Ruxolitinib cream for cutaneous chronic graft-versus-host disease: a small side-by-side trial

Design

Single-centre, randomised, double-blind, intra-patient-controlled phase 2 trial

Population

24 patients aged 12 or older with cutaneous chronic graft-versus-host disease over at least 2% body surface

Primary outcome

Between-side difference in affected body surface area at day 28

Effect

-4.2 percentage points (95% CI -6.8 to -1.5); adjusted -4.0 (-5.5 to -2.4)

At one New York centre, 24 patients with cutaneous chronic graft-versus-host disease covering at least 2% of body surface were randomised so that one side of the body received 1.5% ruxolitinib cream twice daily for 28 days and the other side vehicle. This intra-patient design controls for differences between people.

Among 23 evaluable patients, affected body surface fell from 14.3% to 6.2% on the treated side and from 14.5% to 10.4% on the vehicle side. The between-side difference was -4.2 percentage points (95% CI -6.8 to -1.5), or -4.0 (-5.5 to -2.4) after adjustment. Grade 3 to 5 events were one each of CNS leukaemia, sinusitis and lung infection, with no treatment-related deaths.

Topical corticosteroids remain the mainstay in this condition. This was a 28-day phase 2 trial funded by the manufacturer, so the findings support further study, not a change in first-line treatment.

• Affected body surface fell by 4.2 percentage points more on the treated side.
• Only 24 patients and 28 days of treatment.
• Serious events were few and not attributed to the cream.
• Corticosteroids remain the standard first-line topical.
• Manufacturer-funded phase 2 evidence, so wait for larger trials before changing practice.

Why it matters

It tests a steroid-sparing topical option in a condition where long-term steroid effects are a problem.

Don't overread it

A 28-day, 24-patient, single-centre phase 2 trial paid for by the manufacturer is not evidence for a change in first-line therapy.

The statistics, in plain English

Because each patient serves as their own control, a small sample can still detect a difference, but the result applies only to patches of skin disease treated for four weeks. A reduction of 4 percentage points of body surface is statistically clear; whether it matters to patients was not the question tested.

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