Patisiran

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Patisiran Brand Name– ONPATTRO

What is Patisiran

Patisiran is a parenteral small interfering ribonucleic acid (siRNA) indicated for the treatment of peripheral nerve disease (polyneuropathy) due to hereditary transthyretin-mediated amyloidosis (hATTR) in adults.

In a randomized, placebo-controlled trial, patisiran-treated patients experienced significantly greater improvements in the modified Neuropathy Impairment Score +7 (mNIS+7) and the Quality of Life-Diabetic Neuropathy (QoL-DN) total score after 18 months of treatment compared to patients who received placebo.

Improvements in these scores reflect gains in physiological outcomes (such as muscle strength, reflexes, sensation) and functional outcomes (such as walking, nutritional status, and performance of activities of daily living) with patisiran treatment.

Infusion-related adverse reactions are common with patisiran; premedication is necessary for all patients

Indications

  • amyloidosis-associated polyneuropathy

For the treatment of hereditary transthyretin amyloidosis-associated polyneuropathy

Side Effects

  1. abdominal pain
  2. antibody formation
  3. arthralgia
  4. AV block
  5. back pain
  6. blurred vision
  7. chest pain (unspecified)
  8. chills
  9. cough
  10. dizziness
  11. dyspepsia
  12. dyspnea
  13. edema
  14. erythema
  15. fatigue
  16. flushing
  17. headache
  18. hypertension
  19. hypotension
  20. infection
  21. infusion-related reactions
  22. injection site reaction
  23. muscle cramps
  24. musculoskeletal pain
  25. nausea
  26. palpitations
  27. pharyngitis
  28. phlebitis
  29. rash
  30. rhinitis
  31. sinusitis
  32. syncope
  33. vertigo
  34. xerophthalmia

Monitoring Parameters

  • laboratory monitoring not necessary

Contraindications

  • breast-feeding
  • infusion-related reactions
  • pregnancy
  • vitamin A deficiency

Interactions

There are no drug interactions associated with Patisiran products.

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What is recurrence

What is recurrence?  Recurrence refers to an initial reduction in...

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