Localized scleroderma
ICD-10 L94.0 · ICD-11 EB61.0

Localized Scleroderma with Severe Skin or Musculoskeletal Involvement: What to Do When Methotrexate Has Not Worked

This protocol addresses adults aged 18 and older with localized scleroderma where severe skin or musculoskeletal disease is present and first-line systemic therapy has not achieved the required response.

Clinical scenario

The target population has localized scleroderma with severe skin and/or musculoskeletal involvement — including arthritis, myositis, osteitis, or musculoskeletal pain attributable to the condition — with disease affecting fat tissue, fascia, muscle, joints, and bones, or with widespread skin involvement.

Previous treatment — escalation trigger

The recommended first-line systemic approach in this severe phenotype is methotrexate, with or without concomitant corticosteroid therapy. This protocol applies when that regimen fails to achieve its primary goal: reduction of skin sclerosis within 8 to 12 weeks. Intolerance to or a contraindication for first-line therapy also triggers this step.

Next-step approach (partial overview)

For adults with methotrexate- and/or steroid-refractory disease, a second-line systemic therapy — used off-label — is recommended. The full protocol specifies which agents are indicated, the selection criteria between them, and when each is appropriate.

Complete agent selection, sequencing, and criteria are available via the structured protocol below.

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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.

Arthritis, myositis, osteitis, or depending on the severity, musculoskeletal pain symptoms attributable to LoS.

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).

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