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Deferral of surgery in people having neoadjuvant therapy for rectal cancer
Evidence review C4
NICE Guideline, No. 151
Authors
National Guideline Alliance (UK).Deferral of surgery in people having neoadjuvant therapy for rectal cancer
This evidence review supports recommendations 1.3.4 to 1.3.5.
Review question
Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
Introduction
People whose rectal cancer shows a complete clinical response to neoadjuvant therapy may choose to defer surgery, thereby avoiding the risk of surgical morbidity. However, despite having a complete clinical response some patients following such a watch and wait approach will experience locoregional recurrence or progression. This review question aimed to identify prognostic factors that predict recurrence and survival to better select people for watch and wait management.
Summary of protocol
Please see Table 1 for a summary of the population, prognostic factors, and outcomes (PPO) characteristics of this review.
For full details see the review protocol in appendix A
Methods and process
This evidence review was developed using the methods and process described in Developing NICE guidelines: the manual 2014. Methods specific to this review question are described in the review protocol in appendix A.
Declarations of interest were recorded according to NICE’s 2014 conflicts of interest policy until 31 March 2018. From 1 April 2018, declarations of interest were recorded according to NICE’s 2018 conflicts of interest policy. Those interests declared until April 2018 were reclassified according to NICE’s 2018 conflicts of interest policy (see Register of Interests).
Clinical evidence
Included studies
A systematic review of the clinical literature was conducted but no studies were identified which were applicable to this review question.
See the literature search strategy in appendix B and study selection flow chart in appendix C.
Excluded studies
No studies were identified which were applicable to this review question.
Summary of clinical studies included in the evidence review
No studies were identified which were applicable to this review question (and so there are no evidence tables in appendix D). No meta-analysis was undertaken for this review (and so there are no forest plots in appendix E).
Quality assessment of clinical outcomes included in the evidence review
No studies were identified which were applicable to this review question.
Economic evidence
Included studies
One relevant study was identified in a literature review of published cost-effectiveness analyses on this topic (Rao 2017; see appendix H and appendix I for summary and full evidence tables). The study considered the cost-effectiveness of watch and wait in comparison to radical surgery for patients with rectal cancer after a clinical complete response following chemoradiotherapy. The study considered three patient groups; 60 year old male cohort with no co-morbidities, 80 year old male cohort with no co-morbidities and 80 year old male cohort with significant co-morbidities.
The analysis was a cost-utility analysis measuring effectiveness in terms of quality adjusted life years (QALYs).
Excluded studies
A global search of economic evidence was undertaken for all review questions in this guideline. See Supplement 2 for further information.
Summary of studies included in the economic evidence review
The base case results of Rao 2017 suggest that watch and wait was found to be more effective and more costly than radical surgery in all modelled patient groups. The strategy was therefore dominant in all patient groups.
Uncertainty was assessed using deterministic and probabilistic sensitivity analysis. Results were found to be sensitive to relative recurrence rates after watch and wait (WW) and radical surgery as well as changes in the quality of life (QoL) reduction with radical surgery. It was also found that the model became sensitive to changes in perioperative mortality when the QoL benefit of WW was reduced. In probabilistic sensitivity analysis watch and wait was found to have a 74%, 85% and 90% probability of being cost-effective in the 60 year old male cohort, 80 year old male cohort with no co-morbidities and 80 year old male cohort with significant co-morbidities, respectively.
Despite being a UK study considering the NHS perspective, the study was considered to be only partially applicable. This is because it doesn’t directly address the review question posed in the guideline (but it is partially addressed by the different subgroups considered in the analysis). Whilst the study meets most of the requirements of an adequate economic evaluation (see Developing NICE guidelines: appendix H), it was deemed to have some potentially serious limitations. Most notably, a key aspect of the analysis is the QoL gain with watch and wait and this is based on QoL values from another disease area (prostate cancer).
Economic model
No economic modelling was undertaken for this review because the committee agreed that other topics were higher priorities for economic evaluation.
Evidence statements
Clinical evidence statements
No clinical evidence was identified which was applicable to this review question.
Economic evidence statements
One relevant study was identified in the literature review of published cost effectiveness analyses on this topic (Rao 2017). This was a cost utility study, partially applicable to the decision problem with potentially serious methodological limitations, comparing radical surgery to a ‘watch and wait’ strategy involving outpatient imaging and monitoring in male patients who had had a complete response to neoadjuvant therapy and were suitable for surgery for rectal cancer. ‘Watch and wait’ was the dominant intervention in all subgroups leading to a reduction in both costs (ranging from £6,274 to £8,095) and an increase in QALYs (ranging from 0.56 to 0.72). Probabilistic sensitivity analysis estimated the probability of ‘watch and wait’ being cost effective when QALYs are valued at £20,000 each, is over 74% for all sub-groups.
The committee’s discussion of the evidence
Interpreting the evidence
The outcomes that matter most
Locoregional progression or recurrence was a critical outcome because it typically leads to further treatment with associated treatment related adverse effects. Overall survival and disease free survival were also critical outcomes because a watch and wait strategy (with deferred surgery) would only be safe if it did not impact survival. Organ preservation rate was an important outcome because organ preservation avoids the morbidity and functional consequences of major surgery.
The quality of the evidence
No evidence was identified which was applicable to this review question.
Benefits and harms
Surgery is the gold standard treatment for rectal cancer. However, some people whose rectal cancer shows a complete clinical response to neoadjuvant therapy wish to defer surgery and opt for an organ preserving ‘watch and wait’ strategy instead. The committee acknowledged that while the watch and wait strategy avoids harms due to surgery around one third will experience local regrowth of their tumour and need salvage surgery. Any local regrowth needs to be detected and treated to avoid disease progression, however this involves a surveillance protocol with repeated examinations which may be inconvenient for some patients.
No evidence was identified on the prognostic factors which could predict recurrence or survival, therefore, there is no evidence to help identify groups of patients for whom deferral of surgery would or would not be appropriate. The committee also recognised the lack of agreed definition of complete clinical or radiological response bringing further uncertainty to who might be candidates for deferral of surgery. For these reasons the committee could not recommend deferral of surgery.
The committee agreed that if a person wishes to defer surgery, they should be informed that there is no evidence to help define for whom deferral might be appropriate and that there is a risk of recurrence. If a person still chooses to defer surgery, deferral should only happen in the context of a clinical trial or a national registry where patients are closely monitored in order to detect and treat any local regrowth of their tumour. Patients should be encouraged to enter a clinical trial (for example on going trials OPERA or TRIGGER) and data collection via a national registry should be ensured. This would generate evidence in the future to help define groups that might benefit from deferral of surgery.
