Dietary modifications in patients receiving miglustat.

Area of Science:

• Metabolic disorders
• Gastroenterology
• Pharmacology

Background:

• Miglustat therapy for lysosomal storage diseases is associated with significant weight loss and gastrointestinal disturbances.
• Effective management strategies are crucial for patient adherence and therapeutic outcomes.

Purpose of the Study:

• To evaluate the impact of two dietary interventions on body weight and gastrointestinal tolerability during the initial 6 months of miglustat therapy.
• To compare the efficacy of a low-lactose diet versus a low-disaccharide diet in mitigating miglustat-induced side effects.

Main Methods:

• Retrospective analysis of data from 29 patients (21 children/adolescents, 8 adults) with neurolipidoses treated at two UK centers.
• Comparison of body weight changes and gastrointestinal symptom frequency across three diet groups: unmodified, low-lactose, and low-disaccharide.

Main Results:

• Patients on the low-disaccharide diet exhibited a positive mean body weight change (+2.0%), contrasting with weight loss in unmodified (-8.1%) and low-lactose (-4.1% to -5.2%) diet groups.
• The low-disaccharide diet demonstrated the lowest frequency of gastrointestinal effects, while the low-lactose diet also reduced disturbances compared to an unmodified diet.
• No statistically significant difference in body weight change was observed between the different diets (p = 0.062), despite observed trends.

Conclusions:

• Simple dietary modifications, particularly a low-disaccharide diet, can support weight gain aligned with normal growth potential in young patients with lysosomal storage diseases on miglustat.
• Dietary interventions effectively reduce gastrointestinal disturbances associated with miglustat therapy, improving tolerability.