AMINOHYDROXY PROPYLIDENE BISPHOSPHONATE (APD) TREATMENT IMPROVES THE CLINICAL SKELETAL MANIFESTATIONS OF GAUCHERS-DISEASE

Objective. To evaluate the long-term effects and safety of aminohydrox y propylidene bisphosphonate (APD) treatment on the frequency and seve rity of the clinical skeletal manifestations of Gaucher's disease. Met hodology. Five adolescents who suffered from recurrent bone crisis epi sodes and atraumatic bone fractures due to Gaucher's disease were trea ted with APD for 14 to 83 months. Results. During the 6 years before t reatment, the patients suffered from 6 to 17 bone crisis episodes, or 1 to 2.8 episodes per patient per year. Three patients were free from bone crisis episodes during 14 to 32 months of ADP treatment, while tw o patients had two such episodes during 60 and 83 months of APD treatm ent (these represent a decrease in bone crisis episodes from 1 and 2.8 per year to 0.4 and 0.3 per year, respectively). Although four patien ts suffered from 1 to 3 atraumatic bone fractures during the 6 years p receding treatment (a total of 10 fractures), only one patient sustain ed a fracture on APD treatment (total of 219 months of treatment). Usi ng APD was not associated with clinical side effects, biochemical aber rations, significant changes in liver and kidney function, or changes in serum levels of the hormones regulating mineral metabolism. In all patients, a band-like metaphyseal sclerosis appeared on radiography of the long bone. However, APD did not interfere with bone growth. Concl usions. The marked clinical improvement in the clinical skeletal manif estations of Gaucher's disease and the absence of toxic side effects i n adolescent patients treated with APD support previous findings in th ree adult patients on the efficacy of APD and indicate possibilities f or its use in inducing prolonged remissions in affected patients.