SHORT COMMUNICATION
465
Abatacept Improves Skin-score and Reduces Lesions in Patients with Localized Scleroderma: A Case
Series
Simon WEHNER FAGE, Kristian Bakke ARVESEN and Anne Braae OLESEN
Department of Dermatology, University Hospital of Aarhus, Marselisborg Centret, P.P. Orumsgade 11, DK-8000 Aarhus C, Denmark. E-mail:
[email protected]
Accepted Jan 8, 2018; Epub ahead of print Jan 9, 2018
In 2011 we reported 2 cases of patients with treatment-
resistant, diffuse, deep, localized scleroderma (LoS), who
showed significant improvement of both active disease
and old fibrotic lesions on treatment with abatacept (1).
Since then we have offered abatacept to adult patients
with therapy-resistant, moderate-to-severe, active LoS.
Abatacept is a fusion protein that acts as a T-cell co-
stimulation blocker to inhibits tumour necrosis factor
alpha (TNF-α) and prevent T-cell activation. Another
group has also reported a good effect of abatacept in 3
patients with LoS (2), and there is an ongoing study of
subcutaneous abatacept to treat diffuse cutaneous sys-
temic sclerosis (SSc) (ASSET; https://clinicaltrials.gov/
ct2/show/NCT02161406).
The LoS diseases are a group of sclerosing skin di-
seases of rare occurrence. They mainly affect females
and young adults (3, 4). Adjacent tissue (fascia, muscle,
bone) may also be affected in LoS, but, in contrast to
SSc, there is no involvement of the internal organs (3).
In the subtype called “en coup de sabre” involvement
of the underlying central nervous system (e.g. seizures,
migraine, headache) and abnormal ophthalmological
findings (e.g. uveitis) can occur (5). Untreated LoS ,
especially the linear deep form, may result in joint cont-
ractures, muscle atrophy and growth limitations (5, 6).
Although causing significant morbidity, plaque-type LoS
typically has a benign self-limiting course of 3–5 years
duration of disease activity of each lesion. In general,
the course of the disease can vary, with improvement
and reactivation over some years. Currently, there is no
evidence-based causal (antifibrotic) treatment for LoS,
and there is a lack of clinical trials with regard to other
treatment options (7).
The aim of this study is to further determine whether
abatacept may have a positive effect on disease activity
in otherwise treatment-resistant LoS.
MATERIALS AND METHODS
The scleroderma clinic at the Department of Dermato-Venereology
of Aarhus University Hospital manages approximately 650 patients
with scleroderma and scleroderma-like diseases. Each week a total
of 2–4 new patients are admitted and 25–45 patients attend for
regular control of disease and treatments. Through 2009 to 2016 we
followed up on all adult treatment-resistant LoS patients who were
treated experimentally with abatacept. The patients were treated
with either 500 mg (patients weighing < 60 kg) or 750 mg (> 60
kg) abatacept intravenously on days 1, 15, 30, and thereafter every
4–6 weeks. Some patients switched to subcutaneous injection of
125 mg abatacept/week during treatment. Evaluations, including
either modified Rodnan Skin Score (MRSS) or Localized Sclero-
derma Cutaneous Assessment Tool (LoSCAT) (8), were performed
by well-trained doctors. Medical records review were conducted
to extract data at two time points: At the beginning of abatacept
treatment (the day the first abatacept treatment was injected) 1 st
assessment and either the latest follow up during treatment (the
patient is still treated with abatacept) or at the determination (2
nd assessment) of abatacept treatment. Regarding patients with
generalized LoS, MRSS and LoSCAT, data were collected at
any time, prior to, during, and after abatacept treatment. Primary
endpoints were changes in MRSS, LoSCAT, and/or change of
size of lesions. Secondary endpoints were changes in CRP, C3c,
C4, and/or PIIINP.
RESU