Trial Outcomes & Findings for Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045. (NCT NCT05560555)
NCT ID: NCT05560555
Last Updated: 2024-05-13
Results Overview
NIS (Neuropathy Impairment Score) is a clinically important, sensitive measure of individual neurological function, assessing sensory function, reflexes, and muscle weakness. NIS score ranged from 0 to 244, with higher score indicating greater disability or impairment. The rate of change was calculated from last follow-up.
COMPLETED
5 participants
Baseline and Month 12 (data collected and analyzed over 22 days)
2024-05-13
Participant Flow
Participants with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) from studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain, with cardiac and neurologic exposed to Tafamidis free acid 61 milligrams (mg) for at least 12 months, and who were in neurological follow-up for no less than 12 months were observed in this retrospective study.
Data was collected from medical records and analyzed over 22 days of this study.
Participant milestones
| Measure |
ATTRv
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
3
|
|
Overall Study
COMPLETED
|
2
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=3 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
71.5 Years
n=2 Participants
|
78.0 Years
n=3 Participants
|
75.4 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=2 Participants
|
0 Participants
n=3 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=2 Participants
|
3 Participants
n=3 Participants
|
4 Participants
n=5 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Number of participants according to Genotype
Mutation
|
2 Participants
n=2 Participants
|
0 Participants
n=3 Participants
|
2 Participants
n=5 Participants
|
|
Number of participants according to Genotype
Wild Type
|
0 Participants
n=2 Participants
|
3 Participants
n=3 Participants
|
3 Participants
n=5 Participants
|
|
Age at onset of disease
|
67.0 Years
n=2 Participants
|
75.3 Years
n=3 Participants
|
72.0 Years
n=5 Participants
|
|
Age at diagnosis
|
68.0 Years
n=2 Participants
|
77.0 Years
n=3 Participants
|
73.4 Years
n=5 Participants
|
|
First disease-related sign or symptom
Gastrointestinal disorders
|
0 Participants
n=2 Participants
|
0 Participants
n=3 Participants
|
0 Participants
n=5 Participants
|
|
First disease-related sign or symptom
Urological disorders
|
0 Participants
n=2 Participants
|
0 Participants
n=3 Participants
|
0 Participants
n=5 Participants
|
|
First disease-related sign or symptom
Ophthalmological disorders
|
0 Participants
n=2 Participants
|
0 Participants
n=3 Participants
|
0 Participants
n=5 Participants
|
|
First disease-related sign or symptom
CNS disorders
|
0 Participants
n=2 Participants
|
0 Participants
n=3 Participants
|
0 Participants
n=5 Participants
|
|
First disease-related sign or symptom
Autonomic neuropathy changes
|
1 Participants
n=2 Participants
|
2 Participants
n=3 Participants
|
3 Participants
n=5 Participants
|
|
First disease-related sign or symptom
Peripheral neuropathy
|
0 Participants
n=2 Participants
|
0 Participants
n=3 Participants
|
0 Participants
n=5 Participants
|
|
First disease-related sign or symptom
Heart disorders
|
2 Participants
n=2 Participants
|
3 Participants
n=3 Participants
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and Month 12 (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
NIS (Neuropathy Impairment Score) is a clinically important, sensitive measure of individual neurological function, assessing sensory function, reflexes, and muscle weakness. NIS score ranged from 0 to 244, with higher score indicating greater disability or impairment. The rate of change was calculated from last follow-up.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv
|
1.6 Change in score*months
Interval 0.0 to 3.1
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 6, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
NIS (Neuropathy Impairment Score) is a clinically important, sensitive measure of individual neurological function, assessing sensory function, reflexes, and muscle weakness. NIS score ranged from 0 to 244, with higher score indicating greater disability or impairment. The rate of change was calculated from last follow-up.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Change in NIS for ATTRv
Month 6
|
0.8 Change in score*months
Interval 0.0 to 1.6
|
—
|
|
Change in NIS for ATTRv
Month 18
|
2.4 Change in score*months
Interval 0.0 to 4.7
|
—
|
|
Change in NIS for ATTRv
Month 24
|
3.1 Change in score*months
Interval 0.0 to 6.3
|
—
|
|
Change in NIS for ATTRv
Month 36
|
4.7 Change in score*months
Interval 0.0 to 9.4
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
NIS-LL (Neuropathy Impairment Score Lower Limbs) is a clinically important, sensitive measure of neurological function in individuals, assessing sensory function, reflexes, and muscle weakness of the lower limbo. NÍS-LL assessed muscle weakness, reflexes, sensation. Each item is scored separately for left and right limbs. Components of muscle weakness:0(normal) to4(paralysis), higher score=more weakness; reflexes, sensation:0=normal, 1=decreased, or 2=absent. Total NIS-LL score range 0-88, higher score=more impairment. The rate of change was calculated from last follow-up.
