Treatment of Low Bone Density in Cystic Fibrosis.
Treatment of Low Bone Density in Cystic Fibrosis.
Cystic fibrosis (CF) -- an autosomal recessive genetic disease affecting about 60,000
 individuals worldwide, including about 3,800 in Italy -- is often associated with low bone
 mineral mass. The current aggressive therapies have ensured a much longer survival of CF
 patients but this has led to a higher frequency of osteoporosis and bone fractures, a serious
 problem which not only affects quality of life, but also hinders further therapeutic
 measures.

 The aim of this study, conducted on a large group of children, adolescents and young adults
 with CF, has been the evaluation of bone mass changes after 1 year of a simple treatment with
 RDA-adjusted dietary calcium plus 25-OH vitamin D supplementation, and the feasibility and
 efficacy of alendronate treatment (for another year) in patients not responding to calcium +
 25-OH vitamin D alone.
The study included 2 phases.

 Phase 1 (1-year open-label observational study): following baseline evaluation, bone mass
 changes have been studied with a simple therapy of adequate calcium intake and 25-OH vitamin
 D supplements in all eligible subjects (N=171).

 Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study): the 128
 subjects showing an insufficient response to calcium + 25-OH vitamin D alone (bone mass
 increase <5%) at the end of Phase 1, were randomized into 2 groups and assigned to
 alendronate treatment (N=65) or placebo (N=63) (in addition to calcium and 25-OH vitamin D as
 during Phase 1).

 The study has been carried out by the Coordinator's Institution (Istituto Auxologico
 Italiano)in collaboration with most Regional Reference Centers for CF in Italy.
Inclusion Criteria:

 -  age 2-30 years

 -  clinically stable condition

 -  regular menses in females

 -  low Bone Mineral Apparent Density for age (defined as BMAD Z-score ≤-2.0 if age ≤18
 years or ≤-2.5 if age >18 years).

 Exclusion Criteria:

 -  two or more episodes of hypercalcemia and/or hypercalciuria

 -  contraindications to 25-OH vitamin D or alendronate treatment

 -  recent transplantation

 -  other diseases or medications (glucocorticoids excepted) associated with bone loss.