Methylprednisolone pulse therapy for patients with moderately severe Graves' orbitopathy: a prospective, randomized, placebo-controlled study.
OBJECTIVE
To assess whether methylprednisolone (MP) pulse therapy is efficacious in the treatment of moderately severe Graves' orbitopathy (GO).


METHODS
Prospective, placebo (PL)-controlled, double-blind, randomized study.


METHODS
Fifteen previously untreated patients with active, moderately severe GO participated in the study; 6 patients received MP and 9 patients a PL. Moderately severe disease was defined using the NOSPECS classification of clinical signs of GO . Activity was measured with the clinical activity score (CAS). A dose of 500 mg MP or only solvent was administered intravenously, over three consecutive days, in four cycles at 4 weekly intervals (6 g of MP in total). Qualitatively, a successful treatment outcome was defined as an improvement in one major and/or two minor criteria in the worst eye at week 48. The major criteria were: improvement in diplopia grade; improvement in eye movement; a decrease in CAS of three points. The minor criteria were: decrease of eyelid retraction; decrease of proptosis; improvement in grade of soft tissue swelling; a decrease in CAS of two points.


RESULTS
The qualitative treatment outcome was successful at the end of the trial in five out of six (83%) patients receiving MP and in one out of nine (11%) patients given the PL (relative risk=7.5; (95% confidence interval 1.1-49.3), P=0.005). The treatment was well tolerated.


CONCLUSIONS
In spite of the small number of patients, a significant difference in outcome was observed between MP- and PL-treated patients. We conclude that MP pulse therapy appears to be an effective treatment for active, moderately severe GO.
van Geest
Rob J
RJ
Donders Institute of Ophthalmology, University Medical Center Utrecht, Heidelberglaan 100, 3584 CX Utrecht, The Netherlands. r.j.vangeest@amc.uva.nl
Sasim
Inna V
IV
Koppeschaar
Hans P F
HP
Kalmann
Rachel
R
Stravers
Simone N
SN
Bijlsma
Ward R
WR
Mourits
Maarten P
MP
D000328:Adult; D000368:Aged; D004311:Double-Blind Method; D005260:Female; D005938:Glucocorticoids / Q000008:administration & dosage* / Q000009:adverse effects; D006111:Graves Disease / Q000188:drug therapy*; D006801:Humans; D007262:Infusions, Intravenous; D008297:Male; D008775:Methylprednisolone / Q000008:administration & dosage* / Q000009:adverse effects; D008875:Middle Aged; D011446:Prospective Studies; D020551:Pulse Therapy, Drug / Q000009:adverse effects; D012720:Severity of Illness Index; D016896:Treatment Outcome
D005938:Glucocorticoids; D008775:Methylprednisolone