Fibrous dysplasia/McCune-Albright syndrome (FD/MAS) is a rare genetic disorder initially identified by the triad of multiple fibrous osteodysplasia (FD), precocious puberty, and café-au-lae spots, encompassing various endocrine manifestations such as hyperthyroidism, acromegaly, phosphate wasting, and Cushing's syndrome.
Conditions
Interventions
denosumab:The denosumab group was treated with intravenous infusion of denosumab (1.7 mg/kg, subcutaneous injection) at the appropriate dose for patients at bas
Bisphosphonate group:The bisphosphonate group will receive zoledronic acid injection (0.05 mg/kg, intravenous infusion) at the appropriate dose for patients at baseline, M
Sponsors
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Eligibility
Sex/Gender
All
Age
4 Years to No maximum
Inclusion criteria
Inclusion criteria: 1.Patients over 4 years of age with confirmed fibrous dysplasia/McCune-Albright syndrome and at least one bone lesion; 2.Clinical intervention is required due to previous clinical symptoms, severe deformity or near fracture; 3.Presence of fibrous dysplasia, and has been treated with bisphosphonates for more than 3-6 months and the effect is not good; 4.Normal blood calcium, PTH and vitamin D levels (supplementation is allowed); 5.Baseline bone metabolism marker CTX is elevated; 6.Obtain the understanding of parents or guardians and sign the informed consent; 7.There is no other cause of bone fiber dysplasia;
Exclusion criteria
Exclusion criteria: 1.Pain and clinical manifestations unrelated to FD; 2.Pathological bone pain/fracture caused by endocrine diseases unrelated to FD/MAS; 3.Hypocalcemia, hypophosphatemia, vitamin D deficiency and untreate; 4.Being treated with other drugs that affect the bones, such as other bone metabolism modulators, high-dose steroids, etc. 5.Allergy to denosumab; 6.Other conditions that are not suitable for participation in this study;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Clinical benefit; | — |
Countries
China
Contacts
Public ContactWenli Lu
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed