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ONTT

A Randomized, Controlled Trial of Corticosteroids in the Treatment of Acute Optic Neuritis

Year of Publication: 1992

Authors: Roy W. Beck, Patricia A. Cleary, Malcolm M. Anderson Jr., ..., Constance W. Atwell; and the Optic Neuritis Study Group

Journal: The New England Journal of Medicine

Citation: N Engl J Med 1992;326:581-588


Clinical Question

Does treatment with either oral prednisone or intravenous methylprednisolone improve visual outcome in acute optic neuritis? Does either treatment speed the recovery of vision? What are the complications of treatment in relation to its efficacy?

Bottom Line

Intravenous methylprednisolone followed by oral prednisone accelerates visual recovery compared to placebo, with slightly better visual function at 6 months in contrast sensitivity, visual field, and color vision (but not visual acuity). However, oral prednisone alone is ineffective—providing no benefit in recovery rate or outcome—and unexpectedly increases the risk of new episodes of optic neuritis (relative risk 1.79 vs placebo). Oral prednisone alone, as prescribed in this study, should not be used for acute optic neuritis.

Major Points

  • IV methylprednisolone followed by oral prednisone significantly speeds recovery of visual function vs placebo (P=0.0001 for visual field, P=0.02 for contrast sensitivity, P=0.09 for visual acuity)
  • Oral prednisone alone provides no benefit in rate of recovery or 6-month visual outcome compared to placebo
  • At 6 months, IV methylprednisolone group had slightly better contrast sensitivity (P=0.026), visual field (P=0.054), and color vision (P=0.033), but not visual acuity (P=0.66)
  • Oral prednisone alone unexpectedly increased the rate of new optic neuritis episodes: 27% vs 15% placebo (RR 1.79; 95% CI 1.08-2.95)
  • IV methylprednisolone had lowest rate of new optic neuritis episodes (13%)
  • Multiple sclerosis developed in 14% IV methylprednisolone, 24% oral prednisone, and 20% placebo groups during 6-24 month follow-up (RR for MS with IV methylprednisolone vs placebo: 0.65; 95% CI 0.37-1.16)
  • Maximum treatment benefit occurred during first 15 days; differences between groups decreased over time
  • Patients with worse baseline visual acuity (20/50 to 20/200 or worse) showed greater relative benefit from IV methylprednisolone
  • 6% of IV methylprednisolone patients had visual acuity 20/50 or worse at 6 months vs 7% oral prednisone vs 5% placebo (similar poor outcomes across groups)
  • This trial established that oral prednisone alone should not be used for optic neuritis

Design

Study Type: Multicenter, randomized, controlled, partially double-blind trial

Randomization: 1

Blinding: Partially blinded. Personnel assessing visual function were always unaware of whether patient was assigned to placebo or prednisone group, and as often as possible unaware of whether patient was receiving IV methylprednisolone. Patients in oral-prednisone and placebo groups were blinded to their treatment assignment; those in IV methylprednisolone group were not masked.

Enrollment Period: July 1, 1988 to June 30, 1991

Follow-up Duration: 6 months primary; 6-24 months for new episodes and MS development

Centers: 15

Countries: USA

Sample Size: 457

Analysis: Stratified by baseline visual acuity. Results adjusted for baseline visual loss. Univariate Wilcoxon rank-sum test for 6-month outcomes. Wei-Lachin test for stochastic ordering combining all four measures. Rate of recovery analyzed by Mantel-Haenszel method with Kruskal-Wallis test for censored data. New episodes and MS development analyzed by Kaplan-Meier product-limit method with Mantel log-rank test. Sample size: 145 patients/group for 90% power at α=0.02 to detect 30% reduction in abnormal contrast sensitivity at 6 months; increased by 20% for withdrawal/noncompliance.


Inclusion Criteria

  • Age 18 to 46 years
  • Acute unilateral optic neuritis with visual symptoms lasting 8 days or less
  • Relative afferent pupillary defect in the affected eye
  • Visual-field defect in the affected eye on examination
  • No prior optic neuritis in the same eye
  • No prior history of optic neuritis in the same eye (could have had it in contralateral eye >5 years prior)
  • No clinical evidence of systemic disease other than multiple sclerosis that might cause optic neuritis

Exclusion Criteria

  • Previous optic neuritis in the same eye
  • Prior treatment with corticosteroids for current episode
  • Clinical evidence of systemic disease (other than MS) that might cause optic neuritis (e.g., sarcoidosis, systemic lupus erythematosus, syphilis)
  • Compressive optic neuropathy
  • Connective-tissue diseases

Arms

FieldControlIntravenous MethylprednisoloneOral Prednisone
InterventionOral placebo for 14 daysIntravenous methylprednisolone (Solu-Medrol) 250 mg every 6 hours for 3 days (hospitalized), followed by oral prednisone (Deltasone) 1 mg per kilogram of body weight per day [rounded to nearest 10 mg] for 11 daysOral prednisone 1 mg per kilogram of body weight per day [rounded to nearest 10 mg] for 14 days, with tapered dose (20 mg on day 15, 10 mg on days 16 and 18)
Duration14 days14 days total (3 days IV + 11 days oral)14 days full dose + 4 days taper