Cost effectiveness and resource use
One relevant study was identified in the literature review of published cost effectiveness analyses on this topic (Rao 2017). This was a cost utility study comparing radial surgery to a ‘watch and wait’ strategy involving outpatient imaging and monitoring in male patients who had had a complete response to neoadjuvant therapy and were suitable for surgery for rectal cancer. Three different patient groups were considered - 60 year olds with no comorbidities, 80 year olds with no comorbidities and 80 year olds with significant comorbidities. The model was a decision tree and markov model informed by previous estimates from the literature. All costs were taken from NHS reference costs and the analysis took a NHS & PSS perspective.
‘Watch and wait’ was the dominant intervention in all subgroups leading to a reduction in both costs (ranging from £6,274 to £8,095) and an increase in QALYs (ranging from 0.56 to 0.72). Deterministic sensitivity analysis was conducted in two ways. Alternative scenarios to the base case were explored which involved applying National Comprehensive Cancer Network (NCCN) protocols for follow-up, correlated cost parameters or doubling all costs. Watch and wait remained dominant under all these alternate assumptions.
It was found that the results of the model were sensitive to relative recurrence rates after watch and wait and radical surgery as well as changes in the quality of life reduction with radical surgery. It was also found that the model became sensitive to changes in perioperative mortality when the quality of life benefit of ‘watch and wait’ was reduced. The model was not found to be sensitive to variations in baseline mortality and operative mortality or individual cost parameters. Probabilistic sensitivity analysis estimated the probability of ‘watch and wait’ being cost effective at a £20,000 per QALY threshold at over 74% for all sub-groups.
Despite being a recent UK cost effectiveness study it was deemed only partially applicable to the review questions as it did not directly address the review question posed in the guideline. The question was only partially addressed by the different subgroups considered. It was also deemed to have some potentially serious methodological limitations. Most notably, a key aspect of the analysis is the quality of life gain with ‘watch and wait’ and this is based on values from another disease area (prostate cancer).
The committee found the study to be of limited value in addressing the review question because it didn’t consider the patient factors which were of most interest.
Other factors the committee took into account
The committee were aware of an international registry of patients with rectal cancer managed by a watch and wait strategy after complete clinical response to neoadjuvant therapy. Only a multicentre project like this is likely to collect sufficient patient numbers to answer the question of who is best suited to a watch and wait strategy. Also ongoing trials such as OPERA and TRIGGER may generate evidence in the future on who is most suitable for deferral of surgery. For this reason they chose not to make a research recommendation for a new trial.
References
Rao 2017
Rao C, Sun Myint A, Athanasiou T, et al. (2017) Avoiding Radical Surgery in Elderly Patients With Rectal Cancer Is Cost-Effective. Diseases of the Colon and Rectum 60(1): 30–42 [PubMed: 27926555]
Appendices
Appendix A. Review protocol
Review protocol for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
Table 2Review protocol for deferral of surgery in people having neoadjuvant therapy for rectal cancer
| Field (based on PRISMA-P) | Content |
|---|---|
| Review question | Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery? |
| Type of review question | Prognostic/clinical prediction review |
| Objective of the review | To determine the predictors for people having neoadjuvant chemotherapy or chemoradiotherapy for rectal cancer who do not need surgery. |
| Eligibility criteria – population/disease/condition/issue/domain |
Adults with non-metastatic rectal cancer who have complete clinical response to neoadjuvant radiotherapy or chemoradiotherapy and are fit for, but who have not had, surgery. Rectal cancer: defined as any tumour within 15 cm from anal verge excluding anal canal. |
| Eligibility criteria – intervention(s)/exposure(s)/prognostic factor(s) | Included studies must have at least 5 of the following predictor variables in their models Predictors:
|
| Confounding factors |
Analysis should adjust for important confounding factors, such as:
|
| Outcomes and prioritisation | Critical:
|
| Eligibility criteria – study design | Include published full text papers:
|
| Other inclusion exclusion criteria | Inclusion: English-language All settings will be considered that consider medications and treatments available in the UK Studies published post 2000 Studies published post 2000 will be considered for this review question, as the guideline committee considered that significant advances have occurred in rectal cancer management since this time period and outcomes for patients with rectal cancer prior to 2000 are not the same as post 2000. |
| Proposed sensitivity/sub-group analysis, or meta-regression | In the case of high heterogeneity, the following factors will be considered:
|
| Selection process – duplicate screening/selection/analysis |
Sifting, data extraction and appraisal of methodological quality will be performed by the systematic reviewer. Any disputes will be resolved in discussion with the senior systematic reviewer and the Topic Advisor. Quality control will be performed by the senior systematic reviewer. Dual sifting will be undertaken for this question for a random 10% sample of the titles and abstracts identified by the search. |
| Data management (software) | NGA STAR software will be used for study sifting, data extraction, recording quality assessment using checklists and generating bibliographies/citations. |
| Information sources – databases and dates | Potential sources to be searched (to be confirmed by the Information Scientist): Medline, Medline In-Process, CCTR, CDSR, DARE, HTA, Embase Limits (e.g. date, study design): Apply standard animal/non-English language exclusion Dates: from 2000 |
| Identify if an update | Not an update |
| Author contacts | https://www |
| Highlight if amendment to previous protocol | For details please see section 4.5 of Developing NICE guidelines: the manual |
