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Article detail · 2025

Nutritional management and geno-phenotyping of clinical nutrition in patients with glycogen storage diseases type VI and IX

European Journal of Clinical Nutrition

YÖKSİS OpenAlex Open access · hybrid SJR Q1 JCR Q2 Citations 1
Year
2025
ISSN
0954-3007
Type
article

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Abstract

English (OpenAlex)

BACKGROUND/OBJECTIVES: Glycogen storage diseases type VI (GSD-VI) and type IX (GSD-IX) are rare inherited metabolic disorders caused by enzyme deficiencies that disrupt glycogen metabolism. The aim of this study was to analyze the clinical features, nutritional management and geno-phenotyping of clinical nutrition in a cohort of patients with GSD-VI and GSD-IX. SUBJECTS/METHODS: A retrospective cohort study was conducted with 16 patients with GSD-VI and GSD-IX. Demographic characteristics, clinical and laboratory findings, and nutritional treatment outcomes were collected and analyzed. RESULTS: The mean patient age was 10.57 years (±4.81). The distribution of the diagnoses was as follows: GSD-IXa (3), GSD-IXb (6), GSD-IXc (1), and GSD-VI (6). The average age at diagnosis was 36.5 months (±42.2) (13-114 months) in the GSD-VI group. Among the GSD-IX subgroups, the mean age at diagnosis varied: 23.3months (±4.16) for GSD-IXa, 35.7months (±17.5) for GSD-IXb, and 78months for GSD-IXc. Over the course of the study (4.5 ± 1.77 years), protein intake in GSD VI patients increased by 1.05 g/kg/day (91.3% increase), while in GSD IX patients, it rose by 1.09 g/kg/day (94% rise). Uncooked cornstarch (UCS) started at 1 g/kg/day for GSD-VI and 0.85 g/kg/day for GSD-IX, later reduced to 0.71 g/kg/day (29% decrease) and 0.52 g/kg/day (60% reduction), respectively. CONCLUSION: Overall, this paper provides valuable insights into managing GSDVI and GSDIX patients, emphasizing the role of a high-protein diet aligned with the disease's pathophysiology and the potential of genotyping to enhance nutritional treatment protocols.

Topics

  • Glycogen Storage Diseases and Myoclonus
  • Lysosomal Storage Disorders Research
  • Moyamoya disease diagnosis and treatment

Primary topic Glycogen Storage Diseases and Myoclonus

Authors

  1. SEMA KALKAN UÇAR
  2. ALPEREN ELEK
  3. HAVVA YAZICI
  4. YASEMİN HANDE ATİK ALTINOK İZMİR TINAZTEPE ÜNİVERSİTESİ
  5. AYŞE YÜKSEL YANBOLU
  6. FEHİME ERDEM KARAPINAR
  7. MERVE YOLDAŞ ÇELİK
  8. AYÇA AYKUT
  9. ASUDE DURMAZ
  10. EBRU CANDA
  11. MAHMUT ÇOKER