A retrospective study: Impact of consensus treatment plans on systemic therapy of pediatric morphea

Abstract

Background Morphea is an inflammatory and fibrosing condition that affects the skin and subcutaneous structures. Morphea is managed by dermatologists, rheumatologists, or both. Prior studies have suggested there is significant variability in approach to treatment. In 2012, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) published consensus treatment plans (CTPs) for pediatric morphea to develop more standardized treatment plans for patients requiring systemic therapy. We aimed to assess whether the publication of CTPs has impacted care of patients with morphea at our institution. Methods Data were collected via a retrospective review of medical records of 61 pediatric patients diagnosed with morphea at Seattle Children's Hospital (SCH) from January 1, 2005, to December 12,2017. Results Prior to the publication of CTPs, 2 out of 24 patients (8.3%) were treated with a regimen that matched a subsequent CTP. After publication of CTPs, 29 out of 37 patients (78.4%) were treated with a regimen that matched a CTP (P < 0.001). A subanalysis was performed to assess the number of patients who needed second‐ or third‐line therapies. Of those who followed a CTP therapy plan (n = 26), 3 patients (11.5%) needed a second‐line therapy compared with 11 patients (44%) in the no‐CTP followed group (n = 25), (P = 0.012). Conclusions The publication of CTPs led to a significant change in treatment approach for patients with morphea requiring systemic therapy at SCH. Patients treated with one of the treatment plans recommended by the CTPs were less likely to need second‐line systemic therapy.

Pediatric morphea state‐of‐the‐art literature review: Reframing morphea as a systemic disease

“…Treatment Plans, which are commonly used. 134,135 In a systematic review and meta-analysis comparing the efficacy of MTX and phototherapy (alone or associated with psoralen) for the treatment of pediatric morphea, both treatments were effective in treating active disease. When directly compared via pooled analysis, MTX was effective in 93% of children compared with phototherapy in 71% of cases (P < .003).…”

“…Most pediatric dermatology and pediatric rheumatology experts agree that MTX with or without an initial course of pulsed intravenous or oral corticosteroids is the recommended first‐line treatment for moderate to severe cases. This is exemplified by the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Consensus Treatment Plans, which are commonly used 134,135 . In a systematic review and meta‐analysis comparing the efficacy of MTX and phototherapy (alone or associated with psoralen) for the treatment of pediatric morphea, both treatments were effective in treating active disease.…”

Familial localized scleroderma with paediatric onset: a review

“…The course of the disease may vary, starting with slight erythematous superficial sclerosis of the skin to more severe cases with a decreased quality of life due to pain, skin thickness, as well as muscle and joint involvement [1,2]. Outcome of paediatric-onset localized scleroderma is worse than in adult-onset one because of longer duration, more frequent involvement of deeper and extracutaneous tissues and delayed introduction of treatment [1,3,4]. Although the aetiology and pathogenesis of morphea remain unknown, autoimmune, environmental, and infectious factors have been proposed [1,5].…”

“…The prevalence of autoantibodies in morphea and the general population is usually the same, but anti-single strand DNA, fibrillin-1, histone and topoisomerase II antibody titre may increase in morphea and antinuclear antibody titre in linear or generalized morphea [ 1 , 4 , 6 , 7 ].…”

“…Elevated vascular endothelial growth factor is evidence of vascular damage. An activation of T-cell derived cytokines (interleukin-4, -6, -8, -10, -13, and -17, TGF-b, PDGF, endothelin-1) and fibroblasts cause abnormal collagen synthesis [ 2 , 4 , 8 ]. There are also well-known suppressors of collagen production for example a and g interferon, both of which are produced by Th1 lymphocytes.…”