Palynziq clinic: One year and 43 patients later.

Autor: Lah M; Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis, IN, United States of America. Electronic address: mspurr@iu.edu., McPheron M; Department of Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis, IN, United States of America.
Jazyk: angličtina
Zdroj: Molecular genetics and metabolism [Mol Genet Metab] 2021 Jul; Vol. 133 (3), pp. 250-256. Date of Electronic Publication: 2021 May 19.
DOI: 10.1016/j.ymgme.2021.05.006
Abstrakt: Pegvaliase-pqpz (Palynziq) is an enzyme substitution therapy FDA approved May 2018 to treat phenylketonuria in adults with blood phenylalanine levels greater than 600 μmol/L (10 mg/dL). Pegvaliase is administered via subcutaneous injection and carries a high risk of side effects including anaphylaxis. A consensus statement on its use recommends careful education and monitoring of patients. We established a dedicated Palynziq Clinic in October 2018 with detailed protocols to minimize these risks. In the first year, we evaluated 43 patients, initiated Palynziq in 37 and transitioned two trial patients to commercial drug. 13/37 patients (35.1%) have sustained blood phenylalanine levels <360 μmol/L (6 mg/dL) without adjunct sapropterin dihydrochloride treatment or medical food. The timing and dosage needed to achieve a response did not correlate with patient weight, starting phenylalanine level, starting diet, or co-treatment with sapropterin dihydrochloride. Some patients had consistently low phenylalanine levels <30 μmol/L (0.5 mg/dL) and required doses as low as 20 mg weekly. Anaphylactic episodes were reported by 21.6% (8/37 patients) versus 10% seen in the clinical trial. Rates of other side effects were similar to or less than those in the trial. Adverse reactions commonly occurred shortly after dosage increases. We provide a model for safely introducing and managing pegvaliase in adult patients with PKU.
Competing Interests: Declaration of Competing Interest Dr. Lah has received compensation as a member of advisory boards for BioMarin Pharmaceutical Inc. and as a speaker at a promotional event to discuss her clinical experience with Palynziq. She also serves as a primary investigator on three clinical trials sponsored by BioMarin Pharmaceutical Inc. Dr. McPheron serves as a co-investigator on a clinical trial sponsored by BioMarin. Pharmaceutical Inc.
(Copyright © 2021 Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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