Disabling Pansclerotic Morphea: A century of discovery.

Abstract

Disabling pansclerotic morphea (DPM) is a rare systemic inflammatory disorder at the severe end of the localized scleroderma spectrum which primarily affects children under 14 years of age. The disease is characterized by rapid sclerosis with circumferential involvement that frequently extends to the fascia, muscle, and bone. Disease progression often involves development of sclerotic plaques, chronic skin ulcers, and painful joint contractures leading to patient immobility with a high mortality rate. Internal organ fibrosis is typically absent. The aggressive and systemic nature of the disease leads patients to seek multidisciplinary care where current therapies are targeted towards immunomodulation and measures to preserve mobility while limiting infection, but often have limited efficacy. Here, we summarize all DPM patients in the English literature, common clinical symptoms, laboratory investigations, and treatments reported to date. Assessment of published cases suggest that the number of therapies does not influence disease outcome and that female patients were younger at the time of reported death. Clinician familiarity and awareness of common DPM symptoms are important for an accurate and early diagnosis. Furthermore, knowledge of the treatments that have been reported to be effective in mitigating disease progression may be helpful in expanding the treatment options available to patients.