Paediatric cancer articles within Nature Reviews Clinical Oncology

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  • Review Article |

    Advances in the treatment of childhood cancers have substantially improved cure rates, although the gains in survival are offset by an elevated burden of morbidities and an excess risk of early death owing predominantly to the adverse effects of therapy. In this Review, the authors summarize the evolution of paediatric cancer therapies over the past five decades as well as the associated landscape of treatment-related late and/or long-term health conditions experienced by childhood cancer survivors. In addition, they discuss strategies that are being explored to reduce the overall burden and consequences of these morbidities with the ultimate aim of improving not only the quantity but also the quality of life-years gained for this large, medically vulnerable population.

    • Matthew J. Ehrhardt
    • , Kevin R. Krull
    •  & Melissa M. Hudson

  • Review Article |

    Neuroblastomas are tumours of sympathetic origins typically seen in infants (≤5 years of age). In this Review, the authors describe progress in the treatment of patients with neuroblastoma, which has resulted in considerable improvements in survival outcomes over the past several decades. The authors then summarize ongoing attempts to personalize therapy in patients with high-risk disease, and to safely de-escalate therapy in those with low-risk disease.

    • Bo Qiu
    •  & Katherine K. Matthay

  • News & Views |

    Cerebrospinal fluid liquid biopsies can enable the characterization and monitoring of medulloblastoma. The analysis of copy-number variations in circulating tumour DNA present in these samples can be used as a biomarker to determine the presence of measurable residual disease, and facilitate the optimal treatment and clinical management of patients with medulloblastoma.

    • Joan Seoane
    •  & Laura Escudero

  • Review Article |

    Despite being the most common primary bone cancer in children and young adults, osteosarcoma is a rare cancer, a fact that has complicated efforts to improve patient outcomes. Moreover, the molecular biology of disease is highly heterogeneous and most of the recurrent genetic alterations occur in tumour-suppressor genes that are challenging therapeutic targets. Herein, Gill and Gorlick discuss the new biological discoveries, technologies, and therapeutic agents and approaches that, through collaborative efforts, are poised to generate advances in the treatment of osteosarcoma after more than four decades of stagnation.

    • Jonathan Gill
    •  & Richard Gorlick

  • News & Views |

    The question of whether allogeneic chimeric antigen receptor (CAR) T cells could replace autologous CAR T cell therapy has garnered considerable interest, but limited data have been available for comparisons to date. Now, Benjamin et al. have reported their experience with allogeneic anti-CD19 CAR T cells in 21 paediatric and adult patients with acute lymphoblastic leukaemia.

    • Amanda M. DiNofia
    •  & Stephan A. Grupp

  • Consensus Statement |

    In this Consensus Statement, members from five working groups or societies provide updated comprehensive recommendations to manage toxicities from cancer immunotherapies in children, adolescents and young adults. In their recommendations, they advocate for the adoption of age-based and discipline-specific management criteria, and call for an increased inclusion of young patients with cancer in clinical trials.

    • Dristhi Ragoonanan
    • , Sajad J. Khazal
    •  & Kris M. Mahadeo

  • Review Article |

    Herein, advances in our understanding of the genomic landscape of childhood acute lymphoblastic leukaemia (ALL), encompassing both somatic and germline alterations, are reviewed. The clinical implications of these alterations, particularly those in the germ line, are discussed with regard to susceptibility to ALL, treatment responses and therapy-related toxicities.

    • Ching-Hon Pui
    • , Kim E. Nichols
    •  & Jun J. Yang

  • News & Views |

    The International Society of Paediatric Oncology (SIOP)–Paediatric Oncology in Developing Countries (PODC) Collaborative Wilms Tumour Africa project delivered treatment in eight centres across five countries in sub-Saharan Africa. Setting up a collaboration like this is easier said than done, and herein we share the lessons we learned along the way.

    • Trijn Israels
    •  & Elizabeth M. Molyneux

  • Consensus Statement
    | Open Access

    Chimeric antigen receptor (CAR) T cell therapies have impressive activity in the treatment of cancer but are associated with potentially fatal toxicities. In light of the approval of CAR T cell therapy for paediatric patients, a panel of experts from the Hematopoietic Stem Cell Transplantation (HSCT) Subgroup of the Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network, the CAR T Cell Therapy-Associated Toxicity (CARTOX) Program at The University of Texas MD Anderson Cancer Center, and several other institutions have developed consensus guidelines for the use and management of these treatments in paediatric patients, which are presented herein.

    • Kris M. Mahadeo
    • , Sajad J. Khazal
    •  & Elizabeth J. Shpall

  • News & Views |

    Comprehensive molecular characterization of infant medulloblastoma has uncovered the high degree of heterogeneity of this disease. Recent results from the SJYC07 study elegantly reveal that risk stratification can be improved if DNA methylation profiling data are incorporated into clinicopathological criteria. Importantly, the delineation of disease subgroups potentially has major clinical implications.

