دورية أكاديمية
A core outcome domain set to assess cutaneous neurofibromas related to neurofibromatosis type 1 in clinical trials.
| العنوان: | A core outcome domain set to assess cutaneous neurofibromas related to neurofibromatosis type 1 in clinical trials. |
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| المؤلفون: | Fertitta L; Department of Dermatology.; INSERM, Centre d'Investigation Clinique 1430; National Referral Center for Neurofibromatoses, -Henri-Mondor Hospital, Assistance Publique-Hôpitaux Paris (AP-HP) , 94010 Créteil, France.; INSERM U955 , 94010, Créteil, France., Bergqvist C; Department of Dermatology.; INSERM, Centre d'Investigation Clinique 1430; National Referral Center for Neurofibromatoses, -Henri-Mondor Hospital, Assistance Publique-Hôpitaux Paris (AP-HP) , 94010 Créteil, France., Sarin KY; Department of Dermatology, Stanford Medicine, Stanford University, Redwood City, CA, USA., Plotkin SR; Department of Neurology and Cancer Center, Massachusetts General Hospital, Boston, MA, USA., Moertel C; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA., Petersen AK; Department of Rehabilitation and Development, Randall Children's Hospital at Legacy Emanuel Medical Center, Portland, OR, 97227, USA., Cannon A; School of Health Professions, University of Alabama at Birmingham, Birmingham, AL, USA.; InformedDNA, Inc., St Petersburg, FL, USA., Berman Y; Clinical Genetics, Royal North Shore Hospital, St Leonards, NSW, Australia and University of Sydney, Sydney, Australia., Pichard DC; Dermatology Branch, National Institutes of Arthritis, Musculoskeletal, and Skin Diseases.; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute; National Institutes of Health, Bethesda, MD, USA., Röhl C; NF Patients United - Global Network of NF Support Groups, Vienna, Austria., Lessing A; Neurofibromatosis Northeast, Burlington, MA, USA., Brizion B; Centre de documentation de l'AP-HP, AP-HP, Paris, France., Peiffer B; Department of Dermatology., Ravaud P; Center for Clinical Epidemiology, Hôtel-Dieu Hospital (AP-HP), Paris, France.; Université de Paris, CRESS, INSERM, INRA , F-75004 Paris, France., Tran VT; Center for Clinical Epidemiology, Hôtel-Dieu Hospital (AP-HP), Paris, France.; Université de Paris, CRESS, INSERM, INRA , F-75004 Paris, France., Armand ML; Department of Dermatology., Moryousef S; Department of Dermatology., Ferkal S; Department of Dermatology.; INSERM, Centre d'Investigation Clinique 1430; National Referral Center for Neurofibromatoses, -Henri-Mondor Hospital, Assistance Publique-Hôpitaux Paris (AP-HP) , 94010 Créteil, France., Jannic A; Department of Dermatology., Ezzedine K; Department of Dermatology.; INSERM, Centre d'Investigation Clinique 1430; National Referral Center for Neurofibromatoses, -Henri-Mondor Hospital, Assistance Publique-Hôpitaux Paris (AP-HP) , 94010 Créteil, France.; Université Paris-Est Créteil (UPEC), 94010 Créteil, France., Wolkenstein P; Department of Dermatology.; INSERM, Centre d'Investigation Clinique 1430; National Referral Center for Neurofibromatoses, -Henri-Mondor Hospital, Assistance Publique-Hôpitaux Paris (AP-HP) , 94010 Créteil, France.; INSERM U955, 94010, Créteil, France.; Université Paris-Est Créteil (UPEC), 94010 Créteil, France. |
| مؤلفون مشاركون: | Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) international collaboration |
| المصدر: | The British journal of dermatology [Br J Dermatol] 2024 Jan 23; Vol. 190 (2), pp. 216-225. |
| نوع المنشور: | Systematic Review; Journal Article |
| اللغة: | English |
| بيانات الدورية: | Publisher: Oxford University Press Country of Publication: England NLM ID: 0004041 Publication Model: Print Cited Medium: Internet ISSN: 1365-2133 (Electronic) Linking ISSN: 00070963 NLM ISO Abbreviation: Br J Dermatol Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: 2023- : Oxford : Oxford University Press Original Publication: Oxford [etc.] Published for the British Association of Dermatologists by Blackwell Scientific Publications [etc.] |
| مواضيع طبية MeSH: | Neurofibroma* , Neurofibromatosis 1* , Skin Neoplasms*, Humans ; Delphi Technique ; Research Design |
