Experts Issue Recommendations for Managing Juvenile Localized Scleroderma

Referral to specialized centers, focusing on skin lesions as well as joint and neurological manifestations, and treatment with corticosteroids and disease-modifying antirheumatic drugs (DMARDs), are among new expert recommendations for juvenile localized scleroderma.

The study supporting those recommendations, “Consensus-based recommendations for the management of juvenile localized scleroderma,” was published in the journal Annals of the Rheumatic Diseases.

In 2012, a European project called SHARE was launched to optimize diagnostic and management regimens for children and young adults with rheumatic diseases.

Now, an international committee of 15 experts in pediatric rheumatology (mostly from Europe) focused on recommendations for juvenile localized scleroderma (JLS); 10 of the experts were part of the SHARE consortium, while the remaining five were asked to join given their clinical experience in JLS.

The team searched three online databases in August 2013 and January 2015, selecting 53 studies on diagnosis, assessment, and treatment of JLS. The studies’ validity and level of evidence were evaluated, leading to one overarching principle, and 16 recommendations accepted with at least 80% agreement among the experts.

All experts agreed that, because JLS is a rare disease, suspected cases should be referred to a specialized pediatric rheumatologist for clinical assessment and treatment. This is what the team deemed the overarching principle.

Regarding recommendations, based on the literature and to assess activity and severity of existing lesions, the team recommends the LoSCAT scoring system, which includes a Skin Severity Index and a Skin Damage Index. LoSCAT can be used in daily practice and addresses domains such as body surface area involvement, skin thickness and atrophy (shrinkage), and subcutaneous (under-the-skin) tissue loss.

Another approach to assess lesion activity in JLS is infrared thermography, a non-invasive procedure that detects temperature differences across the body surface. However, the experts cautioned about false positive results from atrophy of the skin, subcutaneous fat and muscle. High-frequency ultrasound also may help detect increased blood flow due to inflammation and other alterations.

Six recommendations addressed extracutaneous manifestations (those not on or under the skin), which are found in nearly 20% of JLS cases. All patients should undergo complete joint examination at diagnosis and during follow-up, with the experts recommending magnetic resonance imaging (MRI) as a helpful tool to assess musculoskeletal involvement.

Also, patients with facial and/or scalp lesions should undergo a head MRI scan to determine central nervous system (CNS, brain and spinal cord) involvement. Signs and symptoms of CNS changes in JLS, which may be distant to the skin lesions and occur mainly in the linear scleroderma subtype, can include seizures, headaches, behavioral changes, and learning disabilities.

Such patients also should be screened and followed up for ocular abnormalities, such as a type of eye inflammation called anterior uveitis. Orthodontic and maxillofacial evaluations are further recommended, as related changes have been associated with linear scleroderma of the face.

Regarding treatment, according to the team, JLS treatment should be defined based on disease subtype, the site of lesions, and degree of activity.

Systemic corticosteroids, such as oral prednisone and intravenous methylprednisolone, may be effective in patients with active disease.

Methotrexate (MTX; 15 mg/m² per week, oral or subcutaneous) is recommended in combination with corticosteroids, with results supporting maintaining this immunosuppressant for at least 12 months after achieving clinical improvement.

In turn, if MTX is ineffective, the disease relapses, or the patients are intolerant to MTX, CellCept (mycophenolate mofetil, by Genentech) can be an alternative DMARD at a dose of 500–1000 mg/m², although the experts cautioned that studies on its safety and effectiveness in JLS are still needed.

Topical treatment with the immune response modifier Aldara (imiquimod) is recommended in selected non-progressive or extended forms of JLS to ease skin thickness in patients with circumscribed (isolated) morphea lesions.

Phototherapy with ultraviolet light also may improve skin softness, despite limited data in children, as well as the risk for long-term effects and a high cumulative dosage of irradiation due to the need for prolonged maintenance therapy.

“To conclude, this SHARE initiative is based on expert opinion informed by the best available evidence and provides recommendations for the diagnosis and treatment of patients with JLS … with a view to improving their outcome in Europe,” researchers wrote.

“We anticipate that these guidelines will likely be adopted by physicians caring for patients with JLS outside Europe” too, the team stated. “It will now be important to broaden discussion and test the reliability of these recommendations to the wider scientific community and to the patients.”