Nearly one in five with generalised myasthenia never reached control over eight years

Design

Delphi consensus definition applied to a retrospective single-centre cohort

Population

220 patients with generalised myasthenia gravis

Primary outcome

Proportion adequately controlled over time

Effect

62.0% at 2 years to 81.9% at 8 years; 17.7% never controlled

Spanish neuromuscular experts used a Delphi process to define adequately controlled generalised myasthenia gravis: stable remission or minimal manifestations for a year, an MG-ADL score of 2 or less, no exacerbation needing a change in therapy or rescue treatment, and no more than mild treatment side effects.

Applied to 220 patients from one referral unit, the proportion controlled rose from 62% at two years to 82% at eight. But 17.7% were never controlled at any assessment, and no clinical feature predicted who. Uncontrolled patients more often stopped drugs because of side effects and cost four to seven times more per year.

A single-centre referral cohort will overrepresent difficult cases. Still, the definition gives a usable target, and the finding that refractory disease cannot be predicted at diagnosis argues for regular review against it.

• Use MG-ADL score of 2 or less, no rescue therapy and tolerable side effects as a practical control target
• Review control at every visit — refractory disease could not be predicted at diagnosis
• Consider early escalation when side effects force repeated drug interruptions
• Expect most patients to reach control within several years

Why it matters

Without a definition, 'stable enough' drifts, and the uncontrolled fifth stays uncontrolled.

The statistics, in plain English

These are descriptive proportions from one specialist centre, not comparisons; they show how often control is reached, not what achieves it.

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