Pediatric Oncology Branch Behavioral Science Core

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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