| Search strategy – for one database | For details please see appendix B. |
| Data collection process – forms/duplicate | A standardised evidence table format was used, see appendix D (clinical evidence tables) and H (economic evidence tables). |
| Data items – define all variables to be collected | For details please see evidence tables in appendix D (clinical evidence tables) or H (economic evidence tables). |
| Methods for assessing bias at outcome/study level | Standard study checklists were used to critically appraise individual studies. For details please see section 6.2 of Developing NICE guidelines: the manual Appraisal of methodological quality: The methodological quality of each study will be assessed using an appropriate checklist:
|
| Criteria for quantitative synthesis (where suitable) | Meta-analyses will be not be conducted for this prognostic review. |
| Methods for analysis – combining studies and exploring (in)consistency | The adjusted odds ratios and 95% confidence intervals will be plotted in RevMan, however pooled results will not be calculated. The forest plots will be used to visually see the studies alongside each other and to explore similarities and differences between studies. |
| Meta-bias assessment – publication bias, selective reporting bias | For details please see section 6.2 of Developing NICE guidelines: the manual. |
| Assessment of confidence in cumulative evidence | For details please see sections 6.4 and 9.1 of Developing NICE guidelines: the manual |
| Rationale/context – Current management | For details please see the introduction to the evidence review. |
| Describe contributions of authors and guarantor | A multidisciplinary committee developed the guideline. The committee was convened by The National Guideline Alliance and chaired by Peter Hoskin in line with section 3 of Developing NICE guidelines: the manual. Staff from The National Guideline Alliance undertook systematic literature searches, appraised the evidence, conducted meta-analysis and cost-effectiveness analysis where appropriate, and drafted the guideline in collaboration with the committee. For details please see Supplement 1. |
| Sources of funding/support | The National Guideline Alliance is funded by NICE and hosted by the Royal College of Obstetricians and Gynaecologists |
| Name of sponsor | The National Guideline Alliance is funded by NICE and hosted by the Royal College of Obstetricians and Gynaecologists |
| Roles of sponsor | NICE funds The National Guideline Alliance to develop guidelines for those working in the NHS, public health, and social care in England |
| PROSPERO registration number | Not registered |
BRAF: v-raf murine sarcoma b-viral oncogene homolog B1; CCTR: Cochrane Controlled Trials Register; CEA: carcinoembryonic antigen; CSDR: Cochrane Database of Systematic Reviews; DARE: Database of Abstracts of Reviews of Effects; HTA: Health Technology Assessment; LVI: lymphovascular invasion; MID: minimal important difference; MSI: microsatellite instability; NGA: National Guidelines Alliance; NHS: National Health Service; NICE: National Institute for Health and Care Excellence; PRISMA-P: Preferred Reporting Items for Systematic Reviews and Meta-Analysis for Protocols; PROSPERO: International prospective register of systematic reviews; QUIPS: Quality in prognostic studies; RAS: rat sarcoma virus oncogene homolog; RCT: randomised controlled trial; ROBINS-I: risk of bias in non-randomised studies of interventions; ROBIS: risk of bias in systematic reviews; TNM: cancer classification system, standing for tumour, nodal and metastasis stages; QUIPS: quality in prognosis studies
Appendix B. Literature search strategies
Literature search strategies for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
Database: Embase/Medline
Last searched on: 12/02/2019
| # | Search |
|---|---|
| 1 | exp Rectal Neoplasms/ use prmz |
| 2 | *rectum cancer/ or *rectum tumor/ |
| 3 | 2 use oemezd |
| 4 | exp Adenocarcinoma/ |
| 5 | (T1 or T2 or N0 or M0).ti,ab. |
| 6 | 1 or 3 |
| 7 | 4 or 5 |
| 8 | 6 and 7 |
| 9 | ((rectal or rectum) adj3 (cancer* or neoplas* or malignan* or tumo?r* or carcinom* or adeno*)).ti,ab. |
| 10 | early rect* cancer.ti,ab. |
| 11 | 6 or 8 or 9 or 10 |
| 12 | exp radiotherapy/ or exp radiation oncology/ or exp external beam radiotherapy/ or exp Brachytherapy/ or exp preoperative care/ or exp neoadjuvant therapy/ or exp multimodality cancer therapy/ or exp chemotherapy/ or exp antineoplastic agent/ or exp drug therapy/ or exp chemoradiotherapy/ or exp fluorouracil/ or exp folinic acid/ or exp capecitabine/ or exp oxaliplatin/ or exp bevacizumab/ or exp methotrexate/ or exp radiation dose fractionation/ or exp tumor recurrence/ or exp radiotherapy dosage/ |
| 13 | 12 use oemezd |
| 14 | exp Radiotherapy/ or exp Radiation Oncology/ or exp Radiotherapy, Computer-Assisted/ or exp Brachytherapy/ or exp Preoperative Care/ or exp Neoadjuvant Therapy/ or exp Combined Modality Therapy/ or exp Chemoradiotherapy/ or exp Antineoplastic Combined Chemotherapy Protocols/ or exp Drug Therapy/ or exp Antineoplastic Agents/ or exp Fluorouracil/ or exp Leucovorin/ or exp Capecitabine/ or exp Bevacizumab/ or exp Methotrexate/ or exp Dose Fractionation/ or exp radiotherapy dosage/ |
| 15 | 14 use prmz |
| 16 | ((radiotherap* or chemoradio* or radiation or brachytherapy* or chemotherapy*) adj (pre?op* or preop* or periop* or neoadjuvant)).ti,ab. |
| 17 | (5-fluorouracil or 5-FU or leucovorin or folinic acid or capecitabine or oxaliplatin or bevacizumab or methotrexate or dose* or fraction* or recurren*).ti,ab. |
| 18 | 13 or 15 or 16 or 17 |
| 19 | exp Organ Preservation/ or Organ Sparing Treatments/ or exp Treatment Outcome/ or exp Disease-Free Survival/ or exp Neoplasm Recurrence, Local/ or exp Neoplasm, Residual/ or exp Lymph Nodes/ or exp Risk Factors/ or exp Prognosis/ or exp Observation/ or exp Watchful Waiting/ or exp Time Factors/ or exp Comorbidity/ or exp Age Factors/ or exp Health Status/ or exp Health Status Indicators/ or exp Morbidity/ or exp Physical Fitness/ |
| 20 | 19 use prmz |
| 21 | exp organ preservation/ or exp conservative treatment/ or exp treatment outcome/ or exp disease free survival/ or exp tumor recurrence/ or exp minimal residual disease/ or lymph node/ or exp lymph node/ or exp risk factor/ or exp prognosis/ or exp observation/ or exp watchful waiting/ or exp time factor/ or exp adjuvant therapy/ or exp cancer control/ or exp comorbidity/ or exp health status indicator/ or exp morbidity/ or age/ or exp performance/ or fitness/ or (exp patient/ and exp health status/) |
