Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy

Maria C Pera; Giorgia Coratti; Nicola Forcina; Elena S Mazzone; Mariacristina Scoto; Jacqueline Montes; Amy Pasternak; Anna Mayhew; Sonia Messina; Maria Sframeli; Marion Main; Robert Muni Lofra; Tina Duong; Danielle Ramsey; Sally Dunaway; Rachel Salazar; Lavinia Fanelli; Matthew Civitello; Roberto de Sanctis; Laura Antonaci; Leonardo Lapenta; Simona Lucibello; Marika Pane; John Day; Basil T Darras; Darryl C De Vivo; Francesco Muntoni; Richard Finkel; Eugenio Mercuri

doi:10.1186/s12883-017-0790-9

Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy

Maria C Pera, Giorgia Coratti, Nicola Forcina, Elena S Mazzone, Mariacristina Scoto, Jacqueline Montes, Amy Pasternak, Anna Mayhew, Sonia Messina, Maria Sframeli, Marion Main, Robert Muni Lofra, Tina Duong, Danielle Ramsey, Sally Dunaway, Rachel Salazar, Lavinia Fanelli, Matthew Civitello, Roberto de Sanctis, Laura AntonaciLeonardo Lapenta, Simona Lucibello, Marika Pane, John Day, Basil T Darras, Darryl C De Vivo, Francesco Muntoni, Richard Finkel, Eugenio Mercuri

School of Health and Social Work

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Abstract

BACKGROUND: Reports on the clinical meaningfulness of outcome measures in spinal muscular atrophy (SMA) are rare. In this two-part study, our aim was to explore patients' and caregivers' views on the clinical relevance of the Hammersmith Functional Motor Scale Expanded- (HFMSE).

METHODS: First, we used focus groups including SMA patients and caregivers to explore their views on the clinical relevance of the individual activities included in the HFMSE. Then we asked caregivers to comment on the clinical relevance of possible changes of HFMSE scores over time. As functional data of individual patients were available, some of the questions were tailored according to their functional level on the HFMSE.

RESULTS: Part 1: Sixty-three individuals participated in the focus groups. This included 30 caregivers, 25 patients and 8 professionals who facilitated the discussion. The caregivers provided a comparison to activities of daily living for each of the HFMSE items. Part 2: One hundred and forty-nine caregivers agreed to complete the questionnaire: in response to a general question, 72% of the caregivers would consider taking part in a clinical trial if the treatment was expected to slow down deterioration, 88% if it would stop deterioration and 97% if the treatment was expected to produce an improvement. Caregivers were informed of the first three items that their child could not achieve on the HFMSE. In response 75% indicated a willingness to take part in a clinical trial if they could achieve at least one of these abilities, 89% if they could achieve two, and 100% if they could achieve more than 2.

CONCLUSIONS: Our findings support the use of the HFMSE as a key outcome measure in SMA clinical trials because the individual items and the detected changes have clear content validity and clinical meaningfulness for patients and their caregivers.

Original language	English
Pages (from-to)	39
Number of pages	10
Journal	BMC Neurology
Volume	17
Issue number	1
DOIs	https://doi.org/10.1186/s12883-017-0790-9
Publication status	Published - 23 Feb 2017

Keywords

spinal muscular atrophy
quality of life
carers
clinical trials

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10.1186/s12883-017-0790-9Licence: CC BY

Published VersionFinal published version, 1.21 MBLicence: CC BY

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Pera, M. C., Coratti, G., Forcina, N., Mazzone, E. S., Scoto, M., Montes, J., Pasternak, A., Mayhew, A., Messina, S., Sframeli, M., Main, M., Lofra, R. M., Duong, T., Ramsey, D., Dunaway, S., Salazar, R., Fanelli, L., Civitello, M., de Sanctis, R., ... Mercuri, E. (2017). Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy. BMC Neurology, 17(1), 39. https://doi.org/10.1186/s12883-017-0790-9