Outcomes

OutcomeTypeControlInterventionHR / OR / RRP-value
Visual field (assessed with Humphrey Field Analyzer) and contrast sensitivity (assessed with Pelli-Robson chart) at 6 months. Recovery defined as return to normal function.PrimaryPlacebo: Contrast sensitivity median 14 (13, 15); Visual field median 14 (13, 15)IV Methylprednisolone: Contrast sensitivity median 15 (14, 15); Visual field median 15 (14, 16)Contrast sensitivity: P=0.026; Visual field: P=0.054 (IV methylprednisolone vs placebo at 6 months)
Visual acuity at 6 months (Snellen ETDRS letter chart)SecondaryPlacebo: 20/16 (20/20, 20/13)IV Methylprednisolone: 20/16 (20/20, 20/13)0.66 (IV vs placebo); 0.35 (oral prednisone vs placebo)
Color vision at 6 months (Farnsworth-Munsell 100-hue error score)SecondaryPlacebo: 100 (220, 37)IV Methylprednisolone: 72 (94, 37)0.033 (IV vs placebo); 0.58 (oral prednisone vs placebo)
Rate of recovery of visual field to normal (Kruskal-Wallis)SecondaryPlacebo chi-squareIV methylprednisolone: chi-square 12.68 (P=0.0004) vs placebo; Oral prednisone: chi-square 2.34 (P=0.13) vs placebo0.0001 (IV vs placebo adjusted); 0.61 (oral prednisone vs placebo adjusted)
Rate of recovery of contrast sensitivity to normalSecondaryPlacebo baselineIV methylprednisolone: chi-square 3.27 (P=0.07) unadjusted, chi-square 5.91 (P=0.02) adjusted; Oral prednisone: chi-square 0.27 (P=0.61) unadjusted0.02 (IV vs placebo adjusted); 0.39 (oral prednisone vs placebo adjusted)
Rate of recovery of visual acuity to normalSecondaryIV methylprednisolone vs placebo: chi-square 16.27 (P=0.0001) adjusted0.0001 (IV vs placebo adjusted); 0.08 (oral prednisone vs placebo adjusted)
Stochastic-ordering summary statistic (all 4 measures combined at 6 months)Secondary0.029 overall
Relative risk of recovery (IV methylprednisolone vs placebo) - Contrast sensitivitySecondaryPlacebo baselineRR 1.17 for all measures; RR 1.14 (95% CI 0.98-1.41) for contrast sensitivityRR 1.14
Poor visual acuity (20/50 or worse) at 6 monthsSecondaryPlacebo: 8 (5.3%)IV Methylprednisolone: 9 (6.0%); Oral Prednisone: 11 (7.1%)NS (similar across groups)
New episodes of optic neuritis in either eye (6-24 months follow-up)SecondaryPlacebo: 24 (15%)IV Methylprednisolone: 20 (13%); Oral Prednisone: 42 (27%)RR 1.79 (oral prednisone vs placebo)0.02 (oral prednisone vs placebo by Mantel log-rank test)
New episodes of optic neuritis in affected eyeSecondaryPlacebo: 16 (10%)IV Methylprednisolone: 14 (9%); Oral Prednisone: 23 (15%)
New episodes of optic neuritis in contralateral eyeSecondaryPlacebo: 8 (5%)IV Methylprednisolone: 8 (5%); Oral Prednisone: 25 (16%)RR 2.50 (oral prednisone vs placebo)
Development of multiple sclerosis (6-24 months follow-up, Poser criteria)SecondaryPlacebo: 28 (20%)IV Methylprednisolone: 20 (14%); Oral Prednisone: 35 (24%)RR 0.65 (IV methylprednisolone vs placebo)NS
General tolerabilityAdverseWell toleratedGenerally well tolerated; IV methylprednisolone group had more weight gain (P<0.001)
Serious adverse effects (IV methylprednisolone group)Adverse02 patients: 1 acute transient depression requiring psychotropic drugs, 1 acute pancreatitis
Weight gainAdverseLess weight gainGreater weight gain in both steroid groups vs placebo (P<0.001)<0.001
Minor side effects (both steroid groups)AdverseMild mood change, stomach upset, facial flushing; more common than placebo
Sleep disturbanceAdverseMore frequent in steroid groups

Subgroup Analysis

Results stratified by baseline visual acuity (20/40 or better; 20/50 to 20/190; 20/200 or worse). The relative risk of recovery was lowest among patients whose vision was 20/40 or better at baseline, higher among those whose vision ranged from 20/50 to 20/190, and highest among those whose vision was 20/200 or worse. This suggests patients with worse baseline visual acuity may derive greater benefit from IV methylprednisolone treatment. At 6 months, poor visual outcome (20/50 or worse) occurred similarly across groups regardless of baseline severity.


Criticisms

  • IV methylprednisolone group was not masked due to hospitalization requirement, introducing potential bias in outcome assessment despite blinded assessors
  • No true IV placebo control - cannot fully separate IV methylprednisolone effect from oral prednisone that followed it
  • Oral prednisone dose (1 mg/kg) may have been too low; higher doses were not tested
  • Increased rate of new optic neuritis with oral prednisone was unexpected and has no clear biologic explanation - may represent chance finding
  • 6-month follow-up may be insufficient to assess long-term visual outcomes and MS risk
  • Results may not apply to patients presenting >8 days after symptom onset
  • Trial not designed to evaluate subgroups - baseline stratification analyses are hypothesis-generating only
  • Cannot determine if IV methylprednisolone alone (without subsequent oral prednisone) would be equally effective
  • Cost and inconvenience of hospitalization for IV treatment is a practical consideration
  • Study conducted before current MS diagnostic criteria and MRI predictors were established

Funding

National Eye Institute, National Institutes of Health (cooperative agreements EY07212, EY07460, EY07461, EY07659, EY07671, EY07673, EY07674, EY07675, EY07676, EY07678, EY07679, EY07680, EY07683, EY07685, EY07687, EY07689, EY07694, and EY07695); Upjohn Company (supplied study medications)

Based on: ONTT (The New England Journal of Medicine, 1992)

Authors: Roy W. Beck, Patricia A. Cleary, Malcolm M. Anderson Jr., ..., Constance W. Atwell; and the Optic Neuritis Study Group

Citation: N Engl J Med 1992;326:581-588

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