Aminohydroxy propylidene bisphosphonate (APD) treatment improves the clinical skeletal manifestations of Gaucher's disease.

OBJECTIVE

To evaluate the long-term effects and safety of aminohydroxy propylidene bisphosphonate (APD) treatment on the frequency and severity of the clinical skeletal manifestations of Gaucher's disease.

METHODOLOGY

Five adolescents who suffered from recurrent bone crisis episodes and atraumatic bone fractures due to Gaucher's disease were treated with APD for 14 to 83 months.

RESULTS

During the 6 years before treatment, 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 two patients had two such episodes during 60 and 83 months of APD treatment (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 patients suffered from 1 to 3 atraumatic bone fractures during the 6 years preceding treatment (a total of 10 fractures), only one patient sustained a fracture on APD treatment (total of 219 months of treatment). Using APD was not associated with clinical side effects, biochemical aberrations, 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.

CONCLUSIONS

The marked clinical improvement in the clinical skeletal manifestations of Gaucher's disease and the absence of toxic side effects in adolescent patients treated with APD support previous findings in three adult patients on the efficacy of APD and indicate possibilities for its use in inducing prolonged remissions in affected patients.