RT Journal Article
SR Electronic
T1 Rituximab in early systemic sclerosis
JF RMD Open
JO RMD Open
FD EULAR
SP e000384
DO 10.1136/rmdopen-2016-000384
VO 3
IS 2
A1 Maaike Boonstra
A1 Jessica Meijs
A1 Annemarie L Dorjée
A1 Nina Ajmone Marsan
A1 Anne Schouffoer
A1 Maarten K Ninaber
A1 Koen D Quint
A1 Femke Bonte-Mineur
A1 Tom W J Huizinga
A1 Hans U Scherer
A1 Jeska K de Vries-Bouwstra
YR 2017
UL http://rmdopen.bmj.com/content/3/2/e000384.abstract
AB 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.