ZOLEDRONIC ACID THERAPY OF PATIENTS WITH PAGET DISEASE OF BONE RESISTANT TO OR WITH UNSUSTAINED REMISSION FOLLOWING...

Area of Science:

• Endocrinology
• Bone Metabolism
• Pharmacology

Background:

• Paget disease (PD) is a chronic bone disorder characterized by abnormal bone remodeling.
• Patients with PD refractory to previous bisphosphonate therapies require alternative treatment options.
• Assessing the efficacy of zoledronic acid (ZA) in such cases is crucial for improving patient outcomes.

Purpose of the Study:

• To evaluate the effectiveness of zoledronic acid (ZA) in patients with Paget disease (PD).
• To assess ZA's impact on patients who did not achieve biochemical remission with prior bisphosphonate therapy or had short remission durations (≤12 months).

Main Methods:

• A cohort of 14 patients (age 54-90) with elevated serum alkaline phosphatase (ALP) and adequate renal function received a single 5 mg infusion of ZA.
• Serum ALP and urine N-telopeptide/creatinine (NTx/Cr) levels were monitored pre-therapy and at regular intervals up to 12 months and beyond.
• Patient data included baseline ALP, remission status, duration of remission, and adverse events.

Main Results:

• Zoledronic acid (ZA) induced biochemical remission in 13 out of 14 patients (93%).
• Nine patients achieved remission for the first time, with durations ranging from 12 to 60 months.
• Four patients with prior remissions ≤12 months experienced more prolonged remissions after ZA therapy.
• One patient showed resistance to ZA therapy despite two treatment courses. Transient hypocalcemia and flu-like symptoms were noted in a few patients.

Conclusions:

• Zoledronic acid (ZA) is highly effective in achieving biochemical remission in patients with Paget disease (PD) resistant to prior bisphosphonate treatments.
• ZA therapy can lead to sustained and prolonged remissions, particularly in patients with a history of short-lived remissions.
• A subset of patients may exhibit resistance to ZA, indicating a need for further research into managing refractory Paget disease.