Outcome measures
| Measure |
ATTRv
n=1 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv
Month 6
|
1.6 Change in score*months
Interval 1.6 to 1.6
|
—
|
|
Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv
Month 12
|
3.1 Change in score*months
Interval 3.1 to 3.1
|
—
|
|
Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv
Month 18
|
4.7 Change in score*months
Interval 4.7 to 4.7
|
—
|
|
Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv
Month 24
|
6.3 Change in score*months
Interval 6.3 to 6.3
|
—
|
|
Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv
Month 36
|
9.4 Change in score*months
Interval 9.4 to 9.4
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
Norfolk Quality of Life Questionnaire for Diabetic Neuropathy is a standardized and validated instrument that assesses the effect of polyneuropathy on the functionality and quality of life of the individual. Norfolk QOL-DN: 35-item participant-rated questionnaire is used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptom was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -4 to 138, where higher score=worse quality of life.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv
|
NA Units on a scale
Data could not be calculated due to absence of data in the records at baseline.
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
COMPASS-31 (Composite Autonomic Symptom Score 31) is a questionnaire designed to assess the severity and functional ability in participants with autonomic dysfunction. COMPASS-31 total score ranged from 0 to 100; where 0=Lesser severity of dysautonomia and 100=Greater severity of dysautonomia.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Change in COMPASS-31 for ATTRv
|
NA Units on a scale
Data could not be calculated due to absence of data in the records at baseline.
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
FAP-RODS is a questionnaire that assessed the effect of neuropathy on daily activities. FAP-RODS total score ranged from 0 to 68; where, 0=Lower ability to perform daily activities and 68=Greater ability to perform daily activities.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv
|
NA Units on a scale
Data could not be calculated due to absence of data in the records at baseline.
|
—
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
FAP is a stage system based on symptom severity and disease progression. FAP stages included: Asymptomatic; Free ambulation (walking without support): stage 1; Supportive ambulation (walking with support): stage 2; Wheelchair-bound or bedridden: stage 3.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
Asymptomatic (Month 24)
|
0 Participants
|
—
|
|
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
Walking without support (Month 24)
|
1 Participants
|
—
|
|
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
Walking with support (Month 24)
|
0 Participants
|
—
|
|
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
Wheelchair or bedridden (Month 24)
|
0 Participants
|
—
|
|
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
Asymptomatic (Month 30)
|
1 Participants
|
—
|
|
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
Walking without support (Month 30)
|
1 Participants
|
—
|
|
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
Walking with support (Month 30)
|
0 Participants
|
—
|
|
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
Wheelchair or bedridden (Month 30)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: From Baseline to Month 30 (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
PND is a staging system that assess the degree of neuropathic dysfunction and its impact on ambulation. PND stages included: Asymptomatic; Stage I: Sensory disturbances, normal gait; Stage II: Sensory disturbances, altered gait not requiring support; IIIA: Gait requiring one support; IIIB: Gait requiring two supports; IV: Wheelchair or bedside. Participants who did not change to higher stages compared to the start of treatment was reported as Unchanged and those who progressed to higher stages were reported under Staging up.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv
Unchanged
|
100.0 Percentage of participants
|
—
|
|
Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv
Staging up
|
0.0 Percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Month 12 (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Percentage of responders to treatment at Month 12 was defined as participants who achieved the change from baseline of less than 4 points in the NIS and participants who achieved the change from baseline of less than 2 points in the NIS-LL were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Percentage of Responders to Treatment for ATTRv
NIS
|
100.0 Percentage of responders
|
—
|
|
Percentage of Responders to Treatment for ATTRv
NIS-LL
|
0.0 Percentage of responders
|
—
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for the specified rows.
