Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy

This method can help answer key questions regarding treatment response and clinical decline overtime in clinical trials for patients with Duchenne Muscular Dystrophy. The main advantages of this technique are its lower dependency on the patient's compliance, and suitability for both ambulatory and nonambulatory patients. Demonstrating the procedure will be Christine Seppi, a trained physiotherapist for motor function measure from the University Children's Hospital Basal.

Perform the clinical assessment of motor function, by having the patient complete the following tasks, beginning with him lying down on his back. First, he should hold his head in midline position, and then turn it from one side to the other. In this and all subsequent motions, score the patient on a scale of zero to three, based on performance.

Next, have the patient remain laying down with his head in the midline position, and ask him to raise his head and maintain this position. Have him bring one knee up to his chest. Now, with one leg flexed both at the hip and knee at approximately 90 degrees, ask him to place his lower leg parallel to the mat with the foot in plantar flexion.

Then, ask him to perform a maximal dorsiflexion of the foot. Next, the patient should place one upper limb beside his body, and bring his hand to the opposite shoulder. Then, still lying down on his back, he should move his lower limbs half flexed, with the feet resting on the mat, slightly apart.

Ask him to maintain this position, and raise his pelvis. Next, ask the patient to lie on his back, and then turn over onto his stomach, and free both of his upper limbs. Then, ask him to lie down on his back and then sit up.

Continue the clinical assessment with the patient sitting on a mat. First, ask him to maintain the seated position while keeping his hands in contact in front of his trunk. While attempting this procedure, it is important to remember to perform it in the given order to avoid unnecessarily repositioning and exhaustion, and to stick to the recommended examination materials and examination steps.

Next, while sitting on the mat, place a tennis ball in front of the patient. He should then touch the ball, and then sit back again. Then, have him sit on the mat with his lower limbs out front, and then stand up.

At this point, ask the patient to stand in front of the chair and then to sit down on the chair and maintain the seated position with his body as straight as possible. Now, ask the patient to sit on the chair with his head in complete flexion. Then, ask him to raise his head, and maintain this position.

At this point in the assessment, ask the patient to sit on the chair in front of a table with his forearms, but not his elbows, placed on the table. Then, ask him to place both hands on top of his head. Now, with the patient in the same position, place a pencil on the table, and ask him to touch the pencil.

Now, place the 10 coins next to his hand, and ask him to pick up coins, and hold them in the same hand. Next, place a CD glued to a piece of cardboard on the table. Ask the patient to place one finger in the center of the CD, and to trace around the edge of the disk with the finger.

Now, hold a pencil and a paper on the table, and ask him to pick up the pencil and draw inside the frame. Then, put a sheet of paper in his hands, and ask him to tear the paper at least four centimeters. Next, place a tennis ball next to the patient's hand.

Ask him to pick up the ball, raise it, and then turn his hand. Then, place a diagram with pictures on the table, and have the patient place a finger in the center of the diagram on the word start and then place his finger on the drawings. At this point, ask the patient to sit on the chair with arms next to his body, and the table at a distance equivalent to the length of his forearm.

Then, ask him to place both hands on the table. Next, ask the patient to sit on equipment adapted to his weight, with both feet on the ground. Then, ask him to stand up.

For this part of the assessment, ask the patient to stand with the upper limbs resting on a piece of equipment for support. Then, ask him to release the support and stand straight. Next, ask the patient to take the same position again, using the equipment for support.

Then, ask him to release the support and raise one foot. For the rest of the assessment, the patient should stand without any support. Ask him to touch the floor with one hand and then stand up again.

Then, ask him to stand and walk 10 steps on his heels. Next, draw a straight line that is about six meters long and two centimeters wide on the floor. Ask the patient to walk along this line.

Then, ask the patient to run. Next, ask the patient to stand on one foot without support, with the other foot off the ground. Ask him to hop in place.

Finally, ask the patient to squat down, and then stand up again. Once the assessment is complete, calculate the motor function measure sub scores by adding the scores of all the items in that domain, dividing it by the maximum score for the domain, and multiplying by 100. Then, to calculate the total score, add the scores of all 32 items, divide the sum by 96, and multiply it by 100.

Here we see images of the motor function measure and six minute walk test, in an eight year old patient with Duchenne Muscular Dystrophy. Numbers in red represent assessment of standing and transfer, and blue and yellow represent assessment of proximal and distal motor function respectively. The first rows represent starting positions, and second rows represent tasks.

Here, the starting position of the six minute walk test is illustrated on the left side, while the right side image shows a patient performing the test on a 30 meter corridor, under supervision of a physiotherapist. This figure shows a representative example of the correlation of baseline quantitative MRI data, with motor function tests in two patients with Duchenne Muscular Dystrophy. Patient 1 with more severe clinical involvement assessed by functional tests, including the motor function measure and the six minute walk test, showed prominent fatty degradation of the thigh muscles, particularly of the abductors.

Patient 2 had better clinical performance, and showed less pronounced fatty degradation of the abductors. For comparison, clinical assessments and qMRI data of all 47 patients at baseline are represented here. Once mastered, this technique can be done in about 45 minutes in ambulatory patients with Duchenne Muscular Dystrophy, if it is performed properly, and there are no further limitations due to the patient's compliance.

Following clinical assessments, objective methods like quantitative muscle MRI can be performed in order to answer additional questions like, subclinical disease progression and their specific treatment. After its development, this technique paved the way for researchers in the field of neuromuscular disorders to perform clinical trials using a sensitive outcome measure that provides reliable information about disease progression, and can predict loss of ambulation. After watching this video, you should have a good understanding on how to perform the motor function measure in a patient with Duchenne Muscular Dystrophy, including assessing standing and transfer, axial and motor function, and distal motor function.

Don't forget that factors that could interfere with your test performance should be avoided, such as wearing uncomfortable clothes, or using slippery examination materials. Further, patient may not use any autotic device while performing this procedure.