Pediatric Oncology Branch Behavioral Science Core

取得的进展：1)由行为健康中心开发和管理的患者报告的结果测量有助于记录疼痛强度和疼痛干扰的改善，这有助于FDA批准selumetinib用于NF1和不能手术的丛状神经纤维瘤儿童。2)为COVID-19大流行期间与慢性疼痛患者合作的精神卫生服务提供者及时发布建议。3)为儿童癌症中心开发和出版实施工具包；该工具包包括一个机构评估工具，用于癌症中心评估他们目前对15个儿童心理社会肿瘤学护理标准和每个标准的附带指南的实施情况。4)出版，《戈伯特羽毛的礼物》（2020年2月，想象出版社），其中包括父母，祖父母，兄弟姐妹，朋友和老师的指南，以帮助解决儿童死亡问题。1)对野生型GIST、琥珀酸脱氢酶缺乏症伴嗜铬细胞瘤、副神经节瘤、1型神经纤维瘤病、2型多发性内分泌瘤、Rasopathies等罕见肿瘤患者进行纵向研究。（pi: Widemann, Yohe, Gross, god）。2)收集受试者报告的症状数据，为正在进行的治疗白血病的CART研究提供神经认知评估和神经毒性评估（PI: Nirali Shah）。3)开展抗fgfr4嵌合抗原受体T细胞治疗复发性或难治性横纹肌肉瘤儿童和青年患者的新一期剂量递增研究的神经毒性评估（PI: Javed Kahn）。4)评估RUNX1型FPD儿童和成人的纵向和自然历史研究的心理社会和行为结果（PI: Paul Liu）。5)儿童和成人肾上腺皮质癌患者的心理社会和行为结果的纵向和自然历史研究（PI: Jaydi Del Rivero）。6)评估丝裂原活化蛋白激酶（MEK1/2）抑制剂Selumetinib （AZD6244硫酸氢）治疗1型神经纤维瘤病（NF1）相关胃肠道瘘瘤（GIST）患者的生活质量（PI: Brigitte Widemann）的II期试验。7)确定患者和家长对使用下一代测序（NGS）诊断和管理癌症的态度，并返回结果和偶然发现（PI: Rosie Kaplan）。8)在一项注册II期临床试验中，收集和分析selumetinib治疗NF1和不能手术的丛状神经纤维瘤儿童的疼痛、身体功能和生活质量的患者报告结局（PRO）数据，这有助于FDA于2020年4月批准selumetinib （koselugo）。在一项II期临床试验中评估selumetinib在NF1和不能手术的丛状神经纤维瘤儿童中的药物依从性。10)研究接受干细胞移植的儿童、青少年和成人镰状细胞性贫血患者的神经认知功能、疼痛和生活质量（pi: Courtney Fitzhugh, John Tisdale）。11)慢性GVHD儿童临床因素的自然历史研究中儿童心理社会特征的评估[j]。12)计划一项神经认知评估，以评估甲脲素在转移性实体瘤患者中的神经毒性，研究其安全性、耐受性、药代动力学、生物学和临床活性（PI: Udo Rudloff）。13)计划对因DOCK8缺陷而接受移植的兄弟姐妹进行移植顺序的定性访谈（PI: Nirali Shah）。15)规划方法和具体措施，以评估使用Apple iPhone和Apple Watch的移动传感器技术的可行性，以评估癌症患者的一般症状（如疲劳、疼痛）和认知功能（如认知灵活性、反应时间）（PI: James Gulley）核心发起的调查：1)建立和传播在COVID-19大流行期间与慢性疼痛患者合作的建议。2)评估父母在孩子死亡前后的社会心理支持需求。3)评估全球范围内纳入姑息治疗和丧亲护理的障碍和促进因素。4)评估强化正念干预对高危癌症儿童及其照顾者的可行性和有效性。5)赞助多机构研究，以开发和验证新的或适应的措施来评估生活质量、疼痛、cGVHD症状和痛苦。6)为患者/护理人员开发电子求救屏幕以及提供者总结报告，以促进转诊。7)通过两阶段的研究方案，接受并与FDA的药物开发工具资格认证项目合作，修改和验证目前患者报告的疼痛强度和疼痛干扰的结果测量，用于NF1临床试验。8)开展一项随机对照试验，评估一种新的体育活动干预措施，以改善接受脑肿瘤放射治疗的青少年的认知晚期效应。9)制定评分量表来评估NF1和丛状或皮肤神经纤维瘤患者的毁容和外观问题。10)评估干预措施以增强干细胞供体准备。11)评估支持丧亲父母的干预措施。12)开发医生讨论尸检的沟通工具。13)探索在癌症/NF1发展过程中如何/何时干预夫妻婚姻/伙伴关系。14)评估儿童时期截肢患者的截肢准备方法。15)确定voice My Choices在患有癌症和其他慢性疾病的AYA患者中的可行性、效用和影响。16)评估儿童造血干细胞供体评估和监测随访的医疗和社会心理实践指南。17)确定将儿科姑息治疗概念整合到现有的儿科肿瘤学项目中。18)评估将丧亲心理社会标准纳入儿科肿瘤学项目。19)开展患者参与调查，评估患有NF的成人和儿童以及这些儿童的父母的想法，以及他们对社会心理临床试验的参与情况，以更好地设计对患者有意义的研究。20)确定兄弟姐妹儿童HSC供体的健康相关生活质量（HRQoL），并将其HRQoL与(1)来自同一家庭的健康非供体兄弟姐妹，(2)患有类似疾病但接受替代治疗（例如，非亲属脐带血移植）的儿童的兄弟姐妹，以及(3)健康年龄、性别和种族/民族匹配对照的HRQoL进行比较。21)开发一种棋盘游戏，为儿童进行CAR - T治疗做准备。22)为电子游戏开发内容，为儿童进行CAR - T治疗做准备。23)为治疗游戏ShopTalk开发可互换卡片，帮助儿童为BMT做准备。24)开展了一项全国性研究，评估COVID-19对儿科姑息治疗提供者和项目的影响。25)对患有NF1、NF2和神经鞘瘤病的成年人进行国际匿名在线调查，以更好地了解他们的社会情感问题，并帮助计划以患者为中心的社会心理试验

Advancements made: 1) Patient-reported outcome measures developed and administered by the Behavioral Health Core were instrumental in documenting improvements in pain intensity and pain interference, which contributed to the FDA approval of selumetinib for children with NF1 and inoperable plexiform neurofibromas. 2) Publication of timely recommendations for mental health providers working with chronic pain patients during the COVID-19 pandemic. 3) Development and publication of an implementation toolkit for pediatric cancer centers: The toolkit includes an Institutional Assessment Tool for cancer centers to assess their current implementation of each of the 15 pediatric psychosocial oncology standards of care and accompanying guidelines for each Standard. 