| 22 | 21 use oemezd |
| 23 | (prognos* or preservation or preserve* or sparing or complete response* or predict* or watch* or wait* or observ* or risk* or regrowth or recurren* or adjuvant or downstag* or downsize* or local control or residual or morbid* or poor perform* or delay* or unfit or fit or (lymph node adj (count or status)) or histolog* or outcome or ((avoid* or suit*) adj3 surger*)).ti,ab. |
| 24 | 20 or 22 or 23 |
| 25 | 11 and 18 and 24 |
| 26 | limit 25 to english language |
| 27 | limit 26 to yr=“2000 -Current” |
| 28 | (conference abstract or letter).pt. or letter/ or editorial.pt. or note.pt. or case report/ or case study/ use oemezd |
| 29 | Letter/ or editorial/ or news/ or historical article/ or anecdotes as topic/ or comment/ or case report/ use prmz |
| 30 | (letter or comment* or abstracts).ti. |
| 31 | or/28–30 |
| 32 | randomized controlled trial/ use prmz |
| 33 | randomized controlled trial/ use oemezd |
| 34 | random*.ti,ab. |
| 35 | or/32–34 |
| 36 | 31 not 35 |
| 37 | (animals/ not humans/) or exp animals, laboratory/ or exp animal experimentation/ or exp models, animal/ or exp rodentia/ use prmz |
| 38 | (animal/ not human/) or nonhuman/ or exp animal experiment/ or exp experimental animal/ or animal model/ or exp rodent/ use oemezd |
| 39 | (rat or rats or mouse or mice).ti. |
| 40 | 36 or 37 or 38 or 39 |
| 41 | 27 not 40 |
Database: Cochrane Library
Last searched on: 12/02/2019
| # | Search |
|---|---|
| 1 | MeSH descriptor: [Rectal Neoplasms] explode all trees |
| 2 | MeSH descriptor: [Adenocarcinoma] explode all trees |
| 3 | T1 or T2 or N0 or M0 |
| 4 | #2 or #3 |
| 5 | #1 and #4 |
| 6 | (rectal or rectum) near (cancer* or neoplas* or malignan* or tumo?r* or carcinom* or adeno*) |
| 7 | early rect* cancer |
| 8 | #1 or #5 or #6 or #7 |
| 9 | MeSH descriptor: [Radiotherapy] explode all trees |
| 10 | MeSH descriptor: [Radiation Oncology] explode all trees |
| 11 | MeSH descriptor: [Radiotherapy, Computer-Assisted] explode all trees |
| 12 | MeSH descriptor: [Brachytherapy] explode all trees |
| 13 | MeSH descriptor: [Preoperative Care] explode all trees |
| 14 | MeSH descriptor: [Neoadjuvant Therapy] explode all trees |
| 15 | MeSH descriptor: [Combined Modality Therapy] explode all trees |
| 16 | MeSH descriptor: [Chemoradiotherapy] explode all trees |
| 17 | MeSH descriptor: [Antineoplastic Combined Chemotherapy Protocols] explode all trees |
| 18 | MeSH descriptor: [Drug Therapy] explode all trees |
| 19 | MeSH descriptor: [Antineoplastic Agents] explode all trees |
| 20 | MeSH descriptor: [Fluorouracil] explode all trees |
| 21 | MeSH descriptor: [Capecitabine] explode all trees |
| 22 | MeSH descriptor: [Bevacizumab] explode all trees |
| 23 | MeSH descriptor: [Methotrexate] explode all trees |
| 24 | MeSH descriptor: [Dose Fractionation] explode all trees |
| 25 | (radiotherap* or chemoradio* or radiation or brachytherapy* or chemotherapy*) near (pre?op* or preop* or periop* or neoadjuvant) |
| 26 | 5-fluorouracil or 5-FU or leucovorin or folinic acid or capecitabine or oxaliplatin or bevacizumab or methotrexate or dose* or fraction* or recurren* |
| 27 | #9 or #10 or #11 or #12 or #13 or #14 or #15 or #16 or #17 or #18 or #19 or #20 or #21 or #22 or #23 or #24 or #25 or #26 |
| 28 | MeSH descriptor: [Organ Preservation] explode all trees |
| 29 | MeSH descriptor: [Organ Sparing Treatments] explode all trees |
| 30 | MeSH descriptor: [Treatment Outcome] explode all trees |
| 31 | MeSH descriptor: [Disease-Free Survival] explode all trees |
| 32 | MeSH descriptor: [Neoplasm Recurrence, Local] explode all trees |
| 33 | MeSH descriptor: [Neoplasm, Residual] explode all trees |
| 34 | MeSH descriptor: [Lymph Nodes] explode all trees |
| 35 | MeSH descriptor: [Risk Factors] explode all trees |
| 36 | MeSH descriptor: [Prognosis] explode all trees |
| 37 | MeSH descriptor: [Observation] explode all trees |
| 38 | MeSH descriptor: [Watchful Waiting] explode all trees |
| 39 | MeSH descriptor: [Time Factors] explode all trees |
| 40 | prognos* or preservation or preserve* or sparing or complete response* or predict* or watch* or wait* or observ* or risk* or regrowth or recurren* or adjuvant or downstag* or downsize* or local control or residual or histolog* or outcome |
| 41 | lymph node near (count or status) |
| 42 | (avoid* or suit*) near surger* |
| 43 | #28 or #29 or #30 or #31 or #32 or #33 or #34 or #35 or #36 or #37 or #38 or #39 or #40 or #41 or #42 |
| 44 | #8 and #27 and #43 Publication Year from 2000 to 2018 |
Appendix C. Clinical evidence study selection
Clinical study selection for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
Figure 1Study selection flow chart
Appendix D. Clinical evidence tables
Clinical evidence tables for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
No clinical evidence was identified which was applicable to this review question.
Appendix E. Forest plots
Forest plots for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
No clinical evidence was identified which was applicable to this review question.
Appendix F. GRADE tables
GRADE tables for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
No clinical evidence was identified which was applicable to this review question.
Appendix G. Economic evidence study selection
Economic evidence study selection for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
A global search of economic evidence was undertaken for all review questions in this guideline. See Supplement 2 for further information.
Appendix H. Economic evidence tables
Economic evidence tables for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
Appendix I. Economic evidence profiles
Economic evidence profiles for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
Appendix J. Economic analysis
Economic evidence analysis for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
No economic analysis was conducted for this review question.
Appendix K. Excluded studies
Excluded clinical studies for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
Table 5Excluded studies and reasons for their exclusion
| Study | Reason for exclusion |
|---|---|
| Abrams, M. J., Koffer, P. P., Leonard, K. L., The emerging non-operative management of non-metastatic rectal cancer: A population analysis, Anticancer Research, 36, 1699–1702, 2016 [PubMed: 27069148] | Patients not selected for complete clinical response |
