Table 5.

Efficacy of Pharmacological Therapy for Active Moderate-to-Severe Thyroid Eye Disease

A. Comparisons of outcomes from baseline to after treatmenta,b
Drug (ref)	Composite outcome (%)	Clinical activity score (%)	Proptosis (%)	Diplopia (%)	Disease relapse (weeks)
IVGC67,68,71,72	23–53	45–83	0–46	0–19	21–40% (week 12)
MMF+IVGC68	63	80	No change	No change	8% (week 12)–11% (week 24)
RTX100	8	31	No change	No change	15% (week 40)
RTX67	60	100	No change	No change	0% (week 40)
TEP91	74	62	77	70	29% (week 51)–37% (week 27- see text)
TCZ112	73	93	27	7	No data
Placebo91,100,112	10–22	22–59	No change	No change	0 (week 12)–8% (week 51)

B. Comparisons of treatment outcomes between groups
Drug (ref), n = no. of randomized	Composite outcome	Clinical activity score	Proptosis	Diplopia	Disease relapse (weeks)
MMF vs. GC106 IVGC n = 78, MMF n = 80	Favored MMF 79% vs. GC 51%	Favored MMF 94% vs. 69%	Favored MMF 69% vs. GC 40%	Favored MMF 90% vs. GC 64%	Favored MMF 0% vs. IVGC 6%
MMF+IVGC vs. IVGC68 MMF+IVGC n = 76, IVGC n = 76	No difference between groups	No difference between groups	No difference between groups	No difference between groups	No difference between groups
	Post hoc MMF+IVGC 67% vs. IVGC 46%
OGC vs. IVGC74 IVGC n = 35, OGC n = 35	Favored IVGC 77% vs. OGC 51%	Favored IVGC 77% vs. OGC 51%	Favored IVGC 60% vs. OGC 40%	No difference between groups	Favored IVGC 0% vs. OGC 11% (week 24)
RTX vs. IVGC67 RTX n = 15, IVGC n = 16	Favored RTX 60% vs. IVGC 38%	Favored RTX 100% vs. IVGC 69%	No difference between groups	No difference between groups	Favored RTX 0% vs. IVGC 31% (week 76)
RTX vs. placebo100 RTX n = 13, placebo n = 12	No difference between groups	No difference between groups	No difference between groups	No difference between groups	No differences between groups (week 50)
Statin+IVGC vs. IVGC71 IVGC, n = 39, IVGC+statin n = 41	Favored atorvastatin+IVGC 51% vs. IVGC 28%	No difference between groups	No difference between groups	No difference between groups	Favored atorvastatin+IVGC 0% vs. IVGC 15%, p = 0.011) (week 24)
TEP vs. placebo91 TEP n = 84 placebo n = 87c	Favored TEP 74% vs. placebo 14%	Favored TEP 62% vs. placebo 22%	Favored TEP 77% vs. placebo 15%	Favored TEP 70% vs. placebo 31%	Data only for TEP 29.4–37% (weeks 27–51)d
TCZ vs. placebo112 TCZ n = 15, placebo n = 17	Favored TCZ 93% vs. placebo 59%	Favored TCZ 73% vs. placebo 29%	Favored TCZe	No difference between groups	No data provided

^a Comparisons of efficacy between treatments are subject to bias due to heterogeneity of patient populations, assessment methodology, end points, definitions of response and relapse, and duration of follow-up. The composite outcome is a combination of activity and severity measures and is variably defined. Proptosis improvement was defined as a reduction ≥2 mm in most studies. Diplopia was assessed using the Gorman scoring system.

^b The figures in A represent statistically significant changes compared with baseline, unless marked “no change.”

^c Pooled data from two randomized controlled trials.^89,90

^d Data for “flares”/relapses available for TEP group only (not placebo group).

^e Proptosis change from baseline TCZ −1.5 mm versus placebo 0.0 mm.

IVGC, intravenous glucocorticoids; MMF, mycophenolate mofetil; OGC, oral glucocorticoids; RTX, rituximab; TCZ, tocilizumab; TEP, teprotumumab.