Effectiveness outcomes | Safety outcomes |
---|---|
Laboratory assessments: • Serum oligosaccharides (µmol/L) • Serum IgG, IgA, and IgM Functional assessments: • 3MSCT (steps/min) • 6MWT (m)a • 2MWT (m)a • FVC (L and % of predicted)a Health Assessment Questionnaires: • EQ-5D-5L • Zarit Burden Interview • QoL questionnaire • Behavior checklists for child and adult Other: • Psychotic events (rate) | • AEs: serious and nonserious • ADRs: including serious and nonserious • AEs leading to treatment discontinuation and death • Any identified risks including anti-VA-IgG antibody, infusion-related reactions, and hypersensitivity • Any potential risk of acute renal failure, loss of consciousness, and medication errors • Vital signs: SBP, DBP, and pulse rate • Electrocardiogram • Laboratory tests (hematology and chemistry) • Physical examination |