MORBIDITY AND MORTALITY IN THE WOLFRAM-SYNDROME

OBJECTIVE - To determine the major causes of morbidity and mortality i n the autosomal recessive Wolfram syndrome, which is defined by diabet es and bilateral progressive optic atrophy with onset in childhood or adolescence. RESEARCH DESIGN AND METHODS - We abstracted and reviewed the medical records of 68 confirmed cases of Wolfram syndrome identifi ed through a nationwide survey of endocrinologists, ophthalmologists, institutes, and homes for the blind. We also reviewed all available au topsy records. RESULTS - The most common causes of morbidity and morta lity were the neurological manifestations of this syndrome and the com plications of urinary tract atony. There was a lower frequency of diab etic ketoacidosis, no histologically proven diabetic glomerulosclerosi s, and less severe, more slowly progressive, diabetic retinopathy than in classic type I diabetic patients. Mortality in Wolfram syndrome is much higher than in type I diabetes; 60% of Wolfram syndrome patients die by age 35. Recognition of these clinical differences from classic type I diabetes is important for the proper management of Wolfram syn drome patients. CONCLUSIONS - Identification of Wolfram syndrome patie nts among all diabetic patients presenting in childhood or adolescence is important because the management of patients with this syndrome is different from that of patients with classic type I diabetes.