Disclosures: Christi F. Ng, MD, MPH: No financial relationships or conflicts of interest

Background and/or Objectives: To describe the effects reported by caregivers of Rett individuals while taking carbidopa/levodopa. To provide clinicians expectations and a framework for prescribing carbidopa/levodopa. Rett individuals often exhibit one or more features of Parkinsonism, such as freezing, rigidity and/or instability. Such symptoms can impair function and the learning of new motor skills. At our multi-disciplinary Rett clinic, we have been prescribing carbidopa/levodopa, and to date there are no known publications describing the use of carbidopa/levodopa to symptomatically treat Parkinsonism in Rett.

Design: Single-arm, retrospective cohort study

Setting: Outpatient

Participants: 25 subjects with Rett and Parkinsonism

Interventions: Enteral administration of carbidopa/levodopa (1/1/2015 to 12/31/2021)

Main Outcome Measures: Caregiver reports of Parkinsonism and side effects

Results: Average age for a patient starting carbidopa/levodopa was 14 yo. Most common Parkinsonian features: freezing (n=22, 88%), masked facies (n=15, 60%) and rigidity/stiffness (n=12, 48%). Twenty (75%) participants with a reduction of Parkinsonism while on carbidopa/levodopa. N&#3f3 (12%) who discontinued the medication because there were no perceived benefits. N&#3f2 (8%) who stopped the medication because it caused side effects, in addition to the lack of improvement. Side effects were common; eighteen (72%) patients had one or more side effects. Most common side effects: constipation (n=9, 36%), reflux/vomiting (n=11, 44%) and/or abdominal cramping/bloating (n=3, 12%). Other less common side effects: changes in mood/agitation, sedation, and hair loss.

Conclusions: Carbidopa/levodopa is a safe and generally well tolerated medication used to reduce Parkinsonian features exhibited by Rett patients. Improvements include more fluid gait, decreased freezing, increased engagement and increased proficiency of Augmentative & Alternative Communication devices. For most, parents felt comfortable managing side effects, such as constipation, in order to continue the benefits. Given the nature of the study design, only broad inferences may be made. However, this does offer insight and a reasonable treatment option for colleagues who may be observing similar features in their Rett patient population.

Level of Evidence: Level IV

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PM&R Meeting Abstracts - https://pmrjabstracts.org/abstract/reduction-of-parkinsonian-features-in-rett-patients-while-on-carbidopa-levodopa/