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5. Computational Pipeline to probe NaV1.7 gain-of-functions variants in neuropathic painful syndromes

Author

Toffano, Alberto, Chiarot, Giacomo, Zamuner, Stefano, Marchi, Margherita, Salvi, Erika, Waxman, Stephen G., Faber, Catharina G., Lauria, Giuseppe, Giacometti, Achille, and Simeoni, Marta

Subjects
Condensed Matter - Soft Condensed Matter
Abstract

Applications of machine learning and graph theory techniques to neuroscience have witnessed an increased interest in the last decade due to the large data availability and unprecedented technology developments. Their employment to investigate the effect of mutational changes in genes encoding for proteins modulating the membrane of excitable cells, whose biological correlates are assessed at electrophysiological level, could provide useful predictive clues. We apply this concept to the analysis of variants in sodium channel NaV1.7 subunit found in patients with chronic painful syndromes, by the implementation of a dedicated computational pipeline empowering different and complementary techniques including homology modeling, network theory, and machine learning. By testing three templates of different origin and sequence identities, we provide an optimal condition for its use. Our findings reveal the usefulness of our computational pipeline in supporting the selection of candidates for cell electrophysiology assay and with potential clinical applications., Comment: 36 pages, 20 figures, Scientific Report in press

Published
2020

13. Second intravenous immunoglobulin dose in patients with Guillain-Barré syndrome with poor prognosis (SID-GBS): a double-blind, randomised, placebo-controlled trial

Author

Walgaard, Christa, Jacobs, Bart C., Lingsma, Hester F., Steyerberg, Ewout W., Van den Berg, Bianca, Doets, Alexandra Y., Leonhard, Sonja E., Verboon, Christine, Huizinga, Ruth, Drenthen, Judith, Arends, Samuel, Kleine Budde, Ilona, Kleyweg, Ruud P., Kuitwaard, Krista, Van der Meulen, Marjon F.G., Samijn, Johnny P.A., Vermeij, Frederique H., Kuks, Jan B.M., Van Dijk, Gert W., Wirtz, Paul W., Eftimov, Filip, Van der Kooi, Anneke J., Garssen, Marcel P.J., Gijsbers, Cees J., De Rijk, Maarten C., Visser, Leo H., Blom, Roderik J., Linssen, Wim H.J.P., Van der Kooi, Elly L., Verschuuren, Jan J.G.M., Van Koningsveld, Rinske, Dieks, Rita J.G., Gilhuis, H. Job, Jellema, Korné, Van der Ree, Taco C., Bienfait, Henriette M.E., Faber, Karin G., Lovenich, Harry, Van Engelen, Baziel G.M., Groen, Rutger J., Merkies, Ingemar S.J., Van Oosten, Bob W., Van der Pol, W. Ludo, Van der Meulen, Willem D.M., Badrising, Umesh A., Stevens, Martijn, Breukelman, Albert-Jan J., Zwetsloot, Casper P., Van der Graaff, Maaike M., Wohlgemuth, Marielle, Hughes, Richard A.C., Cornblath, David R., Van Doorn, Pieter A., Althingh van Geusau, R.B., Van Boheemen, C.J.M., Bronner, I.M., Feenstra, B., Fokke, C., Hoogendoorn, T.A., Van Houten, R., Hovestad, A., Jansen, P.J.H.W., Keuter, E., Krudde, J., Linn, F.H.H., Lion, J., Manschot, S.M., Mellema, S.J., Molenaar, D.S.M., Nieuwkamp, D.J., Oenema, D.G., Van Oostrom, J.C.H., Van Orshoven, N.P., Van der Ploeg, R.J.O., Polman, S., Ruitenberg, A., Ruts, L., Schyns-Soeterboek, A.J.G.M., Trip, R., Jacobs, Bart C, Lingsma, Hester F, Steyerberg, Ewout W, van den Berg, Bianca, Doets, Alexandra Y, Leonhard, Sonja E, Budde, Ilona Kleine, Kleyweg, Ruud P, van der Meulen, Marjon F G, Samijn, Johnny P A, Vermeij, Frederique H, Kuks, Jan B M, van Dijk, Gert W, Wirtz, Paul W, van der Kooi, Anneke J, Garssen, Marcel P J, Gijsbers, Cees J, de Rijk, Maarten C, Visser, Leo H, Blom, Roderik J, Linssen, Wim H J P, van der Kooi, Elly L, Verschuuren, Jan J G M, van Koningsveld, Rinske, Dieks, Rita J G, Gilhuis, H Job, van der Ree, Taco C, Bienfait, Henriette M E, Faber, Catharina G, van Engelen, Baziel G M, Groen, Rutger J, Merkies, Ingemar S J, van Oosten, Bob W, van der Pol, W Ludo, van der Meulen, Willem D M, Badrising, Umesh A, Breukelman, Albert-Jan J, Zwetsloot, Casper P, van der Graaff, Maaike M, Hughes, Richard A C, Cornblath, David R, and van Doorn, Pieter A

