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.
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.
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.
Patients who are ineligible for the MTX arm (due to contraindications), are automatically included in the CsA arm.
Parallel
Methotrexate
DrugMTX is given at a dose of 10-15mg/m2 per week, orally or subcutaneously. Additionally, folate supplementation is administered concurrently as part of standard care, with folate 5-10 mg/week administered 24 hours after MTX intake.
Cyclosporin (CSA)
DrugCSA is given orally (tablet or liquid form) and dosed 3-5mg/kg/day, divided into two doses a day
SALT 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.
SALT 24 weeks
To compare SALT\* (delta- SALT and SALT\<20) between CsA and MTX at 24 weeks. \* 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.
Quality of life (DLQI)
To compare the quality of life c-DLQI\* or DLQI\* between CsA and MTX at 0, 12, 24, 36 weeks. \*DLQI = Dermatology Life Quality Index, measures impact of skin diseases on a patient's life. The score ranges from 0 (no impact) to 30 (extreme impact).
Adverse events
To compare side effects in CSA and MTX group.
Discontinuation of therapy
To compare the proportion of patients who discontinued therapy in CSA and MTX group.
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