Familial Amyloid Cardiomyopathy, Senile Systemic Amyloidosis (SSA), Transthyretin Cardiomyopathy (TTR-CM), Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)
Conditions
Keywords
Survey, burden of illness, caregivers, TTR-FAP, TTR-CM, familial amyloid polyneuropathy, observational, transthyretin familial amyloidosis
Brief summary
This study is an online (web-based) or paper-based survey for patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) and caregivers. The results will be used to describe the emotional, physical, and financial impact of having TTR-FAP or caring for someone who has the disease.
Detailed description
Convenience sample of patients and caregivers recruited through patient advocacy groups. Convenience sample.
Interventions
OTHERNo drug
No drug.
Sponsors
Pfizer
Study design
Observational model
OTHER
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* Adults (ages 18-85 years inclusive) diagnosed with TTR-FAP or TTR-CM and experiencing symptoms or currently providing care for a patient with TTR-FAP or TTR-FAP
Exclusion criteria
* Caregivers who formally care for a TTR-FAP or TTR-CM patient as part of their job description and receive payment for their services.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Demographical Characteristics of Participants | Baseline (Day 1) | Main characteristics included were education level and employment status which were asked from all participants and caregivers. Type of job (full-time, part-time) was asked only from those participants and caregivers who provided their employment status as employed. Those who were unemployed reported their cause of unemployment, whether it was due to ATTR or not. |
| Disease Characteristics of Participants: Disease Duration | Baseline (Day 1) | Duration of disease was defined as the time from diagnosis of disease until baseline visit. This outcome measure was planned to be assessed for reporting arm of participants diagnosed with ATTR. |
| Disease Characteristics of Participants: Mutation Type | Baseline (Day 1) | Genetic mutation leads to misfolding of protein transthyretin (TTR) which results in ATTR. In this outcome, number of participants with each type of resulted mutation type (Val30Met, wild type TTR, Phe64Leu, Ser77Tyr, Thr60Ala or other than these) were reported. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR. |
| Disease Characteristics of Participants: Liver Transplantation Status | Baseline (Day 1) | TTR protein is primarily synthesized in the liver. Liver transplantation was considered as one of the measure to eliminate the main source of variant TTR. In the study, participants who were diagnosed with ATTR were asked for their liver transplantation status (whether they had transplantation or not). In this outcome measure, number of participants with liver transplant status were reported. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR. |
| Disease Characteristics of Participants: Number of Participants With Family History of ATTR | Baseline (Day 1) | Family history of participants diagnosed with ATTR was assessed to determine whether family history of ATTR was a significant risk factor for ATTR or not. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR. |
| Disease Characteristics of Participants: Mobility Status | Baseline (Day 1) | Mobility, i.e., ability to walk was assessed as a part of loss of functioning in the participants diagnosed with ATTR. In this outcome, number of participants with their different mobility status along with the use of mobility aids (able to walk normally, some problems with feet but able to walk without difficulty, some difficulty walking but can walk without help, confined to bed all the time, need 1 cane or crutch to walk, need 2 canes/crutches or a walker to walk) were reported. |
| 12-Item Short-Form Health Survey (SF-12) Scores | Baseline (Day 1) | SF-12 was a patient reported outcome survey that represented overall health status by measuring 8 health-related aspects of an individual: Body pain, general mental health, perception of general health, physical functioning, role limitations caused by mental condition, role limitations caused by a physical condition, social functioning, and vitality. The score range for each of the 8 health aspects was from 0 (poor health) to 100 (better health), higher scores indicating good health condition. Responses on the SF-12 were also used to calculate 2 summary scores: Physical component score (PCS) and mental component score (MCS). The score range for each of these 2 summary scores was from 0 (poor health) to 100 (better health), where 100 indicated good health condition. |
| Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores | Baseline (Day 1) | HADS: participant rated 14-item questionnaire with 2 subscales; HADS-anxiety scale (HADS-A) and HADS-depression scale (HADS-D). HADS-A assesses state of generalized anxiety (anxious mood, restlessness, anxious thoughts, panic attacks); HADS-D assesses state of lost interest and diminished pleasure response (lowering of hedonic tone). Each subscale comprised of 7 items and the participant responds as to how each item applies to him/her over the past week prior to baseline visit, on 4-point response scale. Separate scores were calculated for anxiety and depression with score ranges from 0 (no presence of anxiety or depression) to 3 (severe feeling of anxiety or depression). Total score range was from 0 to 21 for each subscale; higher score indicating greater severity of anxiety and depression symptoms. |
| Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score | Baseline (Day 1) | EQ-5D-3L: participant rated questionnaire to assess generic health status in two parts: single utility score and visual analog scale. For utility score, participants rated their current health state on 5 dimensions: mobility, self-care, usual activities, pain and discomfort, and anxiety and depression with each dimension having three levels of function: 1 indicates no problem; 2 indicates some problem; 3 indicates extreme problem. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total score range of 0.05 to 1.00; higher scores indicating a better health state. |
| Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score | Baseline (Day 1) | EQ-5D: participant rated questionnaire to assess generic health status in two parts: single utility score and visual analog scale. The VAS component rated the current health state on a scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state); higher scores indicating a better health state. |
| Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed | Baseline (Day 1) | The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asked about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Percentage of work time missed of participants were recorded and reported. |
| Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working | Baseline (Day 1) | The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asks about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity. |
| Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment | Baseline (Day 1) | The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asked about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity. |
| Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment | Baseline (Day 1) | The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asks about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity. |
| Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Baseline (Day 1) | Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care). |
| Healthcare Resource Use Survey: Number of Hospitalizations | Baseline (Day 1) | Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care). |
| Healthcare Resource Use Survey: Number of Emergency Care Visits | Baseline (Day 1) | Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care). |
| Healthcare and Resource Use Survey: Symptomatic Treatment of Participants | Baseline (Day 1) | Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs. Number of participants (diagnosed with ATTR) who visited non-medical practitioners (nutrition consultant/dietician, chiropractor, acupuncturist, massage therapist, occupational therapist or other than these) for symptomatic treatments were reported. |
| Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits | Baseline (Day 1) | Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs. Number of visits of participants (diagnosed with ATTR) who visited non-medical practitioners (nutrition consultant/dietician, chiropractor, acupuncturist, massage therapist, occupational therapist or other than these) for symptomatic treatments were reported. |
| Healthcare Resource Use Survey: Out-of-Pocket Costs | Baseline (Day 1) | Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (expenditure on nutritional supplements, non-prescription medications and travel to receive medical care). |
| Participants Pain Score | Baseline (Day 1) | Participants diagnosed with ATTR rated their pain due to the health condition based on 3 items: pain right now, average pain in the past week, and worst pain in the past week prior to baseline visit. All 3 items were rated on an 11-point numeric rating scale ranging from 0=none to 10=severe pain, where higher scores indicated severe pain. |
| Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores | Baseline (Day 1) | Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of neuropathy on the quality of life of participants diagnosed with ATTR. Scoring was based on 35 questions that yield a TQOL as well as 5 subscale scores: activities of daily living, large fiber neuropathy/physical functioning, small fiber neuropathy, autonomic neuropathy, and symptoms. TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life. This outcome measure was planned to be analyzed only for the reporting arm of participants diagnosed with ATTR. |
| Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores | Baseline (Day 1) | Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of neuropathy on the quality of life of participants diagnosed with ATTR. It was summarized in 5 domains: (1) Activities of daily living (score ranges from 0 to 20, where higher score=worse quality of life); (2) Large fiber neuropathy/physical functioning (score ranges from -2 to 58, where higher score=worse condition); (3) Small fiber neuropathy (score ranges from 0 to 16, where higher score=worse condition); (4) Autonomic neuropathy (score ranges from 0 to 12, where higher score=worse condition) and (5) Symptoms (score ranges from 0 to 32, where higher score=less symptoms of disease). Total possible score range= -2 to 138, where higher score=worse quality of life. This outcome measure was analyzed only for the participants diagnosed with ATTR. |
| Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Baseline (Day 1) | KCCQ was a 23-item participant-completed questionnaire that assessed health status and health-related quality of life (HRQoL) in participants with heart failure. It was quantified in to following 10 summary scores: physical limitation, symptom frequency, symptom severity, and symptom stability, total symptoms, quality of life, social interference, self-efficacy, overall summary and clinical summary. Each summary score was scaled to range from 0 (minimum) to 100 (maximum), with higher scores representing greater disability. Total score ranged from 0 to 100, where higher scores indicated better functioning, fewer symptoms, and better disease specific quality of life. |
| Zarit Burden Interview (ZBI): Total Scores | Baseline (Day 1) | ZBI was a 22-item questionnaire designed to evaluate five broad aspects of caregiver burden in terms of personal and role strain associated with caregiving. Five broad aspects were: burden in the relationship, emotional well-being, social and family life, finances, loss of control over one's life. Each item rated on a 5 point scale anchored at 0 for never and 4 for nearly always. Total score ranges from 0-88 with higher scores indicating increased burden of care. |
