Clinical trials or palliative care: Do we have to choose?

Purpose. - Early phases clinical trials for children with neurological diseases have recently spread out. For most of these diseases with neurological impairment, no curative therapy is currently available, thus including the patient in a clinical trial appears as the only alternative. However, uncertainty regarding the disease evolution remains and these patients will face neurological decline and disabilities. The purpose of this study was to analyse whether a paediatric palliative approach is relevant for these patients. Material and methods. - We have analysed retrospectively the resorting to palliative care teams for eight children included in a phase I-II clinical trial designed for metachromatic leukodystrophy. Results. - All of them were facing neurological decline and disabilities. Only four children had met a palliative care team: the team had been called either when they got out of the trial (two patients) or when the clinical trial could be continued closer to their home (two patients). The investigation centre not only handled the trial but was also taking care of all the symptomatic treatments. In this context, it seemed difficult for the investigator to address issues around degradation and death, making the use of a palliative care team pivotal to discuss these questions. Conclusion. - It seems to us possible and crucial to include a palliative approach in patients with neurodegenerative diseases even if included in a clinical trial, whose outcome remains uncertain. Further study will need to evaluate the feasibility, the benefit for patients and possible risks of a precocious recourse to palliative care teams, probably as soon as the inclusion in a clinical trial is considered. (C) 2018 Elsevier Masson SAS. All rights reserved.