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Annals of Surgery Apr 2023To assess the efficacy and safety of intentional watch and wait (W&W) and organ preservation surgery following neoadjuvant chemoradiotherapy plus consolidation CAPEOX in...
Intentional Watch and Wait or Organ Preservation Surgery Following Neoadjuvant Chemoradiotherapy Plus Consolidation CAPEOX for MRI-defined Low-risk Rectal Cancer: Findings From a Prospective Phase 2 Trial (PKUCH-R01 Trial, NCT02860234).
OBJECTIVE
To assess the efficacy and safety of intentional watch and wait (W&W) and organ preservation surgery following neoadjuvant chemoradiotherapy plus consolidation CAPEOX in magnetic resonance imaging (MRI)-defined low-risk rectal cancer.
BACKGROUND
Clinical T2/early T3 rectal cancers can achieve high yield pathological complete response (ypCR) rates after chemoradiotherapy; thus, an intentional W&W or organ preservation strategy for good clinical responders in these subgroups can be further tested.
METHODS
This prospective, single-arm, phase 2 trial enrolled patients with low-risk MRI prestaged rectal cancers, who concurrently received chemoradiation, followed by four 3-weekly cycles of CAPEOX regimen. Following reassessment, clinical complete response (cCR) or near-cCR patients underwent W&W/organ preservation surgery; the primary endpoint was a 3-year organ preservation rate.
RESULTS
Of the 64 participants, 58 completed treatment, with 6.4% and 33.9% grade 3 to 4 toxicities in the radiotherapy and consolidation CAPEOX phases, respectively, during a median 39.5-month follow-up. Initial cCR, and non-cCR occurred in 33, 13, and 18 patients, respectively. Of the 31 cCR and 7 near-cCR cases managed by W&W, local regrowth occurred in 7; of these, 6 received salvage surgery. The estimated 2-year local regrowth rates were 12.9% [95% confidence interval (CI): 1.1%-24.7%] in cCR and 42.9% (95% CI: 6.2%-79.6%) in near-cCR cases, respectively. Eight patients received local excision, including 2 with regrowth salvage. Lung metastases occurred in 3 patients and multiple metastasis occurred in 1 patient; no local recurrence occurred. The estimated 3-year organ preservation rate was 67.2% (95% CI: 55.6%-78.8%). The estimated 3-year cancer-specific survival, non-regrowth disease-free survival, and stoma-free survival were 96.6% (95% CI: 92.1%-100%), 92.2% (95% CI: 85.5%-98.9%), and 82.7% (95% CI: 73.5%-91.9%), respectively.
CONCLUSIONS
Chemoradiotherapy plus consolidation CAPEOX for MRI-defined low-risk rectal cancer can lead to high rates of organ preservation through intentional W&W or local excision. The oncologic safety of this strategy should be further tested.
Topics: Humans; Neoadjuvant Therapy; Organ Preservation; Prospective Studies; Rectal Neoplasms; Chemoradiotherapy; Magnetic Resonance Imaging; Watchful Waiting; Neoplasm Recurrence, Local; Treatment Outcome
PubMed: 35766394
DOI: 10.1097/SLA.0000000000005507 -
Journal of Gastroenterology Feb 2014The management of Barrett's oesophagus and associated neoplasia has evolved considerably in recent years. Modern endoscopic strategies including endoscopic resection and... (Review)
Review
The management of Barrett's oesophagus and associated neoplasia has evolved considerably in recent years. Modern endoscopic strategies including endoscopic resection and mucosal ablation can eradicate dysplastic Barrett's and prevent progression to invasive oesophageal cancer. However, several aspects of Barrett's management remain controversial including the stage in the disease process at which to intervene, and the choice of endoscopic or surgical therapy. A review of articles pertaining to the management of Barrett's oesophagus with or without associated neoplasia, was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Medline, Embase and Cochrane databases were searched to identify literature relevant to eight pre-defined areas of clinical controversy. The following search terms were used: Barrett's oesophagus; dysplasia; intramucosal carcinoma; endotherapy; endoscopic resection; ablation; oesophagectomy. A significant body of evidence exists to support early endoscopic therapy for high-grade dysplasia (HGD). Although not supported by randomised controlled trial evidence, endoscopic therapy is now favoured ahead of oesophagectomy for most patients with HGD. Focal intramucosal (T1a) carcinomas can be managed effectively using endoscopic and surgical therapy, however surgery should be considered the first line therapy where there is submucosal invasion (T1b). Treatment of low grade dysplasia is not supported at present due to widespread over-reporting of the disease. The role of surveillance endoscopy in non-dysplastic Barrett's remains controversial.
