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A4.4 Zoledronate efficacy and safety in active paget’s disease- long-term follow-up and retreatment in clinical practice
  1. E Vieira-Sousa1,2,
  2. A Rodrigues1,2,
  3. J Caetano Lopes2,
  4. S Capela1,
  5. F Ramos1,
  6. R Figueira1,
  7. J Polido-Pereira1,2,
  8. C Ponte1,2,
  9. R Campanilho-Marques1,2,
  10. R Barros1,
  11. J C Romeu1,
  12. J A Pereira da Silva1
  1. 1Rheumatology and Metabolic Bone Diseases Department, Hospital de Santa Maria, Centro Académico de Medicina de Lisboa, Portugal
  2. 2Rheumatology Research Unit, Instituto de Medicina Molecular, Faculdade de Medicina da Universidade de Lisboa, Centro Académico de Medicina de Lisboa, Portugal

Abstract

Background Bisphosphonates are first line therapy in the treatment Paget’s Disease (PD). The objective of this observational study was to assess short and long-term efficacy and safety of zoledronate in the treatment of active PD.

Methods Patients with active PD treated with zoledronate 5 mg were consecutively recruited and followed prospectively. Clinical and laboratory parameters (alkaline phosphatase (ALP), bone specific alkaline phosphatase (BSALP), procollagen type 1 N-terminal propeptide (P1NP), collagen type 1 beta C-terminal telopeptide (b-CTX), seric and urinary calcium and phosphorus and parathormone levels) were determined before, at 3 and every 6 months after treatment. Remission was defined as normalization of ALP. Retreatement was considered when ALP levels increased more than 25% of the upper limit of normal or of the nadir achieve in cases of non normalization of ALP. Adverse events were registered according to clinical protocol.

Results 60 patients (60% males), with a mean age of 68 ± 11 years and a mean disease duration of 11 ± 9 years were included. 69% had polyostotic disease and a mean percentage of skeletal involvement of 10.8 ± 7.6%. 68% were symptomatic: 71% of those referring bone and 54% joint pain attributed to PD. 48.3% had been previously treated with pamidronate (cumulative dose 234 ± 209 mg). The mean follow-up period after zoledronate infusion was of 37 ± 13 months. Only 4 patients (6.6%) required retreatment, on average 30 months after the first zoledronate infusion. A marked reduction of ALP) was observed at 3 (70%) and 6 months (74%) being maximal at 12 months (75%) (p < 0.001). At 3 and 6 months, 95% and 96% of patients respectively, achieved remission. Maximum effect was obtained at 12 months after treatment with 98% of patients being in remission. Significant reductions of the mean levels of BSALP, P1NP, and b-CTX (p < 0.001) were also verified at 3, 6 and 12 months after treatment. 47% of patients reported pain improvement: 89% at 3 months, 7% at 6 months and 4% at 12 months. Transitory side effects were registered in 15 patients, 18% referred flu-like symptoms, 10% showed asymptomatic hypocalcaemia and 30% asymptomatic hypophosphoremia.

Conclusions This study confirms the efficacy and safety of zoledronate in a Portuguese population of patients with active Paget’s disease. Biochemical remission was achieved in 98% of patients at 12 months and improvement of pain in 47%, the majority 3 months after treatment. Furthermore these benefits were long-term sustained with only 6.6% of patients requiring retreatment during an average follow-up of 37 months.

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