Hereditary Transthyretin Amyloidosis (ATTRv), Polyneuropathy
Conditions
Brief summary
A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months with polyneuropathy (PN) have kept going to their multisystemic follow-ups (neuro/ophthalmo/gastrointestinal) ≥12 months.
Interventions
DRUGTafamidis
61 milligrams (mg) as received in studies B3461028 and B3461045
Sponsors
Pfizer
Study design
Observational model
OTHER
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Treatment with tafamidis 61 mg ≥ 12 months * Neurological follow up ≥ 12 months * Diagnosis of transthyretin amyloidosis with polyneuropathy (ATTR-PN) based on one of the following: * Amplitude reduction in, at least, 2 nerves under normal value, excluding median nerve OR 50% amplitude reduction in, at least, 2 nerves on the basal value of the patient, excluding median nerve OR 2 abnormal tests detecting thin fibers alterations (through Sudo scan, RR Interval analysis, etc..)
Exclusion criteria
* Treatment with tafamidis 61 mg \< 12 months * Neurological follow up \< 12 months * Other diagnosis for polyneuropathy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv | Baseline and Month 12 (data collected and analyzed over 22 days) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv | Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv | Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Change in COMPASS-31 for ATTRv | Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv | Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv | From Baseline to Month 30 (data collected and analyzed over 22 days) | 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. |
| Percentage of Responders to Treatment for ATTRv | Month 12 (data collected and analyzed over 22 days) | 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. |
| Number of Participants With R-R Interval Variability for ATTRv | Month 18, 24 and 30 (data collected and analyzed over 22 days) | Number of participants with R-R interval variability (altered/unaltered) was reported in this outcome measure. |
| Modified Body Mass Index (mBMI) for ATTRv | Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days) | 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 \[gram/liter (g/L)\]. |
| Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv | Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Sural sensory nerve action potential amplitude (in microvolts) was measured using electromyography of the left lower limb. |
| Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv | Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Peroneal motor nerve compound muscle action potential amplitude was measured using electromyography of the left lower limb. |
| Change in NIS for ATTRwt | Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Change in NIS-LL for ATTRwt | Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Change in COMPASS-31 for ATTRwt | Baseline, Month 6, 12, 18, 24 and 36 after treatment initiation | 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. |
| Change in FAP-RODs for ATTRwt | Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Change in Norfolk QOL-DN for ATTRwt | Baseline, Month 6, 12, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Change in NIS for ATTRv | Baseline, Month 6, 18, 24 and 36 after the start of treatment (data collected and analyzed over 22 days) | 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. |
| Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt | From Baseline to Month 30 (data collected and analysed over 22 days) | 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. |
| Percentage of Responders to Treatment for ATTRwt | Month 12 (data collected and analyzed over 22 days) | 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. |
| Number of Participants With R-R Interval Variability for ATTRwt | Month 18, 24 and 30 (data collected and analyzed over 22 days) | Number of participants with R-R interval variability (altered/unaltered) was reported in this outcome measure. |
| mBMI for ATTRwt | Month 18, 24 and 30 after start of treatment (data collected and analysed over 22 days) | BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m\^2). |
| Ulnar/Sural SNAP Score for ATTRwt | Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Sural sensory nerve action potential amplitude (in microvolts) was measured using electromyography of the left lower limb. |
| Ulnar/Peroneal CMAP Score for ATTRwt | Month 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Peroneal motor nerve compound muscle action potential amplitude (in millivolts) was measured using electromyography of the left lower limb. |
| Number of Participants With Carpal Tunnel Syndrome | At baseline (data collected and analyzed over 22 days) | Number of participants with carpal tunnel syndrome were reported in this outcome measure. |
| Number of Participants With Lumbar Stenosis | At baseline (data collected and analyzed over 22 days) | Number of participants with lumbar stenosis were reported in this outcome measure. |
| Number of Participants With Gastrointestinal Disturbances | Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Number of participants with gastrointestinal disturbances were reported in this outcome measure. |
| Number of Participants With Unintentional Weight Loss | Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Number of participants with unintentional weight loss were reported in this outcome measure. |
| Number of Participants With Urological Disturbances | Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Number of participants with urological disturbances were reported in this outcome measure. |
| Number of Participants With Ophthalmological Disturbances | Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Number of participants with ophthalmological disturbances were reported in this outcome measure. |
| Number of Participants With Central Nervous System (CNS) Disturbances | Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Number of participants with CNS disturbances were reported in this outcome measure. |
| Number of Participants With Symptoms of Autonomic Neuropathy | Month 18, 24 and 30 after the start of treatment (data collected and analyzed over 22 days) | Number of participants with symptoms of autonomic neuropathy including impaired sweating, sexual dysfunction, orthostatic hypotension were reported in this outcome measure. |
| Number of Participants With Symptoms of Peripheral Neuropathy | At baseline (data collected and analyzed over 22 days) | Number of participants with symptoms of peripheral neuropathy (allodynia and paresthesia) were reported in this outcome measure. |
| Number of Participants According to FAP Stage for ATTRwt | Month 18, 24 and 30 months after the start of treatment (data collected and analyzed over 22 days) | 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. |
Countries
Spain
Participant flow
Recruitment details
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.
