Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' Reports

Frontiers in Pediatrics
Marie HullyIsabelle Desguerre

Abstract

Spinal muscular atrophy type 1 (SMA-1) is a severe neurodegenerative disorder, which in the absence of curative treatment, leads to death before 1 year of age in most cases. Caring for these short-lived and severely impaired infants requires palliative management. New drugs (nusinersen) have recently been developed that may modify SMA-1 natural history and thus raise ethical concerns about the appropriate level of care for patients. The national Hospital Clinical Research Program (PHRC) called "Assessment of clinical practices of palliative care in children with Spinal Muscular Atrophy Type 1 (SMA-1)" was a multicenter prospective study conducted in France between 2012 and 2016 to report palliative practices in SMA-1 in real life through prospective caregivers' reports about their infants' management. Thirty-nine patients were included in the prospective PHRC (17 centers). We also studied retrospective data regarding management of 43 other SMA-1 patients (18 centers) over the same period, including seven treated with nusinersen, in comparison with historical data from 222 patients previously published over two periods of 10 years (1989-2009). In the latest period studied, median age at diagnosis was 3 months [0.6-10.4]. Seventy...Continue Reading

Associated Clinical Trials

References

Sep 1, 2007·Journal of Child Neurology·Ching H WangUNKNOWN Participants of the International Conference on SMA Standard of Care
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Jun 24, 2008·Lancet·Mitchell R Lunn, Ching H Wang
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Oct 8, 2013·Journal of Child Neurology·Rebecca Hurst DavisKathryn J Swoboda
Feb 26, 2014·Palliative & Supportive Care·Alberto García-SalidoRicardo Martino-Alba
Mar 19, 2014·Archives de pédiatrie : organe officiel de la Sociéte française de pédiatrie·C BarnériasI Desguerre
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Oct 30, 2016·Neuromuscular Disorders : NMD·Roberto De SanctisEugenio Mercuri
Dec 28, 2016·Annals of Neurology·Michelle A FarrarMatthew C Kiernan
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Dec 29, 2018·Pediatric Neurology·Michael LeeMichael L McManus
May 2, 2019·Neuromuscular Disorders : NMD·Juliette RoparsIsabelle Desguerre

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Citations

Oct 6, 2020·Frontiers in Pediatrics·Uros Krivec, Serena Caggiano
Apr 7, 2021·Cellular and Molecular Life Sciences : CMLS·Rachel JamesThomas H Gillingwater

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Methods Mentioned

BETA
SMA
antisense oligonucleotides
sedation

Clinical Trials Mentioned

NCT01862042

Software Mentioned

NVivo
Word
R
Excel

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