Linear Scleroderma in Children Under 18: Treatment When Methotrexate and Steroids Have Not Reduced Skin Sclerosis
Clinical scenario
This protocol is for children under 18 years of age with linear localized scleroderma (LoS) presenting with severe skin and/or musculoskeletal involvement — encompassing fat tissue, fascia, muscle, joints, bones, or widespread skin involvement. Linear types of localized scleroderma are more prevalent in childhood than in adults. Because of the risk of late complications — including contractures, growth disturbances, and limb deformities — early potent systemic therapy is indicated.
First-line failure — what triggers this protocol
First-line systemic therapy with methotrexate (off-label), combined with systemic glucocorticosteroids and supplemented by physical and manual therapy where applicable, has not achieved the target goal of reduction of skin sclerosis — an outcome expected to become apparent after at least 8 to 12 weeks of treatment. This failure, or intolerance or contraindication to the first-line regimen, is the escalation trigger for the second-line approach described here.
Second-line approach (partial overview)
When the first-line systemic regimen has not controlled the disease, a second-line off-label systemic agent may be introduced. The full protocol specifies which immunomodulating agents are considered and how they are selected — including agents that work through distinct immunologic mechanisms.
The complete agent list, selection criteria, and management guidance are available in the full protocol below.
Treatment goals
The aim of second-line therapy is clinical improvement, defined as reduction in peripheral erythema and reduction in skin sclerosis.
References
DOI: 10.1111/ddg.15328
- While the limited type of LoS (morphea) primarily occurs in adults, linear types are more prevalent in childhood.
- 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.).
- We recommend methotrexate (MTX) (off-label) as the first-line systemic therapy for LoS with severe skin and/or musculoskeletal involvement.
- We suggest using mycophenolate mofetil, mycophenolic acid, or abatacept as a second-line systemic therapy for LoS in cases of MTX and steroid-refractory courses, contraindication, or intolerance (off-label).
- All patients described in the initial study experienced clinical improvement (reduction in peripheral erythema, reduction in sclerosis), and concomitant steroid dose could be significantly reduced in some cases.
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