Published
2021
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15. Broadening the Genetic Spectrum of Painful Small-Fiber Neuropathy through Whole-Exome Study in Early-Onset Cases.

Author

Misra, Kaalindi, Ślęczkowska, Milena, Santoro, Silvia, Gerrits, Monique M., Mascia, Elisabetta, Marchi, Margherita, Salvi, Erika, Smeets, Hubert J. M., Hoeijmakers, Janneke G. J., Martinelli Boneschi, Filippo Giovanni, Filippi, Massimo, Lauria Pinter, Giuseppe, Faber, Catharina G., and Esposito, Federica

Subjects
NICOTINIC acetylcholine receptors, AGE of onset, PERIPHERAL nervous system, NEUROPATHY, SODIUM channels, TRP channels, OPIOID receptors
Abstract

Small-Fiber Neuropathy (SFN) is a disorder of the peripheral nervous system, characterised by neuropathic pain; approximately 11% of cases are linked to variants in Voltage-Gated Sodium Channels (VGSCs). This study aims to broaden the genetic knowledge on painful SFN by applying Whole-Exome Sequencing (WES) in Early-Onset (EO) cases. A total of 88 patients from Italy (n = 52) and the Netherlands (n = 36), with a disease onset at age ≤ 45 years old and a Pain Numerical Rating Score ≥ 4, were recruited. After variant filtering and classification, WES analysis identified 142 potentially causative variants in 93 genes; 8 are Pathogenic, 15 are Likely Pathogenic, and 119 are Variants of Uncertain Significance. Notably, an enrichment of variants in transient receptor potential genes was observed, suggesting their role in pain modulation alongside VGSCs. A pathway analysis performed by comparing EO cases with 40 Italian healthy controls found enriched mutated genes in the "Nicotinic acetylcholine receptor signaling pathway". Targeting this pathway with non-opioid drugs could offer novel therapeutic avenues for painful SFN. Additionally, with this study we demonstrated that employing a gene panel of reported mutated genes could serve as an initial screening tool for SFN in genetic studies, enhancing clinical diagnostics. [ABSTRACT FROM AUTHOR]

Published
2024
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16. Predicting Outcome in Guillain-Barré Syndrome: International Validation of the Modified Erasmus GBS Outcome Score

