Development of an international core outcome set for peripheral vascular malformations: the OVAMA project

被引:49
作者
Horbach, S. E. R. [1 ]
van der Horst, C. M. A. M. [1 ]
Blei, F. [3 ]
van der Vleuten, C. J. M. [4 ]
Frieden, I. J. [5 ]
Richter, G. T. [6 ]
Tan, S. T. [7 ,8 ]
Muir, T. [9 ]
Penington, A. J. [10 ,11 ]
Boon, L. M. [12 ]
Spuls, P. I. [2 ]
机构
[1] Univ Amsterdam, Dept Plast Reconstruct & Hand Surg, Acad Med Ctr, Amsterdam, Netherlands
[2] Univ Amsterdam, Acad Med Ctr, Dept Dermatol, Amsterdam, Netherlands
[3] Lenox Hill Hosp, Dept Pediat, New York, NY 10021 USA
[4] Radboud Univ Nijmegen, Dept Dermatol, Med Ctr, Nijmegen, Netherlands
[5] Univ Calif San Francisco, Dept Dermatol, San Francisco, CA 94143 USA
[6] Arkansas Childrens Hosp, Dept Otolaryngol, 800 Marshall St, Little Rock, AR 72202 USA
[7] Hutt Hosp, Wellington Reg Plast Maxillofacial & Burns Unit, Wellington, New Zealand
[8] Gillies McIndoe Res Inst, Wellington, New Zealand
[9] James Cook Univ Hosp, Dept Plast & Reconstruct Surg, Middlesbrough, Cleveland, England
[10] Univ Melbourne, Dept Paediat, Melbourne, Vic, Australia
[11] Murdoch Childrens Res Inst, Melbourne, Vic, Australia
[12] Clin Univ St Luc, Ctr Vasc Anomalies, Div Plast & Reconstruct Surg, Brussels, Belgium
关键词
DELPHI CONSENSUS; CLINICAL-TRIALS; LYMPHATIC MALFORMATIONS; CLASSIFICATION; SCLEROTHERAPY; DOMAINS; PATIENT; HEAD; NECK;
D O I
10.1111/bjd.16029
中图分类号
R75 [皮肤病学与性病学];
学科分类号
100206 ;
摘要
BackgroundAn important limitation in vascular malformation research is the heterogeneity in outcome measures used for the evaluation of treatment outcome. ObjectivesTo 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. MethodsThirty-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. Results167 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. ConclusionsOur recommendation is that therapeutic-efficacy studies on peripheral vascular malformations should measure at least these core outcome domains. What's already known about this topic? There is considerable heterogeneity in outcomes used in clinical trials on peripheral vascular malformations. This hampers the interpretation, comparison and aggregation of study data, and in turn the development of evidence-based treatment guidelines. What does this study add? International consensus was reached on the core outcome domains that should be measured in all therapeutic-efficacy studies in this field: radiological assessment, physician-assessed signs, patient-reported pain, overall severity of symptoms, health-related quality of life, patient satisfaction with treatment and outcome, and adverse events. The next step is to reach consensus on how these domains should be measured (core outcome instruments). What are the clinical implications of this work? Development of this core outcome set (COS) should lead to improved standardized outcome reporting. It should allow the development of evidence-based treatment guidelines. Although this COS was primarily developed for clinical research, it may form the basis for a more concise COS that can be implemented in clinical practice. Linked Comment:Thomas. Br J Dermatol 2018; 178:326-327. Plain language summary available online Respond to this article
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收藏
页码:473 / 481
页数:9
相关论文
共 30 条
[1]   Development of a core outcome set for epilepsy in pregnancy (E-CORE): a national multi-stakeholder modified Delphi consensus study [J].
Al Wattar, B. H. ;
Tamilselvan, K. ;
Khan, R. ;
Kelso, A. ;
Sinha, A. ;
Pirie, A. M. ;
McCorry, D. ;
Khan, K. S. ;
Thangaratinam, S. .
BJOG-AN INTERNATIONAL JOURNAL OF OBSTETRICS AND GYNAECOLOGY, 2017, 124 (04) :661-667
[2]   Percutaneous sclerotherapy for lymphatic malformations: A retrospective analysis of patient-evaluated improvement [J].
Alomari, Ahmad I. ;
Karian, Victoria E. ;
Lord, David J. ;
Padua, Horacio M. ;
Burrows, Patricia E. .