Pre-assignment details
Data was collected from medical records and analyzed over 22 days of this study.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 2 |
| 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. | 3 |
| Total | 5 |
Baseline characteristics
| Characteristic | Total | ATTRv | ATTRwt |
|---|---|---|---|
| Age at diagnosis | 73.4 Years | 68.0 Years | 77.0 Years |
| Age at onset of disease | 72.0 Years | 67.0 Years | 75.3 Years |
| Age, Continuous | 75.4 Years | 71.5 Years | 78.0 Years |
| First disease-related sign or symptom Autonomic neuropathy changes | 3 Participants | 1 Participants | 2 Participants |
| First disease-related sign or symptom CNS disorders | 0 Participants | 0 Participants | 0 Participants |
| First disease-related sign or symptom Gastrointestinal disorders | 0 Participants | 0 Participants | 0 Participants |
| First disease-related sign or symptom Heart disorders | 5 Participants | 2 Participants | 3 Participants |
| First disease-related sign or symptom Ophthalmological disorders | 0 Participants | 0 Participants | 0 Participants |
| First disease-related sign or symptom Peripheral neuropathy | 0 Participants | 0 Participants | 0 Participants |
| First disease-related sign or symptom Urological disorders | 0 Participants | 0 Participants | 0 Participants |
| Number of participants according to Genotype Mutation | 2 Participants | 2 Participants | 0 Participants |
| Number of participants according to Genotype Wild Type | 3 Participants | 0 Participants | 3 Participants |
| Race and Ethnicity Not Collected | 0 Participants | — | — |
| Sex: Female, Male Female | 1 Participants | 1 Participants | 0 Participants |
| Sex: Female, Male Male | 4 Participants | 1 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 2 | 0 / 3 |
| other Total, other adverse events | 0 / 2 | 0 / 3 |
| serious Total, serious adverse events | 0 / 2 | 0 / 3 |
Outcome results
Primary
Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv
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.
Time frame: 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.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| ATTRv | Change in Neuropathy Impairment Score (NIS) at Month 12 for ATTRv | 1.6 Change in score*months |
Secondary
Change in COMPASS-31 for ATTRv
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.
Time frame: 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.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| ATTRv | Change in COMPASS-31 for ATTRv | NA Units on a scale |
Secondary
Change in COMPASS-31 for ATTRwt
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.
Time frame: Baseline, Month 6, 12, 18, 24 and 36 after treatment initiation
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.
Secondary
Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv
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.
Time frame: 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.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| ATTRv | Change in Familial Amyloid Polyneuropathy Specific Rasch-Built Overall Disability Scale (FAP-RODs) for ATTRv | NA Units on a scale |
Secondary
Change in FAP-RODs for ATTRwt
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.
Time frame: 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.
