EFZO-FIT: no benefit from efzofitimod, but steroids fell to under 3 mg a day

Design

Phase 3, randomised, double-blind, placebo-controlled

Population

264 adults with pulmonary sarcoidosis requiring treatment

Primary outcome

Change in daily oral corticosteroid dose at week 48

Effect

Placebo-adjusted 0.02 (−1.49 to 1.52) and −0.73 (−2.21 to 0.75) mg/day

EFZO-FIT, a phase 3 double-blind trial at 85 centres in nine countries, randomised 268 adults with pulmonary sarcoidosis requiring treatment to monthly intravenous efzofitimod (3 or 5 mg/kg) or placebo for 48 weeks. Every arm followed a structured 12-week oral corticosteroid taper targeting zero, with dose increases allowed if sarcoidosis worsened.

Efzofitimod did not reduce steroid use beyond placebo: the placebo-adjusted change was 0.02 mg/day (95% CI −1.49 to 1.52) at 3 mg/kg and −0.73 mg/day (−2.21 to 0.75) at 5 mg/kg. Headache, sinusitis and wheeze were more frequent on efzofitimod. Two deaths in the 3 mg/kg group were judged unrelated.

The more useful finding is in the whole trial: mean prednisone fell from 10.7 mg to 2.9 mg a day by week 48, including in the placebo arm. The authors conclude that many patients can taper more aggressively than is common practice. That is an inference from a trial population, not a tested strategy, but it supports a planned taper with monitoring rather than an open-ended maintenance dose.

• Do not use efzofitimod for pulmonary sarcoidosis; it did not reduce steroid need.
• Consider a structured corticosteroid taper towards the lowest effective dose, with planned monitoring.
• Reassess symptoms, spirometry and imaging during the taper and slow it only for objective worsening.
• Discuss steroid side effects with patients as part of the decision to continue maintenance treatment.

Why it matters

Many patients may be carrying steroid harms for doses they no longer need.

Don't overread it

The taper finding is an observation within a trial, not a randomised comparison of taper strategies.

The statistics, in plain English

Both placebo-adjusted differences have intervals spanning zero, so the drug made no measurable difference. The fall from 10.7 to 2.9 mg a day happened across all arms, which is why it reflects the taper, not the drug.

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