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The Impact of a Slow-Release Large Neutral Amino Acids Supplement on Treatment Adherence in Adult Patients with Phenylketonuria.
Burlina, Alessandro P; Cazzorla, Chiara; Massa, Pamela; Loro, Christian; Gueraldi, Daniela; Burlina, Alberto B.
Affiliation
  • Burlina AP; Neurological Unit, St. Bassiano Hospital, Via dei Lotti 40, 36061 Bassano del Grappa, Italy.
  • Cazzorla C; Division of Inherited Metabolic Diseases, Department of Women and Children's Health, University Hospital of Padova, Via Giustiniani 3, 35128 Padova, Italy.
  • Massa P; Division of Inherited Metabolic Diseases, Department of Women and Children's Health, University Hospital of Padova, Via Giustiniani 3, 35128 Padova, Italy.
  • Loro C; Division of Inherited Metabolic Diseases, Department of Women and Children's Health, University Hospital of Padova, Via Giustiniani 3, 35128 Padova, Italy.
  • Gueraldi D; Division of Inherited Metabolic Diseases, Department of Women and Children's Health, University Hospital of Padova, Via Giustiniani 3, 35128 Padova, Italy.
  • Burlina AB; Division of Inherited Metabolic Diseases, Department of Women and Children's Health, University Hospital of Padova, Via Giustiniani 3, 35128 Padova, Italy.
Nutrients ; 12(7)2020 Jul 14.
Article in En | MEDLINE | ID: mdl-32674279
ABSTRACT
The gold standard treatment for phenylketonuria (PKU) is a lifelong low-phenylalanine (Phe) diet supplemented with Phe-free protein substitutes. Adherence to therapy becomes difficult after childhood. Supplementing with large neutral amino acids (LNAAs) has been proposed as an alternative medication to Phe-free protein substitutes (i.e., amino acid mixtures). The aim of this study was to evaluate adherence to therapy and quality of life (QoL) in a cohort of sub-optimally controlled adult PKU patients treated with a new LNAA formulation. Twelve patients were enrolled in a 12-month-trial of slow-release LNAAs (1g/kg/day) plus a Phe-restricted diet. Medication adherence was measured with the Morisky Green Levine Medication Adherence Scale; the QoL was measured using the phenylketonuria-quality of life (PKU-QoL) questionnaire. Phe, tyrosine (Tyr) levels, and Phe/Tyr ratios were measured fortnightly. Before treatment, 3/12 patients self-reported a 'medium' adherence to medication and 9/12 reported a low adherence; 60% of patients reported a full adherence over the past four weeks. After 12 months of LNAA treatment, all patients self-reported a high adherence to medication, with 96% reporting a full adherence. Phe levels remained unchanged, while Tyr levels increased in most patients. The Phy/Tyr ratio decreased. All patients had a significant improvement in the QoL. LNAAs may give patients a further opportunity to improve medication adherence and, consequently, their QoL.
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Full text: 1 Database: MEDLINE Main subject: Phenylalanine / Phenylketonurias / Dietary Proteins / Dietary Supplements / Amino Acids, Neutral / Treatment Adherence and Compliance Type of study: Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Limits: Adult / Female / Humans / Male Language: En Year: 2020 Type: Article

Full text: 1 Database: MEDLINE Main subject: Phenylalanine / Phenylketonurias / Dietary Proteins / Dietary Supplements / Amino Acids, Neutral / Treatment Adherence and Compliance Type of study: Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Limits: Adult / Female / Humans / Male Language: En Year: 2020 Type: Article