Pediatric Oncology Branch Behavioral Science Core

对NF临床试验中评估生活质量和社交技能终点的患者报告结果测量的建议（2021）。2019冠状病毒病对成人神经纤维瘤病患者心理健康和获得医疗服务的影响——来自匿名在线调查（2022年）《2019冠状病毒病对儿科姑息治疗提供者和社会工作者道德困境的影响》发表。第一份关于使用“表达我的选择”的国家结果数据，这是POB为青少年和年轻人制定的一份预先护理计划指南。在《儿童杂志》上共同编辑和出版了一期关于患有罕见疾病的儿童和青少年的社会心理考虑的特刊。开发了一个应用程序，临床中心宝藏之旅，为来到NIH临床中心的儿童做准备，支持内部PI：使用计算机化神经认知测试和神经症状检查表评估神经毒性，我们在I/II期研究中开发了CD33 CAR - t细胞输注在复发/难治性急性髓性白血病（PI）的儿科、青少年和年轻成人患者中。使用计算机化神经认知测试和神经症状清单评估神经毒性，该清单是我们在POB I期CD22 CAR治疗方案中开发的，用于复发性b细胞恶性肿瘤包括中枢神经系统疾病的儿童（PI:Shah）使用计算机化神经认知测试评估神经毒性，并根据POB I期CD19/22 CAR治疗方案中的CD22 CAR结果开发和修订的神经症状清单使用计算机化神经认知测试、神经症状检查表评估神经毒性。开展一项有限地点的试点研究，调查接受CAR - T细胞治疗的复发/难治性血液恶性肿瘤儿童和年轻人神经毒性的认知后遗症（亚急性和长期），包括远程评估（PI:Shalabi）评估接受CAR - T治疗的儿童及其护理人员的患者报告结局自我报告和定性访谈(PI: Haneen Shalabi；赖:收集受试者在输注CD33 CAR - t细胞之前和随后一个月每周报告的症状数据（PI:Shah）评估儿童和青少年甲状腺髓样癌（MTC）的纵向和自然史研究中的社会心理和行为结果（PI:Widemann）评估儿童和成人RUNX1 FPD的纵向和自然史研究中的社会心理和行为结果（PI:Liu）评估在美国国立卫生研究院胃肠道间质瘤（GIST）诊所接受治疗的儿童和成人胃肠道间质瘤（GIST）患者的心理社会因素、压力源和需求评估（PI:Glod）在NIH甲状腺髓样癌（MTC）诊所就诊的儿童和成人的压力源和需求（PI:Glod）调查儿童、青少年和年轻成人患NF1的神经心理功能、生活质量和疼痛的自然史（PI:Widemann）selumetinib在NF1和不能手术的丛状神经纤维瘤儿童中的注册II期临床试验和生活质量，这有助于FDA于2020年4月批准selutib (koselugo) （PI:Widemann）。在一项MEK1/2抑制剂selumetinib （AZD6244硫酸氢）用于1型神经纤维瘤病和皮肤神经纤维瘤成人的试点研究中，对患者报告的结果进行了测量（PI:Gross）评估I型神经纤维瘤病（NF1）相关非典型神经纤维瘤（NF1）相关的周期蛋白依赖性激酶（CDK）4/6抑制剂Abemaciclib的I/II期研究患者报告的结果和神经认知评估（PI:Gross）为NF1和高风险肿瘤患者的一项新的自然史研究（PI:Gross）规划患者报告的结果和神经认知评估（PI:Gross）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：评估全脑辐射对成人脑肿瘤转移患者神经认知功能的影响（PI: Kevin Camphausen）慢性GVHD儿童临床因素的自然史研究中评估儿童的社会心理特征(PI：作为POB自然史罕见肿瘤倡议的一部分，评估儿童、青少年和成人罕见实体瘤患者报告的核心结果（PI: Jaydira Del Rivero）为一项自然史研究进行两组认知测试（低功能和高功能），以检查患有RASopathies的儿童和年轻人的认知、社会和情感发展(PI：对接受DOCK8缺乏症移植的兄弟姐妹的移植顺序进行定性访谈（PI: Nirali Shah）评估相关和非相关供体造血干细胞移植的Dock 8缺乏症患者的症状负担和生活质量（PI: Corina Gonzalez）对Dock 8缺乏症造血干细胞移植前和1年后进行定性访谈(PI：Corina Gonzalez)进行评估，以调查使用Apple iPhone和Apple Watch的移动传感器技术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）的可行性(PI：在一项评估饮食和运动对黑色素瘤患者肠道微生物组和免疫检查点抑制剂治疗反应的方案中设计和实施一项行为健康干预和患者报告的结果（PI: James Gulley）。在一项伊马替尼增加种系RUNX1缺乏症患者RUNX1活性的研究中，评估患者报告的结果和生活质量。（Lea Cunningham）核心发起的调查在COVID-19大流行期间建立和传播与慢性疼痛患者合作的建议评估COVID-19对成人神经纤维瘤病患者心理健康和获得医疗保健的影响（来自匿名在线调查）开展了一项研究评估COVID-19对美国儿科姑息治疗提供者和项目的影响，并开展了第二项研究评估全球影响评估父母在子女死亡前后的心理社会支持需求评估障碍和便利*TRUNCATED*

Recommendations for patient-reported outcome measures assessing quality of life and social skills endpoints in NF clinical trials (2021). Impact of COVID-19 on the impact of COVID-19 on mental health and access to health care in adults with neurofibromatosis from an anonymous online survey (2022) Publication of the impact of COVID-19 on the moral distress of pediatric palliative care providers and social workers. First national outcome data on the use of Voicing My CHOiCES, an advance care planning guide for adolescents and young adult developed in the POB. Co-edited and published a Special Issue in the Journal Children on Psychosocial Considerations for Children and Adolescents Living with a Rare Disease. Developed an app, The Clinical Center Treasure Tour, preparing children coming to the NIH Clinical Center Support Intramural PIs: Assessing neurotoxicity using computerized neurocognitive testing and a neurologic symptom checklist that we developed in a phase I/II study of CD33 CAR T-cell infusions in pediatric and adolescent and young adult patients with relapsed/refractory acute myeloid leukemia (PI: Nirali Shah) Assessing neurotoxicity using computerized neurocognitive testing and a neurologic symptom checklist that we developed in a POB phase I CD22 CAR treatment protocol for children with recurrent B-cell malignancies including CNS disease (PI:Shah) Assessing neurotoxicity using computerized neurocognitive testing and a neurologic symptom checklist that we developed and revised based on the CD22 CAR results in the POB phase I CD19/22 CAR treatment protocol for children with recurrent B-cell malignancies (PI:Shah) Assessing neurotoxicity using computerized neurocognitive testing, neurologic symptom checklist, and patient-reported outcomes on a CD22 CAR trial in adults with Hairy Cell Leukemia (PI:Krietman) Developing a limited-site pilot study to investigate the cognitive aftereffects (subacute and long-term) of neurotoxicity in children and young adults with relapsed/refractory hematologic malignancies who receive CAR T-cell therapy including remote assessments (PI:Shalabi) Assessing patient reported outcomes in children undergoing CAR T therapy and their caregiver through self-report and qualitative interviews (PI: Haneen Shalabi; LAI: Lori Wiener) Collecting subject-reported symptom data prior to infusion of CD33 CAR T-cell infusions and weekly in the month following (PI:Shah) Assessing psychosocial and behavioral outcomes in a longitudinal and natural history study of children and young adults with Medullary Thyroid Carcinoma (MTC) (PI:Widemann) Assessing psychosocial and behavioral outcomes in a longitudinal and natural history study of children and adults with RUNX1 FPD (PI:Liu) Assessing psychosocial and behavioral outcomes in longitudinal and natural history study of children and adults with Adrenal Cortical Cancer (PI:Del Rivero) Assessing psychosocial factors, stressors and needs of children and adults with Gastrointestinal Stromal Tumor (GIST) that attend the NIH GIST clinic (PI:Glod) Assessing psychosocial factors, stressors and needs of children and adults with Medullary Thyroid Carcinoma (MTC) that attend the NIH MTC clinic (PI:Glod) Investigating the natural history of neuropsychological functioning, QOL, and pain in children, adolescents, and young adults with NF1 (PI:Widemann) Analyzing the long-term 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 (PI:Widemann) Assessing patient-reported outcomes of pain, physical function, and quality of life in a phase II clinical trial of selumetinib in adults with NF1 and inoperable plexiform neurofibromas Assessing medication adherence in a phase II clinical trial of selumetinib in children with NF1 and inoperable plexiform neurofibromas (PI:Widemann) Administering the patient-reported outcomes measures for a pilot study of the MEK1/2 inhibitor selumetinib (AZD6244 Hydrogen Sulfate) for adults with neurofibromatosis type 1 and cutaneous neurofibromas (PI:Gross) Assessing patient-reported outcomes measures for a phase I/II study to evaluate the Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas (PI:Gross) Planning the patient-reported outcomes and neurocognitive assessment for a new natural history study of individuals with NF1 and high risk tumors (PI:Gross) 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) 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) Evaluating the effects of whole-brain radiation on the neurocognitive functioning of adults with brain tumor metastases (PI: Kevin Camphausen) Assessing psychosocial characteristics of children in a natural history study of clinical factors in children with chronic GVHD (PI: Steven Pavletic) Assessing a core set of patient-reported outcomes to assess children, adolescents, and adults with rare solid tumors as part of the POB natural history rare tumor initiative (PI: Jaydira Del Rivero) Conducting two cognitive test batteries (low and high functioning) for a natural history study to examine the cognitive, social, and emotional development of children and young adults with RASopathies (PI: Doug Stewart) Conducted qualitative interviews on order of transplant in siblings undergoing transplant for DOCK8 Deficiency (PI: Nirali Shah) Assessing symptom burden and quality of life in patients undergoing related and unrelated donor Hematopoietic Stem Cell Transplant for Dock 8 Deficiency (PI: Corina Gonzalez) Conducting qualitative interviews pre and 1 year post Hematopoietic Stem Cell Transplant for Dock 8 Deficiency (PI: Corina Gonzalez) Conducting evaluations to investigate 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) Designing and implementing a behavioral health intervention and patient-reported outcomes in a protocol assessing the effect of diet and exercise on the gut microbiome and response to immune checkpoint inhibitor therapy in patients with melanoma (PI: James Gulley) Assessing quality of life in a natural history protocol for Myelodysplastic Syndromes (PI: Kathy L McGraw). Assessing patient report outcomes and quality of life in a study of Imatinib to increase RUNX1 activity in patients with germline RUNX1 deficiency (PI: Lea Cunningham) Core-Initiated Investigations Establishing and disseminating recommendations on working with patients with chronic pain during the COVID-19 pandemic Assessing the impact of COVID-19 on mental health and access to health care in adults with neurofibromatosis from an anonymous online survey Developed a study to assess the impact of COVID-19 on pediatric palliative care providers and programs within the US and a 2nd study to assess the impact globally Evaluating the psychosocial support needs of parents before and after the death of their child Assessing the barriers and facilitat *TRUNCATED*