| Zarit Burden Interview: Subscale Scores | Baseline (Day 1) | A questionnaire designed to evaluate aspects of caregiver burden in terms of personal and role strain associated with caregiving. Total score of ZBI scale ranges from 0-88 with higher scores indicating increased burden of care. Five subscale scores were also calculated: (1) Burden in the relationship (consist of 6-items, ranging from 0 to 24 where higher scores indicating increased burden in relationship); (2) Emotional well-being (consisting of 7-items, ranging from 0 to 28 where higher scores indicating worse condition; (3) Social and family life (consisting of 4-items, ranging from 0 to 16 where higher scores indicating worse life condition); (4) Finances (consisting of a single item, scored from 0 to 4 where higher scores indicating worse financial condition); and (5) Loss of control over one's life (consisting of 4-items, ranging from 0 to 16 where higher scores indicating worse control over life). |
| Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR | Baseline (Day 1) | Caregivers completed a series of questions related to the number of hours per week spent on providing care and support to the participants diagnosed with ATTR. |
| Caregiver Burden Items Assessment: Work Time Lost | Baseline (Day 1) | Caregivers completed a series of questions related to the loss in their working time while providing care and support to the participants diagnosed with ATTR. |
| Caregiver Burden Items Assessment: Total Cost | Baseline (Day 1) | Caregivers completed a series of questions related to the total cost spent on providing healthcare support to participants diagnosed with ATTR. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Participants Diagnosed With ATTR Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey. | 60 |
| Caregivers Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not. | 32 |
| Total | 92 |
Baseline characteristics
| Characteristic | Participants Diagnosed With ATTR | Caregivers | Total |
|---|---|---|---|
| Age, Continuous | 58.4 years STANDARD_DEVIATION 12 | 55.9 years STANDARD_DEVIATION 12.8 | 57.5 years STANDARD_DEVIATION 12.2 |
| Sex: Female, Male Female | 13 Participants | 22 Participants | 35 Participants |
| Sex: Female, Male Male | 47 Participants | 10 Participants | 57 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 0 | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 | 0 / 0 |
Outcome results
Primary
12-Item Short-Form Health Survey (SF-12) Scores
SF-12 was a patient reported outcome survey that represented overall health status by measuring 8 health-related aspects of an individual: Body pain, general mental health, perception of general health, physical functioning, role limitations caused by mental condition, role limitations caused by a physical condition, social functioning, and vitality. The score range for each of the 8 health aspects was from 0 (poor health) to 100 (better health), higher scores indicating good health condition. Responses on the SF-12 were also used to calculate 2 summary scores: Physical component score (PCS) and mental component score (MCS). The score range for each of these 2 summary scores was from 0 (poor health) to 100 (better health), where 100 indicated good health condition.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific component for each arm respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Physical Health Summary (n= 56, 28) | 33.6 units on a scale | Standard Deviation 11.5 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Mental Health Summary (n= 56, 28) | 47.1 units on a scale | Standard Deviation 10.8 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Physical Functioning (n= 58, 29) | 32.2 units on a scale | Standard Deviation 11 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Role-Physical (n= 56, 28) | 33.7 units on a scale | Standard Deviation 10.7 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Bodily Pain (n= 57, 29) | 43.0 units on a scale | Standard Deviation 13.3 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | General Health (n= 59, 29) | 37.2 units on a scale | Standard Deviation 12 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Vitality (n= 56, 29) | 41.1 units on a scale | Standard Deviation 10.6 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Social Functioning (n= 57, 29) | 41.0 units on a scale | Standard Deviation 11.6 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Role-Emotional (n= 57, 29) | 39.5 units on a scale | Standard Deviation 13.9 |
| Participants Diagnosed With ATTR | 12-Item Short-Form Health Survey (SF-12) Scores | Mental Health (n= 57, 29) | 48.2 units on a scale | Standard Deviation 10.2 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Social Functioning (n= 57, 29) | 39.5 units on a scale | Standard Deviation 13.8 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Physical Health Summary (n= 56, 28) | 42.2 units on a scale | Standard Deviation 13.7 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | General Health (n= 59, 29) | 39.8 units on a scale | Standard Deviation 13.5 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Mental Health Summary (n= 56, 28) | 39.3 units on a scale | Standard Deviation 11.1 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Mental Health (n= 57, 29) | 41.8 units on a scale | Standard Deviation 11.4 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Physical Functioning (n= 58, 29) | 43.1 units on a scale | Standard Deviation 13.7 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Vitality (n= 56, 29) | 45.7 units on a scale | Standard Deviation 11.5 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Role-Physical (n= 56, 28) | 39.1 units on a scale | Standard Deviation 13.1 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Role-Emotional (n= 57, 29) | 34.3 units on a scale | Standard Deviation 13.6 |
