Browsing by Author "Andersson, Hans C."

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    Lessons learned from 5 years of pegvaliase in US clinics: A case series
    (Elsevier, 2024-12-26) Cooney, Erin; Ammous, Zineb; Bender, Tricia; Clague, Gillian E.; Clifford, Marilyn; Crutcher, Angela; Davis-Keppen, Laura; Havens, Kirsten; Lah, Melissa; Sacharow, Stephanie; Sanchez-Valle, Amarilis; Vucko, Erika; Wardley, Bridget; Wessenberg, Leah; Andersson, Hans C.; Medical and Molecular Genetics, School of Medicine
    Objective: To provide insights and strategies for pegvaliase management in challenging cases with phenylketonuria (PKU) based on the first 5 years of experience with pegvaliase in real-world clinical practice. Methods: Twelve PKU experts gathered during a one-day, in-person meeting to discuss clinical cases illustrating important lessons from their experiences treating patients with pegvaliase in real-world clinical practice. Challenges with pegvaliase experienced prior to and during treatment and corresponding strategies to overcome them were discussed. Results: Twelve cases of adults with PKU (eight females and four males, aged 18 to 68 years) receiving pegvaliase were reviewed and discussed. Challenges of the cases included medical or mental health comorbidities, executive function deficits, challenging social or socioeconomic situations, logistical or geographic barriers, or a combination of these; one was considering pregnancy. Despite challenges, pegvaliase was initiated successfully in most cases. Strategies to overcome barriers included individualized education, including side effect action plans, help from support organizations, collaboration with local providers, and use of telemedicine. Recommendations from the clinicians included that comorbid conditions should be monitored closely after treatment initiation and may require collaboration with other healthcare providers. A collaborative relationship with the clinic, ongoing education, and supportive relatives or friends can help individuals to remain adherent to pegvaliase. Suboptimal adherence may be addressed by a daily reminder system, telemedicine, in-home support, or a modified titration plan. Treated individuals with eating disorders require additional follow-up and support to achieve a healthy relationship with food. In most cases, including late-diagnosed individuals, reduced blood Phe levels resulted in improved PKU-related symptoms, including neurological issues. Conclusion: Experience from the presented cases and 5 years of expert experience with pegvaliase in the real-world setting provide insight and guidance for healthcare professionals initiating and managing pegvaliase treatment in complex PKU cases. These cases demonstrate that, through comprehensive assessment and addressing barriers, pegvaliase treatment can be successful in adults with PKU, regardless of prior treatment success, age, socioeconomic, cognitive, or executive function challenges, as well as in those with comorbidities or considering pregnancy. Ongoing documentation of clinical experience is crucial for advancing the management of individuals receiving this treatment.
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    Use of pegvaliase in the management of phenylketonuria: Case series of early experience in US clinics
    (Elsevier, 2021-08-14) Adams, Darius; Andersson, Hans C.; Bausell, Heather; Crivelly, Kea; Eggerding, Caroline; Lah, Melissa; Lilienstein, Joshua; Lindstrom, Kristin; McNutt, Markey; Ray, Joseph W.; Saavedra, Heather; Sacharow, Stephanie; Starin, Danielle; Tiffany-Amaro, Jennifer; Thomas, Janet; Vucko, Erika; Wessenberg, Leah B.; Whitehall, Kaleigh; Medical and Molecular Genetics, School of Medicine
    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.