| Alongi, F., Mazzola, R., Watch-and-wait versus surgical resection for patients with rectal cancer, The Lancet Oncology, 17, e133–e134, 2016 [PubMed: 27300671] | Letter in response to Renehan (2015) |
| Appelt, A. L., Ploen, J., Harling, H., Jensen, F. S., Jensen, L. H., Jorgensen, J. C. R., Lindebjerg, J., Rafaelsen, S. R., Jakobsen, A., High-dose chemoradiotherapy and watchful waiting for distal rectal cancer: A prospective observational study, The Lancet Oncology, 16, 919–927, 2015 [PubMed: 26156652] | No analysis of prognostic factors |
| Araujo, R. O. C., Valadao, M., Borges, D., Linhares, E., De Jesus, J. P., Ferreira, C. G., Victorino, A. P., Vieira, F. M., Albagli, R., Nonoperative management of rectal cancer after chemoradiation opposed to resection after complete clinical response. A comparative study, European Journal of Surgical Oncology, 41, 1456–1463, 2015 [PubMed: 26362228] | No multivariate prognostic analysis. Univariate data for: distance from anal verge. |
| Bannura, G., Outcome and salvage surgery following “watch and wait” for rectal cancer after neoadjuvant therapy: A systematic review, Revista Chilena de Cirugia, 352, 2017 | Non English language |
| Beets, G. L., Critical appraisal of the ‘wait and see’ approach in rectal cancer for clinical complete responders after chemoradiation, British Journal of Surgery, 99, 910, 2012 [PubMed: 22648639] | Commentary on systematic review (Glynne-Jones. 2012) |
| Beets, G. L., What are We Going to Do with Complete Responses After Chemoradiation of Rectal Cancer?, Annals of Surgical Oncology, 23, 1801–1802, 2016 [PubMed: 26957500] | Expert review |
| Beets, G. L., Figueiredo, N. L., Habr-Gama, A., Van De Velde, C. J. H., A new paradigm for rectal cancer: Organ preservation Introducing the International Watch & Wait Database (IWWD), European Journal of Surgical Oncology, 41, 1562–1564, 2015 [PubMed: 26493223] | Describes watch-and-watch international database. |
| Benezery, K., Chamorey, E., Francois, E., Doyen, J., Gourgou-Bourgade, S., Gerard, J. P., Clinical complete response after neoadjuvant chemoradiotherapy (nCRT) of rectal cancer: A key end point to increase conservative treatment - Findings from the ACCORD12 randomized trial, European Journal of Cancer, 49, S501–S502, 2013 | Conference abstract |
| Bhangu, A., Kiran, R. P., Audisio, R., Tekkis, P., Survival outcome of operated and non-operated elderly patients with rectal cancer: A Surveillance, Epidemiology, and End Results analysis, European Journal of Surgical Oncology, 40, 1510–1516, 2014 [PubMed: 24704032] | Complete clinical response not an inclusion criteria |
| Bhatti, A. B. H., Zaheer, S., Shafique, K., Prognostic role of acellular mucin pools in patients with rectal cancer after pathological complete response to preoperative chemoradiation: Systematic review and meta-analysis, Journal of the College of Physicians and Surgeons Pakistan, 27, 714–718, 2017 [PubMed: 29132485] | Patients had surgery |
| Brooker, R., McKay, M., Crabtree, A., Wong, H., Sripadam, R., Organ sparing radiotherapy in rectal cancer: Definitive chemoradiation is a safe and valid option, Annals of Oncology, 26, iv96, 2015 | Conference abstract |
| Caderillo-Ruiz, G., Diaz, C., Lopez-Basave, H. N., Herrera, M. T., Ruiz Garcia, E., Melchor, J., Trejo, G., Aguilar, J. L., Gomez, A. H., Meneses-Garcia, A., Clinical outcome in patients who did not accept complementary surgery after neoadjuvant chemoradiotherapy (QT-RT) in locally advanced rectal cancer (LARC), Journal of Clinical Oncology. Conference, 34, 2016 | Conference abstract |
| Cotti, G., Nahas, C., Marques, C., Imperiale, A., Ribeiro Jr, U., Nahas, S., Cecconello, I., Hoff, P., Outcomes of nonsurgical treatment in patients with clinical complete response after neoadjuvant therapy for rectal cancer, Diseases of the Colon and Rectum, 59 (5), e262, 2016 | Conference abstract |
| Dattani, M., Heald, R. J., Goussous, G., Broadhurst, J., Sao Juliao, G. P., Habr-Gama, A., Perez, R. O., Moran, B. J., Oncological and Survival Outcomes in Watch and Wait Patients With a Clinical Complete Response After Neoadjuvant Chemoradiotherapy for Rectal Cancer: A Systematic Review and Pooled Analysis, Annals of Surgery, 268, 955–967, 2018 [PubMed: 29746338] | Does not report prognostic analysis |
| Dickson-Lowe, R. A., Hanek, P., Kalaskar, S., Taylor, J., Non-operative management of low rectal cancer with complete response to standard neoadjuvant chemoradiotherapy, Gut, 1), A554–A555, 2015 | Conference abstract |
| Dossa, F., Chesney, T. R., Acuna, S. A., Baxter, N. N., A watch-and-wait approach for locally advanced rectal cancer after a clinical complete response following neoadjuvant chemoradiation: a systematic review and meta-analysis, The Lancet Gastroenterology and Hepatology, 2, 501–513, 2017 [PubMed: 28479372] | Systematic review, does not report prognostic factor analysis. |
| Glynne-Jones, R., Hughes, R., Critical appraisal of the ‘wait and see’ approach in rectal cancer for clinical complete responders after chemoradiation, British Journal of Surgery, 99, 897–909, 2012 [PubMed: 22539154] | Systematic review, does not report prognostic factor analysis. |
| Glynne-Jones, R., Wallace, M., Livingstone, J. I. L., Meyrick-Thomas, J., Complete clinical response after preoperative chemoradiation in rectal cancer: Is a “wait and see” policy justified?, Diseases of the Colon and Rectum, 51, 10–19, 2008 [PubMed: 18043968] | Earlier version of Glynne-Jones (2012) systematic review |
| Gossedge, G., Montazeri, A., Nandhra, A., Wong, H., Artioukh, D., Zeiderman, M., Chipang, A., Myint, A., Complete clinical response to chemoradiotherapy for rectal cancer. Is it safe to ‘watch and wait’?, Colorectal Disease, 2), 20, 2012 | Conference abstract |
| Habr-Gama, A., Gama-Rodrigues, J., Sao Juliao, G. P., Proscurshim, I., Sabbagh, C., Lynn, P. B., Perez, R. O., Local recurrence after complete clinical response and watch and wait in rectal cancer after neoadjuvant chemoradiation: Impact of salvage therapy on local disease control, International Journal of Radiation Oncology Biology Physics, 88, 822–828, 2014 [PubMed: 24495589] | No multivariate prognostic analysis. Univariate data for: T stage, N stage. |
| Habr-Gama, A., Perez, R. O., Nadalin, W., Sabbaga, J., Ribeiro, U., Jr., Silva e Sousa, A. H., Jr., Campos, F. G., Kiss, D. R., GamaRodrigues, J., Operative versus nonoperative treatment for stage 0 distal rectal cancer following chemoradiation therapy: long-term results, Annals of Surgery, 240, 711–7; discussion 717–8, 2004 [PMC free article: PMC1356472] [PubMed: 15383798] | No prognostic factor analysis |