JOURNAL OF VASCULAR AND INTERVENTIONAL RADIOLOGY, 2006, 17 (10) :1639-1648
[3]   Standardized Outcome and Reporting Measures in Pediatric Head and Neck Lymphatic Malformations [J].
Balakrishnan, Karthik ;
Bauman, Nancy ;
Chun, Robert H. ;
Darrow, David H. ;
Grimmer, J. Fredrik ;
Perkins, Jonathan A. ;
Richter, Gresham T. ;
Shin, Jennifer J. ;
Shivaram, Giridhar M. ;
Sidell, Douglas R. ;
Elluru, Ravindhra G. .
OTOLARYNGOLOGY-HEAD AND NECK SURGERY, 2015, 152 (05) :948-953
[4]   Developing Core Outcome Measurement Sets for Clinical Trials: OMERACT Filter 2.0 [J].
Boers, Maarten ;
Kirwan, John R. ;
Wells, George ;
Beaton, Dorcas ;
Gossec, Laure ;
d'Agostino, Maria-Antonietta ;
Conaghan, Philip G. ;
Bingham, Clifton O., III ;
Brooks, Peter ;
Landewe, Robert ;
March, Lyn ;
Simon, Lee S. ;
Singh, Jasvinder A. ;
Strand, Vibeke ;
Tugwell, Peter .
JOURNAL OF CLINICAL EPIDEMIOLOGY, 2014, 67 (07) :745-753
[5]   A Core Outcome Set for the Benefits and Adverse Events of Bariatric and Metabolic Surgery: The BARIACT Project [J].
Coulman, Karen D. ;
Hopkins, James ;
Brookes, Sara T. ;
Chalmers, Katy ;
Main, Barry ;
Owen-Smith, Amanda ;
Andrews, Robert C. ;
Byrne, James ;
Donovan, Jenny L. ;
Mazza, Graziella ;
Reeves, Barnaby C. ;
Rogers, Chris A. ;
Thompson, Janice L. ;
Welbourn, Richard ;
Wordsworth, Sarah ;
Blazeby, Jane M. .
PLOS MEDICINE, 2016, 13 (11)
[6]   ISSVA classification [J].
Dasgupta, Roshni ;
Fishman, Steven J. .
SEMINARS IN PEDIATRIC SURGERY, 2014, 23 (04) :158-161
[7]   Do data characteristics change according to the number of scale points used? An experiment using 5-point, 7-point and 10-point scales [J].
Dawes, John .
INTERNATIONAL JOURNAL OF MARKET RESEARCH, 2008, 50 (01) :61-77
[8]   Developing core outcome set for vitiligo clinical trials: international e-Delphi consensus [J].
Eleftheriadou, Viktoria ;
Thomas, Kim ;
van Geel, Nanja ;
Hamzavi, Iltefat ;
Lim, Henry ;
Suzuki, Tamio ;
Katayama, Ichiro ;
Anbar, Tag ;
Abdallah, Marwa ;
Benzekri, Laila ;
Gauthier, Yvon ;
Harris, John ;
Silva de Castro, Caio Cesar ;
Pandya, Amit ;
Goh, Boon Kee ;
Lan, Cheng-Che E. ;
Oiso, Naoki ;
Al Issa, Ahmed ;
Esmat, Samia ;
Le Poole, Caroline ;
Lee, Ai-Young ;
Parsad, Davinder ;
Taieb, Alain ;
Picardo, Mauro ;
Ezzedine, Khaled .
PIGMENT CELL & MELANOMA RESEARCH, 2015, 28 (03) :363-369
[9]   The COMET Initiative database: progress and activities update (2015) [J].
Gargon, E. ;
Williamson, P. R. ;
Altman, D. G. ;
Blazeby, J. M. ;
Tunis, S. ;
Clarke, M. .
TRIALS, 2017, 18
[10]   Sclerotherapy for low-flow vascular malformations of the head and neck: A systematic review of sclerosing agents [J].
Horbach, Sophie E. R. ;
Lokhorst, Max M. ;
Saeed, Peerooz ;
de Pontouraude, Claire M. F. de Gouyon Matignon ;
Rothova, Aniki ;
van der Horst, Chantal M. A. M. .
JOURNAL OF PLASTIC RECONSTRUCTIVE AND AESTHETIC SURGERY, 2016, 69 (03) :295-304