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. 2024 Feb 6;13(4):921.
doi: 10.3390/jcm13040921.

Use of the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) in Spinal Muscular Atrophy

Laurey Brown  1   2 , Katie Hoffman  1   2 , Chiara Corbo-Galli  3 , Siyuan Dong  4 , Katelyn Zumpf  4 , Christa Weigel  1 , Colleen Blomgren  1 , Hannah Munson  2 , Jessa Bidwell  2 , Vamshi Rao  2   5 , Nancy L Kuntz  2   5 , Abigail Schwaede  2 , Kristin J Krosschell  5   6
Affiliations

Use of the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) in Spinal Muscular Atrophy

Laurey Brown et al. J Clin Med. .

Abstract

Background: Spinal muscular atrophy (SMA) has a remarkable impact on function and participation. Subsequently, the caregivers of individuals with SMA are impacted as well. Providers and the SMA community should be aware of the presence of and likely expectations for the existence of caregiver burden. Methods: The Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) quantifies caregivers' perceptions of function and quality of life pertaining to time, finance and emotion. Analyses were conducted among SMA types and ambulatory and ventilatory status. Participants with SMA had varying ranges of function and were on pharmaceutical treatment. Total ACEND score, longitudinal change in total ACEND score, total quality of life (QOL) score, change in total QOL score and subdomains for QOL, including time, emotion and finance, were all explored. Results: Overall, the ACEND demonstrated discriminant validity and some observed trends. Total ACEND scores improved for caregivers of those with SMA 2, remained stable longitudinally for caregivers of those with SMA 1 and 3 and were not influenced by ventilation status. The caregivers of individuals with SMA 1 had the lowest total quality of life (QOL) score, as did the caregivers of non-ambulatory individuals and those requiring assisted ventilation. Longitudinally, there were no changes in total QOL between caregivers of individuals with different SMA types or ambulatory or ventilation status. There were some differences in emotional needs, but no differences in financial impact between the caregivers of individuals with different types of SMA or ambulatory and ventilatory status. Conclusions: With this information enlightening the presence of caregiver burden and expected changes in burden with pharmaceutical treatment, providers, third party payors and the SMA community at large can better assist, equip and empower those providing the necessary assistance to enable the lives of those with SMA.

Keywords: ACEND; caregiver burden; neuromuscular; pediatric; quality of life; spinal muscular atrophy.

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Conflict of interest statement

L.B. has received personal compensation as a speaker for Biogen and Sarepta, unrelated to this study. K.H. has received personal compensation as an Advisory Board member of Genentech-Roche, outside of the submitted work. K.J.K. has received personal consulting fees for advising, consulting or speaking from Biogen, Genentech-Roche, ASPA Therapeutics and Cure SMA and her institution receives subcontracted support from Robert & Ann H. Lurie Children’s Hospital of Chicago for Biogen, NIH and Scholar Rock clinical trials. All were outside of the submitted work. N.L.K. serves on medical Advisory Boards for Argenx, Biogen, Novartis, Roche and Sarepta. Her institution receives research funds from Biogen, Novartis, Roche and Sarepta. V.K.R. has received personal fees for advising, consulting or speaking from Avexis, Biogen, Genetech-Roche, Scholar Rock, PTC therapeutics, NSPharma, Regenxbio, Sarepta therapeutics, France Foundation, CureSMA and MDa, outside of the submitted work. C.C.-G., S.D., K.Z., C.W., C.B., H.M. and T.B. have nothing to disclose. The funders had no role in the design of the study, the collection, analyses or interpretation of data, the writing of the manuscript or the decision to publish the results.

Figures

Figure 1
Figure 1
Histograms of outcomes by SMA type according to the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND). hashed line: median for type.
Figure 2
Figure 2
Histograms of outcomes by ambulatory status according to the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND). hashed line: median for type.
Figure 3
Figure 3
Histograms of outcomes by ventilation status according to the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND).
See this image and copyright information in PMC

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