| Caregivers | 12-Item Short-Form Health Survey (SF-12) Scores | Bodily Pain (n= 57, 29) | 39.5 units on a scale | Standard Deviation 15.3 |
Primary
Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR
Caregivers completed a series of questions related to the number of hours per week spent on providing care and support to the participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Participants Diagnosed With ATTR | Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR | 72 hours per week |
Primary
Caregiver Burden Items Assessment: Total Cost
Caregivers completed a series of questions related to the total cost spent on providing healthcare support to participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Caregiver Burden Items Assessment: Total Cost | 9313.0 dollars | Standard Deviation 33226.1 |
Primary
Caregiver Burden Items Assessment: Work Time Lost
Caregivers completed a series of questions related to the loss in their working time while providing care and support to the participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Participants Diagnosed With ATTR | Caregiver Burden Items Assessment: Work Time Lost | 22 hours |
Primary
Demographical Characteristics of Participants
Main characteristics included were education level and employment status which were asked from all participants and caregivers. Type of job (full-time, part-time) was asked only from those participants and caregivers who provided their employment status as employed. Those who were unemployed reported their cause of unemployment, whether it was due to ATTR or not.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category for each arm respectively.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Employment Status:Not Currently Employed (n=60,32) | 39 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Unemployed: Not Asked (n= 39, 19) | 0 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Employment Type: Part-Time (n= 20, 11) | 9 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Education Level: Lower Than High School (n=60,32) | 6 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Employment Status: Not Asked (n= 60, 32) | 0 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Education Level: High School/Equivalent(n=60,32) | 8 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Unemployed: Due to ATTR (n= 39, 19) | 25 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Education Level: Associate Degree(n=60,32) | 12 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Employment Status: Skipped (n= 60, 32) | 1 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Education Level: College Degree (n= 60, 32) | 14 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Unemployed: Not due to ATTR (n= 39, 19) | 13 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Education Level: Professional or Graduate(n=60,32) | 20 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Employment Type: Full-Time (n= 20, 11) | 11 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Education Level: Skipped (n= 60, 32) | 0 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Unemployed: Skipped (n= 39, 19) | 1 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Education Level: Not Asked (n= 60, 32) | 0 participants |
| Participants Diagnosed With ATTR | Demographical Characteristics of Participants | Employment Status: Currently Employed (n= 60, 32) | 20 participants |
| Caregivers | Demographical Characteristics of Participants | Education Level: Not Asked (n= 60, 32) | 1 participants |
| Caregivers | Demographical Characteristics of Participants | Employment Status: Currently Employed (n= 60, 32) | 11 participants |
| Caregivers | Demographical Characteristics of Participants | Employment Status:Not Currently Employed (n=60,32) | 19 participants |
| Caregivers | Demographical Characteristics of Participants | Employment Status: Skipped (n= 60, 32) | 1 participants |
| Caregivers | Demographical Characteristics of Participants | Employment Status: Not Asked (n= 60, 32) | 1 participants |
| Caregivers | Demographical Characteristics of Participants | Employment Type: Full-Time (n= 20, 11) | 9 participants |
| Caregivers | Demographical Characteristics of Participants | Employment Type: Part-Time (n= 20, 11) | 2 participants |
| Caregivers | Demographical Characteristics of Participants | Unemployed: Due to ATTR (n= 39, 19) | 0 participants |
| Caregivers | Demographical Characteristics of Participants | Unemployed: Not due to ATTR (n= 39, 19) | 0 participants |
| Caregivers | Demographical Characteristics of Participants | Unemployed: Skipped (n= 39, 19) | 0 participants |
| Caregivers | Demographical Characteristics of Participants | Unemployed: Not Asked (n= 39, 19) | 19 participants |
| Caregivers | Demographical Characteristics of Participants | Education Level: Lower Than High School (n=60,32) | 3 participants |
| Caregivers | Demographical Characteristics of Participants | Education Level: High School/Equivalent(n=60,32) | 6 participants |
| Caregivers | Demographical Characteristics of Participants | Education Level: Associate Degree(n=60,32) | 3 participants |
| Caregivers | Demographical Characteristics of Participants | Education Level: College Degree (n= 60, 32) | 10 participants |
| Caregivers | Demographical Characteristics of Participants | Education Level: Professional or Graduate(n=60,32) | 7 participants |
| Caregivers | Demographical Characteristics of Participants | Education Level: Skipped (n= 60, 32) | 2 participants |
Primary
Disease Characteristics of Participants: Disease Duration
Duration of disease was defined as the time from diagnosis of disease until baseline visit. This outcome measure was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey.
