A critical review of patient and parent caregiver oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in spinal muscular atrophy

被引:32
作者
Messina, Sonia [1 ,2 ]
Frongia, Anna Lia [3 ,4 ]
Antonaci, Laura [3 ,4 ]
Pera, Maria Carmela [3 ,4 ]
Coratti, Giorgia [3 ,4 ]
Pane, Marika [4 ]
Pasternak, Amy [5 ,6 ]
Civitello, Matthew [7 ]
Montes, Jacqueline [8 ,9 ]
Mayhew, Anna [10 ]
Finkel, Richard [7 ]
Muntoni, Francesco [11 ,12 ]
Mercuri, Eugenio [3 ,4 ]
机构
[1] Univ Messina, Dept Clin & Expt Med, Messina, Italy
[2] Ctr Clin Nemo, Messina, Italy
[3] Catholic Univ, Pediat Neurol Unit, Largo Gemelli 8, I-00168 Rome, Italy
[4] Fdn Policlin Univ Agostino Gemelli IRCCS, Ctr Clin Nemo, Rome, Italy
[5] Harvard Med Sch, Dept Neurol, Boston Childrens Hosp, Boston, MA 02115 USA
[6] Harvard Med Sch, Dept Phys & Occupat Therapy, Boston Childrens Hosp, Boston, MA 02115 USA
[7] Univ Cent Florida, Nemours Childrens Hosp, Coll Med, Orlando, FL 32816 USA
[8] Columbia Univ, Irving Med Ctr, Dept Rehabil & Regenerat Med, New York, NY USA
[9] Columbia Univ, Irving Med Ctr, Dept Neurol, New York, NY USA
[10] Newcastle Univ, Inst Med Genet, Newcastle Upon Tyne, Tyne & Wear, England
[11] UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England
[12] Great Ormond St Hosp Trust, NIHR Great Ormond St Hosp Biomed Res Ctr, UCL Great Ormond St Inst Child Hlth, London, England
关键词
Spinal muscular atrophy; Quality of life; Activity of daily living; Health related quality of life; Care burden; Personalized medicine; NEUROMUSCULAR DISORDERS; ACTIVITY LIMITATIONS; OUTCOME MEASURES; FUNCTIONAL ASSESSMENT; RASCH ANALYSIS; SHAM CONTROL; CHILDREN; ADOLESCENTS; QUESTIONNAIRE; RELIABILITY;
D O I
10.1016/j.nmd.2019.10.001
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
The positive outcome of different therapeutic approaches for spinal muscular atrophy (SMA) in clinical trials and in clinical practice have highlighted the need to establish if functional changes are associated with possible changes of patient health-related quality of life or have an effect on activities of daily living and caregiver burden. The aim of this paper is to provide a critical review of the tools previously or currently used to measure quality of life, activity of daily living, and caregiver burden in SMA. We identified 36 measures. Only 6 tools were specifically developed for SMA while the others had been used and at least partially validated in wider groups of neuromuscular disorders including SMA. Twelve of the 36 focused on health-related quality of life, 5 on activities of daily living and 9 on caregiver burden. Ten included a combination of items. The review provides a roadmap of the different tools indicating their suitability for different SMA types or age groups. Scales assessing activities of daily living and care burden can provide patients and carers perspective on functional changes over time that should be added to the observer rated scales used in clinic. (C) 2019 Elsevier B.V. All rights reserved.
引用
收藏
页码:940 / 950
页数:11
相关论文
共 87 条
[1]  
Aartsma-Rus A, 2017, LANCET NEUROL, V16, P341, DOI 10.1016/S1474-4422(17)30041-8
[2]  
Abresch Richard T, 2002, Am J Hosp Palliat Care, V19, P39, DOI 10.1177/104990910201900109
[3]   Caregiver burden-A critical discussion [J].
Bastawrous, Marina .
INTERNATIONAL JOURNAL OF NURSING STUDIES, 2013, 50 (03) :431-441
[4]   How robust is ACTIVLIM for the follow-up of activity limitations in patients with neuromuscular diseases? [J].
Batcho, Charles Sebiyo ;
Van den Bergh, Peter Y. K. ;
Van Damme, Philip ;
Roy, Anna J. ;
Thonnard, Jean-Louis ;
Penta, Massimo .
NEUROMUSCULAR DISORDERS, 2016, 26 (03) :211-220
[5]   Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial [J].
Bertini, Enrico ;
Dessaud, Eric ;
Mercuri, Eugenio ;
Muntoni, Francesco ;
Kirschner, Janbernd ;
Reid, Carol ;
Lusakowska, Anna ;
Comi, Giacomo P. ;
Cuisset, Jean-Marie ;
Abitbol, Jean-Louis ;
Scherrer, Bruno ;
Ducray, Patricia Sanwald ;
Buchbjerg, Jeppe ;
Vianna, Eduardo ;
van der Pol, W. Ludo ;
Vuillerot, Carole ;
Blaettler, Thomas ;
Fontoura, Paulo .
LANCET NEUROLOGY, 2017, 16 (07) :513-522
[6]  
Boyer F., 2006, Annales de Readaptation et de Medecine Physique, V49, P16, DOI 10.1016/j.annrmp.2005.08.001
[7]   Hereditary neuromuscular disease and multicomposite subjective health status: feasibility, internal consistency and test-retest reliability in the French version of the Nottingham Health Profile, the ISPN [J].
Boyer, F ;
Novella, JL ;
Bertaud, S ;
Delmer, F ;
Vesselle, B ;
Etienne, JC .
CLINICAL REHABILITATION, 2005, 19 (06) :644-653
[8]   Cognitive and motor impairments predict functional declines in patients with vascular dementia [J].
Boyle, PA ;
Cohen, RA ;
Paul, R ;
Moser, D ;
Gordon, N .
INTERNATIONAL JOURNAL OF GERIATRIC PSYCHIATRY, 2002, 17 (02) :164-169
[9]  
Broe GA, 1998, INT J GERIATR PSYCH, V13, P667, DOI 10.1002/(SICI)1099-1166(1998100)13:10<667::AID-GPS839>3.0.CO
[10]  
2-G