The Lancet Oncology DECEMBER 07, 2020

Short-course radiotherapy followed by chemotherapy before total mesorectal excision (TME) versus preoperative chemoradiotherapy, TME, and optional adjuvant chemotherapy in locally advanced rectal cancer (RAPIDO): a randomised, open-label, phase 3 trial

Renu R. Bahadoer, Esmée A. Dijkstra, Boudewijn van Etten, Corrie A. M. Marijnen, et al.

Bottom Line

The RAPIDO trial demonstrated that a total neoadjuvant therapy (TNT) approach—consisting of short-course radiotherapy followed by systemic chemotherapy—significantly reduces disease-related treatment failure and distant metastases compared to standard preoperative chemoradiotherapy in patients with high-risk locally advanced rectal cancer.

Key Findings

1. The experimental TNT regimen significantly reduced the 3-year cumulative probability of disease-related treatment failure (DrTF) to 23.7% compared with 30.4% in the standard-of-care arm (hazard ratio 0.76; 95% CI 0.60–0.96; p=0.02).
2. The benefit in DrTF was primarily driven by a significant reduction in distant metastases, with a 3-year cumulative incidence of 19.8% in the experimental arm versus 26.6% in the standard arm (hazard ratio 0.69; 95% CI 0.53–0.89; p=0.004).
3. Pathological complete response (pCR) rates were doubled in the experimental arm at 27.7% compared to 13.8% in the standard arm (odds ratio 2.40; p<0.001).
4. Locoregional failure rates were not significantly different between the two groups, with 8.7% in the experimental arm and 6.0% in the standard arm (hazard ratio 1.45; 95% CI 0.93–2.25; p=0.10).
5. Overall survival at 3 years was comparable between the two arms (approximately 89%).

Study Design

Design
RCT
Open-Label
Sample
920
Patients
Duration
4.5 yr
Median
Setting
Multicenter, Europe
Population Patients with MRI-diagnosed locally advanced rectal cancer featuring high-risk factors (cT4a/b, extramural vascular invasion, cN2, involved mesorectal fascia, or enlarged lateral lymph nodes).
Intervention Short-course radiotherapy (5x5 Gy) followed by six cycles of CAPOX or nine cycles of FOLFOX4 and subsequent total mesorectal excision (TME).
Comparator Preoperative capecitabine-based chemoradiotherapy followed by TME and optional, institutional-policy-dependent postoperative chemotherapy.
Outcome Disease-related treatment failure (DrTF), defined as the first occurrence of locoregional failure, distant metastasis, a new primary colon tumor, or treatment-related death.

Study Limitations

The trial was open-label, which may introduce potential bias, although objective endpoints like DrTF and pCR minimize this risk.
There was a numerical, though not statistically significant, increase in locoregional failure in the experimental arm, which requires ongoing long-term monitoring.
Postoperative chemotherapy in the control arm was optional, reflecting variation in international clinical practice that complicates direct comparisons with rigid adjuvant protocols.
The study specifically focused on high-risk locally advanced rectal cancer, limiting the generalizability of these results to lower-risk populations.

Clinical Significance

The RAPIDO trial establishes total neoadjuvant therapy as a new standard of care for high-risk locally advanced rectal cancer. By shifting chemotherapy to the preoperative setting, this approach achieves higher treatment compliance, higher rates of tumor downstaging (evidenced by increased pCR), and superior control of distant systemic disease compared to the conventional strategy of adjuvant chemotherapy.

Historical Context

Historically, the standard management for locally advanced rectal cancer focused on long-course chemoradiotherapy followed by TME surgery, with adjuvant chemotherapy administered postoperatively to reduce systemic relapse. However, poor adherence to postoperative chemotherapy and the high risk of distant metastases prompted the exploration of total neoadjuvant therapy (TNT) to optimize systemic treatment delivery before surgery.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the physiological and clinical rationale for using a 'Total Neoadjuvant Therapy' (TNT) approach, as seen in the RAPIDO trial, instead of the traditional sequence of preoperative chemoradiotherapy followed by postoperative adjuvant chemotherapy?

Key Response

TNT aims to deliver all systemic therapy early, when the patient is most fit and compliance is highest. Clinically, this treats micrometastatic disease sooner, increases the likelihood of a pathologic complete response (pCR), and avoids the common delays or omissions of adjuvant chemotherapy that often occur due to postoperative complications or slow recovery from surgery.

Resident
Resident

Based on the RAPIDO inclusion criteria, which specific MRI-defined 'high-risk' features would lead you to recommend the experimental short-course radiotherapy/chemotherapy protocol over standard long-course chemoradiotherapy?

Key Response

The RAPIDO trial specifically targeted high-risk locally advanced rectal cancer defined by MRI as having at least one of the following: cT4a or cT4b tumors, extramural venous invasion (EMVI+), cN2 stage, involved mesorectal fascia (MRF+), or enlarged lateral lymph nodes. These patients are at higher risk for distant metastasis, making the systemic-first TNT approach particularly beneficial.