| Habr-Gama, A., Perez, R. O., Proscurshim, I., Campos, F. G., Nadalin, W., Kiss, D., Gama-Rodrigues, J., Patterns of Failure and Survival for Nonoperative Treatment of Stage c0 Distal Rectal Cancer Following Neoadjuvant Chemoradiation Therapy, Journal of Gastrointestinal Surgery, 10, 1319–1329, 2006 [PubMed: 17175450] | No multivariate prognostic analysis. Univariate data for: T stage, N stage. |
| Habr-Gama, A., Sabbaga, J., Gama-Rodrigues, J., Sao Juliao, G. P., Proscurshim, I., Bailao Aguilar, P., Nadalin, W., Perez, R. O., Watch and wait approach following extended neoadjuvant chemoradiation for distal rectal cancer: are we getting closer to anal cancer management?, Diseases of the Colon & RectumDis Colon Rectum, 56, 1109–17, 2013 [PubMed: 24022527] | No multivariate prognostic analysis. Univariate data for: T stage, N stage. |
| Habr-Gama, A., Sao Juliao, G. P., Perez, R. O., Nonoperative management of rectal cancer: Identifying the ideal patients, Hematology/Oncology Clinics of North America, 29, 135–151, 2015 [PubMed: 25475576] | Expert review |
| Heijnen, L. A., Maas, M., Martens, M. H., Lambregts, D. M. J., Van Drie, E., Stassen, L. P. S., Breukink, S. O., Leijtens, J. W. A., Beets-Tan, R. G. H., Beets, G. L., Endoscopy-based follow-up of clinical complete responders after chemoradiation for rectal cancer during a non-operative ‘wait-and-see’ policy, European Journal of Cancer, 2), S485, 2013 | Conference abstract |
| Hupkens, B., Martens, M., Kusters, M., Boelens, P., Meershoek-Klein Kranenbarg, E., Van Gestel, M., Ribeiro, R., Peeters, K., Perez, R., Figueiredo, N., Habr-Gama, A., Van De Velde, C., Beets, G., International watch and wait database: An international database of organ-preservation in rectal cancer, Colorectal Disease, 2), 68, 2015 | Conference abstract |
| Iseas IS, Carballido M, Coraglio M, et al., Moving forward and beyond the standard through a non-operative management in rectal cancer? Our watch and wait approach experience in CoRecto., Proc Am Soc Clin Oncol, 33, 2015 | Conference abstract |
| Jafari, M. D., Weiser, M. R., Personalizing Therapy for Locally Advanced Rectal Cancer, Current Colorectal Cancer Reports, 13, 119–125, 2017 | Expert review |
| Kessler, H., Matzel, K., Merkel, S., Fietkau, R., Hohenberger, W., Results of a “watch and wait” strategy in complete remission of rectal carcinoma after chemoradiotherapy, Diseases of the Colon and Rectum, 56 (4), e205, 2013 | Conference abstract |
| Kim, H. J., Song, J. H., Ahn, H. S., Choi, B. H., Jeong, H., Choi, H. S., Lee, Y. H., Kang, K. M., Jeong, B. K., Wait and see approach for rectal cancer with a clinically complete response after neoadjuvant concurrent chemoradiotherapy, International Journal of Colorectal Disease, 32, 723–727, 2017 [PubMed: 27885479] | Systematic review, does not report prognostic factor analysis. |
| Kong, J. C., Guerra, G. R., Warrier, S. K., Ramsay, R. G., Heriot, A. G., Outcome and Salvage Surgery Following “Watch and Wait” for Rectal Cancer after Neoadjuvant Therapy: A Systematic Review, Diseases of the Colon and Rectum, 60, 335–345, 2017 [PubMed: 28177997] | Systematic review, does not report prognostic factor analysis. |
| Kusters, M., Slater, A., Betts, M., Hompes, R., Guy, R. J., Jones, O. M., George, B. D., Lindsey, I., Mortensen, N. J., James, D. R., Cunningham, C., The treatment of all MRI-defined low rectal cancers in a single expert centre over a 5-year period: is there room for improvement?, Colorectal Disease, 18, O397–O404, 2016 [PubMed: 27313145] | Outcomes not reported for watch |
| Lai, C. L., Lai, M. J., Wu, C. C., Jao, S. W., Hsiao, C. W., Rectal cancer with complete clinical response after neoadjuvant chemoradiotherapy, surgery, or “watch and wait”, International Journal of Colorectal Disease, 31, 413–419, 2016 [PubMed: 26607907] | Does not report prognostic analysis. |
| Lambregts, D. M., Maas, M., Van Der Sande, M., Hupkens, B., Martens, M., Bakers, F., Beets-Tan, R. G. H., Breukink, S. O., Beets, G. L., Improving the selection of complete responders for watchful waiting after chemoradiotherapy for rectal cancer: What can we learn from the ‘missed’ pathologic complete responders after surgery?, United European Gastroenterology Journal, 5 (5 Supplement 1), A324, 2017 | Abstract only. |
| Latif, M, Day, N, Montazeri, A, Wait & see policy following complete clinical response to chemoradiotherapy in rectal cancer, single centre experience, Annals of oncology. Conference: 16th world congress on gastrointestinal cancer, ESMO 2014. Spain. Conference start: 20140625. Conference end: 20140628, 25, ii102–ii103, 2014 | Conference abstract |
| Li, J., Li, L., Yang, L., Yuan, J., Lv, B., Yao, Y., Xing, S., Wait-and-see treatment strategies for rectal cancer patients with clinical complete response after neoadjuvant chemoradiotherapy: A systematic review and meta-analysis, Oncotarget, 7, 44857–44870, 2016 [PMC free article: PMC5190140] [PubMed: 27070085] | Systematic review, No prognostic analysis reported. |
| Li, J., Liu, H., Yin, J., Liu, S., Hu, J., Du, F., Yuan, J., Lv, B., Fan, J., Leng, S., Zhang, X., Wait-and-see or radical surgery for rectal cancer patients with a clinical complete response after neoadjuvant chemoradiotherapy: A cohort study, Oncotarget, 6, 42354–42361, 2015 [PMC free article: PMC4747231] [PubMed: 26472284] | No prognostic analysis reported. |
| Maas, M, Beets-Tan, Rgh, Lambregts, Dmj, Lammering, G, Nelemans, Pj, Engelen, Sme, Dam, Rm, Jansen, Rlh, Sosef, M, Leijtens, Jwa, Hulsewe, Kwe, Buijsen, J, Beets, Gl, Wait-and-see policy for clinical complete responders after chemoradiation for rectal cancer, Journal of Clinical Oncology, 29, 4633–4640, 2011 [PubMed: 22067400] | No multivariate prognostic analysis. Univariate data for: T stage, N stage, distance from anal verge. |
| Martens, M. H., Maas, M., Heijnen, L. A., Lambregts, D. M. J., Leijtens, J. W. A., Stassen, L. P. S., Breukink, S. O., Hoff, C., Belgers, E. J., Melenhorst, J., Jansen, R., Buijsen, J., Hoofwijk, T. G. M., Beets-Tan, R. G. H., Beets, G. L., Long-term outcome of an organ preservation program after neoadjuvant treatment for rectal cancer, Journal of the National Cancer InstituteJ Natl Cancer Inst, 108 (12) (no pagination), 2016 [PubMed: 27509881] | No multivariate prognostic analysis. Univariate data for: T stage, N stage. |