Topics: Ablation Techniques; Adenocarcinoma; Barrett Esophagus; Carcinoma in Situ; Esophageal Neoplasms; Esophagectomy; Esophagoscopy; Humans; Mucous Membrane; Precancerous Conditions; Watchful Waiting
PubMed: 23736794
DOI: 10.1007/s00535-013-0816-z -
Health Affairs (Project Hope) Dec 2013Many patients who seek emergency department (ED) treatment are not well enough for immediate discharge but are not clearly sick enough to warrant full inpatient... (Comparative Study)
Comparative Study Observational Study
Many patients who seek emergency department (ED) treatment are not well enough for immediate discharge but are not clearly sick enough to warrant full inpatient admission. These patients are increasingly treated as outpatients using observation services. Hospitals employ four basic approaches to observation services, which can be categorized by the presence or absence of a dedicated observation unit and of defined protocols. To understand which approach might have the greatest impact, we compared 2010 data from three sources: a case study of observation units in Atlanta, Georgia; statewide discharge data for Georgia; and national survey and discharge data. Compared to patients receiving observation services elsewhere in the hospital, patients cared for in "type 1" observation units-dedicated units with defined protocols-have a 23-38 percent shorter length-of-stay, a 17-44 percent lower probability of subsequent inpatient admission, and $950 million in potential national cost savings each year. Furthermore, we estimate that 11.7 percent of short-stay inpatients nationwide could be treated in a type 1 unit, with possible savings of $5.5-$8.5 billion annually. Policy makers should have hospitals report the setting in which observation services are provided and consider payment incentives for care in a type 1 unit.
Topics: Cost Savings; Databases, Factual; Efficiency, Organizational; Emergency Service, Hospital; Georgia; Health Care Surveys; Health Services Research; Hospital Units; Humans; Length of Stay; Patient Acuity; Patient Admission; Retrospective Studies; Watchful Waiting
PubMed: 24301399
DOI: 10.1377/hlthaff.2013.0662 -
AJR. American Journal of Roentgenology Mar 2017Current clinical guidelines are consistent in supporting annual mammography for women after treatment of primary breast cancer. Surveillance imaging beyond standard... (Review)
Review
OBJECTIVE
Current clinical guidelines are consistent in supporting annual mammography for women after treatment of primary breast cancer. Surveillance imaging beyond standard digital mammography, including digital breast tomosynthesis (DBT), breast ultrasound, and MRI, may improve outcomes. This article reviews the evidence on the performance and effectiveness of breast imaging modalities available for surveillance after treatment of sporadic unilateral primary breast cancer and identifies additional factors to be considered when selecting an imaging surveillance regimen.
CONCLUSION
Evidence review supports the use of mammography for surveillance after primary breast cancer treatment. Variability exists in guideline recommendations for surveillance initiation, interval, and cessation. DBT offers the most promise as a potential modality to replace standard digital mammography as a front-line surveillance test; a single published study to date has shown a significant decrease in recall rates compared with standard digital mammography alone. Most guidelines do not support the use of whole-breast ultrasound in breast cancer surveillance, and further studies are needed to define the characteristics of women who may benefit from MRI surveillance. The emerging evidence about surveillance imaging outcomes suggests that additional factors, including patient and imaging characteristics, tumor biology and gene expression profile, and choice of treatment, warrant consideration in selecting personalized posttreatment imaging surveillance regimens.