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Disease Duration | 5.6 years | Full Range 4 |
Primary
Disease Characteristics of Participants: Liver Transplantation Status
TTR protein is primarily synthesized in the liver. Liver transplantation was considered as one of the measure to eliminate the main source of variant TTR. In the study, participants who were diagnosed with ATTR were asked for their liver transplantation status (whether they had transplantation or not). In this outcome measure, number of participants with liver transplant status were reported. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Liver Transplantation Status | With Liver Transplantation | 31 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Liver Transplantation Status | No Liver Transplantation | 29 participants |
Primary
Disease Characteristics of Participants: Mobility Status
Mobility, i.e., ability to walk was assessed as a part of loss of functioning in the participants diagnosed with ATTR. In this outcome, number of participants with their different mobility status along with the use of mobility aids (able to walk normally, some problems with feet but able to walk without difficulty, some difficulty walking but can walk without help, confined to bed all the time, need 1 cane or crutch to walk, need 2 canes/crutches or a walker to walk) were reported.
Time frame: Baseline (Day 1)
Population: All participants who were included in the study and completed the survey.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mobility Status | Able to Walk Normally | 15 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mobility Status | Some Problems: Able to Walk Without Difficulty | 16 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mobility Status | Some Difficulty: Can Walk Without Help | 14 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mobility Status | Need 1 Cane or Crutch to Walk | 6 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mobility Status | Need 2 Canes/Crutches or a Walker to Walk | 9 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mobility Status | Confined to bed all the Time | 0 participants |
Primary
Disease Characteristics of Participants: Mutation Type
Genetic mutation leads to misfolding of protein transthyretin (TTR) which results in ATTR. In this outcome, number of participants with each type of resulted mutation type (Val30Met, wild type TTR, Phe64Leu, Ser77Tyr, Thr60Ala or other than these) were reported. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mutation Type | Val30Met | 18 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mutation Type | Wild Type TTR | 2 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mutation Type | Phe64Leu | 4 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mutation Type | Ser77Tyr | 3 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mutation Type | Thr60Ala | 11 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mutation Type | Other | 5 participants |
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Mutation Type | Unknown | 17 participants |
Primary
Disease Characteristics of Participants: Number of Participants With Family History of ATTR
Family history of participants diagnosed with ATTR was assessed to determine whether family history of ATTR was a significant risk factor for ATTR or not. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Participants Diagnosed With ATTR | Disease Characteristics of Participants: Number of Participants With Family History of ATTR | 51 participants |
Primary
Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score
EQ-5D-3L: participant rated questionnaire to assess generic health status in two parts: single utility score and visual analog scale. For utility score, participants rated their current health state on 5 dimensions: mobility, self-care, usual activities, pain and discomfort, and anxiety and depression with each dimension having three levels of function: 1 indicates no problem; 2 indicates some problem; 3 indicates extreme problem. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total score range of 0.05 to 1.00; higher scores indicating a better health state.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score | 0.74 units on a scale | Standard Deviation 0.2 |
| Caregivers | Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score | 0.65 units on a scale | Standard Deviation 0.33 |
Primary
Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score
EQ-5D: participant rated questionnaire to assess generic health status in two parts: single utility score and visual analog scale. The VAS component rated the current health state on a scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state); higher scores indicating a better health state.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score | 58.7 units on a scale | Standard Deviation 19.8 |
| Caregivers | Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score | 64.2 units on a scale | Standard Deviation 28.1 |
Primary
Healthcare and Resource Use Survey: Symptomatic Treatment of Participants
Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs. Number of participants (diagnosed with ATTR) who visited non-medical practitioners (nutrition consultant/dietician, chiropractor, acupuncturist, massage therapist, occupational therapist or other than these) for symptomatic treatments were reported.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Participants Diagnosed With ATTR | Healthcare and Resource Use Survey: Symptomatic Treatment of Participants | 12 participants |
Primary
Healthcare Resource Use Survey: Number of Emergency Care Visits
Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Emergency Care Visits | 1.3 emergency care visits | Standard Deviation 0.9 |
| Caregivers | Healthcare Resource Use Survey: Number of Emergency Care Visits | 2.8 emergency care visits | Standard Deviation 1.3 |
Primary
Healthcare Resource Use Survey: Number of Hospitalizations
Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Hospitalizations | 2.1 hospitalization visits | Standard Deviation 2 |
| Caregivers | Healthcare Resource Use Survey: Number of Hospitalizations | 3.5 hospitalization visits | Standard Deviation 1.9 |
Primary
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category for each arm respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Liver Transplant Surgeon (n=6, 5) | 1.2 outpatient visits | Standard Deviation 0.4 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Cardiologist (n=17, 9) | 2.2 outpatient visits | Standard Deviation 2.7 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Nurse/Nurse Practitioner (n=9, 5) | 4.4 outpatient visits | Standard Deviation 5.6 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Neurologist (n=12, 6) | 1.4 outpatient visits | Standard Deviation 0.8 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Urologist (n=9, 1) | 1.9 outpatient visits | Standard Deviation 1.3 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Gastroenterologist (n=9, 3) | 1.3 outpatient visits | Standard Deviation 0.5 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Ophthalmology (n=5, 1) | 1.0 outpatient visits | Standard Deviation 0 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Primary Care Provider/Family Doctor (n=27, 12) | 2.0 outpatient visits | Standard Deviation 1.5 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Other (n=18, 6) | 2.2 outpatient visits | Standard Deviation 2.8 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Hematologist (n=5, 4) | 2.8 outpatient visits | Standard Deviation 0.8 |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Other (n=18, 6) | 1.0 outpatient visits | Standard Deviation 0 |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Primary Care Provider/Family Doctor (n=27, 12) | 1.8 outpatient visits | Standard Deviation 1.5 |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Neurologist (n=12, 6) | 1.2 outpatient visits | Standard Deviation 0.4 |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Cardiologist (n=17, 9) | 1.1 outpatient visits | Standard Deviation 0.3 |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Gastroenterologist (n=9, 3) | 1.3 outpatient visits | Standard Deviation 0.6 |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Liver Transplant Surgeon (n=6, 5) | 1.0 outpatient visits | Standard Deviation 0 |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Nurse/Nurse Practitioner (n=9, 5) | 2.6 outpatient visits | Standard Deviation 2.2 |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Urologist (n=9, 1) | 2.0 outpatient visits | — |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Ophthalmology (n=5, 1) | 2.0 outpatient visits | — |
| Caregivers | Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers | Hematologist (n=5, 4) | 1.8 outpatient visits | Standard Deviation 1.5 |
Primary
Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs. Number of visits of participants (diagnosed with ATTR) who visited non-medical practitioners (nutrition consultant/dietician, chiropractor, acupuncturist, massage therapist, occupational therapist or other than these) for symptomatic treatments were reported.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure and 'n' signifies those participants who were evaluable for specific category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits | Nutrition Consultant/Dietitian (n=2) | 4.0 symptomatic treatment visits | Standard Deviation 2.8 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits | Chiropractor (n=1) | 1.0 symptomatic treatment visits | — |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits | Acupuncturist (n=3) | 3.7 symptomatic treatment visits | Standard Deviation 2.5 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits | Massage Therapist (n=5) | 3.0 symptomatic treatment visits | Standard Deviation 1.9 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits | Occupational Therapist (n=2) | 3.5 symptomatic treatment visits | Standard Deviation 3.5 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits | Other (n=3) | 2.0 symptomatic treatment visits | Standard Deviation 1 |
Primary
Healthcare Resource Use Survey: Out-of-Pocket Costs
Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (expenditure on nutritional supplements, non-prescription medications and travel to receive medical care).
