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2017 ; 3
(2
): e000384
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Rituximab in early systemic sclerosis
#MMPMID28879049
Boonstra M
; Meijs J
; Dorjée AL
; Marsan NA
; Schouffoer A
; Ninaber MK
; Quint KD
; Bonte-Mineur F
; Huizinga TWJ
; Scherer HU
; de Vries-Bouwstra JK
RMD Open
2017[]; 3
(2
): e000384
PMID28879049
show ga
OBJECTIVES: (1) Hypothesis testing of the potency of rituximab (RTX) in
preventing fibrotic complications and (2) assessing acceptability and feasibility
of RTX in early systemic sclerosis (SSc). METHODS: A small, 24-month, randomised,
double-blind, placebo-controlled, single-centre trial in patients with SSc
diagnosed <2 years was conducted. Patients received RTX or placebo infusions at
t=0, t=15 days and t=6 months. Patients were clinically evaluated every 3?months,
with lung function tests and high-resolution CT every other visit. Skin biopsies
were taken at baseline and month 3. Immunophenotyping of peripheral blood
mononuclear cells was performed at every visit, except at months 9 and 18.
Adverse events, course of skin and pulmonary involvement and B cell populations
in skin and peripheral blood were evaluated. RESULTS: In total 16, patients
(rituximab n=8, placebo n=8) were included. Twelve patients had diffuse cutaneous
SSc. Eighty-eight adverse events (RTX n=53, placebo n=35, p=0.22) and 11 serious
adverse events (RTX n=7, placebo n=4, p=0.36) occurred. No unexpected RTX-related
events were observed. Mean skin score over time did not differ between the
groups. Over time, forced vital capacity and extent of lung involvement slightly
improved with RTX, but this difference was insignificant. In peripheral blood B
cells depletion was demonstrated. CONCLUSIONS: No unexpected safety issues were
observed with RTX in early SSc. Although this small trial could not confirm or
reject potential efficacy of RTX in these patients, future placebo-controlled
trials are warranted, specifically in the subgroup of patients with pulmonary
involvement. TRIAL REGISTRATION NUMBER: EudraCT 2008-07180-16; Results.