Current Pharmaceutical Design
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Current Pharmaceutical Design, 2016, 22, 6306-6312
REVIEW ARTICLE
A Review of Clinical Trials of Belimumab in the Management of Systemic Lupus
Erythematosus
Alexis Garcia* and Juan B. De Sanctis
Instituto de Inmunología, Facultad de Medicina, Universidad Central de Venezuela, FOCIS Center of Excellence, Caracas,
Venezuela
A R T I C L E H I S T O R Y
Received: July 1, 2016
Accepted: August 29, 2016
DOI: 10.2174/13816128226661608311
03254
Abstract: There have been few changes over the last 50 years in the treatment of Systemic
lupus erythematosus (SLE), using non-specific anti-inflammatory agents such as: non-
steroidal anti-inflammatory drugs along with the immune cell modulating agent hydroxy-
chloroquine for mild disease, and broad spectrum immunosuppressants plus anti-
inflammatories such as corticosteroids, azathioprine, cyclophosphamide, or mycophenolate
during flares or severe disease with organ involvement. In some patients, the response is
inadequate and side effects appear from mild unpleasant up to severe toxicity. Drug metabo-
lism and clearance may be severely compromised. Therefore, it is a priority to develop better
treatments with fewer adverse events that can be used at different stages of disease activity.
In recent years, a member of the tumor necrosis factor (TNF) family, soluble human B Lym-
phocyte Stimulator protein (BLyS), also referred to as B-cell activating factor (BAFF) and
TNFSF13B has been studied extensively. This protein is synthesized by myeloid cell lines,
specifically interacts with B lymphocytes and increases their life-span. BlyS plays a key role in the selection,
maturation and survival of B cells and it has a significant role in the pathogenesis of SLE.
In this review, we analyzed the role of BLyS as a diagnostic/prognostic marker and/or therapeutic target for lupus
patients, and the different clinical studies published using belimumab.
Keywords: Lupus, B lymphocytes, BlyS, monoclonal antibody therapy.
INTRODUCTION
Systemic lupus erythematosus (SLE) is an autoimmune disease
characterized by the overproduction of autoantibodies against an
array of cytoplasmic and nuclear antigens and affects multiple or-
gans, such as the skin, joints, kidneys, and neuronal tissues [1]. The
disease generates significant symptomatology requiring medical
intervention, and it is also associated with progressive, irreversible,
organ damage and consequently high morbidity and early mortality
[1].
SLE affects mainly women (9:1 ratio and 10 cases per 100,000
people) and its prevalence is higher among Asians, African and
admix populations as compared to Caucasians [2, 3]. The incidence
has increased dramatically in the last 50 years [2, 3] and genetic
factors are important in the genesis of the disease, yet non genetic
factors play an important factor in the progression of the disease [3,
4]. The lack of prompt and adequate treatment increases disease
progression and, in the presence of comorbidities, the subsequent
increase in mortality [4].
Disease activity can be assessed either by clinical parameters
such as The Systemic Lupus Erythematosus Disease Activity Index
(SLEDAI) which is the main standard scale used to evaluate disease
activity or through laboratory tests: anti-double-stranded DNA
(anti-dsDNA), complement determinations (C3, C4, and CH50),
and erythrocyte sedimentation rate (ESR) [5]. Usually, an elevated
ESR along with anti-dsDNA and low C3, C4 levels are associated
with active disease, especially lupus nephritis [5]. Clinically
*Address correspondence to this author at the Instituto de Inmunología,
Facultad de Medicina, Universidad Central de Venezuela, Apartado 50109,
Caracas 1050-A, Venezuela; Tel: +58-212-6934767;
Fax: +58-212-6932815; E-mail: alexisgarcia27@gmail.com
relevant lupus nephritis is associated with a 30% decrease in
creatinine clearance, proteinuria, greater than 1000 mg/dL, and
renal biopsy findings indicative of active nephritis. Anti-nucleo-
some antibodies, that appear early in SLE, have high sensitivity and
specificity for diagnosis, and titers correlate with disease activity.
Anti-C1q antibodies are associated with lupus nephritis; high titers
correlate with active renal disease [6].
Appropriate management of patients with flares using effective
treatments to adequately suppress disproportionate immune system
activation are required to bring about long-term remission of the
disease [7]. Several biological agents were introduced for treatment
of SLE patients in different clinical trials [8]. Nonetheless, most of
the studies are conducted in small controlled-randomized studies
which hamper the analysis of the therapeutic impact on the disease
[8]. New data from research groups assessing inflammatory path-
ways and cellular interactions generated information regarding new
biological targets in patients with SLE [8, 9]. B cells are the critical
cells involved in the pathogenesis of SLE and consequently, neu-
tralization of autoreactive B cells, induction of tolerance, inhibition
of costimulatory signals and modulation of cytokines pathways are
the issues [8, 9]. Current therapeutic approaches targeting of B cells
include direct depletion and inhibition of specific B cell-stimulating
cytokines [8, 9]. More efforts have to be devoted to modulate im-
mune response without hampering basic immune responses.
GENETIC AND EPIGENETIC
Large genome-wide association studies (GWAS) have used
hundreds of thousands of single nucleotide polymorphism (SNP)
markers in order to elaborate a predictive genotype. These studies
have confirmed the importance of gene polymorphisms on immune
response, inflammation, and resolution of the inflammatory re-
sponse [10]. Even though genetic background involved in SLE is
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