Neoplasms

All Conditions

Mouth and Tooth Diseases

Ear, Nose, and Throat Diseases

Rare Diseases

Carcinoma

Nasopharyngeal Carcinoma

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Nasopharyngeal Neoplasms

Pharyngeal Neoplasms

Otorhinolaryngologic Neoplasms

Head and Neck Neoplasms

Nasopharyngeal Diseases

Pharyngeal Diseases

Stomatognathic Diseases

Otorhinolaryngologic Diseases

Patients receiving CCRT from real-world database

Patients receiving IC+CCRT from real-world database

Study results from clinical trials usually have been found not work efficiently in clinical practice. This outcome disparity may be caused by different pragmatic features of medical environment, also known as study setting such as trial setting and real-world setting, in which biases inherent to clinical trial design restrict its applicability even though all confounding factors are avoided.

Trial setting

Jun Ma, Professor

Study results from randomized controlled trials (RCTs) usually have been found not adequately inform practice. A RCT is optimized to determine efficacy, while real-world study is conducted in a routine care setting aimed to determine effectiveness. Thus, it is necessary to evaluate the pragmatism of clinical trials for a better understanding of the external generalizability. Nonetheless, comparative pragmatic features of RCTs and real-world studies still lack well elucidation. By capitalizing on a nasopharyngeal carcinoma (NPC)-specific big-data, real-world database and individual patient data extracted from three landmark RCTs, investigators conducted the direct comparison of NPC cohorts receiving same treatment strategy in clinical trial versus real-world settings, and examined the comparative pragmatic features and their influences on survival outcomes, safety profile, and the probability of returning to society.

Study results from randomized controlled trials (RCTs) usually have been found not adequately inform practice. A RCT is optimized to determine efficacy. Such trials were performed with relatively small samples at sites with experienced investigators and highly selected participants, they could be overestimating benefits and underestimating harm. Real-world study is conducted in a routine care setting aimed to determine effectiveness. Thus, it is necessary to evaluate the pragmatism of clinical trials for a better understanding of the external generalizability. Nonetheless, comparative pragmatic features of RCTs and real-world studies still lack well elucidation. Nasopharyngeal carcinoma (NPC) is a malignant head and neck cancer with the highest incidences in endemic regions such as Southern China, where over 60,600 new cases were diagnosed in 2015 representing 40% of all cases worldwide. Studies conducted in China are critical in optimizing clinical decision-making of NPC. In the past two decades, the recommendation level of induction chemotherapy (IC) + concurrent chemoradiotherapy (CCRT) has been improved evidently from Category 3 to 2A, and CCRT alone has long been a stable (Category 2B) and classic treatment option of NPC and therefore becomes the most commonly used control group in comparative studies. By capitalizing on a NPC-specific big-data, real-world database via a cancer registry in Southern China and individual patient data extracted from the three landmark RCTs, investigators conducted the direct comparison on IC+CCRT cohort or CCRT cohort of clinical trial versus real-world settings, and examined the comparative pragmatic features and their influences on survival outcomes, safety profile, and the probability of returning to society, with the aim to provide new insight into the optimization of trial design and the translation of study evidences into tangible benefits.

Prospective Comparisons of Clinical Trial and Real-world Outcomes in Nasopharyngeal Carcinoma

Prospective Comparisons of Survival Outcomes, Safety Profile, and Probability of Returning to Society Between Three Randomized Controlled Trials and Real-world Evidence in Nasopharyngeal Carcinoma.

Overall survival is measured from day of diagnosis until death due to any cause or the latest known date alive.

Failure-free survival is measured from day of diagnosis to locoregional failure, distant failure, or death from any cause, whichever occurred first.

Locoregional failure-free survival is measured from day of diagnosis to first locoregional failure.

Distant failure-free survival is measured from day of diagnosis to distant failure.

Probability of learning (students who are in college/university/school before illness) or work (staff who are working before illness) stoppage due to illness and treatment.

Probability of returning to learning (students who are in college/university/school before illness) or work (staff who are working before illness) after suspending due to illness and treatment.

Incidence of radioactive brain injury according to the Common Terminology Criteria for Adverse Events (version 4.0).

Incidence of hearing loss according to the Common Terminology Criteria for Adverse Events (version 4.0).

Incidence of blind according to the Common Terminology Criteria for Adverse Events (version 4.0).

Incidence of decreased taste according to the Common Terminology Criteria for Adverse Events (version 4.0).

Incidence of second cancer according to the Common Terminology Criteria for Adverse Events (version 4.0).

Incidence of dysphagia according to the Common Terminology Criteria for Adverse Events (version 4.0).

Incidence of malnutrition according to the Common Terminology Criteria for Adverse Events (version 4.0).

Incidence of hypothyroidism according to the Common Terminology Criteria for Adverse Events (version 4.0).

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Prospective Comparisons of Clinical Trial and Real-world Outcomes in Nasopharyngeal Carcinoma	NCT04108338	Nasopharyngeal ...	Trial setting	8 Years - 79 Years	Sun Yat-sen University
Prospective Comparisons of Clinical Trial and Real-world Outcomes in Nasopharyngeal Carcinoma	NCT04108338	Nasopharyngeal ...	Trial setting	8 Years - 79 Years	Sun Yat-sen University
Prospective Comparisons of Clinical Trial and Real-world Outcomes in Nasopharyngeal Carcinoma	NCT04108338	Nasopharyngeal ...	Trial setting	8 Years - 79 Years	Sun Yat-sen University

Spots Global Cancer Trial Database for return to society