Muscular Dystrophy (MD)

  1. What is the first-line treatment for MD?

    1. glucocorticosteroids

  2. When should we initiate GCs?

    1. before substantial physical decline

  3. What are alternative treatments for MD?

    1. Disease-modifying therapies

  4. What is the goal for the use of GCs?

    1. improve muscle strength

  5. What are the benefits with the use of GCs?

    1. Improve motor function • Improve pulmonary function • Reduce risk of scoliosis •Delay loss of ambulation • May delay progression of cardiomyopathy → improve survival

  6. What are the ADEs with GC use for MS?

    1. Weight gain, Growth suppression, Hirsutism, Delayed puberty, Behavioral changes, Bone fractures, Acne, GI symptoms, Cataracts, & Cushing-like appearance

  7. What do you do if a patient experiences intolerable ADEs?

    1. Decrease dose by 25-33% then Reassess in 2-3 months and avoid rapid withdrawl

  8. what are the types of GCs used for MD?

    1. prednisone, Deflazacort, and Vamorolone

  9. What is the major ADE for prednisone?

    1. More weight gain

  10. What is the major ADE for Deflazacort

    1. More growth suppression

  11. What is the major ADE for Vamorolone

    1. Less adrenal side effects

  12. Which of the following is true about GCs?

    1. GC can lengthen survival

  13. A patient has been treated with prednisone 15 mg daily for 15 months, but experiencing severe GI symptoms and Behavioral problems. which would be the best recommendation?

    1. This decrease the dose of prednisone to 11 mg daily (decrease by 25-30%)

  14. Which medication is the first orally available therapy used in muscular dystrophy?

    1. Givinostat

  15. What should be monitored in patients taking Givinostat?

    1. Baseline platelet count [do not initiate if platelets are < 150 × 109/L], triglycerides • ECG in patients heart isses or on CV meds.

  16. What disease modifying therapies are known as exon-skipping therapies?

    1. Eteplirsen(ETE), Golodirsen (GOL), Vitolarsen(VIT), & Casimersin(CAS)

  17. Which antisense oglionucleotide disease-modifying agent does NOT have an ADE for renal toxicity?

    1. Eteplirsen

  18. Which Antisense Oligonucleotides have Renal Toxicity?

    1. Golodirsen, viltolarsen, casimersen

  19. What do we want to monitor in patients taking Golodirsen, viltolarsen, casimersen?

    1. serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio prior to starting therapy

  20. Why is Creatinine not a reliable measurement of kidney function for patients with MD?

    1. due to low skeletal muscle mass

  21. A neurologist would like to know the best disease-modifying therapy to start in a patient with chronic kidney disease. which of the following is the most important?

    1. Eteplirsen

  22. What are additional considerations for monitoring MD?

    1. bone health, cardiac dysfunction, vaccinations

  23. Which vaccinations should be avoided if the patient is on an antisense oligonucleotide?

    1. Botulinum toxin, Succinylcholine, & Inhalation anesthetics

  24. How can we manage bone health?

    1. use Vitamin D supplementation if sc is < 20ng/mL , Calcium supplementation, and oral bisphosphonates

  25. How can we manage Cardiac dysfuction?

    1. consider diuretics, ACEis, BBs, anticoagulation and starting therapy when EF < 55%

  26. How can we manage vaccinations?

    1. Administer attenuated vaccines > 4 weeks prior to starting immunosuppressive therapy/doses of steroids