Secondary
Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv | Month 6 | 1.6 Change in score*months |
| ATTRv | Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv | Month 12 | 3.1 Change in score*months |
| ATTRv | Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv | Month 18 | 4.7 Change in score*months |
| ATTRv | Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv | Month 24 | 6.3 Change in score*months |
| ATTRv | Change in Neuropathy Impairment Score - Lower Limbs (NIS-LL) for ATTRv | Month 36 | 9.4 Change in score*months |
Secondary
Change in NIS for ATTRv
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Change in NIS for ATTRv | Month 6 | 0.8 Change in score*months |
| ATTRv | Change in NIS for ATTRv | Month 18 | 2.4 Change in score*months |
| ATTRv | Change in NIS for ATTRv | Month 24 | 3.1 Change in score*months |
| ATTRv | Change in NIS for ATTRv | Month 36 | 4.7 Change in score*months |
Secondary
Change in NIS for ATTRwt
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Change in NIS for ATTRwt | Month 6 | 0.4 Change in score*months |
| ATTRv | Change in NIS for ATTRwt | Month 12 | 0.8 Change in score*months |
| ATTRv | Change in NIS for ATTRwt | Month 18 | 1.1 Change in score*months |
| ATTRv | Change in NIS for ATTRwt | Month 24 | 1.5 Change in score*months |
| ATTRv | Change in NIS for ATTRwt | Month 36 | 2.3 Change in score*months |
Secondary
Change in NIS-LL for ATTRwt
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Change in NIS-LL for ATTRwt | Month 6 | 0.2 Change in score*months |
| ATTRv | Change in NIS-LL for ATTRwt | Month 12 | 0.4 Change in score*months |
| ATTRv | Change in NIS-LL for ATTRwt | Month 18 | 0.6 Change in score*months |
| ATTRv | Change in NIS-LL for ATTRwt | Month 24 | 0.8 Change in score*months |
| ATTRv | Change in NIS-LL for ATTRwt | Month 36 | 1.2 Change in score*months |
Secondary
Change in Norfolk QOL-DN for ATTRwt
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.
Time frame: 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.
Secondary
Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv
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.
Time frame: 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.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| ATTRv | Change in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) for ATTRv | NA Units on a scale |
Secondary
mBMI for ATTRwt
BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m\^2).
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | mBMI for ATTRwt | Month 18 | 29.7 Kilogram per meter square*gram/Liter |
| ATTRv | mBMI for ATTRwt | Month 24 | 24.0 Kilogram per meter square*gram/Liter |
| ATTRv | mBMI for ATTRwt | Month 30 | 25.7 Kilogram per meter square*gram/Liter |
Secondary
Modified Body Mass Index (mBMI) for ATTRv
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 \[gram/liter (g/L)\].
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Modified Body Mass Index (mBMI) for ATTRv | Month 24 | 27.7 Kilogram per meter square*gram/Liter |
| ATTRv | Modified Body Mass Index (mBMI) for ATTRv | Month 30 | 24.3 Kilogram per meter square*gram/Liter |
| Unknown | Modified Body Mass Index (mBMI) for ATTRv | Month 18 | — Kilogram per meter square*gram/Liter |
Secondary
Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv
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.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Asymptomatic (Month 24) | 0 Participants |
| ATTRv | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Walking without support (Month 24) | 1 Participants |
| ATTRv | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Walking with support (Month 24) | 0 Participants |
| ATTRv | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Wheelchair or bedridden (Month 24) | 0 Participants |
| ATTRv | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Asymptomatic (Month 30) | 1 Participants |
| ATTRv | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Walking without support (Month 30) | 1 Participants |
| ATTRv | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Walking with support (Month 30) | 0 Participants |
| ATTRv | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Wheelchair or bedridden (Month 30) | 0 Participants |
| Unknown | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Asymptomatic (Month 18) | — Participants |
| Unknown | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Walking without support (Month 18) | — Participants |
| Unknown | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Walking with support (Month 18) | — Participants |
| Unknown | Number of Participants According to Familiar Amyloidotic Polyneuropathy Stage (FAP) for ATTRv | Wheelchair or bedridden (Month 18) | — Participants |
Secondary
Number of Participants According to FAP Stage for ATTRwt
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.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Asymptomatic (Month 18) | 0 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Walking without support (Month 18) | 1 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Walking with support (Month 18) | 0 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Wheelchair or bedridden (Month 18) | 0 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Asymptomatic (Month 24) | 0 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Walking without support (Month 24) | 2 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Walking with support (Month 24) | 0 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Wheelchair or bedridden (Month 24) | 0 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Asymptomatic (Month 30) | 0 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Walking without support (Month 30) | 2 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Walking with support (Month 30) | 0 Participants |
| ATTRv | Number of Participants According to FAP Stage for ATTRwt | Wheelchair or bedridden (Month 30) | 0 Participants |
Secondary
Number of Participants With Carpal Tunnel Syndrome
Number of participants with carpal tunnel syndrome were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| ATTRv | Number of Participants With Carpal Tunnel Syndrome | 2 Participants |
