Efficacy of a Prophylactic Treatment Using Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Adult Patients With Hemophilia. Observational Multicenter Study
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Change from baseline bleeding frequency after one year of follow-up
研究概览
简要总结
Background. Hemophiliac arthropathy is the main long-term physical damage in patients with hemophilia, causing disability and functional limitations.
Objective. Assessment of the efficacy of a prophylactic treatment with EHL products for improving the musculoskeletal health of adult patients with hemophilia.
Study Design. Multicenter observational clinical study. Method. 40 patients with hemophilia A will be included in this study. Patients will be recruited from 5 centers located in various regions of Spain (Community of Madrid, Galicia, Community of Valenciana, Málaga and Vizcaya). The dependent variables will be: bleeding frequency (self-report of bleeding), changes in the dosage of factor VIII with EHL products (rFVIIIFc), pain (measured with the visual analog scale and a pressure Algometer), degree of kinesiophobia (Tampa Scale of Kinesiophobia), degree of adherence to treatment (Veritas-Pro scale), joint health (using the Hemophilia Joint Health Score), muscle strength (using a dynamometer) and functionality (using the 6-Minute Walking Test). Three evaluations will be carried out: baseline, at 6 months, and at the end of the study period, at 12 months. The evolution of quantitative variables shall be analyzed by parametric tests (t-student test) or non-parametric tests (Wilcoxon test). Pearson's correlation coefficient shall be used to obtain the correlation between the dependent and independent variables. By means of a linear regression analysis we will record the percentage influence of the clinical variables relative to the dependent variables measured in this study Expected results. The aim is to establish the effectiveness of the prophylactic treatment in the improvement of the state health, joint pain, muscle strength and functionality in patients with hemophilia.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Patients diagnosed with hemophilia A
- •Patients over 18 years
- •Patients under prophylactic treatment with rFVIIIFc concentrates.
排除标准
- •Patients with neurological or cognitive disorders preventing the understanding of the various measuring instruments
- •Patients who are scheduled for major orthopedic surgery (total arthroplasty)
- •Not signed the Informed Consent Document
结局指标
主要结局
Change from baseline bleeding frequency after one year of follow-up
时间窗: Screening visit, at six months and after one year of follow-up
At baseline, patients will receive a personal self-report of bleeding (hematomas, hemarthrosis and mucous hemorrhagic episodes) where they should indicate the frequency of bleeding during the study period. This record should be delivered to the evaluators in the intermediate assessment and the final evaluation. In this way, changes in the frequency of bleeding throughout the study period will be evaluated
次要结局
- Change from baseline joint pain after one year of follow-up(Screening visit, at six months and after one year of follow-up)
- Change from baseline kinesiophobia after one year of follow-up(Screening visit, at six months and after one year of follow-up)
- Change from baseline joint status after one year of follow-up(Screening visit, at six months and after one year of follow-up)
- Change from baseline dosage of factor VIII with EHL products after one year of follow-up(Screening visit, at six months and after one year of follow-up)
- Change from baseline lower limb functionality after one year of follow-up(Screening visit, at six months and after one year of follow-up)
- Change from baseline joint pain after one year of follow-up: Pressure algometer(Screening visit, at six months and after one year of follow-up)
- Change from baseline muscle strength after one year of follow-up(Screening visit, at six months and after one year of follow-up)
- Change from baseline adherence to prophylactic treatment after one year of follow-up(Screening visit, at six months and after one year of follow-up)