| Mendoza, A. G., Morales, R. D., Russo, L., The first Venezuelan experience in nonoperative management of rectal cancer with complete clinical response following neoadjuvant therapy, Revista Venezolana de Oncologia, 29, 65–75, 2017 | Not English language |
| Myint, As, Smith, F, Whitmarsh, K, Wong, H, Pritchard, M, Non surgical treatment of operable rectal cancer: reducing harm from the standard of care in elderly patients, European journal of surgical oncology. Conference: joint BASO-ACS annual scientific conference and NCRI cancer conference 2016. United kingdom. Conference start: 20161106. Conference end: 20161109, 42, S228–s229, 2016 | Conference abstract |
| Nahas, C. S., Nahas, S. C., Marques, C. F., Ribeiro Jr, U., Bustamante-Lopez, L. A., Cecconello, I., Randomized controlled trial for complete clinical response in patients with locally advanced rectal cancer after neoadjuvant chemoradiotherapy: Observation versus surgical resection, European Journal of Surgical Oncology, 41, S148, 2015 | Conference abstract |
| Nahas, S., Nahas, C., Ribeiro Jr, U., Sparapan Marques, C., Cotti, G. C., Imperiale, A., Ortega, C., Azambuja, R., Chen, A., Hoff, P., Cecconello, I., Observation versus surgical resection in patients with rectal cancer who achieved complete clinical response after neoadjuvant chemoradiotherapy: Preliminary results of a randomized trial (NCT02052921), Diseases of the Colon and Rectum, 58 (5), e103–e104, 2015 | Conference abstract |
| Nahas, Sc, Rizkallah, Nahas Cs, Sparapan, Marques Cf, Ribeiro, U, Cotti, Gc, Imperiale, Ar, Capareli, Fc, Chih, Chen At, Hoff, Pm, Cecconello, I, Pathologic Complete Response in Rectal Cancer: can We Detect It? Lessons Learned From a Proposed Randomized Trial of Watch-and-Wait Treatment of Rectal Cancer, Diseases of the Colon and Rectum, 59, 255–263, 2016 [PubMed: 26953983] | N=4, no recurrence events. |
| Narang, S., Alam, N., Smart, N., Daniels, I., Non-operative management of Rectal Cancer: Too much hype?, Colorectal Disease, 2), 15, 2015 | Conference abstract |
| Neuman, H. B., Elkin, E. B., Guillem, J. G., Paty, P. B., Weiser, M. R., Wong, W. D., Temple, L. K., Treatment for patients with rectal cancer and a clinical complete response to neoadjuvant therapy: A decision analysis, Diseases of the Colon and Rectum, 52, 863–871, 2009 [PubMed: 19502849] | Decision model (not a primary study) - relapse rates during observation alone based on expert opinion. |
| Perez, R. O., Habr-Gama, A., Gama-Rodrigues, J., Proscurshim, I., Juliao, G. P. S., Lynn, P., Ono, C. R., Campos, F. G., Silva, E. Sousa Jr A. H., Imperiale, A. R., Nahas, S. C., Buchpiguel, C. A., Accuracy of positron emission tomography/computed tomography and clinical assessment in the detection of complete rectal tumor regression after neoadjuvant chemoradiation: Long-term results of a prospective trial (National Clinical Trial 00254683), Cancer, 118, 3501–3511, 2012 [PubMed: 22086847] | Combines surgically and non-surgically treated patients |
| Rao, C., Sun Myint, A., Athanasiou, T., Faiz, O., Martin, A. P., Collins, B., Smith, F. M., Avoiding Radical Surgery in Elderly Patients With Rectal Cancer Is Cost-Effective, Diseases of the Colon & RectumDis Colon Rectum, 60, 30–42, 2017 [PubMed: 27926555] | Cost effectiveness study |
| Renehan, A. G., Malcomson, L., Emsley, R., Watch-and-wait approach for rectal cancer: concepts of a subject-specific method, The Lancet Gastroenterology and Hepatology, 2, 627, 2017 [PubMed: 28786383] | Letter in response to Dossa (2017) metaanalysis. |
| Renehan, A. G., Malcomson, L., Emsley, R., Gollins, S., Maw, A., Myint, A. S., Rooney, P. S., Susnerwala, S., Blower, A., Saunders, M. P., Wilson, M. S., Scott, N., O’Dwyer, S. T., Watch-and-wait approach versus surgical resection after chemoradiotherapy for patients with rectal cancer (the OnCoRe project): A propensity-score matched cohort analysis, The Lancet Oncology, 17, 174–183, 2016 [PubMed: 26705854] | No multivariate prognostic analysis for the watch and wait group |
| Renehan, A. G., Malcomson, L., Emsley, R., Scott, N., O’Dwyer, S. T., Watch-and-wait versus surgical resection for patients with rectal cancer - Authors’ reply, The Lancet Oncology, 17, e134–e135, 2016 [PubMed: 27300672] | Authors reply to a letter in response to Rehenan (2015) |
| Sammour, T., Price, B. A., Krause, K. J., Chang, G. J., Nonoperative Management or ‘Watch and Wait’ for Rectal Cancer with Complete Clinical Response After Neoadjuvant Chemoradiotherapy: A Critical Appraisal, Annals of Surgical Oncology, 24, 1904–1915, 2017 [PMC free article: PMC6106774] [PubMed: 28324284] | Systematic review, prognostic analysis not reported |
| Sanchez Loria, F., Iseas, S., O’Connor, J. M., Pairola, A., Chacon, M., Mendez, G., Coraglio, M., Mariani, J., Dieguez, A., Roca, E., Huertas, E., Non-surgical management of rectal cancer. Series of 68 cases, long follow up in two leading centres in Argentina, Digestive and Liver Disease, 48, 1372–1377, 2016 [PubMed: 27260329] | No multivariate prognostic analysis. Univariate data for: T stage, N stage, CEA. |
| Schumacher, A., Rao, A., Loh, B. D., Dudukgian, H., Aboulian, A., McLemore, E. C., Attaluri, V., Rectal cancer: Nonoperative watch and wait vs standard of care surgical total mesorectal excision after complete clinical response to chemoradiation, a prospective cohort study, Journal of the American College of Surgeons, 225 (4 Supplement 1), S45, 2017 | Conference abstract |
| Seshadri, R. A., Kondaveeti, S. S., Jayanand, S. B., John, A., Rajendranath, R., Arumugam, V., Ellusamy, H. R., Sagar, T. G., Complete clinical response to neoadjuvant chemoradiation in rectal cancers: Can surgery be avoided?, Hepato-Gastroenterology, 60, 410–414, 2013 [PubMed: 23635444] | Does not report analysis adjusted for confounders |
| Smith, F. M., Al-Amin, A., Wright, A., Berry, J., Nicoll, J. J., Sun Myint, A., Contact radiotherapy boost in association with ‘watch and wait’ for rectal cancer: initial experience and outcomes from a shared programme between a district general hospital network and a regional oncology centre, Colorectal Disease, 18, 861–870, 2016 [PubMed: 26876570] | Entry criteria were not complete clinical response (some chose nonoperative management for other reasons). |
| Smith, F. M., Rao, C., Perez, R. O., Bujko, K., Athanasiou, T., Habr-Gama, A., Faiz, O., Avoiding radical surgery improves early survival in elderly patients with rectal cancer, demonstrating complete clinical response after neoadjuvant therapy: Results of a decision-analytic model, Diseases of the Colon and Rectum, 58, 159–171, 2015 [PubMed: 25585073] | Decision model (not primary study) - relapse rates during observation alone based on expert opinion. |
