up periods.

EQ-5D-3 L is a generic tool for Patient Reported Outcomes (PRO) measurement that can assess patients' quality of life, irrespective of the disease and includes 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. There are 3 questions per domain where patients respond if they have no problems, mild, or severe problems.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=68 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Anxiety / Depression" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. No problems	72 Participants	62 Participants	49 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Anxiety / Depression" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Mild problems	46 Participants	38 Participants	19 Participants
Proportion of Patients With No Problems, Mild Problems, or Severe Problems in the "Anxiety / Depression" Domain of European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3 L) During the 2 Year Follow-up Period. Severe problems	5 Participants	2 Participants	0 Participants

SECONDARY outcome

Timeframe: 2 years of follow-up

Median T-score for "Anxiety" domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) at baseline, year 1 and year 2.

PROMIS-29 (Version 2.0) is a generic health-related quality of life measure including 7 domains (depression, anxiety, physical function, pain interference, fatigue, sleep disturbance, and ability to participate in social roles and activities).12 Each domain contains questions ranked using a 5-point Likert scale. PROMIS-29 domain scores are converted to T scores and have been standardized to a reference population (mean, 50; SD, 10). Clinically meaningful change is considered to be at least half the SD (5 points). Higher T-score indicates better outcome.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=67 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Median T-score for "Anxiety" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.	53.7 T-score Interval 40.3 to 75.4	51.2 T-score Interval 40.3 to 69.3	51.2 T-score Interval 40.3 to 71.2

SECONDARY outcome

Timeframe: 2 years of follow-up

Median T-score for "Depression" domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) at baseline, year 1, and year 2.

PROMIS-29 (Version 2.0) is a generic health-related quality of life measure including 7 domains (depression, anxiety, physical function, pain interference, fatigue, sleep disturbance, and ability to participate in social roles and activities).12 Each domain contains questions ranked using a 5-point Likert scale. PROMIS-29 domain scores are converted to T scores and have been standardized to a reference population (mean, 50; SD, 10). Clinically meaningful change is considered to be at least half the SD (5 points). Higher T-score indicates better outcome.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=67 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Median T-score for "Depression" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.	41.0 T-score Interval 41.0 to 71.2	41.0 T-score Interval 41.0 to 63.9	41.0 T-score Interval 41.0 to 65.7

SECONDARY outcome

Timeframe: 2 years of follow-up

Median T-score for "Fatigue" domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) at baseline, year 1, and year 2.

PROMIS-29 (Version 2.0) is a generic health-related quality of life measure including 7 domains (depression, anxiety, physical function, pain interference, fatigue, sleep disturbance, and ability to participate in social roles and activities).12 Each domain contains questions ranked using a 5-point Likert scale. PROMIS-29 domain scores are converted to T scores and have been standardized to a reference population (mean, 50; SD, 10). Clinically meaningful change is considered to be at least half the SD (5 points). Higher T-score indicates better outcome.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=67 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Median T-score for "Fatigue" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.	48.6 T-score Interval 33.7 to 75.8	48.6 T-score Interval 33.7 to 75.8	46.0 T-score Interval 33.7 to 75.8

SECONDARY outcome

Timeframe: 2 years of follow-up

Median T-score for "Pain" domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) at baseline, year 1 and year 2.

PROMIS-29 (Version 2.0) is a generic health-related quality of life measure including 7 domains (depression, anxiety, physical function, pain interference, fatigue, sleep disturbance, and ability to participate in social roles and activities).12 Each domain contains questions ranked using a 5-point Likert scale. PROMIS-29 domain scores are converted to T scores and have been standardized to a reference population (mean, 50; SD, 10). Clinically meaningful change is considered to be at least half the SD (5 points). Higher T-score indicates better outcome.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=67 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Median T-score for "Pain" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.	49.6 T-score Interval 41.6 to 75.6	49.6 T-score Interval 41.6 to 75.6	41.6 T-score Interval 41.6 to 71.6

SECONDARY outcome

Timeframe: 2 years of follow-up

Median T-score for "Sleep Disturbance" domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) at baseline, year 1 and year 2.

PROMIS-29 (Version 2.0) is a generic health-related quality of life measure including 7 domains (depression, anxiety, physical function, pain interference, fatigue, sleep disturbance, and ability to participate in social roles and activities).12 Each domain contains questions ranked using a 5-point Likert scale. PROMIS-29 domain scores are converted to T scores and have been standardized to a reference population (mean, 50; SD, 10). Clinically meaningful change is considered to be at least half the SD (5 points). Higher T-score indicates better outcome.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=67 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Median T-score for "Sleep Disturbance" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.	50.5 T-score Interval 43.8 to 61.7	50.5 T-score Interval 43.8 to 63.8	50.5 T-score Interval 43.8 to 63.8

SECONDARY outcome

Timeframe: 2 years of follow-up

Median T-score for "Social" domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) at baseline, year 1 and year 2.

PROMIS-29 (Version 2.0) is a generic health-related quality of life measure including 7 domains (anxiety, depression, fatigue, pain interference, sleep disturbance, and ability to participate in social roles and activities, and physical function).12 Each domain contains questions ranked using a 5-point Likert scale. PROMIS-29 domain scores are converted to T scores and have been standardized to a reference population (mean, 50; SD, 10). Clinically meaningful change is considered to be at least half the SD (5 points). Higher T-score indicates better outcome.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=67 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Median T-score for "Social" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.	51.9 T-score Interval 27.5 to 64.2	51.9 T-score Interval 27.5 to 64.2	64.2 T-score Interval 34.0 to 64.2

SECONDARY outcome

Timeframe: 2 years of follow-up

Median T-score for "Physical Function" domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) at baseline, year 1 and year 2.

PROMIS-29 (Version 2.0) is a generic health-related quality of life measure including 7 domains (anxiety, depression, fatigue, pain interference, sleep disturbance, and ability to participate in social roles and activities, and physical function).12 Each domain contains questions ranked using a 5-point Likert scale. PROMIS-29 domain scores are converted to T scores and have been standardized to a reference population (mean, 50; SD, 10). Clinically meaningful change is considered to be at least half the SD (5 points). Higher T-score indicates better outcome.

Outcome measures

Measure	Baseline to Year 1 n=123 Participants Change assessed from baseline to Year 1. A total of 102 participants completed year 1 follow-up.	Year 1 to Year 2 n=102 Participants Change assessed from year 1 to year 2	Year 2 n=67 Participants Numbers were assessed at year 2. A total of 61 participants completed year 2 follow-up.
Median T-score for "Physical Function" Domain of Patient-Reported Outcome Measurement Information System (PROMIS-29) During the 2 Year Follow-up Period.	56.9 T-score Interval 22.9 to 56.9	56.9 T-score Interval 26.9 to 56.9	56.9 T-score Interval 30.7 to 56.9

Adverse Events

CASH (Cavernous Angiomas With Symptomatic Hemorrhage)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Agnieszka Stadnik

University of Chicago, Medicine and Biological Sciences Division

Phone: (773) 702-8996

Email: astadnik@bsd.uchicago.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place