| مستخلص: | Background: Cutaneous neurofibromas (cNF) are considered one of the highest burdens of neurofibromatosis type 1 (NF1). To date, no medical treatment can cure cNF or prevent their development. In that context, there is an urgent need to prepare and standardize the methodology of future trials targeting cNF. Objectives: The objective was to develop a core outcome domain set suitable for all clinical trials targeting NF1-associated cNF. Methods: The validated approach of this work consisted of a three-phase methodology: (i) generating the domains [systematic literature review (SLR) and qualitative studies]; (ii) agreeing (three-round international e-Delphi consensus process and working groups); and (iii) voting. Results: (i) The SLR and the qualitative studies (three types of focus groups and a French e-survey with 234 participants) resulted in a preliminary list of 31 candidate items and their corresponding definitions. (ii) A total of 229 individuals from 29 countries participated in the first round of the e-Delphi process: 71 patients, relatives or representatives (31.0%), 130 healthcare professionals (HCPs, 56.8%) and 28 researchers, representatives of a drug regulatory authority, industry or pharmaceutical company representatives or journal editors (12.2%). The overall participation rate was 74%. After round 2, five candidate items were excluded. Between rounds 2 and 3, international workshops were held to better understand the disagreements among stakeholders. This phase led to the identification of 19 items as outcome subdomains. (iii) The items were fused to create four outcome domains ('clinical assessment', 'daily life impact', 'patient satisfaction' and 'perception of health') and prioritized. The seven items that did not reach consensus were marked for the research agenda. The final core outcome domain set reached 100% of the votes of the steering committee members. Conclusions: Although numerous outcomes can be explored in studies related to cNF in NF1, the present study offers four outcome domains that should be reported in all trial studies, agreed on by international patients, relatives and representatives of patients; HCPs; researchers, representatives of drug regulatory authorities or pharmaceutical companies and journal editors. The next step will include the development of a set of core outcome measurement instruments to further standardize how these outcomes should be assessed. Competing Interests: Conflicts of interest L.F. is a consultant for Alexion. K.S. is a member of the scientific advisory board of NFlection Therapeutics. S.R.P. is co-founder of NFlection Therapeutics and NF2 Therapeutics, serves on the scientific advisory board of SonALAsense and consults for Akouos. A.J. is a consultant for Alexion. P.W. is a consultant for Alexion, AstraZeneca and Springworks. The other authors have no conflicts of interest to declare. (© The Author(s) 2023. Published by Oxford University Press on behalf of British Association of Dermatologists. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.) |
| التعليقات: | Comment in: Br J Dermatol. 2023 Dec 12;:. (PMID: 38084905) Comment in: Br J Dermatol. 2024 Jan 23;190(2):e20. (PMID: 38262593) |
| فهرسة مساهمة: | Investigator: S Adsit; S Ahlawat; K Alcantara; R Anderson; K Anstett; L Aouad; R Avery; C Barnett-Tapia; B Barton; C Batlle; D Bedwell; TB O'Brian; D Berg; C Bergqvist; Y Berman; C Bettegowda; J Blakeley; R Brainin; SB Dirksen; F Buono; S Buono; W Cai; C Campen; A Cannon; K Carpenter; A Cellucci; N Champollion; LS Chang; P de Blank; A Del Castillo; L Didier; E Dombi; G Erickson; G Evans; K Ezzedine; O Faber; L Fertitta; M Fisher; B Franklin; E Garrett; M Gibson-Williams; M Giovannini; D Glad; D Gold; A Goodkind; K Goto; J Grabowski; V Grasso; A Gross; L Grotti; D Gutmann; D Haberkamp; K Haebich; C Hahn; O Hanemann; C Hingtgen; M Hocking; E Hunt; M Hussey; K Janke; J Janusz; J Jordan; V Kaer; M Karajannis; K Keeling; B Kesterson; A Kim; H Kim; Y Kim; B Klein-Tasman; L Klesse; K Koetsier; E Komlodi-Pasztor; N Larson; A Leier; E Leif; A Lessing; R Listernick; E Loftspring; S Loftspring; K Ina Ly; L Manth; S Martin; S McKnight; M McManus; V Merker; E Morehouse; I Moss; L Mukabenov; P Newman; B Oberlander; J Payne; D Peterson; D Pichard; R Pierpoint; S Plotkin; D Pope; L Popplewell; N Pride; M Ratley; S Rhodes; J Rios; I Ristow; B Riter; C Rohl; C Romo; A Rosenberg; T Rosser; J Samblanet; K Sarin; H Sarnoff; N Schneider; E Schorry; M Sell; S Sheard; D Silverman; B Simpson; M Smith; C Sorman; V Staedtke; P Stavinoha; A Stemmer-Rachamimov; D Stevenson; T Sundby; H Thompson; N Ullrich; M Upadhyaya; M van de Vrie; D Viskochil; J Vitte; J Walker; J Wallace; D Wallis; K Walsh; S Weaver; H Weisman; B Welling; B Widemann; T Wirtanen; P Wolkenstein; P Wolters; M Achtziger Wong; J Zhou |
| تواريخ الأحداث: | Date Created: 20231025 Date Completed: 20240130 Latest Revision: 20240130 |
| رمز التحديث: | 20240130 |
| DOI: | 10.1093/bjd/ljad397 |
| PMID: | 37877514 |
| قاعدة البيانات: | MEDLINE |
| تدمد: | 1365-2133 |
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| DOI: | 10.1093/bjd/ljad397 |