Disease severity in children and adults with Pompe disease related to age and disease duration

Disease severity in children and adults with Pompe disease related to age and disease duration

Hagemans ML; Winkel LP; Hop WC; Reuser AJ; Van Doorn PA; Van der Ploeg AT

Neurology

2005

Child; Cross-Sectional Studies; Female; Humans; infant; Male; Adult; Questionnaires; Aged; Middle Aged; Disease Progression; Age Factors; Respiration; Severity of Illness Index; Activities of Daily Living; adolescent; Preschool; 80 and over; infant; Q3 Literature Search; Newborn; Age of Onset; Muscle; Artificial/statistics & numerical data; Glycogen Storage Disease Type II/diagnosis/epidemiology/physiopathology; Respiratory Insufficiency/epidemiology/etiology/therapy; Skeletal/physiopathology; Wheelchairs/statistics & numerical data

Information about 255 children and adults with Pompe disease was gathered through a questionnaire. Disease severity was associated with disease duration and not with age; an early manifestation of the disease implied earlier wheelchair or ventilator dependency. The patient group under age 15 included a subgroup with a more severe and rapid course of the disease. They require more intensive follow-up and early intervention, before irreversible damage has occurred.

2005

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