Treatment of cystic fibrosis related bone disease
Jagdeesh Ullal1, Katherine Kutney2, Kristen M Williams3
1UPMC Center for Diabetes and Endocrinology, University of Pittsburgh Medical Center, 3601 Fifth Ave, Suite 3B, Falk Medical Building, Pittsburgh, PA 15213, USA.
Insights
Cystic fibrosis bone disease (CFBD) is increasingly recognized due to longer lifespans. Management requires tailored approaches, with ongoing research into new treatments beyond bisphosphonates.
Area of Science:
- Pulmonary Medicine
- Endocrinology
- Bone Biology
Background:
- Effective CFTR modulator therapies improve longevity in cystic fibrosis, increasing focus on non-pulmonary complications like CFBD.
- Cystic fibrosis bone disease (CFBD) is a multifactorial condition characterized by hypomineralized bone, leading to reduced strength, poor quality, and increased fracture risk.
- CFBD affects various age groups, necessitating age-specific management strategies that balance treatment benefits against risks.
Purpose of the Study:
- To review current understanding and management strategies for cystic fibrosis bone disease (CFBD).
- To explore emerging pharmacotherapies and the role of CFTR modulators in bone health for individuals with cystic fibrosis.
- To summarize screening, non-pharmacologic, and pharmacologic treatment options for CFBD.
Main Methods:
- Literature review of current research on cystic fibrosis bone disease.
- Analysis of existing guidelines and emerging therapeutic options for osteoporosis.
- Discussion of the potential impact of CFTR modulators on bone health in cystic fibrosis.
Main Results:
- Bisphosphonates remain a primary treatment for CFBD, but long-term effects in CF patients require further investigation.
- Newer osteoporosis agents, including monoclonal antibodies (Denosumab, Romosozumab) and anabolic therapies (teriparatide, abaloparatide), show promise but lack CF-specific data.
- Screening, non-pharmacologic interventions, and various pharmacotherapies are available, with ongoing research into CFTR modulators' effects on bone.
Conclusions:
- Management of CFBD must be individualized, considering patient age and balancing therapeutic risks and benefits.
- Further clinical trials are needed to evaluate the efficacy and safety of novel bone-targeting agents and CFTR modulators in CFBD.
- A comprehensive approach integrating screening, lifestyle modifications, and tailored pharmacotherapy is essential for managing CFBD.
Abstract:
The advent of highly effective CFTR modulator therapies has slowed the progression of pulmonary complications in people with cystic fibrosis. There is increased interest in cystic fibrosis bone disease (CFBD) due to the increasing longevity of people with cystic fibrosis. CFBD is a complex and multifactorial disease. CFBD is a result of hypomineralized bone leading to poor strength, structure and quality leading to susceptibility to fractures. The development of CFBD spans different age groups. The management must be tailored to each group with nuance and based on available guidelines while balancing therapeutic benefits to risks of long-term use of bone-active medication. For now, the mainstay of treatment includes bisphosphonates. However, the long-term effects of bisphosphonate treatment in people with CF are not fully understood. We describe newer agents available for osteoporosis treatment. Still, the lack of data behooves trials of monoclonal antibodies treatments such as Denosumab and Romozosumab and anabolic bone therapy such as teriparatide and Abaloparatide. In this review, we also summarize screening and non-pharmacologic treatment of CFBD and describe the various options available for the pharmacotherapy of CFBD. We address the prospect of CFTR modulators on bone health while awaiting long-term trials to describe the effects of these medications on bone health.
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