TREATING A PATIENT WITH THE WERNER SYNDROME AND OSTEOPOROSIS USING RECOMBINANT HUMAN INSULIN-LIKE GROWTH-FACTOR

Objective: To assess the safety and effect of recombinant human insuli n-like growth factor 1 (rhIGF-1) on measures of bone metabolism in a h uman model of age-related osteoporosis. Design: 6-month prospective ca se study. Setting: General clinical research center. Patients: 1 patie nt with the Werner syndrome, a low serum IGF-1 level, and osteoporosis . Intervention: Daily subcutaneous administration of rhIGF-1 for 6 mon ths. Measurements: Serum alkaline phosphatase, osteocalcin, type I pro collagen C-peptide and urinary hydroxyproline, calcium, and pyridinoli ne cross-links as measures of bone metabolism and radial shaft, femora l neck, and lumbar bone masses. Results: Serum osteocalcin and type I procollagen C-peptide increased during rhIGF-1 therapy (P < 0.05). Twe nty-four hour urinary calcium, hydroxyproline, and pyridinoline cross- links were also higher after treatment than they were before treatment (P < 0.05). During 6 months of treatment, the bone mineral density of the L2 to L4 vertebrae increased 3%; this value exceeded the coeffici ent of variation of this measurement. Bone density at the femoral neck and radial shaft changed by less than the coefficient of variation of these measurements. No significant changes in serum glucose values or other adverse effects of treatment were noted. Conclusions: Treatment with rhIGF-1 increased both bone formation and resorption in a patien t with the Werner syndrome, a low baseline serum IGF-1 level, and esta blished osteoporosis. Because lumbar bone mass increased without evide nce of bone loss in the appendicular skeleton, a net increase in bone formation (formation greater than resorption) may have been responsibl e.