Challenges in the diagnosis and treatment of disabling pansclerotic morphea of childhood: case-based review

Pansclerotic morphea: a historical case in children

“…In recent years, other therapies that have been trialed to complement the initial treatments include mycophenolate mofetil, sirolimus, biologics, tyrosine kinase inhibitors (TKI), and JAKI. Given that IL-6 plays an integral role in PMC by regulating fibroblast differentiation and stimulating collagen synthesis, abatacept and tocilizumab are strong candidate drugs for managing PMC [4]. Our patient had received oral corticosteroids associated with MTX but without any improvement related to the severity of skin involvement and late presentation.…”

“…Different therapeutic modalities with varying results have been tried: oral steroids, calcitriol, D-penicillamine, low dose methotrexate (MTX), tropical calcipotriene. However, none of these has shown any significant promise until now [3].MTX either in combination with corticosteroid or both with varied successes [4]. In recent years, other therapies that have been trialed to complement the initial treatments include mycophenolate mofetil, sirolimus, biologics, tyrosine kinase inhibitors (TKI), and JAKI.…”

Pansclerotic Morphea Following COVID-19: A Case Report and Review of Literature on Rheumatologic and Non-rheumatologic Dermatologic Immune-Mediated Disorders Induced by SARS-CoV-2

“…Disabling PSM of childhood (DPMC) is a rare subtype of juvenile localized scleroderma (JLS) characterized by pansclerosis mainly affecting children under the age of 14. This aggressive disease has a poor prognosis due to the rapid progression of deep musculoskeletal atrophy resulting in cutaneous ulceration and severe joint contractures ( 13 ).…”

Upcoming treatments for morphea

“…STAT4 deficient mice exhibited reduced fibrosis following bleomycin administration 143 . Ruxolitinb, a JAK1 and JAK2 inhibitor, exerts anti‐fibrotic effects and was initially developed for myelofibrosis 144 . Ruxolitinib has shown minimal responses in children with pan‐sclerotic morphea; however, one study found ruxolitinib was able to decrease scleroderma fibroblast activity downstream of TGFβ 145 .…”

“…Cellular therapies including autologous hematopoietic stem cell transplantation have been effective in improving skin scores in selected SSc patients, and in a case report of two pediatric pansclerotic morphea patients 70–72,151 . Some clinical trials have begun to implement utilization of autologous cells in combination with gene editing therapy.…”