Topical ruxolitinib reduced cutaneous graft-versus-host disease

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

Primary outcome

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

Effect

Adjusted difference about -4 percentage points (95% CI -5.5 to -2.4, P < .001) favouring ruxolitinib

Cutaneous chronic graft-versus-host disease is usually managed with topical corticosteroids, whose use is limited by skin atrophy and other effects. This single-centre phase 2 trial used an intra-patient design in 24 transplant recipients: ruxolitinib 1.5% cream on one body side and vehicle on the matched contralateral side, twice daily for 28 days.

At day 28, affected lesional body surface area fell from 14.3% to 6.2% on the ruxolitinib side and from 14.5% to 10.4% on the vehicle side — an adjusted between-side difference of about 4 percentage points (95% CI -5.5 to -2.4, P < .001). Serious adverse events were few and reflected the underlying haematological illness rather than the cream.

This points to topical ruxolitinib as a steroid-sparing, skin-directed option for epidermal or superficially sclerotic cutaneous graft-versus-host disease. It is small, single-centre and short, so it is a promising second-line signal rather than a settled standard.

• Single-centre phase 2 intra-patient trial, 24 allogeneic transplant recipients with cutaneous graft-versus-host disease.
• Ruxolitinib 1.5% cream vs vehicle, twice daily for 28 days, compared between body sides.
• Adjusted between-side difference in affected skin about 4 percentage points (95% CI -5.5 to -2.4, P < .001).
• Serious adverse events were few and reflected the underlying illness, not the cream.
• Consider it as a steroid-sparing, skin-directed second-line option where available.

Why it matters

It offers a skin-directed alternative to topical steroids in a condition where steroid side-effects limit long-term use.

Don't overread it

This was a small, single-centre, 28-day trial funded by the manufacturer; it shows short-term activity, not long-term benefit.

The statistics, in plain English

The intra-patient design controls for whole-body factors by comparing two sides of the same patient; the difference excludes zero so the effect is real, but 24 patients over 28 days cannot show durability or rarer harms.

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