دورية أكاديمية
أعرض في EDS

Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1.

التفاصيل البيبلوغرافية
العنوان: Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1.
المؤلفون: John L; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Singh G; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Dombi E; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Wolters PL; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Martin S; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Baldwin A; Clinical Research Directorate (CRD), Frederick National Laboratory for Cancer Research, Frederick, MD, USA., Steinberg SM; Biostatistics and Data Management Section, Office of the Clinical Director, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Bernstein J; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Whitcomb P; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Pichard DC; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Dufek A; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Gillespie A; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Heisey K; Clinical Research Directorate (CRD), Frederick National Laboratory for Cancer Research, Frederick, MD, USA., Bornhorst M; Center for Cancer and Blood Disorders, Children's National Hospital, Washington, DC, USA., Fisher MJ; Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Weiss BD; Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Kim A; Center for Cancer and Blood Disorders, Children's National Hospital, Washington, DC, USA., Widemann BC; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Gross AM; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
المصدر: Clinical trials (London, England) [Clin Trials] 2024 Apr; Vol. 21 (2), pp. 189-198. Date of Electronic Publication: 2023 Oct 25.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: SAGE Publications Country of Publication: England NLM ID: 101197451 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1740-7753 (Electronic) Linking ISSN: 17407745 NLM ISO Abbreviation: Clin Trials Subsets: MEDLINE
أسماء مطبوعة: Publication: London : SAGE Publications
Original Publication: London : Arnold, c2004-
مواضيع طبية MeSH: Neurofibroma, Plexiform*/drug therapy , Neurofibromatosis 1*/drug therapy, Child ; Humans ; Prospective Studies ; Reproducibility of Results
مستخلص: Background/aims: We developed an observer disfigurement severity scale for neurofibroma-related plexiform neurofibromas to assess change in plexiform neurofibroma-related disfigurement and evaluated its feasibility, reliability, and validity.
Methods: Twenty-eight raters, divided into four cohorts based on neurofibromatosis type 1 familiarity and clinical experience, were shown photographs of children in a clinical trial (NCT01362803) at baseline and 1 year on selumetinib treatment for plexiform neurofibromas ( n  = 20) and of untreated participants with plexiform neurofibromas ( n  = 4). Raters, blinded to treatment and timepoint, completed the 0-10 disfigurement severity score for plexiform neurofibroma on each image (0 = not at all disfigured, 10 = very disfigured). Raters evaluated the ease of completing the scale, and a subset repeated the procedure to assess intra-rater reliability.
Results: Mean baseline disfigurement severity score for plexiform neurofibroma ratings were similar for the selumetinib group (6.23) and controls (6.38). Mean paired differences between pre- and on-treatment ratings was -1.01 (less disfigurement) in the selumetinib group and 0.09 in the control ( p  = 0.005). For the disfigurement severity score for plexiform neurofibroma ratings, there was moderate-to-substantial agreement within rater cohorts (weighted kappa range = 0.46-0.66) and agreement between scores of the same raters at repeat sessions ( p  > 0.05). In the selumetinib group, change in disfigurement severity score for plexiform neurofibroma ratings was moderately correlated with change in plexiform neurofibroma volume with treatment ( r  = 0.60).
Conclusion: This study demonstrates that our observer-rated disfigurement severity score for plexiform neurofibroma was feasible, reliable, and documented improvement in disfigurement in participants with plexiform neurofibroma shrinkage. Prospective studies in larger samples are needed to validate this scale further.
Competing Interests: Declaration of conflicting interestsDr B.C.W. and Dr A.M.G. have unpaid advisory roles for AstraZeneca and SpringWorks. Dr M.J.F. has paid advisory roles for AstraZeneca and SpringWorks and research support from AstraZeneca, Array BioPharma, and Exelixis for plexiform neurofibroma clinical trials. The funders had no role in the design of the study; in the collection, analyses, or interpretation of data; in the writing of the manuscript; or in the decision to publish the results. Dr M. B. has a paid advisory role on the external advisory board for the Koselugo Registry through Alexion.
References: AMA J Ethics. 2018 Apr 1;20(4):309-323. (PMID: 29671724)
J Clin Oncol. 2021 Mar 1;39(7):797-806. (PMID: 33507822)
N Engl J Med. 2016 Dec 29;375(26):2550-2560. (PMID: 28029918)
J Pediatr. 1989 May;114(5):788-92. (PMID: 2497236)
Psychol Sci. 2006 May;17(5):383-6. (PMID: 16683924)
J Child Neurol. 2002 Aug;17(8):548-54; discussion 571-2, 646-51. (PMID: 12403552)
J Pediatr. 1997 Nov;131(5):678-82. (PMID: 9403645)
J Med Genet. 2002 May;39(5):311-4. (PMID: 12011145)
Science. 2005 Jun 10;308(5728):1623-6. (PMID: 15947187)
Bioanalysis. 2022 Jan;14(2):75-86. (PMID: 34841894)
Anesth Analg. 2007 Nov;105(5):1250-3, table of contents. (PMID: 17959951)
Nat Med. 2021 Jan;27(1):165-173. (PMID: 33442015)
Neurology. 2002 May 28;58(10):1461-70. (PMID: 12041525)
Neuro Oncol. 2018 Nov 12;20(12):1643-1651. (PMID: 29718344)
Pain. 2009 Jun;143(3):223-227. (PMID: 19359097)
Head Neck. 2000 Mar;22(2):132-41. (PMID: 10679900)
Neurology. 2009 Oct 20;73(16):1273-9. (PMID: 19841379)
N Engl J Med. 2020 Apr 9;382(15):1430-1442. (PMID: 32187457)
Childs Nerv Syst. 2021 Jun;37(6):1909-1915. (PMID: 33751171)
Neurology. 2007 Feb 27;68(9):643-7. (PMID: 17215493)
Mol Genet Genomic Med. 2023 Jan;11(1):e2077. (PMID: 36444392)
Neurol Clin. 2002 Aug;20(3):841-65. (PMID: 12432832)
Am J Med Genet. 1999 Mar 26;89(1):31-7. (PMID: 10469434)
Clin Cancer Res. 2021 Aug 1;27(15):4142-4146. (PMID: 33712511)
معلومات مُعتمدة: Z99 CA999999 United States ImNIH Intramural NIH HHS
فهرسة مساهمة: Keywords: Disfigurement; neurofibromatosis type 1; plexiform neurofibroma
سلسلة جزيئية: ClinicalTrials.gov NCT04879160
تواريخ الأحداث: Date Created: 20231025 Date Completed: 20240410 Latest Revision: 20240425
رمز التحديث: 20240425
مُعرف محوري في PubMed: PMC11003851
DOI: 10.1177/17407745231206402
PMID: 37877369
قاعدة البيانات: MEDLINE
الوصف
تدمد:1740-7753
DOI:10.1177/17407745231206402