{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,24]],"date-time":"2026-07-24T06:54:00Z","timestamp":1784876040572,"version":"3.55.0"},"reference-count":37,"publisher":"The Endocrine Society","issue":"12","funder":[{"DOI":"10.13039\/501100005040","name":"Bontius Stichting","doi-asserted-by":"publisher","award":["1"],"award-info":[{"award-number":["1"]}],"id":[{"id":"10.13039\/501100005040","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2019,12,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Context<\/jats:title><jats:p>Fibrous dysplasia\/McCune-Albright syndrome (FD\/MAS) is a rare bone disorder commonly treated with bisphosphonates, but clinical and biochemical responses may be incomplete.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objective<\/jats:title><jats:p>To evaluate the efficacy and tolerability of the receptor activator of nuclear factor-\u03baB ligand inhibitor denosumab in the treatment of patients with FD\/MAS refractory to bisphosphonate therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Design<\/jats:title><jats:p>Case series.<\/jats:p><\/jats:sec><jats:sec><jats:title>Setting<\/jats:title><jats:p>Academic center of expertise for rare bone diseases.<\/jats:p><\/jats:sec><jats:sec><jats:title>Patients<\/jats:title><jats:p>Data were collected from 12 consecutive patients with FD\/MAS with persistent pain and increased biochemical markers of bone turnover (BTMs) after long-term treatment with bisphosphonates (median, 8.8 years) and were treated with subcutaneous denosumab 60 mg at 3- or 6-month intervals with a follow-up for at least 12 months.<\/jats:p><\/jats:sec><jats:sec><jats:title>Main outcome(s)<\/jats:title><jats:p>Sustained reduction of BTMs and bone pain.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A 60 mg dose of denosumab once every 3 months, but not once every 6 months, induced a sustained reduction of BTMs. After a median treatment period of 15.5 months (range, 12 to 19) serum alkaline phosphatase activity and propeptide of type 1 procollagen levels were respectively reduced from 212 \u00b1 39.4 IU\/L to 79 \u00b1 6.0 IU\/L (P = 0.004) and from 346.2 \u00b1 111.1 ng\/mL to 55.7 \u00b1 16.6 ng\/mL (P = 0.023) and normalized in 70% and 75% of patients, respectively. Although not quantitavely measured, 10 patients reported a reduction in bone pain of whom 6 reported complete elimination of pain. Treatment with denosumab was well tolerated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Our results indicate that 60 mg of denosumab every 3 months is a promising, well-tolerated treatment of most patients with FD\/MAS refractory to bisphosphonate therapy. These results together with those of previously published case reports provide the necessary background for the design of a larger, controlled study.<\/jats:p><\/jats:sec>","DOI":"10.1210\/jc.2018-02543","type":"journal-article","created":{"date-parts":[[2019,8,7]],"date-time":"2019-08-07T13:04:49Z","timestamp":1565183089000},"page":"6069-6078","source":"Crossref","is-referenced-by-count":73,"title":["Denosumab in Patients With Fibrous Dysplasia Previously Treated With Bisphosphonates"],"prefix":"10.1210","volume":"104","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1546-1112","authenticated-orcid":false,"given":"Bas C J","family":"Majoor","sequence":"first","affiliation":[{"name":"Department of Internal Medicine, Division of Endocrinology, Center for Bone Quality, Leiden University Medical Center, RC Leiden, Netherlands"},{"name":"Department of Orthopaedic Surgery, Center for Bone Quality, Leiden University Medical Center, RC Leiden, Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Socrates E","family":"Papapoulos","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Division of Endocrinology, Center for Bone Quality, Leiden University Medical Center, RC Leiden, Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P D Sander","family":"Dijkstra","sequence":"additional","affiliation":[{"name":"Department of Orthopaedic Surgery, Center for Bone Quality, Leiden University Medical Center, RC Leiden, Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marta","family":"Fiocco","sequence":"additional","affiliation":[{"name":"Medical Statistics, Department of Biochemical Data Science, Center for Bone Quality, Leiden University Medical Center, RC Leiden, Netherlands"},{"name":"Mathematical Institute, Center for Bone Quality, Leiden University Medical Center, RC Leiden, 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