Number of participants with R-R interval variability (altered/unaltered) was reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=1 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With R-R Interval Variability for ATTRv
Unaltered (Month 30)
|
0 Participants
|
—
|
|
Number of Participants With R-R Interval Variability for ATTRv
Altered (Month 30)
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for the specified rows.
BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m\^2). mBMI was calculated by multiplying BMI by serum albumin levels \[gram/liter (g/L)\].
Outcome measures
| Measure |
ATTRv
n=1 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Modified Body Mass Index (mBMI) for ATTRv
Month 24
|
27.7 Kilogram per meter square*gram/Liter
Interval 27.7 to 27.7
|
—
|
|
Modified Body Mass Index (mBMI) for ATTRv
Month 30
|
24.3 Kilogram per meter square*gram/Liter
Interval 24.3 to 24.3
|
—
|
SECONDARY outcome
Timeframe: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Sural sensory nerve action potential amplitude (in microvolts) was measured using electromyography of the left lower limb.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv
Month 30
|
2.9 Micro volts (μV)
Interval 1.1 to 4.6
|
—
|
SECONDARY outcome
Timeframe: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Peroneal motor nerve compound muscle action potential amplitude was measured using electromyography of the left lower limb.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv
Month 30
|
1.3 Milli Volts (mV)
Interval 1.1 to 1.5
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
NIS (Neuropathy Impairment Score) was a clinically important, sensitive measure of individual neurological function, assessing sensory function, reflexes, and muscle weakness. NIS score ranged from 0 to 244, with higher score indicating greater disability or impairment. The rate of change was calculated from last follow-up.
Outcome measures
| Measure |
ATTRv
n=3 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Change in NIS for ATTRwt
Month 6
|
0.4 Change in score*months
Interval 0.0 to 0.8
|
—
|
|
Change in NIS for ATTRwt
Month 12
|
0.8 Change in score*months
Interval 0.0 to 1.5
|
—
|
|
Change in NIS for ATTRwt
Month 18
|
1.1 Change in score*months
Interval 0.0 to 2.3
|
—
|
|
Change in NIS for ATTRwt
Month 24
|
1.5 Change in score*months
Interval 0.0 to 3.0
|
—
|
|
Change in NIS for ATTRwt
Month 36
|
2.3 Change in score*months
Interval 0.0 to 4.5
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
NIS-LL (Neuropathy Impairment Score Lower Limbs) was clinically important, sensitive measure of neurological function in individuals, assessing sensory function, reflexes, and muscle weakness of the lower limbo. NÍS-LL assessed muscle weakness, reflexes, sensation. Each item scored separately for left and right limbs. Components of muscle weakness:0(normal) to4(paralysis), higher score=more weakness; reflexes, sensation:0=normal, 1=decreased, or 2=absent. Total NIS-LL score range 0-88, higher score=more impairment. The rate of change was calculated from last follow-up.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Change in NIS-LL for ATTRwt
Month 6
|
0.2 Change in score*months
Interval 0.0 to 0.4
|
—
|
|
Change in NIS-LL for ATTRwt
Month 12
|
0.4 Change in score*months
Interval 0.0 to 0.8
|
—
|
|
Change in NIS-LL for ATTRwt
Month 18
|
0.6 Change in score*months
Interval 0.0 to 1.2
|
—
|
|
Change in NIS-LL for ATTRwt
Month 24
|
0.8 Change in score*months
Interval 0.0 to 1.6
|
—
|
|
Change in NIS-LL for ATTRwt
Month 36
|
1.2 Change in score*months
Interval 0.0 to 2.3
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Data was not collected for this outcome measure as data was not available in the medical records.
Norfolk Quality of Life Questionnaire for Diabetic Neuropathy was a standardized and validated instrument that assesses the effect of polyneuropathy on the functionality and quality of life of the individual. Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptom was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -4 to 138, where higher score=worse quality of life.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after treatment initiationPopulation: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Data was not collected for this outcome measure as data was not available in the medical records.
