New England Journal of Medicine November 05, 2020

Colchicine in Patients with Chronic Coronary Disease

Stefan M. Nidorf, Aernoud T.L. Fiolet, Arend Mosterd, et al.

Bottom Line

In patients with chronic coronary disease, low-dose colchicine (0.5 mg once daily) significantly reduced the risk of a composite of cardiovascular death, spontaneous myocardial infarction, ischemic stroke, or ischemia-driven coronary revascularization compared to placebo.

Key Findings

1. The primary composite endpoint (cardiovascular death, spontaneous myocardial infarction, ischemic stroke, or ischemia-driven coronary revascularization) occurred in 6.8% (187/2762) of patients in the colchicine group versus 9.6% (264/2760) in the placebo group (incidence, 2.5 vs. 3.6 events per 100 person-years; HR, 0.69; 95% CI, 0.57 to 0.83; P<0.001).
2. The key secondary composite endpoint (cardiovascular death, spontaneous myocardial infarction, or ischemic stroke) occurred in 4.2% (115 patients) of the colchicine group compared to 5.7% (157 patients) of the placebo group (incidence, 1.5 vs. 2.1 events per 100 person-years; HR, 0.72; 95% CI, 0.57 to 0.92; P=0.007).
3. Incidence rates of the individual components of spontaneous myocardial infarction and ischemia-driven coronary revascularization were also significantly lower with colchicine therapy.
4. The incidence of death from noncardiovascular causes was higher in the colchicine group than in the placebo group (incidence, 0.7 vs. 0.5 events per 100 person-years; HR, 1.51; 95% CI, 0.99 to 2.31).

Study Design

Design
RCT
Double-Blind
Sample
5,522
Patients
Duration
28.6 mo
Median
Setting
Australia, Netherlands
Population Patients aged 35 to 82 years with established chronic coronary disease who were clinically stable for at least 6 months and successfully tolerated a 30-day open-label colchicine run-in phase.
Intervention Colchicine 0.5 mg once daily
Comparator Matching placebo once daily
Outcome A composite of cardiovascular death, spontaneous (nonprocedural) myocardial infarction, ischemic stroke, or ischemia-driven coronary revascularization

Study Limitations

• The study utilized a 30-day open-label run-in phase, which excluded patients who experienced early gastrointestinal intolerance to colchicine (approximately 9% of those initially screened), potentially limiting generalizability and underestimating real-world drug discontinuation rates.
• A borderline concerning trend toward increased noncardiovascular mortality (HR, 1.51; 95% CI, 0.99-2.31) was observed, which warrants careful long-term surveillance.
• The trial did not mandate baseline or follow-up measurements of inflammatory biomarkers, such as high-sensitivity C-reactive protein (hs-CRP), precluding subgroup analyses to determine if treatment benefits correlated with baseline inflammatory risk.
• Women and diverse racial/ethnic groups were underrepresented in the study population, which limits the extrapolation of the findings to broader demographic groups.

Clinical Significance

The LoDoCo2 trial definitively validates the inflammatory hypothesis of atherosclerosis in the chronic setting. It establishes that low-dose colchicine, a widely available and inexpensive generic medication, can be repurposed to provide significant cardiovascular event reduction in patients with stable coronary disease on top of standard secondary prevention therapies. While it offers a valuable new tool in the cardiovascular armamentarium, clinicians must carefully weigh the anti-ischemic benefits against potential gastrointestinal side effects, drug-drug interactions (particularly with CYP3A4 inhibitors), and the trial's small numerical increase in noncardiovascular mortality.

Historical Context

For decades, atherosclerosis was largely viewed as a lipid-storage disease, but mounting evidence implicated underlying inflammation. In 2017, the CANTOS trial provided proof-of-concept that targeting inflammation directly (using the expensive monoclonal antibody canakinumab against IL-1β) reduced cardiovascular events independently of lipid lowering. Seeking a more accessible and cost-effective alternative, investigators turned to colchicine, an ancient drug used for gout. In 2019, the COLCOT trial demonstrated that low-dose colchicine reduced cardiovascular events in patients with a *recent* myocardial infarction. The LoDoCo2 trial (building on the smaller, non-placebo-controlled LoDoCo1 trial in 2013) expanded on this paradigm by proving that the cardiovascular benefits of colchicine extend to a broad population of patients with stable, *chronic* coronary disease.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

How does the mechanism of action of colchicine provide a pathophysiological rationale for its use in preventing events in chronic coronary disease?

Key Response

Colchicine binds to tubulin, inhibiting microtubule polymerization. This action impairs neutrophil migration, chemotaxis, and adhesion. Critically, it also inhibits the assembly of the NLRP3 inflammasome, a multiprotein complex responsible for activating pro-inflammatory cytokines like IL-1beta and IL-6. Since atherosclerotic plaque progression and rupture are highly driven by this local and systemic inflammatory response, inhibiting the NLRP3 inflammasome stabilizes plaques and reduces the risk of acute ischemic events.

Resident
Resident

When considering starting 0.5 mg of colchicine daily in your continuity clinic patients with chronic coronary disease, what specific drug-drug interactions and adverse effects must you screen for prior to initiation?

Key Response

Residents must strictly screen for the concurrent use of moderate-to-strong CYP3A4 inhibitors (e.g., clarithromycin, ketoconazole, diltiazem, verapamil) and P-glycoprotein inhibitors, as colchicine is metabolized and transported by these pathways. Co-administration can lead to fatal colchicine toxicity, manifesting as neuromyopathy, cytopenias, and multiorgan failure. Clinically, they must also counsel patients on gastrointestinal side effects (especially diarrhea), which is the most common reason for drug discontinuation.

