A pilot study of mycophenolate mofetil combined to intravenous methylprednisolone pulses and oral low-dose glucocorticoids in severe early systemic sclerosis.
OBJECTIVE
This pilot study was aimed at evaluating the efficacy and safety of a protocol-based treatment strategy combining mycophenolate mofetil (MMF), intravenous (IV) methylprednisolone (MP) pulses and low-dose glucocorticoids (GC) in early systemic sclerosis (SSc) patients suffering from either active interstitial lung disease (ILD) or extensive skin disease.


METHODS
Sixteen SSc patients were recruited in the study, 9 based on the severity of their skin involvement (modified Rodnan total skin score [TSS] >or= 15) and 7 based on the presence of active ILD. Patients received 3 consecutive daily IV MP pulses, followed by 5 additional monthly IV MP pulses. MMF (0.5 g bid for one week; then, 1 g bid) and low-dose (5-10 mg/day) oral prednisolone were prescribed for one year. Patients were assessed at baseline, month 6 and 12. Statistics were by ANOVA.


RESULTS
TSS and Health Assessment Questionnaire significantly improved over time. In ILD patients, the vital capacity, forced expiratory volume in one second and carbon monoxide diffusing capacity significantly improved. Although the difference was not statistically significant, ground glass lesions decreased, based on semi-quantitative planimetry analyses performed on chest high-resolution computerized tomography. Toxicity was low and none of the patients suffered from renal crisis.


CONCLUSIONS
The results of this pilot study suggest that the combination of MMF, IV MP and low-dose GC might achieve good clinical, functional and radiological results in patients suffering from severe early SSc.
Vanthuyne
M
M
Rheumatology Department, Université Catholique de Louvain, Cliniques Universitaires Saint-Luc, Bruxelles, Belgium.
Blockmans
D
D
Westhovens
R
R
Roufosse
F
F
Cogan
E
E
Coche
E
E
Nzeusseu Toukap
A
A
Depresseux
G
G
Houssiau
F A
FA
D000284:Administration, Oral; D000328:Adult; D004305:Dose-Response Relationship, Drug; D004359:Drug Therapy, Combination; D005260:Female; D005938:Glucocorticoids / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006306:Health Surveys; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D007275:Injections, Intravenous; D017563:Lung Diseases, Interstitial / Q000188:drug therapy / Q000209:etiology; D008297:Male; D008775:Methylprednisolone / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010865:Pilot Projects; D020551:Pulse Therapy, Drug; D012595:Scleroderma, Systemic / Q000150:complications / Q000188:drug therapy*; D012871:Skin Diseases / Q000188:drug therapy / Q000209:etiology; D016896:Treatment Outcome
D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid; D008775:Methylprednisolone