Objective: To determine the efficacy of pamidronate disodium (pamidronate) in treatment of Paget's disease of bone.
Methods: Intravenous drip of pamidronate was given at the dose of 30 - 60 mg per day for 2 - 3 weeks with a total dosage of 90 - 270 (168 +/- 84) mg to 5 patients with Paget's disease of bone, 2 males and 3 females, aged 27 - 74 (61 on average) with the course of disease of 4 - 48 years (24 years on average), all of them having multiple bone lesions, 2 being in grade 4 of pain assessment and bedridden and 3 being in grade 3 with impaired mobility. Pain assessment, biochemical markers of bone turnover, including serum alkaline phosphatase (ALP), carboxy-terminal propeptide of type I collagen (PICP), carboxy-terminal cross-linked telopeptide of type I collagen (ICTP) and urinary hydroxyproline (HOP), and side effects of pamidronate were observed before treatment, by the end of treatment, and 4, 12, 24, and 48 weeks after treatment.
Results: The Pagetic bone pain was significantly reduced by the end of treatment in all patients. The degree of pain assessment declined from grade 4 to grade 2 in 2 patients, and from grade 3 to grade 1 in 3 patients. The mobility was markedly improved without bone pain 12 weeks after treatment and relief of pain lasted for 1 year in all patients. Serum ALP, PICP, ICTP and urinary HOP concentrations were significant decrease by the end of treatment. Moreover, the mean percentage decreases for above biochemical markers at 12 and 24 weeks after treatment were 50% or over. The values of serum ALP, PICP, ICTP and urinary HOP decreased from 398 u/L (median), 818 ng/L (median), (29 +/- 14) ng/L and (71 +/- 16) mg/24 h urine respectively to 159 u/L, 129 ng/L, (12 +/- 4) ng/L and (34 +/- 7) mg/24 h urine respectively 48 weeks after treatment (all P < 0.05). Side effects, including a transient increase in body temperature, skin eruption and pruritus, and a transient increase of serum aspartate aminotransferase concentration, were negligible.
Conclusion: Intravenous infusion of pamidronate is effective on Paget's disease of bone. Pamidronate of the dose of 90 - 270 mg administered within 2 - 3 weeks produces a year-long remission. Side effects of pamidronate are slight and transient.