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OPTIMISTmain

Safety and efficacy of low-intensity versus standard monitoring following intravenous thrombolytic treatment in patients with acute ischaemic stroke (OPTIMISTmain)

Year of Publication: 2025

Authors: Craig S Anderson, Debbie Summers, Menglu Ouyang, ..., for the OPTIMISTmain Investigators

Journal: The Lancet

Citation: Lancet 2025; 405: 1909–22

Link: https://doi.org/10.1016/S0140-6736(25)00549-5


Clinical Question

Is a low-intensity monitoring protocol non-inferior to standard high-intensity monitoring for functional outcome at 90 days in clinically stable patients with mild-to-moderate acute ischemic stroke (NIHSS <10) who received intravenous thrombolysis?

Bottom Line

OPTIMISTmain provides weak evidence that low-intensity monitoring (17 assessments over 24h) is non-inferior to standard monitoring (37 assessments) in stable post-thrombolysis patients with NIHSS <10. The upper bound of the 95% CI (1.15) just touched the non-inferiority margin, with no increase in symptomatic ICH or serious adverse events. Hospitals may consider incorporating this approach according to local circumstances.

Major Points

  • International stepped-wedge cluster-randomized non-inferiority trial across 114 hospitals in 8 countries
  • 4922 participants: 2789 low-intensity monitoring, 2133 standard monitoring
  • Low-intensity protocol reduced assessments from 37 to 17 over 24 hours post-thrombolysis
  • Primary outcome (mRS 2-6 at 90 days): 31.7% vs 30.9%, RR 1.03 (95% CI 0.92-1.15), p_non-inferiority=0.057
  • Upper CI boundary just touches the prespecified non-inferiority margin of 1.15, providing 'weak evidence' of non-inferiority
  • Symptomatic ICH very low in both groups: 0.2% vs 0.4% (RR 0.57)
  • No significant differences in serious adverse events (11.1% vs 11.3%)
  • ICU utilization reduced with low-intensity monitoring, particularly in USA (47.1% vs 77.1%)
  • Process evaluation showed intervention was acceptable, feasible, and well-received by nurses and clinicians

Design

Study Type: International, pragmatic, multicentre, stepped-wedge, cluster-randomised, controlled, blinded-endpoint, non-inferiority trial

Randomization: 1

Blinding: Blinded outcome assessment; open-label intervention (outcome assessors masked to treatment allocation)

Enrollment Period: April 28, 2021 to September 30, 2024

Follow-up Duration: 90 days

Centers: 114

Countries: Australia, Chile, China, Malaysia, Mexico, UK, USA, Viet Nam

Sample Size: 4922

Analysis: Intention-to-treat; log-binomial regression using generalized linear mixed model with fixed effects for group assignment and time (6-month intervals), random effect for hospital cluster; multiple imputation for missing data; non-inferiority margin RR 1.15; SAS Enterprise Guide 9.3 and R version 4.3.1


Inclusion Criteria

  • Age ≥18 years
  • Acute ischaemic stroke receiving IV thrombolysis (alteplase or equivalent) according to local guidelines
  • NIHSS score <10 within 2 hours of thrombolysis initiation
  • Clinically stable

Exclusion Criteria

  • NIHSS ≥10
  • Not clinically stable within 2 hours of thrombolysis
  • Unable to provide consent or opt-out

Arms

FieldLow-intensity monitoringControl
InterventionVital signs and neurological assessment every 15 minutes for 2 hours, every 2 hours for 8 hours, then every 4 hours until 24 hours post-thrombolysis (total 17 assessments). Encouraged to implement outside ICU where possible.Vital signs and neurological assessment every 15 minutes for 2 hours, every 30 minutes for 6 hours, then every 1 hour until 24 hours post-thrombolysis (total 37 assessments).
Duration24 hours monitoring, 90-day follow-up24 hours monitoring, 90-day follow-up

Outcomes

OutcomeTypeControlInterventionHR / OR / RRP-value
Unfavourable functional outcome defined by mRS score 2-6 at 90 daysPrimary606/1963 (30.9%)809/2552 (31.7%)0.057 (non-inferiority)
NIHSS at 7 days or discharge - Mean (SD)Secondary2.1 (3.1)1.9 (2.8)Mean difference -0.11 (-0.36 to 0.13)
Shift in mRS distribution at 90 daysSecondaryOrdinal OR 1.03 (0.89-1.20)
mRS 3-6 at 90 daysSecondary340/1963 (17.3%)401/2562 (15.7%)RR 0.95 (0.79-1.13)
Death at 90 daysSecondary39/2099 (1.9%)60/2716 (2.2%)RR 1.35 (0.84-2.17)
mRS 3-5 at 90 daysSecondary301/1963 (15.3%)341/2552 (13.4%)RR 0.91 (0.75-1.10)
Time to hospital discharge - Median (IQR), daysSecondary7 (4-10)7 (3-10)HR 1.14 (1.04-1.24)
Sleep-related impairment score - Median (IQR)Secondary1.8 (1.4-2.3)1.6 (1.3-2.1)Median difference 0.02 (-0.05 to 0.08)
Patient-reported experience - Median (IQR)Secondary1.1 (1.0-1.5)1.1 (1.0-1.5)Median difference -0.03 (-0.07 to 0.01)
EQ-5D-5L VAS at 90 days - Mean (SD)Secondary81.6 (19.2)82.2 (19.2)Mean difference -0.46 (-1.98 to 1.05)
Time from last monitoring to CT request - Median (IQR), hSecondary16.7 (6.2-23.7)22.4 (14.0-24.0)Median difference 2.3 (-22.5 to 27.1)
Symptomatic intracerebral haemorrhageAdverse8/2122 (0.4%)5/2783 (0.2%)RR 0.57 (0.15-2.13)
Serious adverse events - patients with ≥1 eventAdverse240/2133 (11.3%)309/2789 (11.1%)0.67
Total serious adverse eventsAdverse312 events389 events

Subgroup Analysis

No heterogeneity in treatment effect across prespecified subgroups including age, sex, ethnicity, NIHSS score (<5 vs ≥5), ischaemic stroke subtype, post-thrombolysis care location, and country. All interaction p-values >0.05.


Criticisms

  • Upper bound of 95% CI just touches the non-inferiority margin (1.15), providing only 'weak evidence' of non-inferiority
  • Open-label design may introduce observer bias despite blinded outcome assessment
  • Non-inferiority margin derived from thrombolysis trials in all stroke patients, not specifically mild stroke (NIHSS <10) who have better prognosis
  • Stepped-wedge design with guardian consent may have compromised data quality
  • COVID-19 pandemic disrupted services and created preference for low-intensity monitoring, potentially biasing results
  • Imbalance in group sizes (2789 vs 2133) due to accelerated adoption of low-intensity monitoring
  • Secular confounding from sequential implementation cannot be excluded
  • Modest non-significant increase in mortality with low-intensity monitoring (2.2% vs 1.9%)
  • Approximately half of participants had NIHSS ≤5, where thrombolysis benefit over DAPT is unclear

Funding

National Health and Medical Research Council of Australia (GNT1175861); New South Wales Health Investigator Development Grant; University of New South Wales Medicine Non Communicable Diseases Theme Early-Mid Career Research Seed Grant Scheme; Medical Research Future Fund (ID F02176) for conduct in Australia; Genentech for conduct in USA

Based on: OPTIMISTmain (The Lancet, 2025)

Authors: Craig S Anderson, Debbie Summers, Menglu Ouyang, ..., for the OPTIMISTmain Investigators

Citation: Lancet 2025; 405: 1909–22

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