Clinical efficacy of "Nonphenic", a line of specialty foods for nutritional care in children, adults, and pregnant women with phenylketonuria: study results

Clinical efficacy of "Nonphenic", a line of specialty foods for nutritional care in children, adults, and pregnant women with phenylketonuria: study results

N.A. Semenova¹, E.A. Shestopalova¹, G.V. Baydakova¹, T.P. Zhukova², S.Yu. Ratnikova², E.S. Zaytseva², L.R. Nurgalieva³, S.I. Kutsev¹

¹Research Center for Medical Genetics, Moscow, Russian Federation

²V.N. Gorodkov Ivanovo Research Institute for Maternity and Childhood, Ivanovo, Russian Federation

³Republican Medical Genetic Center, Ufa, Russian Federation

Background: phenylketonuria (PKU) was the first hereditary metabolic disease for which high diet efficacy preventing its symptoms was demonstrated. A low-protein diet and specialty foods lacking phenylalanine should be prescribed as early as possible to control plasma PKU levels. PKU treatment is challenging due to low adherence to the diet. New specialty foods were introduced to improve the quality of medical care for PKU.

Aim: to assess the efficacy of specialty foods for nutritional care in children, adults, and pregnant women with PKU.

Patients and Methods: open-label, multicenter, prospective approbation was performed in outpatient facilities. The study included 60 patients. Nine patients received "Nonphenic^®-13˝ designed for children under one year. Six patients received "Nonphenic^®-20˝ designed for children aged 1–3 years. Nineteen patients received "Nonphenic^®-40˝ designed for children aged 3–7 years. Eighteen patients received "Nonphenic^®-70˝ designed for children over seven years. Eight patients received "Nonphenic^®-75˝ designed for children over seven years, adults, and pregnant women. The treatment duration was 30±2 days. Clinical efficacy and safety were evaluated according to the Good Clinical Practice principles and based on clinical and laboratory findings.

Results: mean PKU levels reduced (significantly in patients who received "Nonphenic^®-70˝ and "Nonphenic^®-75˝) and were within safe ranges. Tyrosine levels were similar at the first and second visits and were within normal ranges. Parents and physicians were completely satisfied with the study products. Moreover, parents reported rapid adaptation to the novel food. No serious adverse events related to the study product which required treatment cancellation were reported during clinical approbation.

Conclusions: this study has demonstrated the clinical efficacy and safety of specialty foods for nutritional care in children, adults, and pregnant women with PKU.

Keywords: phenylketonuria, specialty foods for nutritional care, phenylalanine, diet therapy.

For citation: Semenova N.A., Shestopalova E.A., Baydakova G.V. et al. Clinical efficacy of ˝Nonphenic˝, a line of specialty foods for nutritional care in children, adults, and pregnant women with phenylketonuria: study results. RMJ. 2022;3:4–8.