Abatacept in the treatment of localized scleroderma: A pediatric case series and systematic literature review

Kalampokis, Ioannis; Yi, Belina Y.; Smidt, Aimee C.

doi:10.1016/j.semarthrit.2020.03.020

Cited by 25 publications

(18 citation statements)

References 98 publications

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“…4 On the contrary, abatacept has been reported to be effective against LS because it inhibits matrix metalloproteinase. 5 There are three possibilities for the LS development in our patient. (1) Accidental occurrence, (2) abatacept was ineffective in suppressing autoimmune reactions due to anti-U1 RNP antibodies in MCTD, and (3) LS might have appeared as a paradoxical reaction or an AE due to abatacept use (this seemed most likely in this case).…”

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confidence: 89%

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Localized scleroderma secondary to mixed connective tissue disease during abatacept therapy

Nagaoka

Hayashi

Igawa

2021

J Cutaneous Imm & Allergy

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A 47‐year‐old woman with mixed connective tissue disease was treated with abatacept. After 2 months, a 3‐cm depression with atrophied surface was observed on her back, which had histopathological consistent with the symptoms of localized scleroderma. Although some cases of paradoxical reaction or cutaneous adverse event have been reported from abatacept, no localized scleroderma has been reported, suggesting this to be a unique case.

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confidence: 89%

“…The cutaneous AE due to abatacept is mainly psoriasis lesions; no LS has been reported (Table ) 4 . On the contrary, abatacept has been reported to be effective against LS because it inhibits matrix metalloproteinase 5 …”

Section: Figurementioning

confidence: 99%

Localized scleroderma secondary to mixed connective tissue disease during abatacept therapy

Nagaoka

Hayashi

Igawa

2021

J Cutaneous Imm & Allergy

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“…However, cases refractory to MMF have been observed, and the treatment of recalcitrant morphoea remains largely empirical. Recently abatacept, a recombinant fusion protein interfering with the T‐cell costimulatory pathway, has been reported to be effective in two case reports and three small series of adult and paediatric patients (total of 18 cases) 27–30 . My group shared this good empirical experience with abatacept and presented an abstract of a study on three paediatric patients at the European Society for Pediatric Dermatology Meeting in 2019 31 .…”

Section: Second‐line Therapy For Difficult‐to‐treat Casesmentioning

confidence: 99%

“…The dosing regimen of abatacept in children is 10 mg/kg administered by intravenous infusion at days 0, 14 and 28, and then once monthly thereafter. It may be given as monotherapy or concomitant to MTX or corticosteroids, depending on the individual case and disease course 30 …”

Section: Second‐line Therapy For Difficult‐to‐treat Casesmentioning

confidence: 99%

Diagnosis and management of morphoea in children: an overview

Weibel

2021

Clin Exp Dermatol

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Summary Paediatric morphoea (localized scleroderma) is an inflammatory sclerosing disorder of the skin and subcutis associated with tissue atrophy. It is thought that the disease develops on the background of genetic predisposition (e.g. mosaicism for the common linear variant) initiated by various trigger factors, and that detected autoantibodies and inflammatory cytokines represent secondary epiphenomena. In contrast to the common belief that morphoea is a benign self‐limiting disorder, long‐term data indicate that its chronicity, relapsing nature and extracutaneous complications lead to significant morbidity, particularly when the disease starts in early childhood. Early recognition may be challenging, and the most important clinical clues are band‐like distribution, atrophy of underlying tissue, skin sclerosis, and localized loss of body/scalp hair, eyelashes or eyebrows. Extracutaneous manifestations occur in up to 20% of patients, with arthritis/arthralgia and neurological symptoms being most frequently observed, followed by ophthalmological complications such as uveitis. Corticosteroids and methotrexate are highly effective as first‐line therapy in morphoea, leading to partial reversal of skin manifestations. However, the development of atrophy is not sufficiently prevented by standard therapy. There is a relapse rate of 25%–48% within the first years after stopping treatment, thus long‐term follow‐up is warranted. Mycophenolate mofetil seems to be a beneficial second‐line therapy, and a new drug, abatacept, also seems to be a promising and well‐tolerated second‐line treatment option. Additionally, autologous fat injections are beneficial and may be used as an adjunct to ongoing therapy.

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“…In contrast, numerous published cases exist for the use of abatacept in patients with morphea and show promise for future potential large‐scale trials 119–121 . A retrospective compilation of eight cases of juvenile morphea showed no progression of disease after 30 months treatment with abatacept, and some cases had improvements in pre‐existing lesions 122 . Blocking inducible T cell costimulator (ICOS), another member of the CD28‐superfamily, has shown promising antifibrotic activity in preclinical models of skin fibrosis 123 .…”

Section: Anti‐inflammatory Drugsmentioning

confidence: 99%

Upcoming treatments for morphea

Wenzel

Haddadi

Afshari

et al. 2021

Immunity Inflam & Disease

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Morphea (localized scleroderma) is a rare autoimmune connective tissue disease with variable clinical presentations, with an annual incidence of 0.4–2.7 cases per 100,000. Morphea occurs most frequently in children aged 2–14 years, and the disease exhibits a female predominance. Insights into morphea pathogenesis are often extrapolated from studies of systemic sclerosis due to their similar skin histopathologic features; however, clinically they are two distinct diseases as evidenced by different demographics, clinical features, disease course and prognosis. An interplay between genetic factors, epigenetic modifications, immune and vascular dysfunction, along with environmental hits are considered as the main contributors to morphea pathogenesis. In this review, we describe potential new therapies for morphea based on both preclinical evidence and ongoing clinical trials. We focus on different classes of therapeutics, including antifibrotic, anti‐inflammatory, cellular and gene therapy, and antisenolytic approaches, and how these target different aspects of disease pathogenesis.

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Abatacept in the treatment of localized scleroderma: A pediatric case series and systematic literature review

Cited by 25 publications

References 98 publications

Localized scleroderma secondary to mixed connective tissue disease during abatacept therapy

Localized scleroderma secondary to mixed connective tissue disease during abatacept therapy

Diagnosis and management of morphoea in children: an overview

Upcoming treatments for morphea

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