Supportive care in children with spinal muscular atrophy type 1: Results from a french multicentric study
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
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.
Hully M; Barnerias C; Chabrol B; Vuillerot C; Sabouraud P; Cuisset J M; Cances C; Ropars J; Rivier F; Isapoff A; Desguerre I
Developmental Medicine and Child Neurology
2019
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).
<a href="http://doi.org/10.1111/dmcn.14244" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14244</a>
Analysis Of Death And Palliative Care In A Neonatal Intensive Care Unit
Neonatal Intensive Care Unit; Newborn Death; Palliative Therapy; Analgesia; Analgesic Agent; Article; Clinical Article; Descriptive Research; Female; Follow Up; Hospitalization; Human; Male; Newborn; Pain Assessment; Prematurity; Resuscitation; Retrospective Study; Sedation; Sedative Agent
Objective: To characterize cases of children admitted to the Neonatal Intensive Care Unit of a tertiary university hospital who died in the period ranging from January 01, 2012 to July 31, 2014, and who required palliative care and/or were subjected to it. Methods: A retrospective descriptive study was carried out by reviewing the medical records of these patients to collect data and to perform descriptive statistical analysis. Results: During the study period, 49 children died after at least 48 hours from the time of admission. Of those, 18% children were extremely premature infants and 77% children had malformations. Although necessary for all of the patients in this study, palliative care was provided for only 20% of patients who died. Among the 12 babies who were not resuscitated, 33% of babies were not in palliative care. The Pain and Palliative Care Unit of the institution followed only four neonates in palliative care. These patients were using many invasive devices, had high therapeutic investment, and also altered pain scale scores. Conclusions: This study exhibited a large proportion of newborn infants with serious diseases and health conditions. In a few cases the patients received palliative care, but most of them were not even discussed under palliative point of view. We hope that this study will call attention to the need to propose protocols and implement training for the best treatment of these children.
Marcola L; De Barbosa S M M; Zoboli I; Polastrini R T V; Ceccon M E J
Revista Paulista De Pediatria
2017
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).
10.1590/1984-0462/;2017;35;2;00012