Fellow
Fellow

The LoDoCo2 trial excluded patients with advanced heart failure and severe renal impairment. How do these exclusions impact the extrapolation of the 'anti-inflammatory hypothesis' to the broader cardiovascular population, particularly given colchicine's pharmacokinetic profile?

Key Response

Colchicine is primarily cleared by the kidneys, so its toxicity risk is heavily amplified in severe CKD, precluding its safe use in this demographic without extreme caution. Furthermore, the role of inflammation in HFrEF pathophysiology is complex; while atherosclerosis is driven by the NLRP3 inflammasome, prior anti-inflammatory trials in heart failure (like those using TNF-alpha inhibitors) showed harm. Fellows must recognize that the net benefit of colchicine relies on strict patient selection, avoiding those with poor clearance or non-ischemic drivers of cardiovascular morbidity.

Attending
Attending

With trials like CANTOS, COLCOT, and now LoDoCo2 establishing 'residual inflammatory risk' as a core therapeutic target, how do you balance the impressive relative risk reduction of colchicine against its modest absolute risk reduction and the numerically higher rate of non-cardiovascular mortality when counseling a patient?

Key Response

While the relative risk reduction was nearly 31%, the absolute risk reduction was modest given the event rates in stable chronic CAD. Attendings must weigh the extremely low cost and clear ischemic benefits against a concerning, albeit statistically non-significant, trend toward increased non-cardiovascular death (e.g., from infections or cancers) seen in LoDoCo2. Shared decision-making should frame colchicine as an adjunct for high-risk patients who have already maximized lipid-lowering and antithrombotic therapies but continue to have high residual risk, rather than a universal addition to all stable CAD patients.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

LoDoCo2 utilized a 30-day open-label run-in phase before randomization to confirm patient tolerance to colchicine. How does this specific run-in design influence both the internal validity and the external generalizability of the trial's effect size?

Key Response

An active run-in phase enriches the randomized cohort with 'tolerators,' thereby decreasing crossover, non-adherence, and dropout rates during the double-blind phase. This maximizes statistical power to detect an efficacy signal, strengthening internal validity. However, it severely limits external generalizability; the 'real-world' incidence of early adverse effects (e.g., severe GI intolerance) and subsequent drug discontinuation will be significantly higher in clinical practice than the rates reported in the post-randomization phase of this trial.

Journal Editor
Journal Editor

A notable finding in LoDoCo2 was the numerically higher rate of non-cardiovascular death in the treatment arm (0.7 vs 0.5 events per 100 person-years). As a reviewer, what specific statistical methodology and data breakdowns would you require the authors to provide to ensure this competing risk is transparently addressed?

Key Response

A seasoned reviewer would demand a competing risk analysis (e.g., the Fine-Gray model) for the primary composite endpoint to ensure cardiovascular events are not being artificially reduced by patients dying first from non-CV causes. Furthermore, the reviewer would require a granular, categorized breakdown of non-CV deaths (e.g., specific infections, malignancies) to rule out a biologically plausible harm of chronic systemic immunosuppression, ensuring the abstract's conclusions reflect both the efficacy and potential latent safety signals.

Guideline Committee
Guideline Committee

Given the results of LoDoCo2 and COLCOT, how should guidelines incorporate low-dose colchicine for secondary prevention of ASCVD, and how does this align with the latest AHA/ACC/Multisociety guidelines on the management of chronic coronary disease?

Key Response

Guideline committees must evaluate whether the evidence warrants a Class IIa or IIb recommendation. The 2023 AHA/ACC/ACCP/ASPC/NLA/PCNA Guideline for the Management of Patients With Chronic Coronary Disease officially introduced low-dose colchicine as a Class IIb recommendation for secondary prevention to reduce recurrent ASCVD events in patients who are on optimal lipid-lowering therapy. The committee must weigh the high quality of evidence (multiple RCTs) against the modest absolute risk reduction, the lack of all-cause mortality benefit, and the stringent need to monitor drug-drug interactions when drafting these updates.

Clinical Landscape

Noteworthy Related Trials

2013

LoDoCo Trial

n = 532 · JACC

Tested

Colchicine 0.5mg daily

Population

Patients with stable coronary artery disease

Comparator

No colchicine (open-label control)

Endpoint

Composite of acute coronary syndrome, out-of-hospital cardiac arrest, or noncardioembolic stroke

Key result: Low-dose colchicine was highly effective at reducing cardiovascular events in patients with stable coronary disease.
2017

CANTOS Trial

n = 10,061 · NEJM

Tested

Canakinumab (anti-IL-1beta antibody)

Population

Patients with previous MI and elevated hs-CRP

Comparator

Placebo

Endpoint

Composite of nonfatal MI, nonfatal stroke, or CV death

Key result: Anti-inflammatory therapy with canakinumab led to a significantly lower rate of recurrent cardiovascular events than placebo, independent of lipid lowering.
2019

COLCOT Trial

n = 4,745 · NEJM

Tested

Colchicine 0.5mg daily

Population

Patients with recent myocardial infarction

Comparator

Placebo

Endpoint

Composite of CV death, resuscitated cardiac arrest, MI, stroke, or urgent hospitalization for angina

Key result: Colchicine significantly lowered the risk of ischemic cardiovascular events compared to placebo following a recent MI.

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