Treatment of Localized Scleroderma with Severe Skin or Musculoskeletal Involvement in Patients Under 18
This protocol addresses a high-risk subset of localized scleroderma (LoS): pediatric patients (under 18 years of age) presenting with severe skin or musculoskeletal involvement, where disease extends beyond superficial skin to affect fat tissue, fascia, muscle, joints, or bones — or where widespread cutaneous involvement is present.
Clinical scenario
Severe LoS in a pediatric patient, with arthritis, myositis, osteitis, or musculoskeletal pain attributable to the condition, and/or deep tissue or widespread skin involvement.
In this population, potent systemic therapy must be initiated as early as possible. A longer interval before starting therapy is associated with a higher rate of treatment failure, and delayed treatment risks irreversible complications including contractures, growth disturbances, and limb deformities.
Treatment approach (partial overview)
This is a first-line protocol. Recommended treatment centres on a systemic disease-modifying agent, with an additional systemic anti-inflammatory component during the active phase of disease. The full protocol specifies agent selection, dosing parameters, duration criteria, and the sequencing of therapy — none of which are detailed here.
Treatment goal
Reduction of skin sclerosis — an effect that typically becomes assessable within 8 to 12 weeks of initiating therapy.
References
DOI: 10.1111/ddg.15328
- In subtypes with severe skin or musculoskeletal involvement, systemic therapy with methotrexate is recommended.
- We recommend methotrexate (MTX) (off-label) as the first-line systemic therapy for LoS with severe skin and/or musculoskeletal involvement.
- However, in all other cases and for active linear LoS in childhood, potent systemic therapy should be initiated as early as possible to prevent potential late complications (contractures, growth disturbances, limb deformities, etc.).
- A longer duration until the start of therapy is associated with a higher frequency of treatment failure.
- We suggest considering systemic glucocorticosteroid therapy in addition to the systemic treatment with MTX during the active phase of LoS.
- When assessing the effectiveness of the chosen therapeutic concept, it should be noted that the reduction of sclerosis typically takes at least 8 to 12 weeks in most cases.
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