WHAT THE STUDY ACTUALLY SAYS

Starting Crohn's treatment with infliximab beat a step-up approach in PROFILE

At one year, 79% of newly diagnosed patients given early infliximab plus an immunomodulator were in steroid-free, surgery-free remission, versus 15% treated conventionally.

Sustained steroid-free and surgery-free remission to week 48 in PROFILETop-down (infliximab + immunomodulator): 79%; Accelerated step-up: 15%0%40%80%Top-down (infliximab + immunomodulator)79%Accelerated step-up15%
Sustained steroid-free and surgery-free remission to week 48 in PROFILE
GroupValue (%)
Top-down (infliximab + immunomodulator)79
Accelerated step-up15
Sustained steroid-free and surgery-free remission to week 48 in PROFILE Newly diagnosed active Crohn's disease randomised to early combined immunosuppression (top-down) or accelerated step-up; primary-outcome data available for 379 of 386 patients. Source: The Lancet Gastroenterology & Hepatology

In patients newly diagnosed with active Crohn's disease, starting treatment straight away with the biologic infliximab plus an immunomodulator — a "top-down" strategy — produced far higher rates of lasting remission than the conventional approach of escalating drugs only as the disease demands. In the PROFILE trial, 79% of top-down patients were in sustained steroid-free and surgery-free remission at week 48, against 15% treated with accelerated step-up therapy [s1].

That 64-percentage-point gap (95% CI 57 to 72; p<0.0001) is one of the largest treatment effects reported in inflammatory bowel disease, and it is why the trial's authors argue top-down treatment should be considered standard of care for newly diagnosed active Crohn's [s1].

The two strategies

Crohn's disease has long been treated by escalation: start with steroids and milder drugs, and reach for biologics such as infliximab only when the disease keeps flaring. "Top-down" reverses that order, putting the most effective drugs in first, on the theory that early, decisive control prevents the bowel damage that accumulates during years of trial and error.

PROFILE — published in The Lancet Gastroenterology & Hepatology — was a multicentre, open-label, randomised controlled trial [s1]. It enrolled adults with newly diagnosed active Crohn's disease, defined by a Harvey-Bradshaw Index of 7 or more, a raised C-reactive protein or faecal calprotectin (or both), and endoscopic evidence of active inflammation [s1]. The median time from diagnosis to enrolment was 12 days — these were genuinely new patients [s1].

Between December 2017 and January 2022, 386 patients (mean age 33.6 years) were randomised: 193 to top-down (early combined immunosuppression with infliximab and an immunomodulator) and 193 to accelerated step-up [s1].

What happened

The primary endpoint was sustained steroid-free and surgery-free remission through to week 48, judged by a composite of symptoms and inflammatory markers at every visit — a demanding definition that a patient had to meet continuously, not just once [s1]. Primary-outcome data were available for 379 patients [s1].

Remission was reached by 149 (79%) of 189 top-down patients, against 29 (15%) of 190 step-up patients [s1]. The advantage held regardless of a biomarker the trial had been built around: PROFILE was designed partly to test whether a T-cell-based prognostic assay could identify who most needed aggressive treatment, but there was no biomarker-by-treatment interaction (absolute difference 1 percentage point, 95% CI −15 to 15; p=0.944) — the biomarker did not show clinical utility, while top-down treatment helped across the board [s1].

Fewer complications, not more

A common worry about front-loading powerful drugs is added harm. PROFILE found the opposite. There were fewer adverse events in the top-down group (168 versus 315), fewer serious adverse events (15 versus 42), and fewer complications requiring abdominal surgery (one versus ten), with no difference in serious infections (three versus eight) [s1]. Better disease control, in other words, came with fewer problems, not more — because uncontrolled Crohn's is itself dangerous.

Not the first signal

The direction is not new. Back in 2008, an open randomised trial by D'Haens and colleagues in The Lancet tested early combined immunosuppression against conventional management in 133 newly diagnosed patients [s2]. At week 26, 39 (60.0%) of 65 patients on early combined immunosuppression were in remission without corticosteroids or surgery, against 23 (35.9%) of 64 controls — an absolute difference of 24.1% (95% CI 7.3 to 40.8; p=0.0062) [s2]. Serious adverse events were similar between groups (30.8% versus 25.3%; p=1.0) [s2]. PROFILE, larger and with a stricter remission definition, sharpens a case that has been building for over 15 years.

What it means, and the caveats

The evidence points to early aggressive treatment for newly diagnosed active Crohn's, and away from watchful escalation. Two caveats belong alongside that. PROFILE was open-label, so patients and clinicians knew which strategy they were on — a design that can inflate differences in symptom-based endpoints, though the inflammatory-marker component is harder to bias [s1]. And it was funded in part by PredictImmune Ltd, the company behind the prognostic assay the trial ultimately found unhelpful [s1]. The treatment result stands on its own; the biomarker it was meant to validate did not.

This article describes trial results. It is not advice about any medicine, and nothing here should be used to start, stop or change treatment.

Sources

Sources

  1. A biomarker-stratified comparison of top-down versus accelerated step-up treatment strategies for patients with newly diagnosed Crohn's disease (PROFILE): a multicentre, open-label randomised controlled trial — The Lancet Gastroenterology & Hepatology , February 22, 2024
  2. Early combined immunosuppression or conventional management in patients with newly diagnosed Crohn's disease: an open randomised trial — The Lancet , February 25, 2008

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