Topics: Breast Neoplasms; Evidence-Based Medicine; Female; Humans; Magnetic Resonance Imaging; Mammography; Prognosis; Reproducibility of Results; Sensitivity and Specificity; Treatment Outcome; Watchful Waiting
PubMed: 28075622
DOI: 10.2214/AJR.16.16300 -
Annual Review of Medicine Jan 2019Papillary thyroid microcarcinoma (PMC) is defined as papillary thyroid carcinoma ≤10 mm. Active surveillance of PMC without high-risk features, such as clinical node... (Review)
Review
Papillary thyroid microcarcinoma (PMC) is defined as papillary thyroid carcinoma ≤10 mm. Active surveillance of PMC without high-risk features, such as clinical node metastasis, distant metastasis, and clinical evidence of significant extrathyroid extension, was initiated in two Japanese hospitals in the mid-1990s. This strategy was incorporated into guidelines in Japan in 2010 and in the United States in 2015. In studies conducted by the two hospitals, most PMCs grew very slowly or did not grow, and none of the patients during active surveillance showed distant metastasis or died of thyroid carcinoma. Furthermore, none of the patients who underwent surgery after progression signs were detected showed significant recurrence. Therefore, we conclude that active surveillance should be the first line in management of low-risk PMC, because it is safer and less costly than immediate surgery. Active surveillance helps in avoiding adverse events of surgery and is an economical strategy.
Topics: Carcinoma, Papillary; Disease Management; Disease Progression; Female; Follow-Up Studies; Humans; Japan; Male; Neoplasm Invasiveness; Neoplasm Staging; Patient Selection; Risk Assessment; Survival Rate; Thyroid Neoplasms; Thyroidectomy; Watchful Waiting
PubMed: 30230964
DOI: 10.1146/annurev-med-051517-125510 -
Hamostaseologie Jan 2017Evidence-based medicine is growing in immune thrombocytopenia (ITP), but solid clinical data are still lacking in many areas. A majority of children has self-limited... (Review)
Review
Evidence-based medicine is growing in immune thrombocytopenia (ITP), but solid clinical data are still lacking in many areas. A majority of children has self-limited ITP, but chronic symptomatic ITP exists also in pediatrics. Management includes a watch-and-wait strategy for children with newly diagnosed ITP and no or mild bleeding, and immunoglobulins and corticosteroids, if more bleeding and mucous membrane involvement is present. Treatment endpoints differ in clinical research and in clinical practice. The requirement of platelet enhancing drugs needs to be better defined in guidelines. Second-line therapies for children are rarely required and include thrombopoietin-receptor agonists, rituximab, dexamethasone and immunosuppressants. Thrombopoietin-receptor agonists are successful in adult but also in pediatric ITP. The strategical position of splenectomy differs from that in adults. Although effective in children it is less frequently used because of its life-long cumulative risk of infectious diseases and a higher potential of spontaneous remission in ITP, providing a strong argument to defer splenectomy. The rarity of ITP makes clinical research expensive.
Topics: Adrenal Cortex Hormones; Child; Child, Preschool; Combined Modality Therapy; Diagnosis, Differential; Evidence-Based Medicine; Female; Humans; Immunoglobulins; Immunosuppressive Agents; Infant; Infant, Newborn; Male; Purpura, Thrombocytopenic, Idiopathic; Splenectomy; Treatment Outcome; Watchful Waiting
PubMed: 27699328
DOI: 10.5482/HAMO-16-06-0017 -
Pediatrics Jul 2022Updated guidelines continue to support watchful waiting as an option for uncomplicated acute otitis media (AOM) and provide explicit diagnostic criteria. To determine...
BACKGROUND AND OBJECTIVES
Updated guidelines continue to support watchful waiting as an option for uncomplicated acute otitis media (AOM) and provide explicit diagnostic criteria. To determine treatment prevalence and associated determinants of watchful waiting for AOM in commercially insured pediatric patients.
METHODS
This was a retrospective cohort study using IBM Marketscan Commercial Claims Databases (2005 to 2019) of patients 1 to 12 years old with AOM, without otitis-related complications within 6 months prior, with no tympanostomy tubes, and no other infections around index diagnosis of AOM. We examined monthly antibiotic treatment prevalence (defined as pharmacy dispensing within 3 days of AOM diagnosis) and used multivariable logistic regression models to examine determinants of watchful waiting.
RESULTS
Among 2 176 617 AOM episodes, 77.8% were treated within 3 days. Whereas some clinical characteristics were moderate determinants for watchful waiting, clinician antibiotic prescribing volume and specialty were strong determinants. Low-volume antibiotic prescribers (≥80% of AOM episodes managed with watchful waiting) had 11.61 (95% confidence interval 10.66-12.64) higher odds of using watchful waiting for the index AOM episode than high-volume antibiotic prescribers (≥80% treated). Otolaryngologists were more likely to adopt watchful waiting (odds ratio 5.45, 95% CI 5.21-5.70) than pediatricians, whereas other specialties deferred more commonly to antibiotics.
CONCLUSIONS
Adoption of watchful waiting for management of uncomplicated, nonrecurrent AOM was limited and stagnant across the study period and driven by clinician rather than patient factors. Future work should assess motivators for prescribing and evaluate patient outcomes among clinicians who generally prefer versus reject watchful waiting approaches to guide clinical decision-making.
Topics: Acute Disease; Anti-Bacterial Agents; Child; Child, Preschool; Humans; Infant; Otitis Media; Pediatricians; Retrospective Studies; Watchful Waiting
PubMed: 35726560
DOI: 10.1542/peds.2021-055613 -
Seminars in Thoracic and Cardiovascular... 2011Watchful waiting is an established treatment strategy for asymptomatic patients with severe organic mitral regurgitation. It is based on indications for surgery that are... (Review)
Review
Watchful waiting is an established treatment strategy for asymptomatic patients with severe organic mitral regurgitation. It is based on indications for surgery that are based on current European Society of Cardiology and American Heart Association/American College of Cardiology guideline recommendations, which are defined by symptom onset, impairment of left ventricular function, and left ventricular enlargement. Excellent outcome is achieved when patients are periodically followed with clinical and echocardiographic examinations and when surgery is performed in expert centers. The strategy is based on the recognition of mitral regurgitation at an early symptomatic stage, avoiding a delayed referral of these patients. There is an ongoing debate about whether surgery should be performed in asymptomatic patients with preserved ventricular function. Ultimately, decision-making needs to be individualized and to take individual patient-related factors and local resources (including the natural history of the disease, the risk of surgery, and the likelihood of successful mitral valve repair) into consideration to obtain an optimal outcome with medical and surgical management.
Topics: Atrial Fibrillation; Decision Making; Heart Ventricles; Humans; Mitral Valve; Mitral Valve Insufficiency; Risk Factors; Severity of Illness Index; Systole; Time Factors; Ventricular Function, Left; Watchful Waiting
PubMed: 22172357
DOI: 10.1053/j.semtcvs.2011.09.002 -
Pharmacotherapy Oct 2010This article is the first of a three-part series intended to enhance clinical pharmacists' understanding of methods frequently used in epidemiologic research and their... (Review)
Review
This article is the first of a three-part series intended to enhance clinical pharmacists' understanding of methods frequently used in epidemiologic research and their applications. The basic tenets of epidemiology and uses for data derived from epidemiologic studies are given, along with a high-level overview of the differences between experimental and observational study designs. The defining characteristics of each of the observational study designs (case report or case series, ecologic, cross-sectional, cohort, case-control, nested case-control, and case-cohort) and the resultant strengths and limitations of the study designs are presented. Applications for observational studies in pharmacoepidemiology (including the case-crossover and case-time-control study designs) are discussed. Finally, points to consider when evaluating data from observational studies are addressed.
Topics: Case-Control Studies; Cohort Studies; Cross-Over Studies; Cross-Sectional Studies; Ecosystem; Epidemiologic Methods; Epidemiologic Studies; Humans; Observation; Pharmacoepidemiology; Randomized Controlled Trials as Topic; Research Design; Statistics as Topic
PubMed: 20874034
DOI: 10.1592/phco.30.10.973 -
Seminars in Reproductive Medicine Jan 2020Unexplained infertility represents up to 30% of all cases of infertility. It is a diagnosis of exclusion, where no cause for infertility may be identified in the...
Unexplained infertility represents up to 30% of all cases of infertility. It is a diagnosis of exclusion, where no cause for infertility may be identified in the investigation of the couple, be it anovulation, fallopian tube blockage, or severe male factor. Unexplained infertility therefore cannot be considered a diagnosis to which a specific treatment is directed, rather that it indicates a failure to reach a diagnosis of the true cause of infertility. In this review, we explore the evidence base and potential limitations of the current routine infertility assessment. We also aim to highlight the importance of considering the prognosis of each individual couple through the process of assessment and propose a reconsidered approach to treatment, targeted to the prognosis rather than the diagnosis. Ultimately, a better understanding of the mechanisms of infertility will reduce the number of couples diagnosed with "unexplained" infertility.
Topics: Female; Humans; Infertility; Male; Pregnancy; Prognosis; Reproductive Techniques, Assisted; Watchful Waiting
PubMed: 33058088
DOI: 10.1055/s-0040-1718709