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Use of pegvaliase in the management of phenylketonuria: Case series of early experience in US clinics. | LitMetric

AI Article Synopsis

  • The study presents a case series highlighting the initial experiences of 16 healthcare providers from the US who treated patients with phenylketonuria (PKU) using pegvaliase since its FDA approval in May 2018.
  • The review includes 15 cases of adults with PKU, showcasing various challenges such as financial issues and comorbidities, but emphasizes that successful treatment is still achievable with proper patient education and individualized care.
  • Findings suggest that treatment effectiveness and dietary adjustments should be continuously assessed throughout therapy, and despite some adverse effects, most patients were eager to continue treatment and achieved positive outcomes.

Article Abstract

Objective: To present a case series that illustrates real-world use of pegvaliase based on the initial experiences of US healthcare providers.

Methods: Sixteen healthcare providers from 14 centers across the US with substantial clinical experience in treating patients with phenylketonuria (PKU) with pegvaliase in the two-plus years since FDA approval (May 2018) provided cases that exemplified important lessons from their initial experiences treating patients with pegvaliase. Key lessons from each case and takeaway points were discussed in both live and virtual meetings.

Results: Fifteen cases of adults with PKU (eight males, seven females), representing a spectrum of age (18 to 53 years), previous PKU care, comorbidities, and socioeconomic situations were reviewed and discussed. Full extended case reports are included in the Supplement. The cases showed that treating patients with a daily injectable can be challenging due to a patient's financial problems, treatment challenges, and neuropsychological and psychiatric comorbidities, which can be identified before starting pegvaliase, but do not prohibit successful treatment. The authors agreed that patient education on adverse events (AEs), time to efficacy, dietary changes, and food preparation is an ongoing process that should start prior to initiating pegvaliase treatment. Treatment goals and planned dietary changes once efficacy is reached should be defined prior to treatment initiation and re-evaluated throughout the course of therapy. Each patient's titration schedule and dietary adjustments are unique, depending on occurrence of AEs and individual goals of treatment. Despite the AE profile of pegvaliase, all but two patients remained motivated to continue treatment and achieved efficacy (except one patient in whom titration was still ongoing). AEs occurring early in the treatment pathway may require prolongation of the titration phase and/or concomitant medication use, but do not seem indicative of future tolerability or eventual efficacy. Close follow-up of patients during titration and maintenance to help with dietary changes is important.

Conclusion: This case series provides real-world experience on the use of pegvaliase. Until data from registries and independent research become available, the data presented herein can support appropriate management of patients receiving pegvaliase in clinical practice.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8369061PMC
http://dx.doi.org/10.1016/j.ymgmr.2021.100790DOI Listing

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