Validating Techniques for Measurement of Cutaneous Neurofibromas: Recommendations for Clinical Trials.

Adult Clinical Trials as Topic Humans Male Neurofibroma / diagnostic imaging Neurofibromatosis 1 / diagnostic imaging Photography / methods Reproducibility of Results Skin Neoplasms / pathology

Journal

Neurology
ISSN: 1526-632X
Titre abrégé: Neurology
Pays: United States
ID NLM: 0401060

Informations de publication

Date de publication:
17 08 2021
Historique:
received: 09 10 2020
accepted: 11 05 2021
pubmed: 8 7 2021
medline: 2 9 2021
entrez: 7 7 2021
Statut: ppublish

Résumé

To assess the reliability and variability of digital calipers, 3D photography, and high-frequency ultrasound (HFUS) for measurement of cutaneous neurofibromas (cNF) in patients with neurofibromatosis type 1 (NF1). cNF affect virtually all patients with NF1 and are a major source of morbidity. Reliable techniques for measuring cNF are needed to develop therapies for these tumors. Adults with NF1 were recruited. For each participant, 6 cNF were assessed independently by 3 different examiners at 5 different time points using digital calipers, 3D photography, and HFUS. The intraclass correlation coefficient (ICC) was used to assess intrarater and interrater reliability of linear and volumetric measurements for each technique, with ICC values >0.90 defined as excellent reliability. The coefficient of variation (CV) was used to estimate the minimal detectable difference (MDD) for each technique. Fifty-seven cNF across 10 participants were evaluated. The ICC for image acquisition and measurement was >0.97 within and across examiners for HFUS and 3D photography. ICC for digital calipers was 0.62-0.88. CV varied by measurement tool, linear vs volumetric measurement, and tumor size. HFUS and 3D photography demonstrate excellent reliability whereas digital calipers have good to excellent reliability in measuring cNF. The MDD for each technique was used to create tables of proposed thresholds for investigators to use as guides for clinical trials focused on cNF size. These criteria should be updated as the performance of these end points is evaluated.

Sections du résumé

OBJECTIVE
To assess the reliability and variability of digital calipers, 3D photography, and high-frequency ultrasound (HFUS) for measurement of cutaneous neurofibromas (cNF) in patients with neurofibromatosis type 1 (NF1).
BACKGROUND
cNF affect virtually all patients with NF1 and are a major source of morbidity. Reliable techniques for measuring cNF are needed to develop therapies for these tumors.
METHODS
Adults with NF1 were recruited. For each participant, 6 cNF were assessed independently by 3 different examiners at 5 different time points using digital calipers, 3D photography, and HFUS. The intraclass correlation coefficient (ICC) was used to assess intrarater and interrater reliability of linear and volumetric measurements for each technique, with ICC values >0.90 defined as excellent reliability. The coefficient of variation (CV) was used to estimate the minimal detectable difference (MDD) for each technique.
RESULTS
Fifty-seven cNF across 10 participants were evaluated. The ICC for image acquisition and measurement was >0.97 within and across examiners for HFUS and 3D photography. ICC for digital calipers was 0.62-0.88. CV varied by measurement tool, linear vs volumetric measurement, and tumor size.
CONCLUSIONS
HFUS and 3D photography demonstrate excellent reliability whereas digital calipers have good to excellent reliability in measuring cNF. The MDD for each technique was used to create tables of proposed thresholds for investigators to use as guides for clinical trials focused on cNF size. These criteria should be updated as the performance of these end points is evaluated.

Identifiants

DOI: 10.1212/WNL.0000000000012428 PMID: 34230197
pubmed: 34230197
pii: WNL.0000000000012428
doi: 10.1212/WNL.0000000000012428
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

S32-S41

Informations de copyright

© 2021 American Academy of Neurology.

Auteurs

Raquel D Thalheimer (RD)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Vanessa L Merker (VL)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.
ORCID: 0000-0002-4542-5227

K Ina Ly (KI)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Amanda Champlain (A)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Jennifer Sawaya (J)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Naomi L Askenazi (NL)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Hamilton P Herr (HP)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Jennifer L W Da (JLW)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Justin T Jordan (JT)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Alona Muzikansky (A)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Elizabeth Morehouse Pearce (EM)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Fernanda H Sakamoto (FH)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Jaishri O Blakeley (JO)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

R Rox Anderson (RR)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD.

Scott R Plotkin (SR)

From the Department of Neurology and Cancer Center (R.T., V.L.M., I.L., N.L.A., H.P.H., J.L.W.D., J.T.J., S.R.P.), Wellman Center for Photomedicine (A.C., J.S., E.M.P., F.H.S., R.R.A.), and Biostatistics Center (A.M.), Massachusetts General Hospital, and Department of Dermatology (A.C., J.S., E.M.P., F.H.S., R.R.A.), Harvard Medical School, Boston; and Department of Neurology, Neurosurgery, and Oncology (J.B.), Johns Hopkins University School of Medicine, Baltimore, MD. splotkin@partners.org.
ORCID: 0000-0002-6109-6419

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Validating Techniques for Measurement of...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études cliniques comme sujet › Essais cliniques comme sujet › Validating Techniques for Measurement of...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Validating Techniques for Measurement of...
questionsmedicales.fr › Maladies du système nerveux › Maladies neuromusculaires › Neuropathies périphériques › Tumeurs du système nerveux périphérique › Tumeurs des gaines nerveuses › Neurofibrome › Validating Techniques for Measurement of...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Syndromes néoplasiques héréditaires › Neurofibromatoses › Neurofibromatose de type 1 › Validating Techniques for Measurement of...
questionsmedicales.fr › Techniques d'investigation › Photographie (méthode) › Validating Techniques for Measurement of...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Méthodologie en recherche épidémiologique › Reproductibilité des résultats › Validating Techniques for Measurement of...
questionsmedicales.fr › Maladies de la peau et du tissu conjonctif › Maladies de la peau › Tumeurs cutanées › Validating Techniques for Measurement of...