COMPASS-31 (Composite Autonomic Symptom Score 31) was a questionnaire designed to assess the severity and functional ability in participants with autonomic dysfunction. COMPASS-31 total score ranged from 0 to 100; where 0=Lesser severity of dysautonomia and 100=Greater severity of dysautonomia.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Data was not collected for this outcome measure as data was not available in the medical records.
FAP-RODS is a questionnaire that assessed the effect of neuropathy on daily activities. FAP-RODS total score ranged from 0 to 68; where, 0=Lower ability to perform daily activities and 68=Greater ability to perform daily activities.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for the specified rows.
FAP stage is a staging system based on symptom severity and disease progression. FAP stages included: Asymptomatic; Free ambulation (Walking without support): stage 1; Supportive ambulation (Walking with support): stage 2; Wheelchair-bound or bedridden: stage 3.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants According to FAP Stage for ATTRwt
Asymptomatic (Month 18)
|
0 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Walking without support (Month 18)
|
1 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Walking with support (Month 18)
|
0 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Wheelchair or bedridden (Month 18)
|
0 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Asymptomatic (Month 24)
|
0 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Walking without support (Month 24)
|
2 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Walking with support (Month 24)
|
0 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Wheelchair or bedridden (Month 24)
|
0 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Asymptomatic (Month 30)
|
0 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Walking without support (Month 30)
|
2 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Walking with support (Month 30)
|
0 Participants
|
—
|
|
Number of Participants According to FAP Stage for ATTRwt
Wheelchair or bedridden (Month 30)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: From Baseline to Month 30 (data collected and analysed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
PND was a simple staging system according to the degree of neuropathic dysfunction and its impact on ambulation. PND stages included: Asymptomatic: I Sensory disturbances, normal gait: II Sensory disturbances, altered gait not requiring support: IIIA Gait requiring one support: IIIB Gait requiring two supports: IV Wheelchair or bedside). Participants who did not change to higher stages compared to the start of treatment was reported as Unchanged and those who progressed to higher stages were reported under Staging up.
Outcome measures
| Measure |
ATTRv
n=3 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt
Staging up
|
33.3 Percentage of participants
|
—
|
|
Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt
Unchanged
|
66.7 Percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Month 12 (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Percentage of responders to treatment was defined as participants who achieved the change from baseline of less than 4 points in the NIS and participants who achieved the change from baseline of less than 2 points in the NIS-LL were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=3 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Percentage of Responders to Treatment for ATTRwt
NIS-LL
|
100.0 Percentage of responders
|
—
|
|
Percentage of Responders to Treatment for ATTRwt
NIS
|
100.0 Percentage of responders
|
—
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Number of participants with R-R interval variability (altered/unaltered) was reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=1 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With R-R Interval Variability for ATTRwt
Unaltered (Month 24)
|
0 Participants
|
—
|
|
Number of Participants With R-R Interval Variability for ATTRwt
Altered (Month 24)
|
1 Participants
|
—
|
|
Number of Participants With R-R Interval Variability for ATTRwt
Unaltered (Month 30)
|
0 Participants
|
—
|
|
Number of Participants With R-R Interval Variability for ATTRwt
Altered (Month 30)
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for the specified rows.
BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m\^2).
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
mBMI for ATTRwt
Month 18
|
29.7 Kilogram per meter square*gram/Liter
Interval 29.7 to 29.7
|
—
|
|
mBMI for ATTRwt
Month 24
|
24.0 Kilogram per meter square*gram/Liter
Interval 22.1 to 26.0
|
—
|
|
mBMI for ATTRwt
Month 30
|
25.7 Kilogram per meter square*gram/Liter
Interval 22.1 to 29.4
|
—
|
SECONDARY outcome
Timeframe: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure.
Sural sensory nerve action potential amplitude (in microvolts) was measured using electromyography of the left lower limb.
Outcome measures
| Measure |
ATTRv
n=1 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Ulnar/Sural SNAP Score for ATTRwt
Month 24
|
17.0 Micro volts (μV)
Interval 17.0 to 17.0
|
—
|
|
Ulnar/Sural SNAP Score for ATTRwt
Month 30
|
11.9 Micro volts (μV)
Interval 11.9 to 11.9
|
—
|
SECONDARY outcome
Timeframe: Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Overall Number of Participants Analyzed' signifies participants evaluable for this outcome measure.