4) Publication of, The Gift of Gerbert's Feather (February 2020, Magination Press), which includes a guide for parents, grandparents, siblings, friends and teachers to help address death to children. Projects: Support for: 1)The longitudinal study of patients with rare tumors including Wild Type GIST, Pheochromocytoma and Paraganglioma associated with Succinate Dehydrogenase Deficiency, Neurofibromatosis type 1, multiple endocrine neoplasia type 2, and Rasopathies. (PIs: Widemann, Yohe, Gross, Glod). 2) Collecting subject-reported symptom data and providing neurocognitive assessment and evaluation of neurotoxicity for ongoing CART studies designed to treat leukemias (PI: Nirali Shah). 3) Developing the neurotoxicity assessment for a new Phase I Dose Escalation Study of Anti-FGFR4 Chimeric Antigen Receptor T Cells in Children and Young Adults with Recurrent or Refractory Rhabdomyosarcoma (PI: Javed Kahn). 4) Assessing psychosocial and behavioral outcomes in a longitudinal and natural history study of children and adults with RUNX1 FPD (PI: Paul Liu). 5) Assessing psychosocial and behavioral outcomes in longitudinal and natural history study of children and adults with Adrenal Cortical Cancer (PI: Jaydi Del Rivero). 6) Assessing quality of life outcomes in a Phase II trial of the Mitogen Activated Protein Kinase Kinase (MEK1/2) inhibitor Selumetinib (AZD6244 hydrogen sulfate) in Patients with Neurofibromatosis Type 1 (NF1) related Gastrointestinal Stomal Tumors (GIST) (PI: Brigitte Widemann). 7) Identifying patient and parent attitudes toward the use of next generation sequencing (NGS) for diagnosing and managing cancer and the return of results and incidental findings (PI: Rosie Kaplan). 8) Collecting and analyzing patient-reported outcome (PRO) data of pain, physical function, and quality of life in a registration phase II clinical trial of selumetinib in children with NF1 and inoperable plexiform neurofibromas, which contributed to FDA approval of selumetib (koselugo) in April 2020. 9) Assessing medication adherence in a phase II clinical trial of selumetinib in children with NF1 and inoperable plexiform neurofibromas. 10) Investigating neurocognitive functions, pain, and quality of life in several protocols of children, adolescents, and adults with sickle cell anemia undergoing stem cell transplant (PIs: Courtney Fitzhugh, John Tisdale). 11) Assessing psychosocial characteristics of children in a natural history study of clinical factors in children with chronic GVHD (PI: Steven Pavletic). 12) Planning a neurocognitive evaluation to assess for neurotoxicities on a first-in-human phase I trial to investigate the safety, tolerability, pharmacokinetics, biological and clinical activity of metarrestin in subjects with metastatic solid tumors (PI: Udo Rudloff). 13) Planning to conduct qualitative interviews on order of transplant in siblings undergoing transplant for DOCK8 Deficiency (PI: Nirali Shah). 15) Planning the methodology and specific measures to evaluate the feasibility of mobile sensor technologies using an Apple iPhone and Apple Watch to assess general symptomology (e.g., fatigue, pain) and cognitive function (e.g., cognitive flexibility, reaction time) of cancer patients (PI: James Gulley) Core-Initiated Investigations: 1) Establishing and disseminating recommendations on working with patients with chronic pain during the COVID-19 pandemic. 