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2015 ; 10
(11
): e0133702
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English Wikipedia
Efficacy of Rituximab in Refractory Inflammatory Myopathies Associated with Anti-
Synthetase Auto-Antibodies: An Open-Label, Phase II Trial
#MMPMID26539981
Allenbach Y
; Guiguet M
; Rigolet A
; Marie I
; Hachulla E
; Drouot L
; Jouen F
; Jacquot S
; Mariampillai K
; Musset L
; Grenier P
; Devilliers H
; Hij A
; Boyer O
; Herson S
; Benveniste O
PLoS One
2015[]; 10
(11
): e0133702
PMID26539981
show ga
OBJECTIVE: Anti-synthetase syndrome (anti-SS) is frequently associated with
myositis and interstitial lung disease (ILD). We evaluated prospectively, in a
multicenter, open-label, phase II study, the efficacy of rituximab on muscle and
lung outcomes. METHODS: Patients were enrolled if they were refractory to
conventional treatments (prednisone and at least 2 immunosuppressants). They
received 1 g of rituximab at D0, D15, and M6. The primary endpoint was muscular
improvement based on manual muscular testing (MMT10, Kendall score in 10 muscles)
at M12. Secondary endpoints were normalization of creatine kinase (CK) level, ILD
improvement based on forced vital capacity and/or diffuse capacity for carbon
monoxide, and number and/or doses of associated immunosuppressants. RESULTS:
Twelve patients were enrolled, and 10 completed the study. Only 2 patients
presented an improvement of at least 4 points on at least two muscle groups
(primary end-point). Overall, seven patients had an increase of at least 4 points
on MMT10. CK level decreased from 399 IU/L (range, 48-11,718) to 74.5 IU/L
(range, 40-47,857). Corticosteroid doses decreased from 52.5 mg/d (range, 10-70)
to 9 mg/d (range, 7-65) and six patients had a decrease in the burden of their
associated immunosuppressants. At baseline, all 10 patients presented with ILD.
At M12, improvement of ILD was observed in 5 out of the 10 patients,
stabilization in 4, and worsening in 1. CONCLUSIONS: This pilot study of
rituximab treatment in patients with refractory anti-SS provided data on
evolution of muscular and pulmonary parameters. Rituximab should now be evaluated
in a larger, controlled study for this homogenous group of patients. TRIAL
REGISTRATION: Clinicaltrials.gov NCT00774462.