Peroneal motor nerve compound muscle action potential amplitude (in millivolts) was measured using electromyography of the left lower limb.
Outcome measures
| Measure |
ATTRv
n=1 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Ulnar/Peroneal CMAP Score for ATTRwt
Month 24
|
5.9 Milli Volts (mV)
Interval 5.9 to 5.9
|
—
|
|
Ulnar/Peroneal CMAP Score for ATTRwt
Month 30
|
2.7 Milli Volts (mV)
Interval 2.7 to 2.7
|
—
|
SECONDARY outcome
Timeframe: At baseline (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
Number of participants with carpal tunnel syndrome were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=3 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Carpal Tunnel Syndrome
|
2 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: At baseline (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
Number of participants with lumbar stenosis were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=3 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Lumbar Stenosis
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Number of participants with gastrointestinal disturbances were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=3 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Gastrointestinal Disturbances
Month 18
|
—
|
1 Participants
|
|
Number of Participants With Gastrointestinal Disturbances
Month 24
|
0 Participants
|
1 Participants
|
|
Number of Participants With Gastrointestinal Disturbances
Month 30
|
0 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. Here, 'Overall Number Analyzed' signifies participants evaluable for this outcome measure and 'Number Analyzed' signifies participants evaluable for the specified rows.
Number of participants with unintentional weight loss were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=2 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Unintentional Weight Loss
Month 18
|
—
|
0 Participants
|
|
Number of Participants With Unintentional Weight Loss
Month 24
|
—
|
0 Participants
|
|
Number of Participants With Unintentional Weight Loss
Month 30
|
—
|
0 Participants
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. All participants in the 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Number of participants with urological disturbances were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=3 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Urological Disturbances
Month 18
|
0 Participants
|
0 Participants
|
|
Number of Participants With Urological Disturbances
Month 24
|
0 Participants
|
0 Participants
|
|
Number of Participants With Urological Disturbances
Month 30
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. All participants in the 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Number of participants with ophthalmological disturbances were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=3 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Ophthalmological Disturbances
Month 18
|
0 Participants
|
0 Participants
|
|
Number of Participants With Ophthalmological Disturbances
Month 24
|
0 Participants
|
0 Participants
|
|
Number of Participants With Ophthalmological Disturbances
Month 30
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. All participants in the 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Number of participants with CNS disturbances were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=3 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Central Nervous System (CNS) Disturbances
Month 18
|
0 Participants
|
0 Participants
|
|
Number of Participants With Central Nervous System (CNS) Disturbances
Month 24
|
0 Participants
|
0 Participants
|
|
Number of Participants With Central Nervous System (CNS) Disturbances
Month 30
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study. All participants in the 'Overall Number of Participants Analyzed' contributed data to the table but may not have evaluable data for every row. Here, 'Number Analyzed' signifies participants evaluable for the specified rows.
Number of participants with symptoms of autonomic neuropathy including impaired sweating, sexual dysfunction, orthostatic hypotension were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=2 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Symptoms of Autonomic Neuropathy
Month 18
|
0 Participants
|
1 Participants
|
|
Number of Participants With Symptoms of Autonomic Neuropathy
Month 24
|
0 Participants
|
2 Participants
|
|
Number of Participants With Symptoms of Autonomic Neuropathy
Month 30
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: At baseline (data collected and analyzed over 22 days)Population: Analysis population included all eligible participants whose data was retrieved and analyzed in this study.
Number of participants with symptoms of peripheral neuropathy (allodynia and paresthesia) were reported in this outcome measure.
Outcome measures
| Measure |
ATTRv
n=2 Participants
Participants diagnosed with ATTRv in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
ATTRwt
n=3 Participants
Participants diagnosed with ATTRwt in studies B3461028 (NCT01994889) and B3461045 (NCT02791230) in Spain who presented with multisystem involvement (cardiac and neurological) and received tafamidis free acid 61 mg for at least 12 months were included.
|
|---|---|---|
|
Number of Participants With Symptoms of Peripheral Neuropathy
|
1 Participants
|
0 Participants
|
Adverse Events
ATTRv
ATTRwt
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER