Abstract
Pegvaliase (Palynziq®) was FDA approved in 2018 as an enzyme substitution therapy in patients with Phenylketonuria. However, various drug induced hypersensitivity adverse events (HAEs) have been reported. We present a case of Pegvaliase (Palynziq®) induced anaphylaxis and successful desensitization. A 13-step desensitization protocol was performed using three solution concentrations of Palynziq with premedication of diphenhydramine and prednisone in an outpatient setting. The patient tolerated the desensitization and was able to continue Palynziq.
Highlights
Phenylketonuria (PKU) is an autosomal recessive disorder caused by a deficiency of phenylalanine hydroxylase (PAH), which converts the amino acid phenylalanine into tyrosine
We present a 13-step desensitization procedure, which was successfully implemented in the outpatient setting
While a spectrum of hypersensitivity reactions may occur with pegvaliase-pqpz, acute systemic hypersensitivity reactions develop in 0.02%–0.08% of patients [4]
Summary
Phenylketonuria (PKU) is an autosomal recessive disorder caused by a deficiency of phenylalanine hydroxylase (PAH), which converts the amino acid phenylalanine into tyrosine. In patients with PKU, excessive levels of phenylalanine (PHE) can lead to neurological, cognitive, developmental and psychiatric problems. The dietary treatment alone or combined with sapropterin dihydrochloride (Kuvan®) has been used to lower the PHE levels. The poor compliance with diet or lack of responsiveness to Kuvan in some patients limited the treatment success. In 2018, the FDA approved Pegvaliase-pqpz (Palynziq®), the first treatment that lowers the blood PHE level by substituting for the PAH enzyme and offers the possibility to normalize the diet. Various HAEs may occur, ranging from local injection site reactions to systemic anaphylaxis. There is currently no desensitization protocol published for this newly approved pegvaliase-pqpz in the scenario of anaphylaxis. We present a 13-step desensitization procedure, which was successfully implemented in the outpatient setting
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