Development of an international core outcome set for peripheral vascular malformations (OVAMA project)

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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Aim

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.

Contributors

Horbach 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

Publication

Journal: British Journal of Dermatology
Volume:
Issue:
Pages: -
Year: 2018
DOI: 10.1111/bjd.16029

Further Study Information

Current Stage: Not Applicable
Date: October 2015 - February 2016
Funding source(s): Academic scholarship


Health Area

Disease Category: Heart & circulation, Skin

Disease Name: Vascular malformations

Target Population

Age Range: 0 - 100

Sex: Either

Nature of Intervention: Any

Stakeholders Involved

- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Patient/ support group representatives
- Researchers

Study Type

- COS for clinical trials or clinical research
- Recommendations for outcome measures (measurement/how)

Method(s)

- 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.