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category.
| Arm | Measure | Group | Value (MEDIAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Out-of-Pocket Costs | Nutritional Supplements (n=13) | 150 dollars | Full Range 121 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Out-of-Pocket Costs | Non-Prescription Medications (n=46) | 28 dollars | Full Range 76 |
| Participants Diagnosed With ATTR | Healthcare Resource Use Survey: Out-of-Pocket Costs | Travel to Receive Medical Care (n=45) | 30 dollars | Full Range 823 |
Primary
Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores
HADS: participant rated 14-item questionnaire with 2 subscales; HADS-anxiety scale (HADS-A) and HADS-depression scale (HADS-D). HADS-A assesses state of generalized anxiety (anxious mood, restlessness, anxious thoughts, panic attacks); HADS-D assesses state of lost interest and diminished pleasure response (lowering of hedonic tone). Each subscale comprised of 7 items and the participant responds as to how each item applies to him/her over the past week prior to baseline visit, on 4-point response scale. Separate scores were calculated for anxiety and depression with score ranges from 0 (no presence of anxiety or depression) to 3 (severe feeling of anxiety or depression). Total score range was from 0 to 21 for each subscale; higher score indicating greater severity of anxiety and depression symptoms.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores | Depression Sub-Scale | 5.9 units on a scale | Standard Deviation 3.4 |
| Participants Diagnosed With ATTR | Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores | Anxiety Sub-Scale | 5.9 units on a scale | Standard Deviation 3.9 |
| Caregivers | Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores | Depression Sub-Scale | 8.1 units on a scale | Standard Deviation 5.3 |
| Caregivers | Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores | Anxiety Sub-Scale | 10.8 units on a scale | Standard Deviation 4.5 |
Primary
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
KCCQ was a 23-item participant-completed questionnaire that assessed health status and health-related quality of life (HRQoL) in participants with heart failure. It was quantified in to following 10 summary scores: physical limitation, symptom frequency, symptom severity, and symptom stability, total symptoms, quality of life, social interference, self-efficacy, overall summary and clinical summary. Each summary score was scaled to range from 0 (minimum) to 100 (maximum), with higher scores representing greater disability. Total score ranged from 0 to 100, where higher scores indicated better functioning, fewer symptoms, and better disease specific quality of life.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Physical Limitation Score | 41.7 units on a scale | Standard Deviation 29.2 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Symptom Stability Score | 48.5 units on a scale | Standard Deviation 10.7 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Symptom Frequency Score | 47.7 units on a scale | Standard Deviation 25.1 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Symptom Burden Score | 51.0 units on a scale | Standard Deviation 24.5 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Total Symptom Score | 49.3 units on a scale | Standard Deviation 23.8 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Self-Efficacy Score | 78.7 units on a scale | Standard Deviation 21.1 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Quality of Life Score | 50.0 units on a scale | Standard Deviation 18.6 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Social Limitation Score | 31.8 units on a scale | Standard Deviation 25 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Overall Summary Score | 43.2 units on a scale | Standard Deviation 20 |
| Participants Diagnosed With ATTR | Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores | Clinical Summary Score | 45.5 units on a scale | Standard Deviation 25.2 |
Primary
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of neuropathy on the quality of life of participants diagnosed with ATTR. Scoring was based on 35 questions that yield a TQOL as well as 5 subscale scores: activities of daily living, large fiber neuropathy/physical functioning, small fiber neuropathy, autonomic neuropathy, and symptoms. TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life. This outcome measure was planned to be analyzed only for the reporting arm of participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores | 54.6 units on a scale | Standard Deviation 32.8 |
Primary
Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of neuropathy on the quality of life of participants diagnosed with ATTR. It was summarized in 5 domains: (1) Activities of daily living (score ranges from 0 to 20, where higher score=worse quality of life); (2) Large fiber neuropathy/physical functioning (score ranges from -2 to 58, where higher score=worse condition); (3) Small fiber neuropathy (score ranges from 0 to 16, where higher score=worse condition); (4) Autonomic neuropathy (score ranges from 0 to 12, where higher score=worse condition) and (5) Symptoms (score ranges from 0 to 32, where higher score=less symptoms of disease). Total possible score range= -2 to 138, where higher score=worse quality of life. This outcome measure was analyzed only for the participants diagnosed with ATTR.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure and 'n' signifies those participants who were evaluable for specific category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores | Symptoms (n= 50) | 11.5 units on a scale | Standard Deviation 7.4 |
