University of Hertfordshire

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Old measures and new scores in spinal muscular atrophy patients

Research output: Contribution to journalArticlepeer-review

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Old measures and new scores in spinal muscular atrophy patients. / Mazzone, Elena; Montes, Jacqueline; Main, Marion; Mayhew, Anna; Ramsey, Danielle; Glanzman, Allan M; Dunaway, Sally; Salazar, Rachel; Pasternak, Amy; Quigley, Janet; Pane, Marika; Pera, Maria C; Scoto, Mariacristina; Messina, Sonia; Sframeli, Maria; D'amico, Adele; Van Den Hauwe, Marleen; Sivo, Serena; Goemans, Nathalie; Darras, Basil T; Kaufmann, Petra; Bertini, Enrico; De Vivo, Darryl C; Muntoni, Francesco; Finkel, Richard; Mercuri, Eugenio.

In: Muscle & nerve, Vol. 52, No. 3, 12.08.2015, p. 435-7.

Research output: Contribution to journalArticlepeer-review

Harvard

Mazzone, E, Montes, J, Main, M, Mayhew, A, Ramsey, D, Glanzman, AM, Dunaway, S, Salazar, R, Pasternak, A, Quigley, J, Pane, M, Pera, MC, Scoto, M, Messina, S, Sframeli, M, D'amico, A, Van Den Hauwe, M, Sivo, S, Goemans, N, Darras, BT, Kaufmann, P, Bertini, E, De Vivo, DC, Muntoni, F, Finkel, R & Mercuri, E 2015, 'Old measures and new scores in spinal muscular atrophy patients', Muscle & nerve, vol. 52, no. 3, pp. 435-7. https://doi.org/10.1002/mus.24748

APA

Mazzone, E., Montes, J., Main, M., Mayhew, A., Ramsey, D., Glanzman, A. M., Dunaway, S., Salazar, R., Pasternak, A., Quigley, J., Pane, M., Pera, M. C., Scoto, M., Messina, S., Sframeli, M., D'amico, A., Van Den Hauwe, M., Sivo, S., Goemans, N., ... Mercuri, E. (2015). Old measures and new scores in spinal muscular atrophy patients. Muscle & nerve, 52(3), 435-7. https://doi.org/10.1002/mus.24748

Vancouver

Mazzone E, Montes J, Main M, Mayhew A, Ramsey D, Glanzman AM et al. Old measures and new scores in spinal muscular atrophy patients. Muscle & nerve. 2015 Aug 12;52(3):435-7. https://doi.org/10.1002/mus.24748

Author

Mazzone, Elena ; Montes, Jacqueline ; Main, Marion ; Mayhew, Anna ; Ramsey, Danielle ; Glanzman, Allan M ; Dunaway, Sally ; Salazar, Rachel ; Pasternak, Amy ; Quigley, Janet ; Pane, Marika ; Pera, Maria C ; Scoto, Mariacristina ; Messina, Sonia ; Sframeli, Maria ; D'amico, Adele ; Van Den Hauwe, Marleen ; Sivo, Serena ; Goemans, Nathalie ; Darras, Basil T ; Kaufmann, Petra ; Bertini, Enrico ; De Vivo, Darryl C ; Muntoni, Francesco ; Finkel, Richard ; Mercuri, Eugenio. / Old measures and new scores in spinal muscular atrophy patients. In: Muscle & nerve. 2015 ; Vol. 52, No. 3. pp. 435-7.

Bibtex

@article{9c73aaadc87940fb9ba37d133802e4d9,
title = "Old measures and new scores in spinal muscular atrophy patients",
abstract = "INTRODUCTION: A recent Rasch analysis performed on the Hammersmith Functional Motor Scale-Expanded (HFMSE) in patients with spinal muscular atrophy (SMA) identified issues impacting scale validity, redundant items, and disordered thresholds on some items.METHODS: We modified the HMFSE scoring based on the Rasch analysis and on expert consensus to establish whether the traditional scoring overestimated the number of patients with changes within 2 points from baseline. Data were collected retrospectively from multicenter data sets in 255 type 2 and 3 SMA patients.RESULTS: The mean 12-month changes using the new and the traditional scoring system did not differ significantly (P > 0.05). The numbers of patients who improved or decreased by >2 points were also similar.CONCLUSIONS: The presence of outliers using the traditional scoring system was not due to overestimation of changes in activities that were tested bilaterally or to discrepancies in the scoring hierarchy of individual items.",
keywords = "Adolescent, Adult, Child, Child, Preschool, Female, Humans, Male, Middle Aged, Muscular Atrophy, Spinal, Psychometrics, Retrospective Studies, Severity of Illness Index, Spinal Muscular Atrophies of Childhood, Young Adult, Journal Article, Research Support, Non-U.S. Gov't",
author = "Elena Mazzone and Jacqueline Montes and Marion Main and Anna Mayhew and Danielle Ramsey and Glanzman, {Allan M} and Sally Dunaway and Rachel Salazar and Amy Pasternak and Janet Quigley and Marika Pane and Pera, {Maria C} and Mariacristina Scoto and Sonia Messina and Maria Sframeli and Adele D'amico and {Van Den Hauwe}, Marleen and Serena Sivo and Nathalie Goemans and Darras, {Basil T} and Petra Kaufmann and Enrico Bertini and {De Vivo}, {Darryl C} and Francesco Muntoni and Richard Finkel and Eugenio Mercuri",
note = "{\textcopyright} 2015 Wiley Periodicals, Inc.",
year = "2015",
month = aug,
day = "12",
doi = "10.1002/mus.24748",
language = "English",
volume = "52",
pages = "435--7",
journal = "Muscle & nerve",
issn = "1097-4598",
publisher = "John Wiley and Sons Inc.",
number = "3",

