Leptomeningeal disease (LMD), previously known as neoplastic meningitis, or meningeal carcinomatosis (if arising from solid tumors) or leukemic meningitis (if arising from hematological malignancies), represents a stage of metastatic cancer when tumor cells have seeded the leptomeninges and cerebrospinal fluid (CSF)-containing subarachnoid space. Despite decades of efforts by the neuro-oncology community, response assessment remains challenging, and the prognosis of this whole neuroaxial disease remains extremely poor, ranging typically between 3-8 months despite standard of care therapy. The extensive heterogeneity in outcomes observed in randomized controlled trials (RCTs) for LMD has been thoroughly documented by prior consensus reviews and systematic reviews, warranting the development and implementation of a standardized core outcome set (COS) for LMD. This project aims to develop a COS for LMD (LeptoCOS) through a multidisciplinary, international initiative, underpinned by an updated systematic review and Delphi-based efforts, following COS-STAD and COMET guidelines for developing COS. The COS developed will be relevant primarily to later-phase clinical effectiveness trials of LMD but will have relevance for design and reporting for non-randomized studies and real-world outcomes research in patients with LMD. With the endorsement of professional societies and non-profit organizations in neuro-oncology, the LeptoCOS project will aim to standardize the use and reporting of outcomes reported in future clinical studies of LMD.
ContributorsSUPERVISOR and SENIOR INVESTIGATOR:
Alireza Mansouri, MD, MSc, FRCS; amansouri@pennstatehealth.psu.edu;
Director of Clinical and Translational Research in Skull Base and Neuro-Oncology and Assistant Professor, Department of Neurosurgery; Department of Neurosurgery, Penn State Health Milton S. Hershey Medical Center, Hershey, PA, United States
Disease Category: Cancer
Disease Name: Leptomeningeal disease
Age Range: 18 - 120
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (caregivers)
- Epidemiologists
- Journal editors
- Methodologists
- Researchers
- Statisticians
- COS for clinical trials or clinical research
- Consensus conference
- Consensus meeting
- Delphi process
- Literature review
- Survey
- Systematic review
The present study aims to develop core outcome set (COS) for LMD (named LeptoCOS) using current best practices and in line with most updated methodological guidance, including the COMET Handbook and the COS-STAD guidelines. We plan to first generate a long-list of outcomes of potential relevance based on updated systemic review of published literature and searches of trial registries. Using eDelphi process and consensus meetings, we will generate short list of outcomes for inclusion in the LeptoCOS. If the expert panel deems appropriate, separate COS will be generated for trials of patients with solid tumor LMD and trials of patients with LMD from hematological malignancies.