. 2019 Sep 10;322(10):936–945. doi: 10.1001/jama.2019.12618

Table 2. Six-Month Results From a Randomized Clinical Trial Comparing Methotrexate and Mycophenolate Mofetil for Noninfectious Uveitis.

Patient-Level Characteristics	Methotrexate (n = 96)	Mycophenolate Mofetil (n = 98)	Absolute Risk Difference for Treatment Success, % (95% CI)	OR Estimate for Treatment Success (95% CI)^a	P Value
Primary Analysis
Treatment success, No. (%)^b	64 (66.7)	56 (57.1)	9.5 (−5.3 to 21.8)	1.50 (0.81 to 2.81)	.20
Treatment failure, No. (%)	32 (33)	42 (43)
Secondary Analyses
Reason for treatment failure, no./No. (%)
Efficacy^c	26/32 (81)	38/42 (90)			.55
Intolerability^d	3/32 (9)	2/42 (5)
Safety^e	3/32 (9)	2/42 (5)
Treatment success by anatomical location, no./No. (%)
Anterior uveitis and intermediate uveitis/intermediate uveitis only	6/18 (33.3)	14/22 (63.6)	−30.3 (−51.6 to 1.1)	0.29 (0.08 to 1.05)	.07^f
Posterior uveitis/panuveitis	58/78 (74.4)	42/76 (55.3)	19.1 (3.6 to 30.6)	2.35 (1.16 to 4.90)	.02^f
Treatment success at 12 mo, no./No. (%)
Continued on randomized antimetabolite^g	48/60 (80)	40/54 (74)	5.9 (−12.2 to 17.0)	1.40 (0.57 to 3.56)	.47
Switched to other antimetabolite^h	20/29 (69)	7/20 (35)	34.2 (6.6 to 52.6)	4.17 (1.32 to 13.16)	.02
Missed doses, mean (SD), %ⁱ	4.6 (1.0)	4.3 (0.5)			.87
Eye-Level Characteristics	Methotrexate (n = 185 eyes)	Mycophenolate Mofetil (n = 181 eyes)			P Value^j
Eye-Level Characteristics	Methotrexate (n = 185 eyes)	Mycophenolate Mofetil (n = 181 eyes)			P Value^j	Change in logMAR visual acuity, median (IQR)^k	−0.10 (−0.32 to 0.00)	−0.12 (−0.31 to 0.00)	.83
Reduction in central macular thickness, median (IQR) [No.], μm^l	−26.00 (−89.00 to 5.00) [42]	−14.00 (−80.00 to 3.25) [55]			.95

Abbreviations: IQR, interquartile range; logMAR, logarithm of the minimum angle of resolution; OR, odds ratio.

^{^a}

Logistic regression with treatment group as a fixed effect and study site as a prespecified random effect.

^{^b}

Treatment success was defined by achieving corticosteroid-sparing control of inflammation in both eyes at the month 6 visit.

^{^c}

Treatment failure due to efficacy was defined by not achieving the treatment success definition at month 6 or could be declared earlier if patient had persistent worsening inflammation.

^{^d}

Treatment failure due to intolerability was declared if a patient was unable or unwilling to continue medication due to adverse effects.

^{^e}

Treatment failure due to safety was declared if a patient had an abnormal laboratory result that met the serious adverse event threshold.

^{^f}

Interaction between anatomical subtype and treatment group was significant (P = .004).

^{^g}

Of patients who achieved treatment success at 6 months, 60 patients originally randomized to methotrexate and 54 patients originally randomized to mycophenolate mofetil continued on same antimetabolite through 12 months.

^{^h}

Of patients who had treatment failure at 6 months, 29 patients originally randomized to mycophenolate mofetil switched to methotrexate, and 20 patients originally randomized to methotrexate switched to mycophenolate mofetil.

^ⁱ

Indicates the number of missed doses over total expected doses throughout a patient’s enrollment in the trial.

^{^j}

P values computed from a linear mixed-effects model.

^{^k}

Decrease indicates gain in visual acuity.

^{^l}

Indicates eyes with macular edema excluding patients who had a serous retinal detachment in the setting of Vogt-Koyanagi-Harada disease.