}

RIS

TY - JOUR

T1 - Old measures and new scores in spinal muscular atrophy patients

AU - Mazzone, Elena

AU - Montes, Jacqueline

AU - Main, Marion

AU - Mayhew, Anna

AU - Ramsey, Danielle

AU - Glanzman, Allan M

AU - Dunaway, Sally

AU - Salazar, Rachel

AU - Pasternak, Amy

AU - Quigley, Janet

AU - Pane, Marika

AU - Pera, Maria C

AU - Scoto, Mariacristina

AU - Messina, Sonia

AU - Sframeli, Maria

AU - D'amico, Adele

AU - Van Den Hauwe, Marleen

AU - Sivo, Serena

AU - Goemans, Nathalie

AU - Darras, Basil T

AU - Kaufmann, Petra

AU - Bertini, Enrico

AU - De Vivo, Darryl C

AU - Muntoni, Francesco

AU - Finkel, Richard

AU - Mercuri, Eugenio

N1 - © 2015 Wiley Periodicals, Inc.

PY - 2015/8/12

Y1 - 2015/8/12

N2 - INTRODUCTION: A recent Rasch analysis performed on the Hammersmith Functional Motor Scale-Expanded (HFMSE) in patients with spinal muscular atrophy (SMA) identified issues impacting scale validity, redundant items, and disordered thresholds on some items.METHODS: We modified the HMFSE scoring based on the Rasch analysis and on expert consensus to establish whether the traditional scoring overestimated the number of patients with changes within 2 points from baseline. Data were collected retrospectively from multicenter data sets in 255 type 2 and 3 SMA patients.RESULTS: The mean 12-month changes using the new and the traditional scoring system did not differ significantly (P > 0.05). The numbers of patients who improved or decreased by >2 points were also similar.CONCLUSIONS: The presence of outliers using the traditional scoring system was not due to overestimation of changes in activities that were tested bilaterally or to discrepancies in the scoring hierarchy of individual items.

AB - INTRODUCTION: A recent Rasch analysis performed on the Hammersmith Functional Motor Scale-Expanded (HFMSE) in patients with spinal muscular atrophy (SMA) identified issues impacting scale validity, redundant items, and disordered thresholds on some items.METHODS: We modified the HMFSE scoring based on the Rasch analysis and on expert consensus to establish whether the traditional scoring overestimated the number of patients with changes within 2 points from baseline. Data were collected retrospectively from multicenter data sets in 255 type 2 and 3 SMA patients.RESULTS: The mean 12-month changes using the new and the traditional scoring system did not differ significantly (P > 0.05). The numbers of patients who improved or decreased by >2 points were also similar.CONCLUSIONS: The presence of outliers using the traditional scoring system was not due to overestimation of changes in activities that were tested bilaterally or to discrepancies in the scoring hierarchy of individual items.

KW - Adolescent

KW - Adult

KW - Child

KW - Child, Preschool

KW - Female

KW - Humans

KW - Male

KW - Middle Aged

KW - Muscular Atrophy, Spinal

KW - Psychometrics

KW - Retrospective Studies

KW - Severity of Illness Index

KW - Spinal Muscular Atrophies of Childhood

KW - Young Adult

KW - Journal Article

KW - Research Support, Non-U.S. Gov't

U2 - 10.1002/mus.24748

DO - 10.1002/mus.24748

M3 - Article

C2 - 26111847

VL - 52

SP - 435

EP - 437

JO - Muscle & nerve

JF - Muscle & nerve

SN - 1097-4598

IS - 3

ER -