    • Marc Remke
    •  & Vijay Ramaswamy

  • Comment |

    A call for action has been made for the establishment of a global fund for cancer control, but potential donors have not reacted yet, possibly because this task would require an annual expenditure of billions of dollars. Herein, I suggest a less onerous starting point: targeting childhood cancer in Africa guided by well-designed national cancer control plans.

    • Cristina Stefan

  • Opinion |

    Precision medicine has dramatically changed the landscape of drug development in oncology, but this paradigm shift remains to be adopted in early phase clinical trials of molecularly targeted agents and immunotherapeutic agents in children with cancer. The authors, members of the Innovative Therapies for Children with Cancer (ITCC) Consortium, describe trial design strategies to enable drugs with promising activity to progress rapidly to randomized studies and, therefore, substantially accelerate drug development for children and adolescents with cancer.

    • Lucas Moreno
    • , Andrew D. J. Pearson
    •  & Gilles Vassal

  • News & Views |

    To improve the survival rates globally for paediatric patients with cancer, looking beyond level A evidence to treat children in low and middle-income countries is imperative. Herein, we discuss why metronomic chemotherapy, which is less toxic and more affordable, offers a pragmatic approach to provide new standards for patients with relapsed disease who have no alternatives.

    • Nicolas André
    • , Shripad Banavali
    •  & Eddy Pasquier

  • Review Article |

    The development and implementation of more effective genome analysis technologies has enabled substantial improvements in our understanding of the genomic changes that take place in patients with acute lymphoblastic leukaemia (ALL). This Review provides a detailed summary of advances in our understanding of the genomics of ALL, and describes how these advances might lead to improved patient outcomes.

    • Kathryn G. Roberts
    •  & Charles G. Mullighan

  • News & Views |

    The Collaborative Wilms Tumour Africa Project comprises eight centres in sub-Saharan Africa, which are implementing a treatment guideline that has been developed for local conditions. Uniform outcome evaluation, communication and training are all part of the project remit and will ultimately serve to improve cancer care for children in Africa.

    • Trijn Israëls
    •  & Elizabeth M. Molyneux

  • Opinion |

    Numerous calls for improvement in the quality of follow-up care provided to cancer survivors, particularly survivors of childhood cancer have been issued by key health organizations in the USA; the Passport for Care (PFC) is a web-based support system for survivor care that was developed in response. In this Perspectives, key individuals involved in the development of this support tool for clinical decision making outline the programme, describe how it compares with similar guidance tools, and discuss the potential implications of the PFC for survivorship care. The challenges encountered and the lessons learned during development and deployment of the PFC are also highlighted.

    • David G. Poplack
    • , Michael Fordis
    •  & Marc E. Horowitz

  • Review Article |

    Immunotherapies for cancer are demonstrating increasing success, and efforts are underway to generate and test new immunotherapies in a wider array of paediatric cancers. Mackall and coauthors discuss the emerging science driving this rapidly moving field and highlight the most pressing challenges that must be overcome for continued progress in this arena, including how to optimize management of novel toxicities associated with these agents and how best to incorporate these therapies into standard treatment paradigms.

    • Crystal L. Mackall
    • , Melinda S. Merchant
    •  & Terry J. Fry

  • Review Article |

    Medulloblastoma is the most-common form of paediatric brain cancer. Advances in our understanding of the molecular basis of medulloblastoma indicate that it is not a single disease, but a collection of four distinct molecular tumour subtypes. This knowledge has important implications for medulloblastoma research and treatment. In this Review, the characteristic demographic, clinical and genetic features of the four molecular subtypes of medulloblastoma are described, and the implications of molecular distinctions on therapy are discussed.

    • Amar J. Gajjar
    •  & Giles W. Robinson

  • Review Article |

    Recent studies of neuroblastoma have shed light on the dramatic heterogeneity in its clinical behaviour, which spans from spontaneous regression or differentiation to relentless disease progression. This Review describes the different mechanisms of spontaneous neuroblastoma regression—including neurotrophin deprivation, humoral or cellular immunity, loss of telomerase activity and alterations in epigenetic regulation—and the consequent therapeutic approaches, as a better understanding of these mechanisms might help to identify optimal therapies.

    • Garrett M. Brodeur
    •  & Rochelle Bagatell

  • Opinion |

    Many cancers, particularly those that arise in childhood, have a hereditary component. In this Perspectives article, the authors provide their views on how the increased adoption of high-throughput DNA sequencing technologies, which produce vast genetic data that is not necessarily limited to known cancer-susceptibility loci, and cancer surveillance strategies are influencing the clinical management of familial cancer. Important ethical issues relating to genetic counselling and disclosure of genetic information on disease susceptibility are discussed, and strategies for approaching these ethical dilemmas are proposed.

    • Nardin Samuel
    • , Anita Villani
    •  & David Malkin

  • Science and Society |

    In this Science & Society article, the authors provide their personal experience of treating children in low and middle-income countries in sub-Saharan Africa. The authors discuss how conducting clinical trials in this environment can be used for the benefit of all paediatric patients, and outline the measures that need to be put in place to ensure that the outcomes in improved care are met.

    • Trijn Israëls
    • , Joyce Kambugu
    •  & Elizabeth M. Molyneux

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