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Introduction <p>Fibrous dysplasia, alone or associated with McCune-Albright syndrome, is a rare bone disorder that can cause pain, deformity, fractures, and thus affect quality of life. Various treatments have been tried, starting with bisphosphonate administration, and now targeted therapies are being developed. Due to the rarity of the disease, there are only a few randomised clinical trials, making it difficult to understand the efficacy of each treatment. The aim of this systematic review is to provide an overview of what has been done in the past and what has been published recently on new targeted therapies.</p> Methods <p>A systematic search was conducted in MEDLINE and Web of Science up to February 3, 2026 for all oral, subcutaneous or intravenous therapies in patients with fibrous dysplasia. Data extraction and analysis followed PRISMA guidelines. The review was registered on PROSPERO, no. 42024602268.</p> Results <p>Fifty-six studies were included, most focusing on nitrogen-containing bisphosphonates, followed by new targeted therapies, mainly denosumab, a monoclonal antibody targeting RANKL. Overall, almost three-quarters of the studies showed a reduction in lesion size or activity, with only a few studies quantifying this reduction by various means (X-rays, CT, MRI, or Na[18F]F PET-CT). Almost all showed a reduction in circulating bone markers (<i>n</i> = 45/47), with a weighted mean reduction of 31 ± 18% in alkaline phosphatase (<i>n</i> = 30). The new targeted therapies looked promising, in particular denosumab showing a 66 ± 15% weighted mean reduction in fibrous dysplasia lesion activity on Na[18F]F PET-CT, but some patients have experienced serious side effects, which need to be better understood in order to prevent them more effectively.</p> Conclusion <p>Denosumab has shown promising results in recent years in terms of reducing the size and activity of fibrous dysplasia lesions and improving bone markers. Further research is needed to better prevent the serious side effects sometimes associated with this treatment.</p>

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Comparison of traditional and new treatments for fibrous dysplasia: a systematic review and meta-analysis

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月6日

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  • Lucie Levaillant,
  • Guillaume Mabilleau,
  • Adèle Malagié,
  • Sixtine Donat,
  • Régis Coutant,
  • Patrice Rodien,
  • Claire Briet

摘要

Introduction

Fibrous dysplasia, alone or associated with McCune-Albright syndrome, is a rare bone disorder that can cause pain, deformity, fractures, and thus affect quality of life. Various treatments have been tried, starting with bisphosphonate administration, and now targeted therapies are being developed. Due to the rarity of the disease, there are only a few randomised clinical trials, making it difficult to understand the efficacy of each treatment. The aim of this systematic review is to provide an overview of what has been done in the past and what has been published recently on new targeted therapies.

Methods

A systematic search was conducted in MEDLINE and Web of Science up to February 3, 2026 for all oral, subcutaneous or intravenous therapies in patients with fibrous dysplasia. Data extraction and analysis followed PRISMA guidelines. The review was registered on PROSPERO, no. 42024602268.

Results

Fifty-six studies were included, most focusing on nitrogen-containing bisphosphonates, followed by new targeted therapies, mainly denosumab, a monoclonal antibody targeting RANKL. Overall, almost three-quarters of the studies showed a reduction in lesion size or activity, with only a few studies quantifying this reduction by various means (X-rays, CT, MRI, or Na[18F]F PET-CT). Almost all showed a reduction in circulating bone markers (n = 45/47), with a weighted mean reduction of 31 ± 18% in alkaline phosphatase (n = 30). The new targeted therapies looked promising, in particular denosumab showing a 66 ± 15% weighted mean reduction in fibrous dysplasia lesion activity on Na[18F]F PET-CT, but some patients have experienced serious side effects, which need to be better understood in order to prevent them more effectively.

Conclusion

Denosumab has shown promising results in recent years in terms of reducing the size and activity of fibrous dysplasia lesions and improving bone markers. Further research is needed to better prevent the serious side effects sometimes associated with this treatment.