2) Evaluating the psychosocial support needs of parents before and after the death of their child. 3) Assessing the barriers and facilitators to incorporating palliative and bereavement care worldwide. 4) Evaluating the feasibility and efficacy of an enhanced mindfulness intervention for children with high risk cancer and their caregivers. 5) Sponsoring multi-institutional studies to develop and validate new or adapted measures to assess QOL, pain, cGVHD symptoms, and distress. 6) Developing an electronic distress screen for patients/caregivers alongside a provider summary report to facilitate referrals. 7) Accepted into and working with the FDA's Drug Development Tool Qualification Program to modify and validate current patient-reported outcome measures of pain intensity and pain interference for use in NF1 clinical trials through a two-phase research protocol. 8) Conducting a randomized controlled trial to evaluate a novel physical activity intervention to improve cognitive late effects in youth who received radiation for a brain tumor. 9) Developing rating scales to assess disfigurement and appearance concerns in individuals with NF1 and plexiform or cutaneous neurofibromas. 10) Assessing interventions to enhance stem cell donor preparedness. 11) Evaluating an intervention to support bereaved parents. 12) Developing a communication tool for physicians on discussing autopsy. 13) Exploring how/when to intervene with couples marriages/partnerships during the cancer/NF1 trajectory. 14) Evaluating methods of preparation for amputation in individuals who underwent an amputation as a child. 15) Determining the feasibility, utility, and impact of Voicing My Choices in AYA living with cancer and other chronic illnesses. 16) Evaluating medical and psychosocial practice guidelines for the assessment and surveillance follow-up of pediatric hematopoetic stem cell donors. 17) Determining integration of pediatric palliative care concepts into existing pediatric oncology programs. 18) Evaluating integration of bereavement psychosocial standards of care into pediatric oncology programs. 19) Developing a patient engagement survey to assess the thoughts of adults and children with NF, and the parents of these children, and about their participation in psychosocial clinical trials to better design studies that are meaningful to patients. 20) Determining the health related quality of life (HRQoL) of sibling pediatric HSC donors and to compare their HRQoL to that of (1) healthy non-donor sibling from the same family, (2) siblings of children with similar diseases who receive alternate treatments (e.g., unrelated cord blood transplants), and (3) healthy age, gender, and race/ethnicity-matched controls. 21)Developing a board game to prepare children for CAR T therapy. 22) Developing content for an electronic game to prepare children for CAR T therapy. 23) Developing interchangeable cards for the therapeutic game ShopTalk to prepare children for BMT. 24) Developed a nationwide study to assess the impact of COVID-19 on pediatric palliative care providers and programs. 25) Conducting an international anonymous online survey for adults with NF1, NF2, and schwannomatosis to better understand their social-emotional concerns and to help plan patient-centered psychosocial trials