Background/Purpose: Juvenile Localized Scleroderma (JLS), includes a number of conditions characterized by skin thickening with varying degree of severity. Many patients, particularly those with the linear subtype, develop deformities and joint limitation or, as in scleroderma of head/face, functional and cosmetic problems as well as involvement of eyes and brain [1]. Although represents the first-line treatment for JLS [2], for some patients Methotrexate (MTX) is ineffective or not well tolerated. We investigated the safety and efficacy of mycophenolate mofetil (MMF) in a cohort of patients with severe or MTX-refractory JLS.

Methods: Consecutive JLS patients with severe or MTX-refractory followed at our Pediatric Rheumatology Unit were treated with MMF at doses varying from 600-1200 mg/m2/day bid. The disease was defined as active by the appearance of new lesions or increasing size of pre-existing ones, clinical signs of active inflammation, such as erythema, and/or by detection of disease activity by thermography when the affected area was more than 0.5°C warmer than the contralateral side or the surrounding skin depending on the site of the lesion itself. Outcome was defined as active disease (A), clinical remission on medication (CRM) (inactive disease for at least 6 consecutive months ON treatment) and Clinical Remission (CR) (inactive disease for at least 24 consecutive months OFF treatment).

Results: Twentyone patients (9M, 12F) entered the study. The JLS clinical subtypes [3] were circumscribed (C) morphea (2 pts), generalized morphea (GM) (1), linear scleroderma limbs (LSL) (5), linear scleroderma head/face (LSHF) (4), pansclerotic morphea (PM) (3), mixed subtype (MiX) (6). The age at onset of the disease was 8.2 yrs (range 0–14), disease duration at diagnosis was 10 months (range 0–3 years). Before starting  MMF, 19 patients have been treated with MTX and low dose corticosteroids oral in 13 pts, IV pulse in 6. In this group, MMF was started because of MTX lost efficacy (10) or relapse after MTX withdrawal (9). In one patient MMF was started because of MTX-related anaphylaxis and one patient with linear head/face congenital scleroderma with cerebral involvement. After 7.4 year follow up (range 2-14.7) CRM was present in 8 patients (38%) and sustained CR in 8 (38%). Five patients (24%)  (1 PM, 2 LSHF, 2 MiX) have still active disease despite MMF.

Conclusion: MMF is effective in severe and/or MTX-refractory JLS and generally well tolerated. Our study, although with the limits of being small-sized and retrospective, confirms that MMF represents a valid alternative to MTX, particularly in severe cases, when extracutaneous manifestations are present, or as complementary to MTX, when it loses efficacy. Further controlled studies are needed to confirm these data or to proof its efficacy as first line treatment of JLS.

References

1. Zulian F, Arthritis Rheum 2005; 52(9):2873-81
2. Zulian F, SHARE Consensus-based recommendations for JLS Ann Rheum Dis. Ahead Pub 2019.
3. Laxer RM § Zulian F Curr Opin Rheumatol 2006;18:606.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/mycophenolate-mofetil-for-the-treatment-of-severe-or-methotrexate-refractory-juvenile-localized-scleroderma/