| ATTRwt | Number of Participants With Carpal Tunnel Syndrome | 2 Participants |
Secondary
Number of Participants With Central Nervous System (CNS) Disturbances
Number of participants with CNS disturbances were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants With Central Nervous System (CNS) Disturbances | Month 18 | 0 Participants |
| ATTRv | Number of Participants With Central Nervous System (CNS) Disturbances | Month 24 | 0 Participants |
| ATTRv | Number of Participants With Central Nervous System (CNS) Disturbances | Month 30 | 0 Participants |
| ATTRwt | Number of Participants With Central Nervous System (CNS) Disturbances | Month 18 | 0 Participants |
| ATTRwt | Number of Participants With Central Nervous System (CNS) Disturbances | Month 24 | 0 Participants |
| ATTRwt | Number of Participants With Central Nervous System (CNS) Disturbances | Month 30 | 0 Participants |
Secondary
Number of Participants With Gastrointestinal Disturbances
Number of participants with gastrointestinal disturbances were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants With Gastrointestinal Disturbances | Month 24 | 0 Participants |
| ATTRv | Number of Participants With Gastrointestinal Disturbances | Month 30 | 0 Participants |
| ATTRwt | Number of Participants With Gastrointestinal Disturbances | Month 18 | 1 Participants |
| ATTRwt | Number of Participants With Gastrointestinal Disturbances | Month 24 | 1 Participants |
| ATTRwt | Number of Participants With Gastrointestinal Disturbances | Month 30 | 2 Participants |
Secondary
Number of Participants With Lumbar Stenosis
Number of participants with lumbar stenosis were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| ATTRv | Number of Participants With Lumbar Stenosis | 0 Participants |
| ATTRwt | Number of Participants With Lumbar Stenosis | 1 Participants |
Secondary
Number of Participants With Ophthalmological Disturbances
Number of participants with ophthalmological disturbances were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants With Ophthalmological Disturbances | Month 18 | 0 Participants |
| ATTRv | Number of Participants With Ophthalmological Disturbances | Month 24 | 0 Participants |
| ATTRv | Number of Participants With Ophthalmological Disturbances | Month 30 | 0 Participants |
| ATTRwt | Number of Participants With Ophthalmological Disturbances | Month 18 | 0 Participants |
| ATTRwt | Number of Participants With Ophthalmological Disturbances | Month 24 | 0 Participants |
| ATTRwt | Number of Participants With Ophthalmological Disturbances | Month 30 | 0 Participants |
Secondary
Number of Participants With R-R Interval Variability for ATTRv
Number of participants with R-R interval variability (altered/unaltered) was reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants With R-R Interval Variability for ATTRv | Altered (Month 30) | 1 Participants |
| ATTRv | Number of Participants With R-R Interval Variability for ATTRv | Unaltered (Month 30) | 0 Participants |
| Unknown | Number of Participants With R-R Interval Variability for ATTRv | Unaltered (Month 18) | — Participants |
| Unknown | Number of Participants With R-R Interval Variability for ATTRv | Altered (Month 18) | — Participants |
| Unknown | Number of Participants With R-R Interval Variability for ATTRv | Unaltered (Month 24) | — Participants |
| Unknown | Number of Participants With R-R Interval Variability for ATTRv | Altered (Month 24) | — Participants |
Secondary
Number of Participants With R-R Interval Variability for ATTRwt
Number of participants with R-R interval variability (altered/unaltered) was reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants With R-R Interval Variability for ATTRwt | Unaltered (Month 24) | 0 Participants |
| ATTRv | Number of Participants With R-R Interval Variability for ATTRwt | Altered (Month 24) | 1 Participants |
| ATTRv | Number of Participants With R-R Interval Variability for ATTRwt | Unaltered (Month 30) | 0 Participants |
| ATTRv | Number of Participants With R-R Interval Variability for ATTRwt | Altered (Month 30) | 1 Participants |
| Unknown | Number of Participants With R-R Interval Variability for ATTRwt | Unaltered (Month 18) | — Participants |
| Unknown | Number of Participants With R-R Interval Variability for ATTRwt | Altered (Month 18) | — Participants |
Secondary
Number of Participants With Symptoms of Autonomic Neuropathy
Number of participants with symptoms of autonomic neuropathy including impaired sweating, sexual dysfunction, orthostatic hypotension were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants With Symptoms of Autonomic Neuropathy | Month 18 | 0 Participants |
| ATTRv | Number of Participants With Symptoms of Autonomic Neuropathy | Month 24 | 0 Participants |
| ATTRv | Number of Participants With Symptoms of Autonomic Neuropathy | Month 30 | 1 Participants |
| ATTRwt | Number of Participants With Symptoms of Autonomic Neuropathy | Month 18 | 1 Participants |
| ATTRwt | Number of Participants With Symptoms of Autonomic Neuropathy | Month 24 | 2 Participants |
| ATTRwt | Number of Participants With Symptoms of Autonomic Neuropathy | Month 30 | 2 Participants |
Secondary
Number of Participants With Symptoms of Peripheral Neuropathy
Number of participants with symptoms of peripheral neuropathy (allodynia and paresthesia) were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| ATTRv | Number of Participants With Symptoms of Peripheral Neuropathy | 1 Participants |
| ATTRwt | Number of Participants With Symptoms of Peripheral Neuropathy | 0 Participants |
Secondary
Number of Participants With Unintentional Weight Loss
Number of participants with unintentional weight loss were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRwt | Number of Participants With Unintentional Weight Loss | Month 18 | 0 Participants |
| ATTRwt | Number of Participants With Unintentional Weight Loss | Month 24 | 0 Participants |
| ATTRwt | Number of Participants With Unintentional Weight Loss | Month 30 | 0 Participants |
Secondary
Number of Participants With Urological Disturbances
Number of participants with urological disturbances were reported in this outcome measure.
