Abstract
Introduction: The top research priority for cavernoma, identified by a James Lind Alliance Priority setting partnership was 'Does treatment (with neurosurgery or stereotactic radiosurgery) or no treatment improve outcome for people diagnosed with a cavernoma?' This pilot randomised controlled trial (RCT) aims to determine the feasibility of answering this question in a main phase RCT.
Methods and analysis: We will perform a pilot phase, parallel group, pragmatic RCT involving approximately 60 children or adults with mental capacity, resident in the UK or Ireland, with an unresected symptomatic brain cavernoma. Participants will be randomised by web-based randomisation 1:1 to treatment with medical management and with surgery (neurosurgery or stereotactic radiosurgery) versus medical management alone, stratified by prerandomisation preference for type of surgery. In addition to 13 feasibility outcomes, the primary clinical outcome is symptomatic intracranial haemorrhage or new persistent/progressive focal neurological deficit measured at 6 monthly intervals. An integrated QuinteT Recruitment Intervention (QRI) evaluates screening logs, audio recordings of recruitment discussions, and interviews with recruiters and patients/parents/carers to identify and address barriers to participation. A Patient Advisory Group has codesigned the study and will oversee its progress.
Ethics and dissemination: This study was approved by the Yorkshire and The Humber-Leeds East Research Ethics Committee (21/YH/0046). We will submit manuscripts to peer-reviewed journals, describing the findings of the QRI and the Cavernomas: A Randomised Evaluation (CARE) pilot trial. We will present at national specialty meetings. We will disseminate a plain English summary of the findings of the CARE pilot trial to participants and public audiences with input from, and acknowledgement of, the Patient Advisory Group.
Methods and analysis: We will perform a pilot phase, parallel group, pragmatic RCT involving approximately 60 children or adults with mental capacity, resident in the UK or Ireland, with an unresected symptomatic brain cavernoma. Participants will be randomised by web-based randomisation 1:1 to treatment with medical management and with surgery (neurosurgery or stereotactic radiosurgery) versus medical management alone, stratified by prerandomisation preference for type of surgery. In addition to 13 feasibility outcomes, the primary clinical outcome is symptomatic intracranial haemorrhage or new persistent/progressive focal neurological deficit measured at 6 monthly intervals. An integrated QuinteT Recruitment Intervention (QRI) evaluates screening logs, audio recordings of recruitment discussions, and interviews with recruiters and patients/parents/carers to identify and address barriers to participation. A Patient Advisory Group has codesigned the study and will oversee its progress.
Ethics and dissemination: This study was approved by the Yorkshire and The Humber-Leeds East Research Ethics Committee (21/YH/0046). We will submit manuscripts to peer-reviewed journals, describing the findings of the QRI and the Cavernomas: A Randomised Evaluation (CARE) pilot trial. We will present at national specialty meetings. We will disseminate a plain English summary of the findings of the CARE pilot trial to participants and public audiences with input from, and acknowledgement of, the Patient Advisory Group.
| Original language | English |
|---|---|
| Article number | e075187 |
| Journal | BMJ Open |
| Volume | 13 |
| Issue number | 8 |
| DOIs | |
| Publication status | Published - 9 Aug 2023 |
Bibliographical note
Funding Information:Conceptualisation: RA-SS and NK, supported by JJML, VG, PSH, KH, PJH, EK, SL, CM, ARN, MR, JS, AS, CT, JWa, DWh, and PW. Methodology: JJML, VG, PSH, KH, PJH, EK, SL, CM, ARN, MR, JS, AS, CT, JWa, DWh, PW, NK and RA-SS. Project administration: JJML, ABj, JvB, PB, ABu, NB, DB, JC, EC, FC, MD, RD, RJE, LFo, LFe, IF, VG, PG, NG, KH, LSH, TH, AH, DH, PJH, AI, EK, SM, CM, NM, RN, MCP, MR, AR, SR-K, MT, CT, JWa, DWh, DWa, PW, JWi, OWW, CU, SU, RV, NK and RA-SS. Funding Acquisition: RA-SS, supported by LFo, EK, and NK. Writing—original draft: JJML and RA-SS. Writing—review and editing: All. Supervision: RA-SS and NK.
Publisher Copyright:
© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY. Published by BMJ.
Keywords
- adult neurology
- clinical trial
- neurosurgery
- paediatric neurology
- stroke