درس هایی از کاربرد «pegvaliase-pqpz (PALYNZIQ®)» در بیماری فنیل کتونوری: هزینه ها، منافع و نقشه راه آینده
کد: G-1058
نویسندگان: Seyedeh Maryam Abdollahzadeh © ℗
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دانلود: دانلود پوستر
خلاصه مقاله:
خلاصه مقاله
Abstract Known to be recognized as the first FDA-approved enzyme substitution therapy for treatment of phenylketonuria (PKU) on May 2018, pegvaliase-pqpz (Palynziq®, BioMarin Pharmaceutical Inc.) can be considered an evolution in treatment of the disease. Since then, several adult and a few younger patients were successfully treated with pegvaliase. Almost 6 years of world-wide experience with the novel treatment of PKU learned us much about the new achievements in therapeutics for the inborn error of metabolism. Though may be life-threatening anytime during the treatment period due to anaphylaxis, several advantages including, sustained improvement of phenylalanine (Phe) levels, neurological sequelae, and quality of life, while on a regular (NOT a low-protein) diet has been reported regarding the costs and benefits of the treatment. In this review, the mechanism of action, pharmacokinetic properties, clinical efficacy, and log-term safety, and tolerability profile of pegvaliase are discussed. Indication of prescription, dosing, titration, and discontinuation patterns of pegvaliase, related adverse events (including anaphylaxis and the proposed desensitization protocol), and special considerations of nutrition management of patients on pegvaliase therapy are further reviewed. Moreover, this article provides an overview of patient medication guide and education, instruction for use, safety information, monitoring protocol, models suggested for safely introducing and managing pegvaliase and also evidence- and consensus-based recommendations for the use of pegvaliase. Keywords: Pegvaliase; phenylketonuria; costs; benefits
