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Advances in Neurofibromatosis in Children, Adolescents, and Young Adults

A special issue of Cancers (ISSN 2072-6694).

Deadline for manuscript submissions: 1 June 2026 | Viewed by 1197

Special Issue Editors


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Guest Editor
Pediatric Oncology Branch, National Cancer Institute, Bethesda, MD 20814, USA
Interests: patient reported outcomes; neurocognitive outcomes; quality of life; psychosocial well-being; survivorship; late effects; behavioral health interventions; pediatric brain tumors; pediatric cancer predisposition syndromes; pediatric ALL

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Guest Editor
National Cancer Institute (NCI), Rockville, MD, USA
Interests: pediatric oncology; neurofibromatosis; clinical trial design

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Guest Editor
1. National Cancer Institute (NCI), Rockville, MD, USA
2. Nemours Children's Hospital, Delaware 1600 Rockland Road, Wilmington, DE 19803, USA
Interests: neurofibromatosis; MPNST; genomics; cell free DNA; proteomics; epigenetics; liquid biopsy; peripheral nerve sheath tumors

Special Issue Information

Dear Colleagues,

Neurofibromatosis type 1 (NF1), NF2-related schwannomatosis (NF2), and non-NF2-related schwannomatosis (SWN) are rare genetic tumor predisposition syndromes in which various types of tumor may develop in the central and peripheral nervous systems. Individuals with these conditions also may experience various non-tumor-related phenotypes that may arise in childhood, such as juvenile cataracts in NF2 and bone abnormalities and cognitive impairments in NF1. This Special Issue will focus on all types of research focusing on children, adolescents, and young adults with NF1, NF2, or SWN. Manuscripts should describe interventional trials, the development of novel assessment tools, and results from natural history studies for these genetic conditions. Such studies are needed to evaluate new drug treatments or behavioral interventions, to better understand the development of both benign and malignant tumors and other clinical manifestations (e.g., bone, neurologic, cognitive, and social–emotional issues, pain, hearing loss, and physical problems), to investigate the underlying mechanism related to the pathology of these manifestations, and to develop new patient-centered outcomes and biomarkers to monitor changes over time related to these disorders and with treatment specifically focused on children, adolescents, and young adults. Studies that are exclusively bioinformatic, such as those that identify candidate biomarkers, will require rigorous cross-validation and, when possible, should be validated in an independent dataset. Additional clinical validation in patient samples and/or functional validation in vitro or in vivo is strongly encouraged.

Dr. Pamela L. Wolters
Dr. Andrea M. Gross
Dr. Russell Taylor Sundby
Guest Editors

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Keywords

  • neurofibromatosis
  • schwannomatosis
  • natural history
  • clinical trials
  • targeted therapy
  • outcomes
  • psychosocial
  • biomarkers
  • pediatric

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Published Papers (1 paper)

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Research

17 pages, 788 KB  
Article
Age-Specific ADHD and Internalizing/Externalizing Comorbidity in Children with Neurofibromatosis Type 1: A Multi-Site Study
by Dan Liu, Pamela L. Wolters, Bonita P. Klein-Tasman, Karin S. Walsh, Jonathan M. Payne, Natalie Pride, Stephanie M. Morris and Yang Hou
Cancers 2026, 18(3), 529; https://doi.org/10.3390/cancers18030529 - 6 Feb 2026
Cited by 1 | Viewed by 877
Abstract
Objective: The current study tested (1) how ADHD symptoms and internalizing or externalizing problems covaried across ages 3–18 in children with neurofibromatosis type 1 (NF1), and (2) whether demographic and NF1-specific factors moderated the associations. Method: We analyzed integrated cross-sectional data [...] Read more.
Objective: The current study tested (1) how ADHD symptoms and internalizing or externalizing problems covaried across ages 3–18 in children with neurofibromatosis type 1 (NF1), and (2) whether demographic and NF1-specific factors moderated the associations. Method: We analyzed integrated cross-sectional data of 685 observations from 455 children and adolescents with NF1 (Mage = 9.79 years, SD = 3.88; 43% female) across six institutions in the United States and Australia. ADHD symptoms (inattention and hyperactivity/impulsivity) and internalizing/externalizing problems were assessed via parent-report measures. Time-varying effect modeling was employed to examine the age-specific associations between ADHD symptoms and internalizing/externalizing problems. Moderation analyses tested effects of sex, parental education, and NF1 inheritance mode (familial vs. sporadic). Results: Inattention and hyperactivity/impulsivity symptoms were associated with greater internalizing and externalizing problems across ages 3–17. Inattention links were similar across ages, while the hyperactivity/impulsivity-externalizing link was stronger in early childhood than during adolescence. NF1 inheritance mode significantly moderated the inattention-externalizing link, with stronger associations observed among children with familial NF1. Other moderators were nonsignificant. Conclusions: ADHD symptoms are robustly linked to internalizing and externalizing problems from childhood to middle adolescence in children with NF1, with familial NF1 emerging as a potentially elevated risk factor. Future longitudinal and experimental research is needed to inform integrated intervention approaches, especially for those with familial NF1. Full article
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