Fellow
Fellow

Compare the 'Short-Course RT to Chemotherapy' sequence used in RAPIDO with the 'Chemotherapy to Long-Course CRT' sequence used in PRODIGE 23. What are the trade-offs regarding local control and toxicity?

Key Response

RAPIDO used 5x5 Gy followed by CAPOX/FOLFOX, showing excellent systemic control but a slightly higher 5-year local recurrence rate (10% vs 6%). PRODIGE 23 used FOLFIRINOX followed by long-course CRT (50 Gy with capecitabine), which generally provides more potent radiosensitization for local control. Fellows must weigh the convenience and systemic intensity of RAPIDO against the potentially superior local downstaging of long-course-based TNT.

Attending
Attending

The 5-year follow-up of the RAPIDO trial revealed a higher rate of locoregional recurrence in the experimental TNT group compared to the standard of care. How does this finding influence your multidisciplinary discussion for a patient with a very low rectal tumor where an R0 resection is borderline?

Key Response

The increased local recurrence (10% vs 6%) in the TNT arm suggests that while short-course RT followed by chemotherapy is excellent for preventing distant disease, it may be less effective at ensuring long-term local pelvic control than long-course chemoradiotherapy. In cases where the circumferential resection margin (CRM) is threatened or for very low tumors where sphincter preservation is the goal, an attending might favor long-course CRT to maximize local regression.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The primary endpoint of RAPIDO was 'disease-related treatment failure' (DrTF). Critically evaluate the limitations of using this composite endpoint in a surgical oncology trial where local and distant failure modes have different clinical implications and salvage possibilities.

Key Response

Composite endpoints like DrTF (including local recurrence, metastases, and death) increase statistical power by capturing more events, but they assume that all components are of equal clinical weight. In rectal cancer, a distant metastasis is often terminal, whereas a local recurrence might be surgically salvageable. By grouping them, the trial may mask a treatment that improves one while worsening the other, requiring researchers to carefully deconstruct the components in secondary analyses.

Journal Editor
Journal Editor

One notable observation in the RAPIDO control arm was that only 58% of patients received the intended adjuvant chemotherapy. To what extent does this 'pragmatic' failure of the control arm jeopardize the internal validity of the trial's conclusion regarding the superiority of TNT?

Key Response

A tough reviewer would argue that the trial compares TNT not just to 'standard timing,' but to an 'under-treated' control group. If the benefit of TNT is merely that patients actually received the chemotherapy they were supposed to get, the study proves the logistical superiority of neoadjuvant delivery rather than a biological advantage of the timing itself. This highlights the difficulty of executing 'standard' adjuvant arms in modern colorectal trials.

Guideline Committee
Guideline Committee

Given the results of RAPIDO and the PRODIGE 23 trials, how should current guidelines (such as NCCN or ESMO) be updated regarding the 'Level of Evidence' for TNT in high-risk LARC, and should short-course RT-based TNT be preferred over long-course-based TNT?

Key Response

Both RAPIDO and PRODIGE 23 provide Category 1/Level 1a evidence for TNT as a standard of care for high-risk LARC. However, guidelines must remain nuanced; the NCCN already lists both as options. The committee must decide whether to issue a 'preference' based on the 5-year local recurrence data from RAPIDO, potentially recommending long-course CRT-based TNT (PRODIGE 23 style) for patients where local control is the primary concern (e.g., MRF+ or low tumors).

Clinical Landscape

Noteworthy Related Trials

2004

CAO/ARO/AIO-94 Trial

n = 823 · NEJM

Tested

Preoperative chemoradiotherapy (5-FU based)

Population

Patients with clinical T3 or T4 rectal cancer

Comparator

Postoperative chemoradiotherapy

Endpoint

Disease-free survival

Key result: Preoperative chemoradiotherapy resulted in lower local recurrence rates and reduced toxicity compared to postoperative treatment.
2014

Polish II Trial

n = 515 · Lancet Oncol

Tested

Preoperative chemoradiotherapy (oxaliplatin/5-FU)

Population

Patients with clinical T3 or T4 rectal cancer

Comparator

Preoperative radiotherapy alone

Endpoint

3-year disease-free survival

Key result: Preoperative chemoradiotherapy did not improve disease-free survival compared to radiotherapy alone, though it increased pathological complete response rates.
2021

PRODIGE 23 Trial

n = 461 · Lancet Oncol

Tested

Neoadjuvant FOLFIRINOX followed by chemoradiotherapy and TME

Population

Patients with locally advanced rectal cancer

Comparator

Standard preoperative chemoradiotherapy and TME

Endpoint

Disease-free survival

Key result: The addition of neoadjuvant FOLFIRINOX significantly improved disease-free survival and metastasis-free survival compared to the standard approach.

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