| Smith, F., Rao, C., Perez, R., Bujko, K., Athanasiou, T., Habr-Gama, A., Faiz, O., Avoiding radical surgery improves survival in elderly patients with rectal cancer demonstrating complete clinical response following neoadjuvant therapy: Results of a decision analytical model, Colorectal Disease, 2), 63, 2014 [PubMed: 25585073] | Conference abstract |
| Smith, J. D., Ruby, J. A., Goodman, K. A., Saltz, L. B., Guillem, J. G., Weiser, M. R., Temple, L. K., Nash, G. M., Paty, P. B., Nonoperative management of rectal cancer with complete clinical response after neoadjuvant therapy, Annals of Surgery, 256, 965–972, 2012 [PubMed: 23154394] | No multivariate prognostic analysis. Univariate data for: T stage, N stage |
| Smith, J. J., Chow, O. S., Gollub, M. J., Nash, G. M., Temple, L. K., Weiser, M. R., Guillem, J. G., Paty, P. B., Avila, K., Garcia-Aguilar, J., Rectal Cancer, Consortium, Organ Preservation in Rectal Adenocarcinoma: a phase II randomized controlled trial evaluating 3-year disease-free survival in patients with locally advanced rectal cancer treated with chemoradiation plus induction or consolidation chemotherapy, and total mesorectal excision or nonoperative management, BMC Cancer, 15, 767, 2015 [PMC free article: PMC4619249] [PubMed: 26497495] | Protocol for a phase II study. |
| Smith J, Strombom P, Chow O, et al. Assessment of a Watch-and-Wait Strategy for Rectal Cancer in Patients with a Complete Response after Neoadjuvant Therapy, JAMA Oncology., 2018 [PMC free article: PMC6459120] [PubMed: 30629084] | No multivariable prognostic analysis |
| Smith, J., Ruby, J., Goodman, K., Saltz, L., Guillem, J., Weiser, M., Temple, L., Nash, G., Paty, P., Non-operative management of rectal cancer with complete clinical response following neoadjuvant therapy, Irish Journal of Medical Science, 6), S183, 2012 | Conference abstract |
| Souza, J., Guimaraes, R., Siqueira, M. B., Gil, R., Araujo, R., Valadao, M., Watch and wait versus surgery with pathological complete response: Single institution experience, Annals of Oncology, 28 (Supplement 5), v204, 2017 | Conference abstract |
| Spiegel, D., Boyer, M. J., Hong, J. C., Willaims, C. D., Kelley, M. J., Salama, J. K., Palta, M., Non-operative management for locally advanced rectal cancer in the veterans health administration, International Journal of Radiation Oncology Biology Physics, 99 (2 Supplement 1), S67–S68, 2017 | Conference abstract |
| Sposato, L. A., Lam, Y., Karapetis, C., Vatandoust, S., Roy, A., Hakendorf, P., Dwyer, A., de Fontgalland, D., Hollington, P., Wattchow, D., Observation of “complete clinical response” in rectal cancer after neoadjuvant chemoradiation: The Flinders experience, Asia-Pacific Journal of Clinical Oncology, 14, 439–445, 2018 [PubMed: 29932278] | Does not report prognostic analysis |
| Torres-Mesa, P. A., Oliveros, R., Mesa, J., Olaya, N., Sanchez, R., Outcomes of the non-surgical management of locally advanced rectal cancer after neoadjuvant treatment. [Spanish], Revista Colombiana de Cancerologia, 18, 109–119, 2014 | Spanish language |
| Vaccaro, C. A., Yazyi, F. J., Ojra Quintana, G., Santino, J. P., Sardi, M. E., Beder, D., Tognelli, J., Bonadeo, F., Lastiri, J. M., Rossi, G. L., Locally advanced rectal cancer: Preliminary results of rectal preservation after neoadjuvant chemoradiotherapy, Cirugia espanola, 94, 274–9, 2016 [PubMed: 26980259] | No multivariate prognostic analysis. Univariate data for: initial MRI stage. |
| van der Valk M, Hilling D, Bastiaannet E, et al. Long-term outcomes of clinical complete responders after neoadjuvant treatment for rectal cancer in the International Watch & Wait Database (IWWD): an international multicentre registry study, The Lancet, 391, 2537–2545, 2018 [PubMed: 29976470] | Does not report prognostic analysis |
| Vatandoust S, Lam YH, Roy AC, Wattchow D, Hollington P, Karapetis C Retrospective study of patients (pts) who were managed with watch and wait strategy (W&W) after neoadjuvant chemoradiation (NCRT) for locally advanced rectal cancer (LARC)., Proc Am Soc Clin Oncol, 33: , 2015 | Abstract only |
| Vitelli, C. E., Stipa, F., De Paula, U., Is a policy of watch and wait after a complete response following neoadjuvant treatment for locally advanced rectal adenocarcinoma justified? Should we reset the limit?, Updates in surgery, 66, 7–8, 2014 [PubMed: 24288010] | Expert review |
Appendix L. Research recommendations
Research recommendations for review question: Which people having neoadjuvant radiotherapy or chemoradiotherapy for rectal cancer do not need surgery?
No research recommendations were made for this review question.
Tables
Table 1Summary of the protocol (PFO table)
| Population | Adults with non-metastatic rectal cancer who have complete clinical response to neoadjuvant radiotherapy or chemoradiotherapy and are fit for, but who have not had, surgery. |
|---|---|
| Factors |
|
| Outcomes | Critical
|
BRAF: v-raf murine sarcoma b-viral oncogene homolog B1; CEA: carcinoembryonic antigen; LVI: lymphovascular invasion; MSI: microsatellite instability; RAS: rat sarcoma virus oncogene homolog; TRG: tumour regression grade.
Final
Evidence reviews
Developed by the National Guideline Alliance part of the Royal College of Obstetricians and Gynaecologists
Disclaimer: The recommendations in this guideline represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, professionals are expected to take this guideline fully into account, alongside the individual needs, preferences and values of their patients or service users. The recommendations in this guideline are not mandatory and the guideline does not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian.
Local commissioners and/or providers have a responsibility to enable the guideline to be applied when individual health professionals and their patients or service users wish to use it. They should do so in the context of local and national priorities for funding and developing services, and in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities. Nothing in this guideline should be interpreted in a way that would be inconsistent with compliance with those duties.
NICE guidelines cover health and care in England. Decisions on how they apply in other UK countries are made by ministers in the Welsh Government, Scottish Government, and Northern Ireland Executive. All NICE guidance is subject to regular review and may be updated or withdrawn.