The Effectiveness of Cyclosporine Versus Methotrexate in the Treatment of Pediatric Alopecia Areata in Routine Clinical Care: a Patient Preference Trial
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- SALT 36 weeks
研究概览
简要总结
Rationale: Since the introduction of Janus kinase (JAK) inhibitors, there has been a significant advancement in the treatment of pediatric alopecia areata. Eligibility for this treatment, in the Netherlands, requires prior failure of systemic therapies such as cyclosporin or methotrexate. However, the choice between methotrexate and cyclosporin as first-line systemic treatment is not supported by robust comparative studies.
Therefore, the investigators conduct a patient preference trial with a long-term follow-up provided in the Pediatric Systemic Alopecia Areata Registry (STA2R-Pediatric). This study will evaluate the effectiveness of Cyclosporin (CsA) and Methotrexate (MTX) in children and adolescents with moderate-to-severe AA.
Objective(s): To investigate the effectiveness of CsA and MTX in the treatment of children and adolescents with alopecia areata in routine clinical care.
Study type: This is a prospective, patient preference clinical trial with a duration up to 36 weeks in accordance with the routine clinical care guidelines.
Study population: This study will include children and adolescents (2-17 years old) diagnosed with AA who start first-line systemic treatment.
Methods: Patients and their parents will choose between CsA and MTX treatment as in routine clinical care, receiving follow-up in accordance with standard clinical practices. The participants will not be randomized. The primary endpoint is the measurement of the Severity of Alopecia Tool (SALT) at 9-months with a secondary endpoint at 24 weeks. SALT scores will be measured by a blinded assessor. The (Children) - Dermatology Life Quality Index ((C)-DLQI) questionnaire will be conducted at each visit (0, 3, 6, 9 months), allowing evaluation of the impact on patients' quality of life.
详细描述
Rationale: Alopecia areata (AA) is a dermatological disorder characterized by non-scarring hair loss, significantly affecting the quality of life of patients. While some patients experience spontaneous hair regrowth or respond well to localized treatments, patients with moderate-to-severe AA represent a subgroup that requires systemic therapies for effective management. If the disease is severe and systemic treatment is desired, cyclosporine or methotrexate is initiated. Currently, the clinical management of moderate-to-severe AA is primarily based on expert opinions. There is a lack of research on systemic treatments for moderate-to-severe AA, comprising only a small number of randomized controlled trials and observational studies. There is a clear absence of long-term, prospective, and comparative data on these systemic therapies. Most conventional systemic treatments for AA are prescribed off-label, underlining the importance of gaining a more comprehensive understanding of their effectiveness and safety.
Since the introduction of Janus kinase (JAK) inhibitors, there has been a significant advancement in the treatment of pediatric alopecia areata. As of 2024, Ritlecitinib-a selective JAK3 inhibitor-has been approved for reimbursement in adolescents aged 12 years and older with severe disease (defined as SALT50 or greater). Eligibility for this treatment requires prior failure of systemic therapies such as cyclosporin or methotrexate. However, the choice between methotrexate and cyclosporin as first-line systemic treatment is not supported by robust comparative studies and is largely based on expert opinion, resulting in considerable (inter)national variation in clinical practice.
Therefore, the investigators conduct a patient preference trial with a long-term follow-up provided in the Pediatric Systemic Alopecia Areata Registry (STA2R-Pediatric). This study will evaluate the effectiveness of Cyclosporin (CsA) and Methotrexate (MTX) in children and adolescents with moderate-to-severe AA. Aiming to provide valuable insights into the effectiveness, safety, and long-term outcomes of these therapies. By collecting and analyzing such data, this study endeavors to contribute to evidence-based clinical management, ultimately improving care for children and adolescents with AA.
Objective(s): To investigate the effectiveness of CsA and MTX in the treatment of children and adolescents with alopecia areata in routine clinical care.
Study type: This is a prospective, patient preference clinical trial with a duration up to 36 weeks in accordance with the routine clinical care guidelines after which patients will be followed in the STA2R-Pediatric (PANAMA-ID 11347).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •In order to be eligible to participate in this study, a subject must meet all of the following criteria:
- •Age 2-17 years
- •Clinical diagnosis of AA by a certified dermatologist
- •Willingness of participant (in case 12-17 years) and parents to provide informed consent for participation in the study.
排除标准
- •A potential subject who meets any of the following criteria will be excluded from participation in this study:
- •Inability to adhere to the study protocol, including medication intake, clinic visits, and questionnaire completion.
- •Patients who are ineligible for the CsA arm (due to contraindications), are automatically included in the MTX arm.
- •Contra-indications CsA:
- •Impaired kidney function. Poorly controlled hypertension Active infections. Presence of a malignancy. Nephrotic syndrome combined with poorly controlled hypertension, infection or malignancy.
- •Kidney disorders, except in cases of nephrotic syndrome with mild to moderate renal impairment.
- •Patients who are ineligible for the MTX arm (due to contraindications), are automatically included in the CsA 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
Methotrexate (MTX)
Methotrexate is administered orally or intramusculairly and dosed 10-15mg/m2, 1 times a week, with folate suppletion 24 hours after MTX intkae
干预措施: Methotrexate (Drug)
Cyclosporine (Cyclo)
Cyclosporine is given orally, dosed 3-5mg/kg/day and divided in to two doses/ day
干预措施: Cyclosporin (CSA) (Drug)
结局指标
主要结局
SALT 36 weeks
时间窗: 36 weeks
To compare SALT\* (delta-SALT and SALT\<20) between baseline and 36 weeks of treatment between the CsA and MTX group. \* The Severity of Alopecia Tool (SALT) score is a standardized, numerical measure (0-100) used by dermatologists to quantify scalp hair loss in patients with alopecia areata. A score of 0 indicates no hair loss, while 100 indicates total scalp hair loss.
次要结局
- SALT 24 weeks(24 weeks)
- Quality of life (DLQI)(24, 36 weeks)
- Adverse events(0, 12, 24, 36 weeks)
- Discontinuation of therapy(0, 12, 24, 36 weeks)
研究者
Rick Waalboer-Spuij, MD, PhD
Deputy Head of Department of Dermatology, MD, PhD.
Erasmus Medical Center