Role of Sclerostin in Mastocytosis Bone Disease
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- plasma sclerostin measurements (in pmol/l) SOST gene expression by Real-Time PCR dimensions of osteolytic lesions on low-dose computed tomography (in mm) dimensions of osteosclerotic lesions on low-dose computed tomography (in mm)
研究概览
简要总结
Mastocytosis is very rare and highly heterogeneous group of disorders, characterized by the accumulation of clonal mast cells which can infiltrate several organs and tissues.
Bones are the most frequent localization of systemic mastocytosis. The aim of our research was to explain the potential role of sclerostin in the pathogenesis of bone disease in mastocytosis.
详细描述
Mastocytosis is a heterogeneous group of disorders, characterized by the accumulation of clonal mast cells which can infiltrate several organs, such as the skin, bone marrow or liver. The skeleton is the most frequent localization of systemic mastocytosis (SM). Bone involvement occurs in approximately 70% of SM patients. Pathogenesis of mastocytosis bone disease is poorly understood.
The aim of our research is to explain the potential role of sclerostin, a recently discovered bone tissue protein, in the pathogenesis of bone changes in patients with mastocytosis.
The study group consists of adult patients with mastocytosis divided according to their clinical variants of disease (aggressive systemic mastocytosis - ASM, systemic mastocytosis with an associated hematological neoplasms SM-AHN, smouldering systemic mastocytosis - SSM, indolent systemic mastocytosis - ISM and cutaneous mastocytosis - CM; and group of healthy volunteers.
The concentration of sclerostin, bioactive sclerostin and expression of the SOST gene in human plasma and HMC-1.2 human mast cell culture supernatants is assessed. The Real-Time PCR method is used to evaluate the expression of sclerostin at the mRNA level, while the concentration of the sclerostin protein and its bioactive form is assessed using the enzyme immunoassay ELISA method. The obtained results are correlated with selected demographic, clinical, laboratory and radiological findings. Low-dose CT scan is used to assess bone changes.
These preliminary results could serve that sclerostin may be a new therapeutic target in patients with mastocytosis.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age > 18 years
- •Mastocytosis defined according to WHO criteria
- •Known KIT mutation status
排除标准
- •History of organ transplant
- •Inability to give informed consent
- •Pregnancy, Breastfeeding
- •Vulnerable Patient, defined as: patient with another uncontrolled severe disease; patient under juridical protection
结局指标
主要结局
plasma sclerostin measurements (in pmol/l) SOST gene expression by Real-Time PCR dimensions of osteolytic lesions on low-dose computed tomography (in mm) dimensions of osteosclerotic lesions on low-dose computed tomography (in mm)
时间窗: 1 year
The Primary Outcome Measures concern: * the measurements of plasma levels of sclerostin and its bioactive form in patients with mastocytosis and healthy volunteers * the measurements of levels of sclerostin and its bioactive form in HMC-1.2 human mast cells unstimulated and stimutaled with Il-6
dimensions of osteolytic lesions (in mm)
时间窗: 1 year
The Primary Outcome Measures concern: - the measurements of the dimensions of osteolytic bone lesions on low-dose computed tomography in patients with mastocytosis
dimensions of osteosclerotic lesions (in mm)
时间窗: 1 year
The Primary Outcome Measures concern: - the measurements of the dimensions of osteosclerotic bone lesions on low-dose computed tomography in patients with mastocytosis
次要结局
未报告次要终点
研究者
Aneta Szudy Szczyrek
Doctor of Medicine
Medical University of Lublin