Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS

被引：16

作者：

Cornett, Kayla M. D. ^{[1
]}

Menezes, Manoj P. ^{[2
]}

Bray, Paula ^{[1
]}

Shy, Rosemary R. ^{[3
]}

Moroni, Isabella ^{[4
]}

Pagliano, Emanuela ^{[4
]}

Pareyson, Davide ^{[4
]}

Estilow, Tim ^{[5
]}

Yum, Sabrina W. ^{[6
]}

Bhandari, Trupti ^{[7
,8
]}

Muntoni, Francesco ^{[7
,8
,9
]}

Laura, Matilde ^{[9
]}

Reilly, Mary M. ^{[9
]}

Finkel, Richard S. ^{[10
]}

Eichinger, Katy J. ^{[11
]}

Herrmann, David N. ^{[11
]}

Shy, Michael E. ^{[12
]}

Burns, Joshua ^{[1
]}

机构：

[1] Univ Sydney, Childrens Hosp Westmead, Sch Hlth Sci, Sydney, NSW, Australia

[2] Univ Sydney, Childrens Hosp Westmead, Paediat & Child Hlth, Sydney, NSW, Australia

[3] Univ Iowa, Dept Pediat, Carver Coll Med, Iowa City, IA 52242 USA

[4] Fdn IRCCS Ist Neurol Carlo Besta, Milan, Italy

[5] Childrens Hosp Philadelphia, Dept Occupat Therapy, Philadelphia, PA 19104 USA

[6] Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Div Neurol, Philadelphia, PA 19104 USA

[7] Great Ormond St Hosp Sick Children, UCL Inst Child Hlth, Biomed Res Ctr, London, England

[8] Great Ormond St Hosp Sick Children, Natl Inst Hlth Res, Biomed Res Ctr, London, England

[9] UCL, MRC Ctr Neuromuscular Dis, UCL Queen Sq Inst Neurol, London, England

[10] St Jude Childrens Res Hosp, Translat Neurosci Pediat, 332 N Lauderdale St, Memphis, TN 38105 USA

[11] Univ Rochester, Dept Neurol, Rochester, NY USA

[12] Univ Iowa, Dept Neurol, Carver Coll Med, Iowa City, IA 52242 USA

来源：

ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY | 2020年 / 7卷 / 09期

基金：

英国医学研究理事会;

关键词：

MARIE-TOOTH DISEASE; VALIDATION;

D O I：

10.1002/acn3.51145

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

The CMT Pediatric Scale (CMTPedS) is a reliable, valid, and responsive clinical outcome measure of disability in children with CMT. The aim of this study was to identify the most responsive patient subset(s), based on the standardized response mean (SRM), to optimize the CMTPedS as a primary outcome measure for upcoming clinical trials. Analysis was based on a 2-year natural history data from 187 children aged 3-20 years with a range of CMT genetic subtypes. Subsets based on age (3-8 years), disability level (CMTPedS score 0-14), and CMT type (CMT1A) increased the SRM of the CMTPedS considerably. Refining the inclusion criteria in clinical trials to younger, mildly affected cases of CMT1A optimizes the responsiveness of the CMTPedS.

引用

页码：1713 / 1715

页数：3

共 8 条

[1] Transitioning outcome measures: relationship between the CMTPedS and CMTNSv2 in children, adolescents, and young adults with Charcot-Marie-Tooth disease [J].

Burns, Joshua ;

Menezes, Manoj ;

Finkel, Richard S. ;

Estilow, Tim ;

Moroni, Isabella ;

Pagliano, Emanuela ;

Laura, Matilde ;

Muntoni, Francesco ;

Herrmann, David N. ;

Eichinger, Kate ;

Shy, Rosemary ;

Pareyson, Davide ;

Reilly, Mary M. ;

Shy, Michael E. .

JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, 2013, 18 (02) :177-180

[2] Validation of the Charcot-Marie-Tooth disease pediatric scale as an outcome measure of disability [J].

