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Real-World Experience With VMAT2 Inhibitors.

Nicki Niemann1, Joseph Jankovic

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Vesicular monoamine transporter 2 (VMAT2) inhibitors effectively treat hyperkinetic movement disorders, showing significant improvement in patient-reported illness severity. However, access in the U.S. is limited for unapproved uses.

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Area of Science:

  • Neurology
  • Pharmacology
  • Movement Disorders

Background:

  • Vesicular monoamine transporter 2 (VMAT2) inhibitors, including tetrabenazine (TBZ), deutetrabenazine (DTBZ), and valbenazine (VBZ), are used to manage hyperkinetic movement disorders.
  • Patient access and adherence to these VMAT2 inhibitors can be challenging due to factors like insurance, physician inexperience, efficacy, and side effects.

Purpose of the Study:

  • To evaluate the real-world effectiveness and safety of VMAT2 inhibitors (TBZ, DTBZ, VBZ) in treating hyperkinetic movement disorders.
  • To identify barriers to VMAT2 inhibitor access and adherence.

Main Methods:

  • A retrospective chart review was conducted.
  • Patient data were supplemented with questionnaire responses.
  • The study period was from January 1, 2017, to August 30, 2018.

Main Results:

  • 135 patients received 178 VMAT2 inhibitor prescriptions, with DTBZ being the most common (58.4%).
  • Tourette syndrome/tics was the most frequent diagnosis (49.6%).
  • VMAT2 inhibitors significantly improved hyperkinetic movement disorder symptoms, with 60.9%-71.9% of patients showing improvement on a Likert scale. Side effects were generally mild and manageable.

Conclusions:

  • VMAT2 inhibitors demonstrate effectiveness and safety across various hyperkinetic movement disorders.
  • Patient access to VMAT2 inhibitors in the U.S. is restricted for conditions not approved by the FDA.