Decision-making for Infants with Complex Life-Threatening Conditions

摘要患有生命危险疾病的婴儿，在早期可能会在生命的头几个月内死亡，现在正在接受高度复杂的，有时是实验性的治疗，旨在治愈疾病或延长生命。然而，这些治疗有不确定的结果，婴儿有很高的死亡风险，如果他们活下来，将面临显著的发病率。患有这些疾病的婴儿经历了一个不确定的轨迹，通常涉及多重健康危机，需要父母和卫生保健提供者就治疗的类型和水平做出关键决定。决策跨越了从是否开始治疗，如何改变治疗以应对医疗危机，是否从积极的治疗护理转向以症状为重点的姑息治疗，到是否退出治疗的连续体。这项为期5年的研究将采用纵向的案例研究设计来检查接受生命维持治疗的婴儿对复杂危及生命的疾病（CLTC）的决策轨迹。三组具有特别高的死亡风险和显著的寿命发病率的cltc婴儿是本研究的重点：极早产儿（妊娠<26周），复杂心脏异常的婴儿，以及因遗传诊断需要进行造血干细胞移植（HSCT）的婴儿。将研究40个案例，每个案例将包括婴儿、父母、提供者和照顾的实际环境。在病例成员出生或诊断后，至少每月进行一次叙述性访谈和自我报告，直到死亡或入组后1年。病例内和跨病例分析将用于探索这些婴儿的决策轨迹，并检查决策如何以及何时跨越疾病轨迹。该研究将确定整个轨迹的转折点，并确定影响决策的因素，例如父母希望的参与程度；渴望更多或更少参与的父母的特征；以及医疗服务提供者在决策过程中所扮演的角色。研究结果将用于发展cltc婴儿的决策类型和疾病轨迹过渡点。这将为制定干预措施提供基础，以促进对这些婴儿的决策。公共卫生相关性：本研究将采用纵向多案例研究设计来检查患有复杂危及生命疾病（CLTC）的婴儿的决策轨迹，以及从出生或诊断到死亡或12个月对父母决策的影响。这项研究将是第一个在多个cltc婴儿群体中，对所有类型的健康相关决策（治疗开始和加强、治疗从治愈性护理转向姑息性护理、停止或退出治疗）在一年的疾病轨迹中进行前瞻性研究，包括死亡和存活的婴儿。此外，极早产儿（妊娠<26周）、患有复杂先天性心脏病（CHD）的婴儿和需要进行造血干细胞移植（HSCT）的遗传诊断的婴儿的样本将允许探索在三种最常见的儿童死亡原因中接受实验性治疗的婴儿的决策是如何相似和不同的。我们将确定决策轨迹上的过渡点，并确定对决策的影响，例如父母想要的参与程度；渴望更多或更少参与的父母的特征；以及医疗服务提供者在决策过程中所扮演的角色。目标将是确定整个轨迹的过渡点，并为制定干预措施提供基础，以促进父母对这些婴儿的决策。

DESCRIPTION (provided by applicant): Decision-Making For Infants with Complex Life-Threatening Conditions Abstract Infants with life-threatening conditions who earlier would have died in the first months of life are now receiving highly complex and sometimes experimental treatments designed to cure the condition or prolong life. However, these treatments have uncertain outcomes, the infants are at high risk for death, and if they live face a life-span of significant morbidity. Infants with these conditions experience an uncertain trajectory that typically involves multiple health crises requiring parents and health care providers to make critical decisions about the type and level of treatment. Decisions span the continuum from whether to initiate treatment, how to alter the treatment to respond to a medical crisis, whether to shift from aggressive curative care to symptom- focused palliative care, to whether to withdraw treatment. This 5-year study will use a longitudinal, case study design to examine the trajectory of decision making for infants undergoing life-sustaining treatment for complex life-threatening conditions (CLTC). Three groups of infants with CLTCs who are at particularly high risk for death and significant life-span morbidity are the focus of this study: extremely preterm infants (<26 weeks gestation), infants with complex cardiac anomalies, and infants with genetic diagnoses requiring a hematopoietic stem cell transplant (HSCT). Forty cases will be studied and each case will include the infant, parents, providers and the physical context of caregiving. Narrative interviews and self-report measures will be used with case members following birth or diagnosis and at least monthly thereafter until death or 1 year following enrollment. A within-case and cross-case analysis will be used to explore the trajectory of decision making for these infants and examine how and when decisions are across the illness trajectory. The study will identify transition points across the trajectory and identify factors influencing decision making, such as the level of involvement that parents' want; characteristics of parents who desire more or less involvement; and roles health care providers can play in decision making. The findings will be used to develop typologies of decision making and illness trajectory transition points for infants with CLTCs. These will provide a foundation for developing interventions to facilitate the decision making for these infants. PUBLIC HEALTH RELEVANCE: This study will use a longitudinal multiple case study design to examine the trajectory of decision making for infants with complex life-threatening conditions (CLTC) and influences on parents' decision making from birth or diagnosis until death or 12 months. This study will be the first study to prospectively examine the trajectory of all types of health-related decisions (treatment initiation and intensification, shifts in treatment from curative care to palliative care, withholding or withdrawing treatment) across a year-long illness trajectory, in multiple populations of infants with CLTCs, both those who die and those who survive. In addition, the sample of extremely premature infants (<26 weeks gestation), infants with complex congenital heart disease (CHD), and infants with genetic diagnoses requiring a hematopoietic stem cell transplant (HSCT) will allow for exploration of how decision making is both similar and different for infants undergoing experimental treatment for the three most common causes of pediatric death. We will identify transition points across the trajectory of decision making and identify the influences on decision making, such as the level of involvement that parents' want; characteristics of parents who desire more or less involvement; and roles health care providers play in decision making. The goal will be to identify transition points across the trajectory and provide a foundation for developing interventions to facilitate parents' decision-making for these infants.