Pediatric Localized Scleroderma with Severe Skin or Musculoskeletal Involvement After Methotrexate and Glucocorticoid Failure

Clinical scenario

Localized scleroderma in a patient under 18 years of age with severe involvement — affecting fat tissue, fascia, muscle, joints, or bones — or widespread skin involvement with symptoms of arthritis, myositis, osteitis, or musculoskeletal pain attributable to LoS.

First-line treatment — target not reached

The previous line used Methotrexate combined with glucocorticoids (intravenous methylprednisolone or oral prednisone). The required goal — reduction of skin sclerosis within 8 to 12 weeks — was not achieved, or the regimen was not tolerated or was contraindicated. This protocol addresses the next step after that failure.

Second-line approach (partial overview)

When first-line systemic therapy has not controlled disease activity, certain immunomodulatory agents are indicated as a second-line option. The complete protocol specifies which agents apply and under which conditions — available in full below.

Instant Access to Structured Evidence-Based Regimens

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 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).

View source ↗