未经批准:对Dravet综合征(DS)患者的适当管理具有挑战性,考虑到症状的严重程度以及疾病对患者和护理人员的负担。本研究旨在确定,通过定性的方法论和德尔菲共识驱动的过程,一套DS管理建议,以指导临床医生评估DS患者的临床状况和生活质量(QoL),特别关注患者和护理人员报告的结果(PRO)。
未经评估:本研究分五个阶段进行,由包括儿科神经学家在内的多学科科学委员会(SC)领导,癫痫学家,神经心理学家,一个癫痫护士,和DS患者倡导者的成员。在第1和第2阶段,护理人员和SC准备并回答了与患者QoL相关的问卷。在阶段3,SC产生,基于这些答案和焦点小组讨论,一份70项的德尔菲问卷,以九点李克特量表涵盖六个主题类别。在第四阶段,32名小组成员,来自不同的西班牙机构,具有多学科背景,回答了问卷。如果≥80%和67-79%的小组成员,则获得共识并定义为强或中等,分别,对报表评分≥7。第5阶段包括手稿的准备。
UNASSIGNED:小组成员同意总共69个项目(98.6%),54(77.14%),和15(21.43%)具有强烈和中等的共识,分别。专家建议包括需要频繁评估患者和护理人员的QoL参数。专家们一致认为,应通过涵盖不同领域的具体问卷来评估生活质量。同样,结果显示,关于与神经发育相关的几个临床参数的定期评估的共识,注意,行为,其他合并症,癫痫突然意外死亡(SUDEP)。还就文书达成了共识,具体参数,和护理人员在DS患者常规临床管理中的教育。
未经评估:这一共识产生了一套临床和QoL参数评估的建议,包括PROs,与QoL的一般评估有关,神经发育,注意,行为,影响QoL的其他合并症,SUDEP,护理人员/亲属和DS患者的QoL。
UNASSIGNED: The appropriate management of patients with Dravet Syndrome (DS) is challenging, given the severity of symptoms and the burden of the disease for patients and caregivers. This study aimed to identify, through a qualitative methodology and a Delphi consensus-driven process, a set of recommendations for the management of DS to guide clinicians in the assessment of the clinical condition and quality of life (QoL) of DS patients, with a special focus on patient- and caregiver-reported outcomes (PROs).
UNASSIGNED: This study was conducted in five phases, led by a multidisciplinary scientific committee (SC) including pediatric neurologists, epileptologists, a neuropsychologist, an epilepsy nurse, and members of DS patient advocates. In phases 1 and 2, a questionnaire related to patients\' QoL was prepared and answered by caregivers and the SC. In phase 3, the SC generated, based on these answers and on a focus group discussion, a 70-item Delphi questionnaire, covering six topic categories on a nine-point Likert scale. In phase 4, 32 panelists, from different Spanish institutions and with a multidisciplinary background, answered the questionnaire. Consensus was obtained and defined as strong or moderate if ≥80% and 67-79% of panelists, respectively, rated the statement with ≥7. Phase 5 consisted of the preparation of the manuscript.
UNASSIGNED: The panelists agreed on a total of 69 items (98.6%), 54 (77.14%), and 15 (21.43%) with strong and moderate consensus, respectively. The experts\' recommendations included the need for frequent assessment of patient and caregivers QoL parameters. The experts agreed that QoL should be assessed through specific questionnaires covering different domains. Likewise, the results showed consensus regarding the regular evaluation of several clinical parameters related to neurodevelopment, attention, behavior, other comorbidities, and sudden unexpected death in epilepsy (SUDEP). A consensus was also reached on the instruments, specific parameters, and caregivers\' education in the routine clinical management of patients with DS.
UNASSIGNED: This consensus resulted in a set of recommendations for the assessment of clinical and QoL parameters, including PROs, related to the general evaluation of QoL, neurodevelopment, attention, behavior, other comorbidities affecting QoL, SUDEP, and QoL of caregivers/relatives and patients with DS.