S E R Horbach1, C M A M van der Horst1, F Blei2, C J M van der Vleuten3, I J Frieden4, G T Richter5, S T Tan6,7, T Muir8, A J Penington9,10, L M Boon11, P I Spuls12. 1. Department of Plastic, Reconstructive and Hand Surgery, Academic Medical Center (AMC), University of Amsterdam, the Netherlands. 2. Department of Pediatrics, Lenox Hill Hospital, New York, NY, U.S.A. 3. Department of Dermatology, Radboud University Medical Center, Nijmegen, the Netherlands. 4. Department of Dermatology, University of California San Francisco, San Francisco, U.S.A. 5. Department of Otolaryngology, Arkansas Children's Hospital, Little Rock, AR, U.S.A. 6. Wellington Regional Plastic, Maxillofacial and Burns Unit, Hutt Hospital, Wellington, New Zealand. 7. Gillies McIndoe Research Institute, Wellington, New Zealand. 8. Department of Plastic and Reconstructive Surgery, James Cook University Hospital, Middlesbrough, U.K. 9. Department of Paediatrics, University of Melbourne, Melbourne, Australia. 10. Murdoch Children's Research Institute, Melbourne, Australia. 11. Center for Vascular Anomalies, Division of Plastic and Reconstructive Surgery, Cliniques Universitaires Saint-Luc, Brussels, Belgium. 12. Department of Dermatology, Academic Medical Center (AMC), University of Amsterdam, the Netherlands.
Abstract
BACKGROUND: An important limitation in vascular malformation research is the heterogeneity in outcome measures used for the evaluation of treatment outcome. OBJECTIVES: 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 three-round e-Delphi process using online surveys, participants repeatedly rated the importance of these domains on a five-point Likert scale. Participants could also propose other relevant domains. This process was performed for LM, VM and AVM separately. Consensus was predefined as 80% agreement on the importance of a domain among both the physician group and the patient/parent group. Outcomes were then re-evaluated 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. CONCLUSIONS: Our recommendation is that therapeutic-efficacy studies on peripheral vascular malformations should measure at least these core outcome domains.
BACKGROUND: An important limitation in vascular malformation research is the heterogeneity in outcome measures used for the evaluation of treatment outcome. OBJECTIVES: 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 three-round e-Delphi process using online surveys, participants repeatedly rated the importance of these domains on a five-point Likert scale. Participants could also propose other relevant domains. This process was performed for LM, VM and AVM separately. Consensus was predefined as 80% agreement on the importance of a domain among both the physician group and the patient/parent group. Outcomes were then re-evaluated 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. CONCLUSIONS: Our recommendation is that therapeutic-efficacy studies on peripheral vascular malformations should measure at least these core outcome domains.
Authors: Paula R Williamson; Iain A Bruce; Bilal Alkhaffaf; Jane M Blazeby; Aleksandra Metryka; Anne-Marie Glenny; Ademola Adeyeye; Paulo Matos Costa; Ismael Diez Del Val; Suzanne S Gisbertz; Ali Guner; Simon Law; Hyuk-Joon Lee; Ziyu Li; Koji Nakada; Rafael Mauricio Restrepo Nuñez; Daniel Reim; John V Reynolds; Peter Vorwald; Daniela Zanotti; William Allum; M Asif Chaudry; Ewen Griffiths Journal: Trials Date: 2021-06-21 Impact factor: 2.279
Authors: M M Lokhorst; S E R Horbach; M Waner; T M O; C J M van der Vleuten; L B Mokkink; C M A M van der Horst; P I Spuls Journal: Br J Dermatol Date: 2019-12-02 Impact factor: 9.302
Authors: Sophie E R Horbach; Amber P M Rongen; Roy G Elbers; Chantal M A M van der Horst; Cecilia A C Prinsen; Phyllis I Spuls Journal: Qual Life Res Date: 2019-09-23 Impact factor: 4.147
Authors: Frédérique C M Bouwman; Silje S Kooijman; Bas H Verhoeven; Leo J Schultze Kool; Carine J M van der Vleuten; Sanne M B I Botden; Ivo de Blaauw Journal: Eur J Pediatr Date: 2020-10-13 Impact factor: 3.183