Author

Doets, Alex Y, Lingsma, Hester F, Walgaard, Christa, Islam, Badrul, Papri, Nowshin, Davidson, Amy, Yamagishi, Yuko, Kusunoki, Susumu, Dimachkie, Mazen M, Waheed, Waqar, Kolb, Noah, Islam, Zhahirul, Mohammad, Quazi Deen, Harbo, Thomas, Sindrup, Soren H, Chavada, Govindsinh, Willison, Hugh J, Casasnovas, Carlos, Bateman, Kathleen, Miller, James AL, van den Berg, Bianca, Verboon, Christine, Roodbol, Joyce, Leonhard, Sonja E, Benedetti, Luana, Kuwabara, Satoshi, Van den Bergh, Peter, Monges, Soledad, Marfia, Girolama A, Shahrizaila, Nortina, Galassi, Giuliana, Péréon, Yann, Bürmann, Jan, Kuitwaard, Krista, Kleyweg, Ruud P, Marchesoni, Cintia, Sedano Tous, María J, Querol, Luis, Illa, Isabel, Wang, Yuzhong, Nobile-Orazio, Eduardo, Rinaldi, Simon, Schenone, Angelo, Pardo, Julio, Vermeij, Frederique H, Lehmann, Helmar C, Granit, Volkan, Cavaletti, Guido, Gutiérrez-Gutiérrez, Gerardo, Barroso, Fabio A, Visser, Leo H, Katzberg, Hans D, Dardiotis, Efthimios, Attarian, Shahram, van der Kooi, Anneke J, Eftimov, Filip, Wirtz, Paul W, Samijn, Johnny PA, Gilhuis, H Jacobus, Hadden, Robert DM, Holt, James KL, Sheikh, Kazim A, Karafiath, Summer, Vytopil, Michal, Antonini, Giovanni, Feasby, Thomas E, Faber, Catharina G, Gijsbers, Cees J, Busby, Mark, Roberts, Rhys C, Silvestri, Nicholas J, Fazio, Raffaella, van Dijk, Gert W, Garssen, Marcel PJ, Straathof, Chiara SM, Gorson, Kenneth C, and Jacobs, Bart C

Published
2021
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23. Development, validation and feasibility of a Patient Satisfaction Questionnaire for evaluating the quality performance of a diagnostic small fibre neuropathy service: A qualitative study.

Author

Geerts, Margot, Hoeijmakers, Janneke G. J., Essers, Brigitte A. B., Merkies, Ingemar S. J., Faber, Catharina G., and Goossens, Mariëlle E. J. B.

Subjects
NEUROPATHY, HEALTH services accessibility, SCALE analysis (Psychology), QUALITATIVE research, DIAGNOSTIC services, OUTPATIENT services in hospitals, MEDICAL quality control, CRONBACH'S alpha, NEUROLOGISTS, EXECUTIVES, HEALTH status indicators, QUESTIONNAIRES, RESEARCH methodology evaluation, PILOT projects, RESEARCH evaluation, INTERVIEWING, CONTENT analysis, CONTINUUM of care, DESCRIPTIVE statistics, CHEST X rays, ANXIETY, EXPERIMENTAL design, SOUND recordings, ELECTROCARDIOGRAPHY, RESEARCH methodology, NEUROLOGY, QUALITY assurance, PATIENT satisfaction, DELPHI method, PHYSICIANS, CONFIDENCE intervals, PATIENTS' attitudes, COGNITION, MEDICAL referrals, NERVE conduction studies, MENTAL depression
Abstract

Introduction and Aim: Small fibre neuropathy (SFN) is a peripheral neuropathy, leading to neuropathic pain and autonomic dysfunction. An evidence‐based standardized patient diagnostic SFN service has been implemented in the Netherlands for improving patient‐centred SFN care. However, the quality of care of this diagnostic SFN service has never been assessed from a patient perspective. The aim of this study was to develop and validate an SFN‐Patient Satisfaction Questionnaire (SFN‐PSQ) to measure the quality performance of a standardized diagnostic SFN service. Methods: A descriptive qualitative study to create the SFN‐PSQ was performed using the (COREQ (Consolidated Criteria for Reporting Qualitative Research) checklist. For item generation and content development, domains and/or items from validated PSQs were selected. The content development and content validity were performed using a Delphi method with SFN expert caregivers with different backgrounds. By using the three‐step‐test method in individual cognitive interviews, the content validity by patients was finalized. Results: In one online Delphi panel round, the content of the first concept of the SFN‐PSQ was validated, which resulted in the second concept of the SFN‐PSQ. From July 2019 till March 2020, nine patients consented to participate in the individual cognitive interviews. The most significant changes of the new questionnaire were adding domains and items concerning the waiting list, the diagnostic services and consultation by the hospital psychiatrist. Also, a differentiation was made for both an inpatient and outpatient diagnostic SFN service. Furthermore, the clarity and intelligibility of the domains/items were improved, resulting in an increased comprehension of the SFN‐PSQ. Ultimately, the new developed SFN‐PSQ consisted of 10 domains and 51 items, suitable for measuring patient satisfaction of the neurological analysis in patients with SFN. Conclusion: Through item generation, expert opinions and interviews with patients, the SFN‐PSQ was developed and validated, and feasibility was confirmed. The structure of the questionnaire, based on the logistic and diagnostic SFN pathway, could be used as a model in other hospitals to improve the quality, continuity and access of SFN care and other chronic diseases taking into account potential cross‐cultural differences. Patient or Public Contribution: Caregivers were involved in the item generation and content development of the questionnaire. Patients were directly involved in testing the content validity and feasibility of the SFN‐PSQ. Clinical Trial Registration: Not applicable. [ABSTRACT FROM AUTHOR]

Published
2024
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26. Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial

Author

Kierkegaard, Marie, Okkersen, Kees, Jimenez-Moreno, Cecilia, Wenninger, Stephan, Daidj, Ferroudja, Glennon, Jeffrey, Cumming, Sarah, Littleford, Roberta, Monckton, Darren, Lochmüller, Hanns, Catt, Michael, Faber, Catharina, Hapca, Adrian, Donnan, Peter, Gorman, Gráinne, Bassez, Guillaume, Schoser, Benedikt, Knoop, Hans, Treweek, Shaun, van Engelen, Baziel, Maas, Daphne, Nikolaus, Stephanie, Cornelissen, Yvonne, van Nimwegen, Marlies, Klerks, Ellen, Bouman, Sacha, Heskamp, Linda, Heerschap, Arend, Rahmadi, Ridho, Groot, Perry, Heskes, Tom, Kapusta, Katarzyna, Abghari, Shaghayegh, Aschrafi, Armaz, Poelmans, Geert, Raaphorst, Joost, Trenell, Michael, van Laar, Sandra, Wood, Libby, Cassidy, Sophie, Newman, Jane, Charman, Sarah, Steffaneti, Renae, Taylor, Louise, Brownrigg, Allan, Day, Sharon, Atalaya, Antonio, Hogarth, Fiona, Schüller, Angela, Stahl, Kristina, Künzel, Heike, Wolf, Martin, Jelinek, Anna, Lignier, Baptiste, Couppey, Florence, Delmas, Stéphanie, Deux, Jean-François, Hankiewicz, Karolina, Dogan, Celine, Minier, Lisa, Chevalier, Pascale, Hamadouche, Amira, Adam, Berit, Hannah, Michael, McKenzie, Emma, Rauchhaus, Petra, Van Hees, Vincent, Catt, Sharon, Schwalber, Ameli, Merkies, Ingemar, Dittrich, Juliane, Monckton, Darren G, Faber, Catharina G, Donnan, Peter T, and van Engelen, Baziel G M

Published
2018
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27. Oral fingolimod for chronic inflammatory demyelinating polyradiculoneuropathy (FORCIDP Trial): a double-blind, multicentre, randomised controlled trial

Author

Day, Timothy, Spies, Judith, Roberts, Leslie, Van Damme, Philip, Van den Bergh, Peter YK, Maertens de Noordhout, Alain, Dionne, Annie, Larue, Sandrine, Massie, Rami, Melanson, Michel, Camu, William, De Seze, Jérôme, Le Masson, Gwendal, Pouget, Jean, Schmidt, Jens, Kimiskidis, Vasilios K, Chapman, Joab, Drory, Vivian E, Fazio, Raffaella, Gallia, Francesca, Kusunoki, Susumu, Mori, Masahiro, Iijima, Masahiro, Okamoto, Tomoko, Baba, Masayuki, Faber, Catharina G, van Schaik, Ivo N, Fryze, Waldemar, Motta, Ewa, Selmaj, Krzysztof, Casasnovas, Carlos, Sola, Antonio Guerrero, Illa, Isabel, Holt, James, Miller, James AL, Lunn, Michael P, Brannagan, Thomas H, III, Brown, Martin, Kelemen, John, Iyadurai, Stanley, Rezania, Kourosh, Sharma, Khema R, Tandan, Rup, Gudesblatt, Mark, Lawson, Victoria, Amato, Anthony A, Hughes, Richard, Dalakas, Marinos C, Merkies, Ingemar, Latov, Norman, Léger, Jean-Marc, Nobile-Orazio, Eduardo, Sobue, Gen, Genge, Angela, Cornblath, David, Merschhemke, Martin, Ervin, Carolyn Marie, Agoropoulou, Catherine, and Hartung, Hans-Peter

Published
2018
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29. Idiopathic distal sensory polyneuropathy: ACTTION diagnostic criteria

Author

Freeman, Roy, Gewandter, Jennifer S., Faber, Catharina G., Gibbons, Christopher, Haroutounian, Simon, Lauria, Giuseppe, Levine, Todd, Malik, Rayaz A., Singleton, J. Robinson, Smith, A. Gordon, Bell, Josh, Dworkin, Robert H., Feldman, Eva, Herrmann, David N., Hoke, Ahmet, Kolb, Noah, Mansikka, Heikki, Oaklander, Anne Louise, Peltier, Amanda, Polydefkis, Michael, Ritt, Elissa, Russell, James W., Sainati, Stephen, Steiner, Deborah, Treister, Roi, and Üçeyler, Nurcan

Published
2020
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30. Peripheral Pain Captured Centrally: Altered Brain Morphology on MRI in Small Fiber Neuropathy Patients With and Without an SCN9AGene Variant

Author

van Gool, Raquel, Far, Amir, Drenthen, Gerhard S., Jansen, Jacobus F.A., Goijen, Celine P., Backes, Walter H., Linden, David E.J., Merkies, Ingemar S.J., Faber, Catharina G., Upadhyay, Jaymin, and Hoeijmakers, Janneke G.J.

Abstract

The current study aims to characterize brain morphology of pain as reported by small fiber neuropathy (SFN) patients with or without a gain-of-function variant involving the SCN9Agene and compare these with findings in healthy controls without pain. The Neuropathic Pain Scale was used in patients with idiopathic SFN (N = 20) and SCN9A-associated SFN (N = 12) to capture pain phenotype. T1-weighted, structural magnetic resonance imaging (MRI) data were collected in patients and healthy controls (N = 21) to 1) compare cortical thickness and subcortical volumes and 2) quantify the association between severity, quality, and duration of pain with morphological properties. SCN9A-associated SFN patients showed significant (P < .017, Bonferroni corrected) higher cortical thickness in sensorimotor regions, compared to idiopathic SFN patients, while lower cortical thickness was found in more functionally diverse regions (eg, posterior cingulate cortex). SFN patient groups combined demonstrated a significant (Spearman’s ρ = .44–.55, P = .005–.049) correlation among itch sensations (Neuropathic Pain Scale-7) and thickness of the left precentral gyrus, and midcingulate cortices. Significant associations were found between thalamic volumes and duration of pain (left: ρ = −.37, P = .043; right: ρ = −.40, P = .025). No associations were found between morphological properties and other pain qualities. In conclusion, in SCN9A-associated SFN, profound morphological alterations anchored within the pain matrix are present. The association between itch sensations of pain and sensorimotor and midcingulate structures provides a novel basis for further examining neurobiological underpinnings of itch in SFN.

Published
2024
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35. TRPA1 rare variants in chronic neuropathic and nociplastic pain patients

Author

Marchi, Margherita, primary, Salvi, Erika, additional, Andelic, Mirna, additional, Mehmeti, Elkadia, additional, D'Amato, Ilaria, additional, Cazzato, Daniele, additional, Chiappori, Federica, additional, Lombardi, Raffaella, additional, Cartelli, Daniele, additional, Devigili, Grazia, additional, Dalla Bella, Eleonora, additional, Gerrits, Monique, additional, Almomani, Rowida, additional, Malik, Rayaz A., additional, Ślęczkowska, Milena, additional, Mazzeo, Anna, additional, Gentile, Luca, additional, Dib-Hajj, Sulayman, additional, Waxman, Stephen G., additional, Faber, Catharina G., additional, Vecchio, Eleonora, additional, de Tommaso, Marina, additional, and Lauria, Giuseppe, additional

Published
2023
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37. Integrative miRNA–mRNA profiling of human epidermis: unique signature of SCN9A painful neuropathy

Author

Andelic, Mirna, primary, Salvi, Erika, additional, Marcuzzo, Stefania, additional, Marchi, Margherita, additional, Lombardi, Raffaella, additional, Cartelli, Daniele, additional, Cazzato, Daniele, additional, Mehmeti, Elkadia, additional, Gelemanovic, Andrea, additional, Paolini, Matilde, additional, Pardo, Carlotta, additional, D’Amato, Ilaria, additional, Hoeijmakers, Janneke G J, additional, Dib-Hajj, Sulayman, additional, Waxman, Stephen G, additional, Faber, Catharina G, additional, and Lauria, Giuseppe, additional

Published
2023
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43. Peripheral Ion Channel Genes Screening in Painful Small Fiber Neuropathy

Author

Ślęczkowska, Milena, primary, Almomani, Rowida, additional, Marchi, Margherita, additional, Salvi, Erika, additional, de Greef, Bianca T A, additional, Sopacua, Maurice, additional, Hoeijmakers, Janneke G J, additional, Lindsey, Patrick, additional, Waxman, Stephen G, additional, Lauria, Giuseppe, additional, Faber, Catharina G, additional, Smeets, Hubert J M, additional, and Gerrits, Monique M, additional

Published
2022
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45. IgM Anti MAG± Peripheral Neuropathy (IMAGiNe) Study Protocol: An international, observational, prospective registry of patients with IgM M-protein Peripheral Neuropathies

Author

Hamadeh, Tatiana, van Doormaal, Perry T C, Pruppers, Mariëlle H J, van de Mortel, Johannes P M, Hoeijmakers, Janneke G J, Cornblath, David R, Vrancken, Alexander F J E, Faber, Catharina G, Notermans, Nicolette C, Marasca, Manuele, Salvalaggio, Alessandro, Briani, Chiara, and Merkies, Ingemar S J

Subjects
anti MAG antibodies, clinimetry, paraproteinemic neuropathies, IgM neuropathies, Monoclonal gammopathy of undetermined significance
Published
2023

47. Prospective Evaluation of Health Care Provider and Patient Assessments in Chemotherapy-Induced Peripheral Neurotoxicity

Author

Alberti, Paola, Bernasconi, Davide P, Cornblath, David R, Merkies, Ingemar S J, Park, Susanna B, Velasco, Roser, Bruna, Jordi, Psimaras, Dimitri, Koeppen, Susanne, Pace, Andrea, Dorsey, Susan G, Argyriou, Andreas A, Kalofonos, Haralabos P, Briani, Chiara, Schenone, Angelo, Faber, Catharina G, Mazzeo, Anna, Russo, Massimo, Grisold, Wolfgang, Valsecchi, Mariagrazia, Cavaletti, Guido, RS: MHeNs - R1 - Cognitive Neuropsychiatry and Clinical Neuroscience, Klinische Neurowetenschappen, MUMC+: MA Med Staf Spec Neurologie (9), Alberti, P, Bernasconi, D, Cornblath, D, Jose Merkies, I, Park, S, Velasco, R, Bruna, J, Psimaras, D, Susanne, K, Pace, A, Dorsey, S, Argyriou, A, Kalofonos, H, Briani, C, Schenone, A, Faber, C, Mazzeo, A, Grisold, W, Valsecchi, M, and Cavaletti, G

Subjects
Adult, medicine.medical_specialty, Adolescent, Patients, Psychometrics, TOTAL NEUROPATHY SCORE, neuropathy, CIPN, outcome measures, TNSc, TNSn, clinical trial, PATHOGENESIS, Medizin, Diagnostic Techniques, Neurological, Nurses, Antineoplastic Agents, CHILDREN, Gynecologic oncology, Severity of Illness Index, RESPONSIVENESS, Aged, Aged, 80 and over, Humans, Middle Aged, Neoplasms, Neurotoxicity Syndromes, Peripheral Nervous System Diseases, Physicians, Prospective Studies, Young Adult, Internal medicine, 80 and over, Medicine, media_common.cataloged_instance, THALIDOMIDE, European union, VALIDITY, Prospective cohort study, media_common, business.industry, Minimal clinically important difference, Gold standard, OUTCOME MEASURES, ASSESSMENT TOOLS, Chemotherapy regimen, Clinical trial, Diagnostic Techniques, MINIMALLY IMPORTANT DIFFERENCES, Neurological, Observational study, Neurology (clinical), business
Abstract

Background and ObjectiveThere is no agreement on the gold standard for detection and grading of chemotherapy-induced peripheral neurotoxicity (CIPN) in clinical trials. The objective is to perform an observational prospective study to assess and compare patient-based and physician-based methods for detection and grading of CIPN.MethodsConsecutive patients, aged 18 years or older, candidates for neurotoxic chemotherapy, were enrolled in the United States, European Union, or Australia. A trained investigator performed physician-based scales (Total Neuropathy Score–clinical [TNSc], used to calculate Total Neuropathy Score–nurse [TNSn]) and supervised the patient-completed questionnaire (Functional Assessment of Cancer Treatment/Gynecologic Oncology Group–Neurotoxicity [FACT/GOG-NTX]). Evaluations were performed before and at the end of chemotherapy. On participants without neuropathy at baseline, we assessed the association between TNSc, TNSn, and FACT/GOG-NTX. Considering a previously established minimal clinically important difference (MCID) for FACT/GOG-NTX, we identified participants with and without a clinically important deterioration according to this scale. Then, we calculated the MCID for TNSc and TNSn as the difference in the mean change score of these scales between the 2 groups.ResultsData from 254 participants were available: 180 (71%) had normal neurologic status at baseline. At the end of the study, 88% of participants developed any grade of neuropathy. TNSc, TNSn, and FACT/GOG-NTX showed good responsiveness (standardized mean change from baseline to end of chemotherapy >1 for all scales). On the 153 participants without neuropathy at baseline and treated with a known neurotoxic chemotherapy regimen, we verified a moderate correlation in both TNSc and TNSn scores with FACT/GOG-NTX (Spearman correlation index r = 0.6). On the same sample, considering as clinically important a change in the FACT/GOG-NTX score of at least 3.3 points, the MCID was 3.7 for TNSc and 2.8 for the TNSn.ConclusionsMCID for TNSc and TNSn were calculated and the TNSn can be considered a reliable alternative objective clinical assessment if a more extended neurologic examination is not possible. The FACT/GOG-NTX score can be reduced to 7 items and these items correlate well with the TNSc and TNSn.Classification of EvidenceThis study provides Class III evidence that a patient-completed questionnaire and nurse-assessed scale correlate with a physician-assessed scale.

Published
2021

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