Muscular Dystrophy, Myopathy, Myositis
Conditions
Brief summary
The objective of the proposed study is to evaluate the clinical utility of muscle ultrasonography for improving the diagnostic yield and safety of core muscle biopsy. Our facility currently uses core (needle) biopsy to obtain muscle samples in patients 18 years old or older. Currently, there is no imaging tool used to guide the actual biopsy. As muscle biopsy is an invasive and potentially painful procedure, improving the diagnostic yield of this test is important.
Interventions
PROCEDUREUltrasound
The ultrasound guided group will have an ultrasound to identify the optimal site for biopsy and guide the procedure for safety purposes.
Sponsors
Duke University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Consecutive subjects scheduled for a biopsy at our EMG laboratory will be asked if they would like to participate until a total of 40 participants are obtained.
Exclusion criteria
* Age under 18 years. * Inability to provide consent for participation.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Amount of Tissue Obtained | At time of biopsy | This data will be analyzed to determine if ultrasound guidance improves muscle yield (as measured by pathology determined volume and mass). |
| Number of Patients Receiving Diagnosis From Muscle Biopsy | At time of biopsy | The rate of achieving a specific final diagnosis in ultrasound guided muscle biopsies vs. unguided biopsies will be examined. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events Related to Muscle Biopsy | Patient involvement limited to the time of biopsy; Records analyzed up to 10 months after biopsy | Data will be examined to determine if ultrasound guidance reduces the rate of adverse events in muscle biopsies. |
| Number of Times Biopsy Needle Was Inserted to Obtain Biopsy Tissue | At time of biopsy | For core or needle biopsies, the physician passes the needle into the muscle until they feel that adequate tissue samples have been obtained. This outcome measure is the number of passes required in each study arm. |
| Number of Participants With Inadequate Biopsy Samples | At time of pathology review | The presence of an inadequate sample was determined by the blinded pathologist reading the muscle biopsies. This reflects the sample having enough preserved muscle tissue for histologic analysis. It is separate from the number of participants receiving a diagnosis. A sample may be adequate, but non-diagnostic. Only one biopsy was performed in each patients, so the number of biopsies is the same as the number of participants. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Ultrasound Guided Biopsy Ultrasound guided biopsy will be used in all patients.
Ultrasound: The ultrasound guided group will have an ultrasound to identify the optimal site for biopsy and guide the procedure for safety purposes. | 16 |
| Non-Ultrasound Guided Group The control group will have non-ultrasound guided biopsies performed, which is the current standard of care. | 24 |
| Total | 40 |
Baseline characteristics
| Characteristic | Non-Ultrasound Guided Group | Ultrasound Guided Biopsy | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 3 Participants | 10 Participants | 13 Participants |
| Age, Categorical Between 18 and 65 years | 21 Participants | 6 Participants | 27 Participants |
| Age, Continuous | 55.375 years STANDARD_DEVIATION 12.8 | 56.1875 years STANDARD_DEVIATION 19.8 | 55.7 years STANDARD_DEVIATION 15.7 |
| Gender Female | 17 Participants | 9 Participants | 26 Participants |
| Gender Male | 7 Participants | 7 Participants | 14 Participants |
| Region of Enrollment United States | 24 participants | 16 participants | 40 participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 16 | 0 / 24 |
| serious Total, serious adverse events | 0 / 16 | 0 / 24 |
Outcome results
Primary
Amount of Tissue Obtained
This data will be analyzed to determine if ultrasound guidance improves muscle yield (as measured by pathology determined volume and mass).
Time frame: At time of biopsy
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ultrasound Guided Biopsy | Amount of Tissue Obtained | 0.29 grams | Standard Deviation 0.23 |
| Non-Ultrasound Guided Group | Amount of Tissue Obtained | 0.28 grams | Standard Deviation 0.21 |
Primary
Number of Patients Receiving Diagnosis From Muscle Biopsy
The rate of achieving a specific final diagnosis in ultrasound guided muscle biopsies vs. unguided biopsies will be examined.
Time frame: At time of biopsy
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ultrasound Guided Biopsy | Number of Patients Receiving Diagnosis From Muscle Biopsy | 7 participants |
| Non-Ultrasound Guided Group | Number of Patients Receiving Diagnosis From Muscle Biopsy | 12 participants |
Secondary
Number of Participants With Adverse Events Related to Muscle Biopsy
Data will be examined to determine if ultrasound guidance reduces the rate of adverse events in muscle biopsies.
Time frame: Patient involvement limited to the time of biopsy; Records analyzed up to 10 months after biopsy
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ultrasound Guided Biopsy | Number of Participants With Adverse Events Related to Muscle Biopsy | 0 participants |
| Non-Ultrasound Guided Group | Number of Participants With Adverse Events Related to Muscle Biopsy | 0 participants |
Secondary
Number of Participants With Inadequate Biopsy Samples
The presence of an inadequate sample was determined by the blinded pathologist reading the muscle biopsies. This reflects the sample having enough preserved muscle tissue for histologic analysis. It is separate from the number of participants receiving a diagnosis. A sample may be adequate, but non-diagnostic. Only one biopsy was performed in each patients, so the number of biopsies is the same as the number of participants.
Time frame: At time of pathology review
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ultrasound Guided Biopsy | Number of Participants With Inadequate Biopsy Samples | 1 Inadequate Biopsy Samples |
| Non-Ultrasound Guided Group | Number of Participants With Inadequate Biopsy Samples | 2 Inadequate Biopsy Samples |
Secondary
Number of Times Biopsy Needle Was Inserted to Obtain Biopsy Tissue
For core or needle biopsies, the physician passes the needle into the muscle until they feel that adequate tissue samples have been obtained. This outcome measure is the number of passes required in each study arm.
Time frame: At time of biopsy
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ultrasound Guided Biopsy | Number of Times Biopsy Needle Was Inserted to Obtain Biopsy Tissue | 2.57 needle passes | Standard Deviation 0.756 |
| Non-Ultrasound Guided Group | Number of Times Biopsy Needle Was Inserted to Obtain Biopsy Tissue | 2.5 needle passes | Standard Deviation 0.761 |