Thorax. 2001 Apr;56(4):314-6.

Randomised controlled trial; parallel design.

Inclusion criteria: CF; no organ transplantation; 70% of all eligible participants in a longitudinal BMD study recruited after one year of follow-up; no prior treatment with bone sparing agents; BMD Z-score of < or = -2 at lumbar spine, proximal femur or N = 31 (15 in treatment group); 9 female (more in treatment group but exact number not reported). Mean (SD) age 26.1 (5.8) years; BMI 21.1 (2.7) kg/m2; FEV1 50.9 (20.3) % of predicted treatment. Groups similar with respect to age, initial BMD, bone biochemistry and respiratory disease severity. Single centre, UK. 3 participants (2 from treatment group) withdrew.

All participants with pancreatic insufficiency (relevant to all except one in control group) continued long term oral vitamin D (900 IU/day); all participants in both groups received oral calcium (1g daily). Intravenous pamidronate 30 mg every 3 months for 6 months (2 doses).

BMD (distal radius, ultradistal radius; 0, 6 months; SXA); Adverse events (bone pain); Withdrawals (total, due to adverse events); BMD (lumbar spine; proximal femur (total hip); 0, 6 months; DXA Hologic QDR 4500 Waltham MA); Survival.

Trial duration planned for 1 year, but was shortened to 6 months because of adverse events. After 6 months of treatment the pamidronate group (n=13) showed a significant increase in absolute BMD compared with the control group (n=15) in the lumbar spine (mean difference 5.8% (CI 2.7% to 8.9%)) and total hip (mean difference 3.0% (CI 0.3% to 5.6%)). However, the pamidronate group showed a reduction in BMD compared with the control group in the distal forearm (mean difference -1.7% (CI -3.7% to 0.3%)). The use of pamidronate was associated with a high incidence of bone pain in non-corticosteroid treated individuals.

Intravenous pamidronate increases axial BMD in adults with cystic fibrosis, but the high incidence of bone pain associated with this treatment might limit its use.