Family Decision Making in Pediatric Bone Marrow Transplant

描述（由申请人提供）：与健康同胞供体进行异基因骨髓移植（BMT）是一种潜在的治愈性，尽管危及生命，治疗某些高危儿科疾病。进行移植的决定是复杂的。这种手术可以挽救患病儿童的生命，但健康的兄弟姐妹却要承受疼痛、受伤、感染等身体风险，还要承担起拯救患病兄弟姐妹的责任的心理负担。孩子们通常是未成年人，所以父母必须权衡每个孩子的风险和利益，使他们处于矛盾的位置。如果征求儿童的意见，他们不太可能拒绝，因为患病儿童极有可能在不治疗的情况下死亡。令人惊讶的是，人们对家庭如何处理这一重大决定以及他们所感知和考虑的风险和利益知之甚少。移植界和法院普遍认为，捐赠者的兄弟姐妹从移植中受益。然而，初步的儿科研究表明，患有癌症的儿童的兄弟姐妹有适应问题的风险。此外，四项回顾性研究表明，少数捐助者感到被排除在决策过程之外。这四个网站，纵向试点研究的目的是18-22个家庭的描述，使用扎根理论技术，父母/s，患者，捐助者和非捐助者的兄弟姐妹在决策过程中的经验，注意他们的利益和风险的看法。我们将发展一个实质性的理论与治疗决策与家庭作为我们的分析单位。具体目标是：（1）使用扎根理论技术，在考虑为癌症儿童进行异基因骨髓移植的家庭中，建立一个基于数据的家庭决策过程的实质性理论;（2）确定从儿童与未成年同胞供体进行异基因骨髓移植的家庭中积累和收集数据的可行性。目的是描述家庭决策过程，并确定可能导致家庭成员不良结果的因素。这些数据将有助于指导干预措施的设计，以改善移植期间所有家庭成员的结果。此外，本研究将提供初步数据，以告知使用同胞供体的伦理理由和此类供体的监管框架。总之，该项目将为假设检验大型多中心研究提供坚实的基础，然后将其转化为干预研究。公共卫生相关性：有关异基因儿科骨髓移植的家庭决策信息将适用于并有助于设计干预措施，以改善其他儿科危及生命疾病的家庭决策结果，并可能在其他高压力的家庭情况下。虽然幸运的是，危及生命的儿科疾病相对罕见，但它们对整个大家庭甚至社区的影响是巨大的。

DESCRIPTION (provided by applicant): Allogeneic bone marrow transplantation (BMT) with a healthy sibling donor is a potentially curative, though life-threatening, treatment for certain high risk pediatric diseases. The decision to proceed with transplant is complicated. The procedure may save the life of the ill child, but subjects the healthy sibling to the physical risks of pain, injury, infection and the psychological burden of feeling responsible for saving the ill sibling. The children are typically minors, so the parents must weigh the risks and benefits for each child , placing them in a conflicted position. If the children are consulted, they are unlikely to refuse given the grave possibility that the ill child may die without treatment. Surprisingly, little is known about how families approach this momentous decision and the risks and benefits they perceive and consider. It is widely assumed by the transplant community and the courts that donor siblings benefit from transplant. However, preliminary pediatric research demonstrates that siblings of children with cancer are at risk for adjustment problems. Further, four retrospective studies suggest that minor donors feel excluded from the decision-making process. The purpose of this four-site, longitudinal pilot study of 18-22 families is to describe, using grounded theory techniques, the experiences of parent/s, patients, donor and non-donor siblings during the decision-making process with attention to their perceptions of benefits and risks. We will develop a substantive theory related to treatment decision- making with the family as our unit of analysis. The specific aims are (1) to develop a data-based, substantive theory of the family decision-making process in families considering an allogeneic bone marrow transplant for a child with cancer using grounded theory techniques; (2) to determine the feasibility of accruing and collecting data from families in which a child is undergoing an allogeneic bone marrow transplant with a minor sibling donor. The goal is to describe the family decision-making process and also to identify factors that may lead to poor outcomes for family members. These data will help guide the design of interventions to improve outcomes for all family members during transplantation. In addition, this study will provide preliminary data to inform the ethical justification for using sibling donors and the regulatory framework for such donation. In sum, this project will provide the firm foundation for a hypothesis- testing large multi-site study that will then translate into an intervention study. PUBLIC HEALTH RELEVANCE: Information on family decision-making about an allogeneic pediatric bone marrow transplant will be applicable to and helpful in designing interventions to improve the outcomes of family decision-making in other pediatric life threatening diseases, and potentially in other high stress family situations. Although, fortunately, life-threatening pediatric diseases are relatively rare, their impact on entire extended families and even communities is immense.