Background
An important limitation in vascular malformation research is the heterogeneity in outcome measures used for the evaluation of treatment outcome.
Objective
The Outcome measures for VAscular MAlformations (OVAMA) project aimed to reach international consensus on a core outcome set (COS) for clinical research on peripheral vascular malformations: lymphatic (LM), venous (VM) and arteriovenous malformations (AVM). In this consensus study, we determined what domains should constitute the COS.
Methods
Thirty-six possibly relevant outcome domains were proposed to an international group of physicians, patients and the parents of patients. In a 3-round e-Delphi process using online surveys, participants repeatedly rated the importance of these domains on a 5-point Likert scale. Participants could also propose other relevant domains. This process was performed for LM, VM and AVM separately. Consensus was pre-defined as 80% agreement on the importance of a domain amongst both the physician group and the patient/parent group. Outcomes were then reevaluated in an online consensus meeting.
Results
167 physicians and 134 patients and parents of patients with LM (n=50), VM (n=71) and AVM (n=29) participated in the study. After three rounds and a consensus meeting, consensus was reached for all three types of vascular malformations on the core domains of radiological assessment, physician-reported location-specific signs, patient-reported severity of symptoms, pain, quality of life, satisfaction and adverse events. Vascular malformation type-specific signs and symptoms were included for LM, VM and AVM, separately.
Conclusion
It is recommended to measure at least these core outcome domains in therapeutic-efficacy studies on peripheral vascular malformations.
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The Outcome measures for VAscular MAlformations (OVAMA) project aimed to reach international consensus on a core outcome set (COS) for clinical research on peripheral vascular malformations: lymphatic (LM), venous (VM) and arteriovenous malformations (AVM). In this consensus study, we determined what domains should constitute the COS.
ContributorsHorbach SER, van der Horst CMAM, Blei F, van der Vleuten CJM, Frieden IJ, Richter GT, Tan ST, Muir T, Penington A, Boon LM, Spuls PI; OVAMA consensus group.
Sophie E.R. Horbach MD, PhD candidate Plastic and reconstructive surgery
Prof. Chantal M.A.M. van der Horst MD, plastic surgeon
Prof. Phyllis I. Spuls MD, dermatologist
Disease Category: Heart & circulation, Skin
Disease Name: Vascular malformations
Age Range: 0 - 100
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Patient/ support group representatives
- Researchers
- COS for clinical trials or clinical research
- Recommendations for outcome measures (measurement/how)
- Consensus meeting
- Delphi process
- Systematic review
In our recently performed systematic review, we discovered that there is a lot of heterogeneity in outcomes used (S.E.R. Horbach, I.M. Rigter, J.H. Sillevis Smitt, J.A. Reekers, P.I. Spuls, C.M.A.M. van der Horst. entitled "Intralesional Bleomycin Injections for Vascular Malformations: A Systematic Review and Meta-Analysis". Accepted recently in Plastic and Reconstructive Surgery PRS-D-15-01154R3).
In order to reach an international consensus on a core outcome set, we will perform an international three-round e-Delphi consensus study in which all stakeholders will be involved (patients, parents, medical specialists and researchers).The final list of proposed outcome measures will be discussed in a consensus meeting of the OVAMA consensus group. Once there is an international consensus on the COS (of outcome domains), we will perform a systematic review on the measurement instruments for these domains. If necessary, a new disease specific (possibly patient-reported) outcome measure will be developed.