The Effectiveness of Hydroxychloroquine Versus Methotrexate in the Treatment of Lichen Planopilaris in Routine Clinical Care: a Patient Preference Trial
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 56
- 试验地点
- 1
- 主要终点
- LPPAI
研究概览
简要总结
Rationale Lichen planopilaris (LPP) is a prevalent form of cicatricial alopecia, predominantly affecting women and causing irreversible hair loss. Hydroxychloroquine (HCQ) and methotrexate (MTX) are the most frequently used systemics for treatment of LPP in daily practice. Due to the absence of well-established treatment guidelines, this study aims to evaluate the effectiveness of HCQ and MTX in routine clinical care.
Objective(s) To investigate the effectiveness of HCQ and MTX in the treatment of adults with lichen planopilaris in routine clinical care.
Study type Prospective, patient preference clinical trial with a duration up to 48 weeks in accordance with the routine clinical care guidelines.
Study population This study will include adults (≥18 years) diagnosed with LPP.
Methods Patients will choose between HCQ and MTX treatment as in routine clinical care, receiving follow-up in accordance with standard clinical practices. They will not be randomized. The primary endpoint is the measurement of the Lichen Planopilaris Activity Index (LPPAI) at the 6-months, providing a quantitative assessment of the disease's activity and response to the selected treatment. The Skindex-29 questionnaire will be conducted at each visit, allowing evaluation of the impact on patients' quality of life.
详细描述
Treatment of LPP remains a challenge due to limited evidence-based guidelines and a lack of randomized controlled trials, leading to the absence of standardized therapeutic protocols and outcome measures. Currently, the treatment goal for LPP primarily focuses on halting disease progression to minimize further hair loss and alleviate associated symptoms. Treatment options for LPP include topical/intralesional corticosteroids, and systemic therapies such as hydroxychloroquine (HCQ), methotrexate (MTX), and other immunosuppressive agents. However, the absence of daily practice studies makes it challenging to establish therapeutic recommendations.
This patient preferred trial aims to fill a crucial knowledge gap in LPP management. Existing evidence lacks comprehensive guidelines for treating LPP effectively, leaving current approaches largely empirical. Aligning with the urgent need for answers highlighted by the 'Nederlandse Vereniging voor Dermatologie en Venereologie Kennisagenda Dermatologie 2019': 'Q9 - Wat is de effectiviteit van systemische behandeling bij patiënten met cicatriciële alopeciëen?' (What is the most effective systemic treatment for cicatricial alopecias) (13) underscores the pressing concern regarding the effectiveness of systemic therapy in cicatricial alopecia. This study seeks to contribute novel insights to the field by comparing the effectiveness of the two most commonly used systemic treatments, HCQ and MTX in treating LPP. By comparing the effectiveness and safety profiles of these systemics, this study endeavours to provide valuable information that can guide evidence-based treatment decisions and enhance the overall understanding of LPP management.
Objective(s)
The main objective is to assess the effectiveness in routine clinical care of hydroxychloroquine (HCQ) and methotrexate (MTX) in the treatment of adults diagnosed with lichen planopilaris (LPP) by evaluating the impact on the Lichen Planopilaris Activity Index (LPPAI) after a 6-month treatment period.
Primary objective:
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age of 18 years and older.
- •Diagnosed with LPP.
- •Willingness to provide informed consent for participation in the study.
- •No contraindications or known allergies to HCQ or MTX.
排除标准
- •Histopathological different diagnosis than LPP.
- •Inability to adhere to the study protocol, including medication intake, clinic visits, and questionnaire completion.
- •Patients who are ineligible for the HCQ arm (due to contraindications), are automatically included in the MTX arm.
- •Contraindications HCQ:
- •retinopathy and/or maculopathy
- •myasthenia gravis
- •body weight less than 35 kg
- •Patients who are ineligible for the MTX arm (due to contraindications), are automatically included in the HCQ arm.
- •Contraindications MTX:
- •Conception (both male and female) and lactation
- •Severe kidney or liver dysfunction (fibrosis, cirrhosis) or alcohol abuse
- •Bone marrow hypoplasia, immunodeficiency
- •Anemia, leukopenia, or thrombocytopenia
- •Poor nutritional status (low albumin)
- •Hypersensitivity or allergy to MTX
- •Lung toxicity due to MTX or significant reduction in lung function.
研究组 & 干预措施
Hydroxychloroquine (HCQ)
HCQ is administered orally at a dosage of 400 mg daily.
干预措施: Hydroxychloroquine (Drug)
Methotrexate (MTX)
MTX is given at a dose of 15 mg per week, orally or subcutaneously. Additionally, folate supplementation is administered concurrently as part of standard care, with folate 10 mg/week administered 24 hours after MTX intake.
干预措施: Methotrexate (Drug)
结局指标
主要结局
LPPAI
时间窗: at 24 weeks
The main study parameter is the change in Lichen PlanoPilaris Activity Index (LPPAI) measured at 6 months (0-10).
次要结局
- Skindex-29(at 0, 12, 24, 36, 48 weeks)
- Side effects(at 0, 12, 24, 36, 48 weeks)
- LPPAI(at 0, 12, 36, 48 weeks)
研究者
DirkJan Hijnen
MD, PhD
Erasmus Medical Center