Vitiligo Registry for Adults and Children in the UK (VIRTUAL-UK)
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Multi-centred registry will be developed, with an aim to establish short- and long-term safety (pharmacovigilance) and effectiveness of vitiligo therapies
研究概览
简要总结
Vitiligo is the most common depigmentation disorder affecting around 1% of the population worldwide. Fifty two percent of patients develop vitiligo before the age of 20 and around 80% develop vitiligo before the age of 30 years old.1 Vitiligo often presents in childhood and tends to be a lifelong disease, requiring prolonged courses of phototherapy.
Currently no national or international registry for patients with vitiligo exists. Individual dermatologists maintain a database of such patients, however no coordinated efforts have been made to combine these individual registries into a broader national registry. Finally, recently published British Association of Dermatologists (BAD) guideline for the management of vitiligo, recommended the development of a national registry for people with vitiligo undergoing systemic or light therapy to identify outcomes and safety.
详细描述
Vitiligo is the most common depigmentation disorder affecting around 1% of the population worldwide. Fifty two percent of patients develop vitiligo before the age of 20 and around 80% develop vitiligo before the age of 30 years old. Vitiligo often presents in childhood and tends to be a lifelong disease, requiring prolonged courses of phototherapy.
Vitiligo is an autoimmune skin disorder and is often associated with multiple other autoimmune disorders such as thyroid disorders, diabetes, IBS, pernicious anemia, alopecia areata amongst others. Current treatments include topical corticosteroids and calcineurin inhibitors, phototherapy, combination of phototherapy with oral corticosteroids, laser and surgical interventions. Other treatment modalities such as Methotrexate have also been trialed. Currently available treatments are being used off license with uncertain therapeutic success.
Previously, it has been shown that vitiligo has an inverse relationship with melanoma, which means that people with vitiligo are less likely to develop melanoma. A recent systematic review and meta-analysis looking into the risk of skin cancer in people with vitiligo showed that compared with people without vitiligo, people with vitiligo had a significantly lower risk of nonmelanoma skin cancer. The same pattern occurred for melanoma (but was not statistically significant). However, this review was limited by the small number of included studies and high heterogeneity due to methodological and clinical differences between the included studies. In addition, there is lack of data on the skin cancer risk for vitiligo patients who receive prolonged phototherapy courses, especially regarding high cumulative exposures in children with less deeply pigmented skin. This is especially important when NB-UVB is combined with topical tacrolimus treatment, due to theoretical increased risk of skin cancer.
In addition, there is an urgent need for new, effective and licensed treatment for vitiligo. In the dawn on new therapeutic era of new emerging treatments such as topical and oral JAK inhibitors, evidence will be needed on the effectiveness, safety and cost effectiveness of such therapies compared to other treatments. In order to gather evidence on clinical effectiveness of vitiligo treatments and to assist with updating of clinical guidance pathway, as well as to gather information on possible risk factors, adverse events, maintenance regime and assist with conduction of post marketing studies, prospective multicentre registry is needed for vitiligo patients who are treated with phototherapy, combination treatments (with phototherapy), oral agents (such as oral corticosteroids) including new emerging JAK inhibitors (once there are available in the UK).
Oral corticosteroids combined with phototherapy are recommended by the recently published BAD guidelines for cases of rapidly progressive vitiligo. Case reports were published on initiation of methotrexate and oral JAK inhibitors for the treatment of vitiligo.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Paediatric and adult patients with vitiligo under management in secondary care.
- •Written informed consent for study participation obtained from the patient or parents / legal guardian, with assent as appropriate by the patient, depending on the level of understanding.
- •Participants consent to participate in long-term follow up and access to all relevant medical records as needed by the study team
- •Clinical diagnosis of vitiligo made by a dermatologist
- •Willingness to comply with all study requirements.
- •Competent use of English language, according to patient's age (capable of understanding patient questionnaires).
排除标准
- •Insufficient understanding of the study by the patient and/or parent/guardian.
- •Patients with vitiligo/their parents or guardians, who do not provide consent for participation.
结局指标
主要结局
Multi-centred registry will be developed, with an aim to establish short- and long-term safety (pharmacovigilance) and effectiveness of vitiligo therapies
时间窗: 12 months
VIRTUAL-UK is a small registry and in collaboration with the BAD, aims to utilize and modify one of the existing platforms (software), in order to assist the end user (i.e. Dermatologists) in accessing the platform and reducing the time needed for training and data input.
次要结局
- To document the clinical and socio-demographic characteristics, co-morbidities including cancers, previous treatments of patients included in the cohort as assessed by the case report forms (CRFs).(12 months)
- To document disease trajectory and its relationship to demographic, phenotypic and treatment factors(12 months)
- To assess short- and long-term safety (pharmacovigilance) of these therapies(12 months)
- To describe the clinical and socio-demographic characteristics, co-morbidities including cancers, previous treatments of patients included in the cohort(12 months)
- To evaluate the efficacy and safety of currently used therapies for vitiligo (including combination therapies), providing a basis for better shared decision making and guidelines.(12 months)
- To evaluate the impact of these therapies on vitiligo patients' quality of life(12 months)
- To attempt to identify risk factors associated with treatment failure(12 months)
- To develop predictors of rapidly progressive vitiligo(12 months)