| Participants Diagnosed With ATTR | Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores | Activities of Daily Living (n= 50) | 5.7 units on a scale | Standard Deviation 6.1 |
| Participants Diagnosed With ATTR | Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores | Small Fiber Neuropathy (n= 49) | 6.0 units on a scale | Standard Deviation 4.2 |
| Participants Diagnosed With ATTR | Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores | Large Fiber Neuropathy (n= 47) | 26.5 units on a scale | Standard Deviation 16.8 |
| Participants Diagnosed With ATTR | Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores | Autonomic Neuropathy (n= 50) | 4.2 units on a scale | Standard Deviation 3.1 |
Primary
Participants Pain Score
Participants diagnosed with ATTR rated their pain due to the health condition based on 3 items: pain right now, average pain in the past week, and worst pain in the past week prior to baseline visit. All 3 items were rated on an 11-point numeric rating scale ranging from 0=none to 10=severe pain, where higher scores indicated severe pain.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | Participants Pain Score | Pain Right now | 3.1 units on a scale | Standard Deviation 3.1 |
| Participants Diagnosed With ATTR | Participants Pain Score | Average Pain Past Week | 3.4 units on a scale | Standard Deviation 3 |
| Participants Diagnosed With ATTR | Participants Pain Score | Worst Pain Past Week | 4.3 units on a scale | Standard Deviation 3.4 |
Primary
Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment
The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asks about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment | 49.8 units on a scale | Standard Deviation 30.5 |
| Caregivers | Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment | 41.2 units on a scale | Standard Deviation 30.4 |
Primary
Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working
The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asks about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working | 32.8 units on a scale | Standard Deviation 32.7 |
| Caregivers | Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working | 36.3 units on a scale | Standard Deviation 31.6 |
Primary
Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment
The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asked about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment | 37.3 units on a scale | Standard Deviation 36.1 |
| Caregivers | Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment | 40.4 units on a scale | Standard Deviation 35.6 |
Primary
Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed
The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asked about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Percentage of work time missed of participants were recorded and reported.
Time frame: Baseline (Day 1)
Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed | 2 percentage of work time missed | Full Range 26 |
| Caregivers | Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed | 0 percentage of work time missed | Full Range 21.8 |
Primary
Zarit Burden Interview: Subscale Scores
A questionnaire designed to evaluate aspects of caregiver burden in terms of personal and role strain associated with caregiving. Total score of ZBI scale ranges from 0-88 with higher scores indicating increased burden of care. Five subscale scores were also calculated: (1) Burden in the relationship (consist of 6-items, ranging from 0 to 24 where higher scores indicating increased burden in relationship); (2) Emotional well-being (consisting of 7-items, ranging from 0 to 28 where higher scores indicating worse condition; (3) Social and family life (consisting of 4-items, ranging from 0 to 16 where higher scores indicating worse life condition); (4) Finances (consisting of a single item, scored from 0 to 4 where higher scores indicating worse financial condition); and (5) Loss of control over one's life (consisting of 4-items, ranging from 0 to 16 where higher scores indicating worse control over life).
Time frame: Baseline (Day 1)
Population: All participants who were included in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Participants Diagnosed With ATTR | Zarit Burden Interview: Subscale Scores | Burden in Relationship (n=28) | 7.6 units on a scale | Standard Deviation 5 |
| Participants Diagnosed With ATTR | Zarit Burden Interview: Subscale Scores | Emotional Well-Being (n=29) | 8.3 units on a scale | Standard Deviation 5.9 |
| Participants Diagnosed With ATTR | Zarit Burden Interview: Subscale Scores | Social and Family Life (n=28) | 5.1 units on a scale | Standard Deviation 3.4 |
| Participants Diagnosed With ATTR | Zarit Burden Interview: Subscale Scores | Finances (n=29) | 1.9 units on a scale | Standard Deviation 1.4 |
| Participants Diagnosed With ATTR | Zarit Burden Interview: Subscale Scores | Loss of Control Over Life (n=29) | 6.5 units on a scale | Standard Deviation 3.5 |
Primary
Zarit Burden Interview (ZBI): Total Scores
ZBI was a 22-item questionnaire designed to evaluate five broad aspects of caregiver burden in terms of personal and role strain associated with caregiving. Five broad aspects were: burden in the relationship, emotional well-being, social and family life, finances, loss of control over one's life. Each item rated on a 5 point scale anchored at 0 for never and 4 for nearly always. Total score ranges from 0-88 with higher scores indicating increased burden of care.
Time frame: Baseline (Day 1)
Population: All participants who were included in the study and completed the survey.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Diagnosed With ATTR | Zarit Burden Interview (ZBI): Total Scores | 28.8 units on a scale | Standard Deviation 17.5 |