Time frame: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ATTRv | Number of Participants With Urological Disturbances | Month 18 | 0 Participants |
| ATTRv | Number of Participants With Urological Disturbances | Month 24 | 0 Participants |
| ATTRv | Number of Participants With Urological Disturbances | Month 30 | 0 Participants |
| ATTRwt | Number of Participants With Urological Disturbances | Month 18 | 0 Participants |
| ATTRwt | Number of Participants With Urological Disturbances | Month 24 | 0 Participants |
| ATTRwt | Number of Participants With Urological Disturbances | Month 30 | 1 Participants |
Secondary
Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv
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.
Time frame: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| ATTRv | Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv | Unchanged | 100.0 Percentage of participants |
| ATTRv | Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRv | Staging up | 0.0 Percentage of participants |
Secondary
Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt
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.
Time frame: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| ATTRv | Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt | Unchanged | 66.7 Percentage of participants |
| ATTRv | Percentage of Participants Who do Not Have Stage Progression in the PND Score for ATTRwt | Staging up | 33.3 Percentage of participants |
Secondary
Percentage of Responders to Treatment for ATTRv
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.
Time frame: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| ATTRv | Percentage of Responders to Treatment for ATTRv | NIS | 100.0 Percentage of responders |
| ATTRv | Percentage of Responders to Treatment for ATTRv | NIS-LL | 0.0 Percentage of responders |
Secondary
Percentage of Responders to Treatment for ATTRwt
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.
Time frame: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| ATTRv | Percentage of Responders to Treatment for ATTRwt | NIS | 100.0 Percentage of responders |
| ATTRv | Percentage of Responders to Treatment for ATTRwt | NIS-LL | 100.0 Percentage of responders |
Secondary
Ulnar/Peroneal CMAP Score for ATTRwt
Peroneal motor nerve compound muscle action potential amplitude (in millivolts) was measured using electromyography of the left lower limb.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Ulnar/Peroneal CMAP Score for ATTRwt | Month 24 | 5.9 Milli Volts (mV) |
| ATTRv | Ulnar/Peroneal CMAP Score for ATTRwt | Month 30 | 2.7 Milli Volts (mV) |
Secondary
Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv
Peroneal motor nerve compound muscle action potential amplitude was measured using electromyography of the left lower limb.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv | Month 30 | 1.3 Milli Volts (mV) |
| Unknown | Ulnar/Peroneal Compound Muscle Action Potential Amplitude (CMAP) for ATTRv | Month 24 | — Milli Volts (mV) |
Secondary
Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv
Sural sensory nerve action potential amplitude (in microvolts) was measured using electromyography of the left lower limb.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv | Month 30 | 2.9 Micro volts (μV) |
| Unknown | Ulnar/Sural Sensory Nerve Action Potential Amplitude (SNAP) for ATTRv | Month 24 | — Micro volts (μV) |
Secondary
Ulnar/Sural SNAP Score for ATTRwt
Sural sensory nerve action potential amplitude (in microvolts) was measured using electromyography of the left lower limb.
Time frame: 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.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| ATTRv | Ulnar/Sural SNAP Score for ATTRwt | Month 24 | 17.0 Micro volts (μV) |
| ATTRv | Ulnar/Sural SNAP Score for ATTRwt | Month 30 | 11.9 Micro volts (μV) |