Burns, Joshua ;

Ouvrier, Robert ;

Estilow, Tim ;

Shy, Rosemary ;

Laura, Matilde ;

Pallant, Julie F. ;

Lek, Monkol ;

Muntoni, Francesco ;

Reilly, Mary M. ;

Pareyson, Davide ;

Acsadi, Gyula ;

Shy, Michael E. ;

Finkel, Richard S. .

ANNALS OF NEUROLOGY, 2012, 71 (05) :642-652

[3] Natural History of Charcot-Marie-Tooth Disease During Childhood [J].

Cornett, Kayla M. D. ;

Menezes, Manoj P. ;

Shy, Rosemary R. ;

Moroni, Isabella ;

Pagliano, Emanuela ;

Pareyson, Davide ;

Estilow, Timothy ;

Yum, Sabrina W. ;

Bhandari, Trupti ;

Muntoni, Francesco ;

Laura, Matilde ;

Reilly, Mary M. ;

Finkel, Richard S. ;

Eichinger, Kate J. ;

Herrmann, David N. ;

Bray, Paula ;

Halaki, Mark ;

Shy, Michael E. ;

Burns, Joshua .

ANNALS OF NEUROLOGY, 2017, 82 (03) :353-359

[4] Phenotypic Variability of Childhood Charcot-Marie-Tooth Disease [J].

Cornett, Kayla M. D. ;

Menezes, Manoj P. ;

Bray, Paula ;

Halaki, Mark ;

Shy, Rosemary R. ;

Yum, Sabrina W. ;

Estilow, Timothy ;

Moroni, Isabella ;

Foscan, Maria ;

Pagliano, Emanuela ;

Pareyson, Davide ;

Laura, Matilde ;

Bhandari, Trupti ;

Muntoni, Francesco ;

Reilly, Mary M. ;

Finkel, Richard S. ;

Sowden, Janet ;

Eichinger, Katy J. ;

Herrmann, David N. ;

Shy, Michael E. ;

Burns, Joshua .

JAMA NEUROLOGY, 2016, 73 (06) :645-651

[5] Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives [J].

Dangouloff, Tamara ;

Servais, Laurent .

THERAPEUTICS AND CLINICAL RISK MANAGEMENT, 2019, 15 :1153-1161

[6] Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study [J].

De Vivo, Darryl C. ;

Bertini, Enrico ;

Swoboda, Kathryn J. ;

Hwu, Wuh-Liang ;

Crawford, Thomas O. ;

Finkel, Richard S. ;

Kirschner, Janbernd ;

Kuntz, Nancy L. ;

Parsons, Julie A. ;

Ryan, Monique M. ;

Butterfield, Russell J. ;

Topaloglu, Haluk ;

Ben-Omran, Tawfeg ;

Sansone, Valeria A. ;

Jong, Yuh-Jyh ;

Shu, Francy ;

Staropoli, John F. ;

Kerr, Douglas ;

Sandrock, Alfred W. ;

Stebbins, Christopher ;

Petrillo, Marco ;

Braley, Gabriel ;

Johnson, Kristina ;

Foster, Richard ;

Gheuens, Sarah ;

Bhan, Ishir ;

Reyna, Sandra P. ;

Fradette, Stephanie ;

Farwell, Wildon .

NEUROMUSCULAR DISORDERS, 2019, 29 (11) :842-856

[7] MRI biomarker assessment of neuromuscular disease progression: a prospective observational cohort study [J].

Morrow, Jasper M. ;

Sinclair, Christopher D. J. ;

Fischmann, Arne ;

Machado, Pedro M. ;

Reilly, Mary M. ;

Yousry, Tarek A. ;

Thornton, John S. ;

Hanna, Michael G. .

LANCET NEUROLOGY, 2016, 15 (01) :65-77

[8] Development and validation of a novel rating system for scoring standing foot posture: The Foot Posture Index [J].

Redmond, AC ;

Crosbie, J ;

Ouvrier, RA .

CLINICAL BIOMECHANICS, 2006, 21 (01) :89-98

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