Denosumab as a Potential Disease-Modifying Treatment in Indolent Systemic Mastocytosis-Related Osteoporosis

地诺单抗作为惰性系统性肥大细胞增多症相关骨质疏松症的潜在疾病修饰疗法

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Abstract

Indolent systemic mastocytosis (ISM) is a rare clonal disorder characterized by mast cell proliferation and release of inflammatory mediators affecting multiple organs, including the skeleton, frequently causing osteoporosis. We present the case of a 78-year-old woman with a longstanding history of cutaneous mastocytosis who developed systemic symptoms including gastrointestinal discomfort and flushing. Laboratory evaluation revealed elevated serum tryptase level of 28.7 ng/mL (SI: 28.7 μg/L) (reference range, <11 ng/mL [SI: < 11 μg/L]), a positive KIT D816V pathogenic variant (testing to confirm somatic vs germline origin was not available), and increased urinary prostaglandin D2. Bone marrow biopsy confirmed ISM. Despite long-term bisphosphonate therapy, she sustained a low-trauma vertebral fracture, prompting transition to denosumab in 2021. Over 3 years of treatment, the patient experienced a 7% increase in lumbar spine bone mineral density and a sustained decrease in serum tryptase levels to 12.0 to 14.3 ng/mL (SI: 12.0-14.3 μg/L), suggesting improved bone health and potential reduction in systemic mast cell burden. This case highlights the challenges in managing ISM-related osteoporosis and suggests a dual benefit of denosumab in improving bone mineral density and potentially reducing mast cell activity.

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