Treatment principles in adults and development of patient-reported outcomes in cyclic vomiting syndrome

Abstract

Background: Cyclic vomiting syndrome is a chronic disorder of gut-brain interaction that is present in both adults and children. It is characterized by severe nausea, vomiting, abdominal pain, and several non-GI symptoms. It is also associated with several comorbid conditions such as anxiety and depression, which affect overall health care outcomes.

Methods: This article delineates treatment principles, encompassing both abortive interventions and prophylactic regimens currently recommended for CVS. However, it underscores a critical concern: the absence of FDA-approved medications for CVS treatment, with existing therapies relying on retrospective and open-labeled trials.

Key results: This article emphasizes the pressing need for the development of CVS-specific outcome assessment tools to facilitate more accurate evaluation and robust data collection for the future studies. In exploring this deficiency, the manuscript also presents the up-to-date data and development that enhances our comprehension of patient-centric concepts, and the challenges faced in creating CVS-specific tools, and presents a roadmap for their development. Addressing this gap is crucial for advancing our understanding of CVS and optimizing patient care.

Conclusions and inferences: This elucidates the current state of CVS management but also advocates for a future where tailored tools enhance our ability to measure and improve the outcomes for individuals with this debilitating disorder.

Keywords: clinical outcomes; cyclic vomiting; disease severity; patient‐reported outcomes; treatment.

FIGURE 1

Severity of cyclic vomiting syndrome.

FIGURE 2

FDA's roadmap to patient‐focused outcome measurement in clinical trials. US FDA, “Patient‐Focused Drug Development: Selecting, Developing, or Modifying Fit‐for‐Purpose Clinical Outcome Assessments,” Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholder.