Resumen
We developed a disease-specific, 10-point functional rating scale for patients with inclusion body myositis (IBMFRS). The IBMFRS was utilized as a secondary outcome measure in a multicenter pilot trial of the clinical safety and tolerability of high-dose beta interferon-1a. In this trial, 28 IBM patients completed the IBMFRS at baseline and monthly for 6 months. The IBMFRS showed statistically significant correlations (P < 0.001) with maximal voluntary isometric contraction, manual muscle testing, handgrip dynamometry, and the amyotrophic lateral sclerosis (ALS) functional rating scale (ALSPRS). Compared to these other outcome measures, the IBMFRS was also the most sensitive measure of change over the course of the study.
| Idioma original | English (US) |
|---|---|
| Páginas (desde-hasta) | 473-476 |
| Número de páginas | 4 |
| Publicación | Muscle and Nerve |
| Volumen | 37 |
| N.º | 4 |
| DOI | |
| Estado | Published - abr 1 2008 |
ASJC Scopus subject areas
- Clinical Neurology
- Physiology (medical)
- Cellular and Molecular Neuroscience
- Physiology
Acceder al documento
Otros archivos y enlaces
Huella
Profundice en los temas de investigación de 'Inclusion body myositis functional rating scale: A reliable and valid measure of disease severity'. En conjunto forman una huella única.Citar esto
- APA
- Standard
- Harvard
- Vancouver
- Author
- BIBTEX
- RIS
En: Muscle and Nerve, Vol. 37, N.º 4, 01.04.2008, p. 473-476.
Producción científica: Article › revisión exhaustiva
}
TY - JOUR
T1 - Inclusion body myositis functional rating scale
T2 - A reliable and valid measure of disease severity
AU - Jackson, C. E.
AU - Barohn, R. J.
AU - Gronseth, G.
AU - Pandya, S.
AU - Herbelin, L.
AU - Tawil, Rabi
AU - Griggs, Robert
AU - Thornton, Charles
AU - Kissel, John
AU - Mendell, Jerry
AU - Genge, Angela
AU - Karpati, George
AU - Jackson, Carlayne
AU - Rose, Michael
AU - Amato, Anthony
AU - Barohn, Richard
AU - Saperstein, David
AU - McDermott, Michael P.
AU - Pandya, Shree
AU - Wahlen, Deborah
AU - King, Wendy
AU - Venturini, Adriana
AU - Myers, Deborah
AU - Prisley, Samantha
AU - Tagerman, Michelle
AU - O'Brien, Meredith
AU - Herbelin, Laura
AU - Cos, Lynn
AU - Downing, Karen
AU - Plesiak, Roza
AU - Barefield, Frieda
AU - Morrison, Catherine
AU - Briemberg, Hannah
AU - Holloway, Robert
AU - Peterson, Derick
AU - Janciuras, Joanne
AU - Martens, William
AU - Blood, Christine
N1 - Funding Information: Benjamin O Anderson is supported by the Susan G Komen Leadership Grant Research Project, award number SAC160001. Itamar S Santos reports grants from FAPESP (Brazilian Public Agency), outside the submitted work. Carl Abelardo T Antonio reports grants, personal fees, and non-financial support from Johnson & Johnson (Philippines), outside the submitted work. Rafael Tabarés-Seisdedos and Ferrán Catalá-López are supported in part by grant PROMETEOII/2015/021 from Generalitat Valenciana, and Rafael Tabarés-Seisdedos is supported by the national grant PI14/00894 from ISCIII-FEDER. Pablo M Lavados reports grants, personal fees, and non-financial support from Bayer, non-financial support from Boehringer Ingelheim, grants and personal fees from AstraZeneca, grants from CONICYT, and grants from The George Institute for Global Health, outside the submitted work. Jasvinder A Singh serves as the principal investigator for an investigator-initiated study funded by Horizon pharmaceuticals through a grant to DINORA, a 501c3 entity, and is on the steering committee of OMERACT, an international organization that develops measures for clinical trials and receives arms-length funding from 36 pharmaceutical companies. Ai Koyanagi's work is supported by the Miguel Servet contract financed by the CP13/00150 and PI15/00862 projects, integrated into the National R+D+I and funded by the ISCIII General Branch Evaluation and Promotion of Health Research and the European Regional Development Fund (ERDF-FEDER). Aletta E Schutte is funded by the Medical Research Council of South Africa, and the South African Research Chair Initiative by the National Research Foundation. Ana Maria Nogales Vasconcelos and her team in Brazil has received funding from Ministry of Health (process number: 25000192049/2014-14). Mahesh PA acknowledges the Indian Council of Medical Research for financial support (ICMR Grant number 5/8/4-4 (Env)/2003-NCD-I). Katharine J Looker has received funding from the World Health Organization for the HSV-2 seroprevalence review which informs this work; during the study, KJL also received separate funding from the World Health Organization, USAID/PATH, Sexual Health 24, and the National Institute for Health Research (NIHR) Health Protection Research Unit (HPRU) in Evaluation of Interventions at the University of Bristol. Donal Bisanzio is supported by Bill & Melinda Gates Foundation (#OPP1068048). Thomas Fürst received financial support from the Swiss National Science Foundation (SNSF; project no P300P3-154634. Jost B Jonas reports personal fees from Consultant for Mundipharma (Cambridge, UK); from Patent holder with Biocompatibles UK (Franham, Surrey, UK; Treatment of eye diseases using encapsulated cells encoding and secreting neuroprotective factor and/or anti-angiogenic factor; Patent number: 20120263794), patent application with University of Heidelberg (Heidelberg, Germany; Agents for use in the therapeutic or prophylactic treatment of myopia or hyperopia; Europäische Patentanmeldung 15 000 771.4), outside the submitted work. Rodrigo Sarmiento-Suarez receives institutional support from Universidad de Ciencias Aplicadas y Ambientales, UDCA, Bogotá, Colombia. Stefanos Tyrovolas's work is supported by the Foundation for Education and European Culture (IPEP), the Sara Borrell postdoctoral programme (reference no CD15/00019 from the Instituto de Salud Carlos III (ISCIII—Spain) and the Fondos Europeo de Desarrollo Regional (FEDER). Beatriz Paulina Ayala Quintanilla acknowledges institutional support of PRONABEC (National Program of Scholarship and Educational Loan), provided by the Peruvian Government, while studying for her doctoral course at the Judith Lumley Centre of La Trobe University funded by PRONABEC. Manami Inoue is the beneficiary of a financial contribution from the AXA Research fund as chair holder of the AXA Department of Health and Human Security, Graduate School of Medicine, The University of Tokyo from Nov 1, 2012; the AXA Research Fund has no role in this work. Laith J Abu-Raddad acknowledges the support of Qatar National Research Fund (NPRP 9-040-3-008) who provided the main funding for generating the data provided to the GBD-IHME effort. Yogeshwar Kalkonde is a Wellcome Trust/ DBT India Alliance Intermediate Fellow in Public Health. Sarah Derrett reports grants and fees from the Euroqol Foundation, outside the submitted work. Dan J Stein reports personal fees from Lundbeck, Novartis, AMBRF, Biocodex, Sevier, SUN, and CIPLA, and grants from NRGF and MRC, outside the submitted work. Tea Lallukka reports funding from The Academy of Finland, grant 287488, as an Academy Research Fellow. Charles D A Wolfe's research was funded/supported by the National Institute for Health Research (NIHR) Biomedical Research Centre based at Guy's and St Thomas' NHS Foundation Trust and King's College London. The views expressed are those of the authors and not necessarily those of the NHS, the NIHR, the UK Department of Health, Public Health England, or the World Health Organization. All other authors declare no competing interests. Funding Information: We would like to thank the countless individuals who have contributed to the Global Burden of Disease Study 2015 in various capacities. The data reported here have been supplied by the US Renal Data System (USRDS). Data for this research was provided by MEASURE Evaluation, funded by the United States Agency for International Development (USAID). Collection of these data was made possible by the US Agency for International Development (USAID) under the terms of cooperative agreement GPO-A-00-08-000_D3-00. Views expressed do not necessarily reflect those of USAID, the US Government, or MEASURE Evaluation. Parts of this material are based on data and information provided by the Canadian institute for Health Information. However, the analyses, conclusions, opinions and statements expressed herein are those of the author and not those of the Canadian Institute for Health information. The Palestinian Central Bureau of Statistics granted the researchers access to relevant data in accordance with license no SLN2014-3-170, after subjecting data to processing aiming to preserve the confidentiality of individual data in accordance with the General Statistics Law, 2000. The researchers are solely responsible for the conclusions and inferences drawn upon available data. This paper uses data from SHARE Waves 1, 2, 3 (SHARELIFE), 4 and 5 (DOIs: 10.6103/SHARE.w1.500, 10.6103/SHARE.w2.500, 10.6103/SHARE.w3.500, 10.6103/SHARE.w4.500, 10.6103/SHARE.w5.500), see Börsch-Supan and colleagues, 2013, for methodological details. The SHARE data collection has been primarily funded by the European Commission through FP5 (QLK6-CT-2001-00360), FP6 (SHARE-I3: RII-CT-2006-062193, COMPARE: CIT5-CT-2005-028857, SHARELIFE: CIT4-CT-2006-028812) and FP7 (SHARE-PREP: number 211909, SHARE-LEAP: number 227822, SHARE M4: number 261982). Additional funding from the German Ministry of Education and Research, the US National Institute on Aging (U01_AG09740-13S2, P01_AG005842, P01_AG08291, P30_AG12815, R21_AG025169, Y1-AG-4553-01, IAG_BSR06-11, and OGHA_04-064) and from various national funding sources is gratefully acknowledged. This study has been realised using the data collected by the Swiss Household Panel (SHP), which is based at the Swiss Centre of Expertise in the Social Sciences FORS. The project is financed by the Swiss National Science Foundation. The following individuals would like to acknowledge various forms of institutional support: Simon I Hay is funded by a Senior Research Fellowship from the Wellcome Trust (#095066), and grants from the Bill & Melinda Gates Foundation (OPP1119467, OPP1093011, OPP1106023 and OPP1132415). Amanda G Thrift is supported by a fellowship from the National Health and Medical Research Council (GNT1042600). Panniyammakal Jeemon is supported by the Wellcome Trust-DBT India Alliance, Clinical and Public Health, Intermediate Fellowship (2015–2020). Boris Bikbov, Norberto Percio, and Giuseppe Remuzzi acknowledge that work related to this paper has been done on the behalf of the GBD Genitourinary Disease Expert Group supported by the International Society of Nephrology (ISN). Amador Goodridge acknowledges funding from Sistema Nacional de Investigadores de Panamá-SNI. José das Neves was supported in his contribution to this work by a Fellowship from Fundação para a Ciência e a Tecnologia, Portugal (SFRH/BPD/92934/2013). Lijing L Yan is supported by the National Natural Sciences Foundation of China grants (71233001 and 71490732). Olanrewaju Oladimeji is an African Research Fellow at Human Sciences Research Council (HSRC) and Doctoral Candidate at the University of KwaZulu-Natal (UKZN), South Africa, and would like to acknowledge the institutional support by leveraging on the existing organisational research infrastructure at HSRC and UKZN. Nicholas Steel received funding from Public Health England as a Visiting Scholar in the Institute for Health Metrics and Evaluation in 2016. No individuals acknowledged received additional compensation for their efforts.
PY - 2008/4/1
Y1 - 2008/4/1
N2 - We developed a disease-specific, 10-point functional rating scale for patients with inclusion body myositis (IBMFRS). The IBMFRS was utilized as a secondary outcome measure in a multicenter pilot trial of the clinical safety and tolerability of high-dose beta interferon-1a. In this trial, 28 IBM patients completed the IBMFRS at baseline and monthly for 6 months. The IBMFRS showed statistically significant correlations (P < 0.001) with maximal voluntary isometric contraction, manual muscle testing, handgrip dynamometry, and the amyotrophic lateral sclerosis (ALS) functional rating scale (ALSPRS). Compared to these other outcome measures, the IBMFRS was also the most sensitive measure of change over the course of the study.
AB - We developed a disease-specific, 10-point functional rating scale for patients with inclusion body myositis (IBMFRS). The IBMFRS was utilized as a secondary outcome measure in a multicenter pilot trial of the clinical safety and tolerability of high-dose beta interferon-1a. In this trial, 28 IBM patients completed the IBMFRS at baseline and monthly for 6 months. The IBMFRS showed statistically significant correlations (P < 0.001) with maximal voluntary isometric contraction, manual muscle testing, handgrip dynamometry, and the amyotrophic lateral sclerosis (ALS) functional rating scale (ALSPRS). Compared to these other outcome measures, the IBMFRS was also the most sensitive measure of change over the course of the study.
KW - Beta-interferon
KW - Functional scale
KW - Inclusion-body myositis
KW - Outcome measure
KW - Quality of life
UR - https://www.scopus.com/pages/publications/41849098898
UR - https://www.scopus.com/pages/publications/41849098898#tab=citedBy
U2 - 10.1002/mus.20958
DO - 10.1002/mus.20958
M3 - Article
C2 - 18236463
AN - SCOPUS:41849098898
SN - 0148-639X
VL - 37
SP - 473
EP - 476
JO - Muscle and Nerve
JF - Muscle and Nerve
IS - 4
ER -