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title = "Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy",

abstract = "BACKGROUND: Reports on the clinical meaningfulness of outcome measures in spinal muscular atrophy (SMA) are rare. In this two-part study, our aim was to explore patients' and caregivers' views on the clinical relevance of the Hammersmith Functional Motor Scale Expanded- (HFMSE).METHODS: First, we used focus groups including SMA patients and caregivers to explore their views on the clinical relevance of the individual activities included in the HFMSE. Then we asked caregivers to comment on the clinical relevance of possible changes of HFMSE scores over time. As functional data of individual patients were available, some of the questions were tailored according to their functional level on the HFMSE.RESULTS: Part 1: Sixty-three individuals participated in the focus groups. This included 30 caregivers, 25 patients and 8 professionals who facilitated the discussion. The caregivers provided a comparison to activities of daily living for each of the HFMSE items. Part 2: One hundred and forty-nine caregivers agreed to complete the questionnaire: in response to a general question, 72% of the caregivers would consider taking part in a clinical trial if the treatment was expected to slow down deterioration, 88% if it would stop deterioration and 97% if the treatment was expected to produce an improvement. Caregivers were informed of the first three items that their child could not achieve on the HFMSE. In response 75% indicated a willingness to take part in a clinical trial if they could achieve at least one of these abilities, 89% if they could achieve two, and 100% if they could achieve more than 2.CONCLUSIONS: Our findings support the use of the HFMSE as a key outcome measure in SMA clinical trials because the individual items and the detected changes have clear content validity and clinical meaningfulness for patients and their caregivers.",

keywords = "spinal muscular atrophy, quality of life, carers, clinical trials",

author = "Pera, {Maria C} and Giorgia Coratti and Nicola Forcina and Mazzone, {Elena S} and Mariacristina Scoto and Jacqueline Montes and Amy Pasternak and Anna Mayhew and Sonia Messina and Maria Sframeli and Marion Main and Lofra, {Robert Muni} and Tina Duong and Danielle Ramsey and Sally Dunaway and Rachel Salazar and Lavinia Fanelli and Matthew Civitello and {de Sanctis}, Roberto and Laura Antonaci and Leonardo Lapenta and Simona Lucibello and Marika Pane and John Day and Darras, {Basil T} and {De Vivo}, {Darryl C} and Francesco Muntoni and Richard Finkel and Eugenio Mercuri",

note = "{\textcopyright} The Author(s). 2017 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated",

year = "2017",

month = feb,

day = "23",

doi = "10.1186/s12883-017-0790-9",

language = "English",

volume = "17",

pages = "39",

journal = "BMC Neurology",

issn = "1471-2377",

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Pera, MC, Coratti, G, Forcina, N, Mazzone, ES, Scoto, M, Montes, J, Pasternak, A, Mayhew, A, Messina, S, Sframeli, M, Main, M, Lofra, RM, Duong, T, Ramsey, D, Dunaway, S, Salazar, R, Fanelli, L, Civitello, M, de Sanctis, R, Antonaci, L, Lapenta, L, Lucibello, S, Pane, M, Day, J, Darras, BT, De Vivo, DC, Muntoni, F, Finkel, R & Mercuri, E 2017, 'Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy', BMC Neurology, vol. 17, no. 1, pp. 39. https://doi.org/10.1186/s12883-017-0790-9

TY - JOUR

T1 - Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy

AU - Pera, Maria C

AU - Coratti, Giorgia

AU - Forcina, Nicola

AU - Mazzone, Elena S

AU - Scoto, Mariacristina

AU - Montes, Jacqueline

AU - Pasternak, Amy

AU - Mayhew, Anna

AU - Messina, Sonia

AU - Sframeli, Maria

AU - Main, Marion

AU - Lofra, Robert Muni

AU - Duong, Tina

AU - Ramsey, Danielle

AU - Dunaway, Sally

AU - Salazar, Rachel

AU - Fanelli, Lavinia

AU - Civitello, Matthew

AU - de Sanctis, Roberto

AU - Antonaci, Laura

AU - Lapenta, Leonardo

AU - Lucibello, Simona

AU - Pane, Marika

AU - Day, John

AU - Darras, Basil T

AU - De Vivo, Darryl C

AU - Muntoni, Francesco

AU - Finkel, Richard

AU - Mercuri, Eugenio

N1 - © The Author(s). 2017 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated

PY - 2017/2/23

Y1 - 2017/2/23

N2 - BACKGROUND: Reports on the clinical meaningfulness of outcome measures in spinal muscular atrophy (SMA) are rare. In this two-part study, our aim was to explore patients' and caregivers' views on the clinical relevance of the Hammersmith Functional Motor Scale Expanded- (HFMSE).METHODS: First, we used focus groups including SMA patients and caregivers to explore their views on the clinical relevance of the individual activities included in the HFMSE. Then we asked caregivers to comment on the clinical relevance of possible changes of HFMSE scores over time. As functional data of individual patients were available, some of the questions were tailored according to their functional level on the HFMSE.RESULTS: Part 1: Sixty-three individuals participated in the focus groups. This included 30 caregivers, 25 patients and 8 professionals who facilitated the discussion. The caregivers provided a comparison to activities of daily living for each of the HFMSE items. Part 2: One hundred and forty-nine caregivers agreed to complete the questionnaire: in response to a general question, 72% of the caregivers would consider taking part in a clinical trial if the treatment was expected to slow down deterioration, 88% if it would stop deterioration and 97% if the treatment was expected to produce an improvement. Caregivers were informed of the first three items that their child could not achieve on the HFMSE. In response 75% indicated a willingness to take part in a clinical trial if they could achieve at least one of these abilities, 89% if they could achieve two, and 100% if they could achieve more than 2.CONCLUSIONS: Our findings support the use of the HFMSE as a key outcome measure in SMA clinical trials because the individual items and the detected changes have clear content validity and clinical meaningfulness for patients and their caregivers.

AB - BACKGROUND: Reports on the clinical meaningfulness of outcome measures in spinal muscular atrophy (SMA) are rare. In this two-part study, our aim was to explore patients' and caregivers' views on the clinical relevance of the Hammersmith Functional Motor Scale Expanded- (HFMSE).METHODS: First, we used focus groups including SMA patients and caregivers to explore their views on the clinical relevance of the individual activities included in the HFMSE. Then we asked caregivers to comment on the clinical relevance of possible changes of HFMSE scores over time. As functional data of individual patients were available, some of the questions were tailored according to their functional level on the HFMSE.RESULTS: Part 1: Sixty-three individuals participated in the focus groups. This included 30 caregivers, 25 patients and 8 professionals who facilitated the discussion. The caregivers provided a comparison to activities of daily living for each of the HFMSE items. Part 2: One hundred and forty-nine caregivers agreed to complete the questionnaire: in response to a general question, 72% of the caregivers would consider taking part in a clinical trial if the treatment was expected to slow down deterioration, 88% if it would stop deterioration and 97% if the treatment was expected to produce an improvement. Caregivers were informed of the first three items that their child could not achieve on the HFMSE. In response 75% indicated a willingness to take part in a clinical trial if they could achieve at least one of these abilities, 89% if they could achieve two, and 100% if they could achieve more than 2.CONCLUSIONS: Our findings support the use of the HFMSE as a key outcome measure in SMA clinical trials because the individual items and the detected changes have clear content validity and clinical meaningfulness for patients and their caregivers.

KW - spinal muscular atrophy

KW - quality of life

KW - carers

KW - clinical trials

U2 - 10.1186/s12883-017-0790-9

DO - 10.1186/s12883-017-0790-9

M3 - Article

C2 - 28231823

SN - 1471-2377

VL - 17

SP - 39

JO - BMC Neurology

JF - BMC Neurology

IS - 1

ER -

Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy

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