1
40
2
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Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
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August 2019 List
Text
A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text.
Citation List Month
August 2019 List
URL Address
<a href="http://doi.org/10.1111/dmcn.14244" target="_blank" rel="noreferrer noopener">http://doi.org/10.1111/dmcn.14244</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
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Supportive care in children with spinal muscular atrophy type 1: Results from a french multicentric study
Publisher
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Developmental Medicine and Child Neurology
Date
A point or period of time associated with an event in the lifecycle of the resource
2019
Subject
The topic of the resource
death; child; female; human; major clinical study; male; palliative therapy; controlled study; conference abstract; prospective study; caregiver; clinical research; drug therapy; France; gastrostomy; history; intensive care unit; new drug; noninvasive ventilation; nusinersen; nutrition supplement; physician; sedation; sedative agent; standardization; Werdnig Hoffmann disease
Creator
An entity primarily responsible for making the resource
Hully M; Barnerias C; Chabrol B; Vuillerot C; Sabouraud P; Cuisset J M; Cances C; Ropars J; Rivier F; Isapoff A; Desguerre I
Description
An account of the resource
Introduction: This National Hospital Clinical Research Program (PHRC) was conducted in France between 2012 and 2016 to depict palliative practices in spinal muscular atrophy type 1 (SMA-1). New drugs (Nusinersen) have been developed and may modify its natural history. We thus present data about supportive care for patients included in that PHRC, comparing them to patients not included concomitantly. Patients and Methods: Supportive care data (enteral nutrition, non-invasive ventilation [NIV], sedation), age and place of death were collected prospectively from a specific health-book and a survey about conditions of death for the patients included in the PHRC and retrospectively by questioning physicians of the French Pediatric Neuromuscular Network for the other patients, some of them receiving Nusinersen. Result(s): In 18 centres, 38 patients were included in the PHRC, 43 were not; including 7 receiving Nusinersen. Mean age at diagnosis was 3.9 months (SD 2.4). 77 patients died at 7.5 months (SD 4,96), 32% at home, 8% in an intensive care unit. 85% patients received enteral nutrition, some through a gastrostomy (8%). 16% had a NIV. 70% received sedative treatment. No statistical difference was found between the two groups. However only all 3 patients (4%) receiving Nusinersen had both a gastrostomy and a NIV, without any sedation. Conclusion(s): Our data confirm that palliative care is essential in the management of ASI-1 patients, that is so far still a fatal disorder. Our data suggest that Nusinersen treatment was accompanied by more invasive supportive care, claiming for a standardization of practices in the children and caregivers' best-interest.
Identifier
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<a href="http://doi.org/10.1111/dmcn.14244" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14244</a>
Rights
Information about rights held in and over the resource
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
2019
August 2019 List
Barnerias C
Cances C
Caregiver
Chabrol B
Child
Clinical Research
conference abstract
Controlled Study
Cuisset J M
Death
Desguerre I
Developmental Medicine and Child Neurology
Drug Therapy
Female
France
Gastrostomy
History
Hully M
Human
Intensive Care Unit
Isapoff A
Major Clinical Study
Male
new drug
Noninvasive Ventilation
nusinersen
nutrition supplement
Palliative Therapy
Physician
Prospective Study
Rivier F
Ropars J
Sabouraud P
Sedation
Sedative Agent
standardization
Vuillerot C
Werdnig Hoffmann disease
-
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
January 2018 List
Text
A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text.
Citation List Month
January 2018 List
URL Address
<a href="http://doi.org/10.1016/j.ejpn.2017.04.1310" target="_blank" rel="noreferrer">http://doi.org/10.1016/j.ejpn.2017.04.1310</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
Palliative care in children with spinal muscular atrophy type 1: How do they die? Results from a French multicentric study (National Hospital clinical Research Program)
Publisher
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European Journal Of Paediatric Neurology
Date
A point or period of time associated with an event in the lifecycle of the resource
2017
Subject
The topic of the resource
clinical research; palliative therapy; Werdnig Hoffmann disease; 50-48-6 (amitriptyline); 52-26-6 (morphine); 57-27-2 (morphine); 549-18-8 (amitriptyline); Amitriptyline; benzodiazepine derivative; Child; Clinical Article; clinical practice; Diagnosis; Drug Therapy; Female; follow up; Human; infant; Intensive care unit; Interview; Male; Morphine; multicenter study; nasogastric tube; Noninvasive Ventilation; psychologist; quantitative analysis; Resuscitation; time of death
Creator
An entity primarily responsible for making the resource
Hully M; Barnerias C; Vanesse S; Viallard ML; Desguerre I
Description
An account of the resource
Objective: 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 conducted to depict palliative practices in that fatal disease, in which death up to now occurs few weeks or months after the diagnosis. We here report data about the conditions of death for the patients included. Methods: In this French multicentric study, patients were included from june 2012 to june 2016. Parents and physicians filled in a specific health book during the follow up, the physician in charge filled in a survey concerning the patient's management over the last 48 hours before death, then a semidirected interview of the parents was conducted by a trained psychologist 6 to 18 months after the child's death. We here report the quantitative analysis of data obtained from the survey about patient's management around death. Results: 38 patients were included in the study (17 centres), data were available for 36 dead patients. Median age at inclusion was 3 months (0,6-10,4), death occurred at a median 5,5 month of age (1,5-16,4), i.e a median follow-up of 2 months (0,2-12,8). 39% of patients died at home, 6% in an intensive care unit. At the time of death, patients received morphine (56%), benzodiazepines (39%), amitriptyline (39%). Treatments were given through a nasogastric tube (83%), and oxygenotherapy was delivered (76%). 6% patients received noninvasive ventilation at the time of death. No resuscitation recommendations had been prepared for most patients (97%), written in 85%, after a multidisciplinary meeting in most cases (79%). Conclusion: Our data confirm current knowledge about natural outcome in SMA 1, death occurring very soon after the diagnosis, claiming for an effective palliative management of the patients, including the involvement of parents in medical care at home.
Identifier
An unambiguous reference to the resource within a given context
<a href="http://doi.org/10.1016/j.ejpn.2017.04.1310" target="_blank" rel="noreferrer">10.1016/j.ejpn.2017.04.1310</a>
Rights
Information about rights held in and over the resource
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
2017
50-48-6 (amitriptyline)
52-26-6 (morphine)
549-18-8 (amitriptyline)
57-27-2 (morphine)
Amitriptyline
Barnerias C
benzodiazepine derivative
Child
Clinical Article
Clinical Practice
Clinical Research
Desguerre I
Diagnosis
Drug Therapy
European Journal of Paediatric Neurology
Female
Follow Up
Hully M
Human
Infant
Intensive Care Unit
Interview
January 2018 List
Male
Morphine
Multicenter Study
nasogastric tube
Noninvasive Ventilation
Palliative Therapy
Psychologist
quantitative analysis
Resuscitation
time of death
Vanesse S
Viallard ML
Werdnig Hoffmann disease