paediatric

ACCELERATE is a Brussels-based forum where children's cancer doctors, drug companies, the EMA and FDA and parents agree which new cancer drugs should be tested in children and how, so that medicines developed for adults are not left untested in childhood cancers.

ACTION is the first placebo-controlled phase 3 trial ever run in diffuse midline glioma, the childhood brain-stem tumour that radiotherapy alone has never cured. It asks whether taking dordaviprone after radiotherapy lengthens life.

Acute myeloid leukaemia in children carries gene fusions rather than the mutations of ageing, is treated with four or five intensive courses of chemotherapy, and cures around two thirds of children. Adding gemtuzumab ozogamicin lowered relapse in the AAML0531 trial, and the menin inhibitor revumenib is the first targeted drug approved for the KMT2A-rearranged form common in young children.

People diagnosed between 15 and 39 get different cancers from children and from older adults, and for years their survival improved more slowly than either. Since 2000 that has changed: five-year survival gains for this group have paralleled those of childhood cancers. The obstacles that remain are trial enrolment, access and insurance, and support that fits the age.

A childhood-cancer charity started by a four-year-old patient's lemonade stand that now funds hundreds of grants and runs an open-science data lab for paediatric cancer genomics.

Led the St. Jude medulloblastoma trials that introduced molecular subgroups into risk-adapted treatment.

ATRT is an aggressive brain tumour of babies and toddlers caused by loss of a single gene, SMARCB1, part of the machinery that opens and closes DNA. Intensive chemotherapy with stem-cell rescue, and radiotherapy where age allows, now cure a meaningful share of children who once had little chance, and drugs aimed at the epigenetic consequence of SMARCB1 loss (EZH2 inhibitors) are in trials.

A child treated during the years in which bone is laid down may never reach the peak bone mass they would have had, which is a different problem from an adult losing bone already built. Thirty per cent of adult survivors of childhood leukaemia had low bone density, most strongly associated with growth hormone deficiency and smoking, both treatable.

A girl who had radiotherapy to the chest carries a risk of breast cancer by age 50 of about 30 per cent. It is not only about dose: a low dose to the whole lung gave a higher standardised incidence than a high dose to a smaller field, because volume matters. Surveillance is recommended from early adulthood, decades before ordinary screening starts.

Britain's own survivor cohort, built on national registration rather than on hospital volunteers, which is why it can follow people for half a century and count deaths that nobody reported. Its central finding is that what kills survivors changes with time: recurrence early, second cancers and heart disease late.

Burkitt lymphoma is the fastest-growing human tumour, driven by a single rearrangement that switches on the MYC gene. That speed makes it exquisitely sensitive to chemotherapy: short, intense courses, now with the antibody rituximab, cure the great majority of children in well-resourced settings. The remaining task is to bring the same cure to the African children who make up most cases.

Germ cell tumours of the brain grow near the pineal gland or above the pituitary in teenagers. The commonest kind, germinoma, is so sensitive to radiation and chemotherapy that most patients are cured; the other kinds need stronger chemotherapy and radiotherapy, and doctors measure two proteins in the blood and spinal fluid to tell them apart and to follow treatment.

The largest study of what happens to children after cancer is cured. Following tens of thousands of survivors for decades, it showed that heart damage, second cancers and other late effects were common after older treatments, and that gentler modern protocols have already halved late deaths.

Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available.

Primary lung tumours in children are rare and unlike adult lung cancer. Pleuropulmonary blastoma starts as a lung cyst in infants and results from a faulty DICER1 gene that also predisposes to thyroid, ovarian and kidney tumours; removing cysts early, guided by an international registry and family gene testing, prevents progression to the aggressive solid forms.

Led the St. Jude Total Therapy studies that cure over 90% of children with leukaemia without cranial radiation.

Chordoma is a slow-growing bone cancer (a sarcoma) of the skull base and spine that arises from leftover embryonic notochord cells. Complete surgery followed by high-dose proton or carbon-ion radiotherapy controls most tumours, and the whole disease depends on a single transcription factor, brachyury, which vaccines and degraders are now trying to hit.

Adding the antibody-drug conjugate gemtuzumab ozogamicin to chemotherapy lowered the chance of relapse in children with acute myeloid leukaemia. Years after the drug had been withdrawn from the US market, this trial helped bring it back for children.

This trial asked whether children with average-risk medulloblastoma could safely receive less radiation. Shrinking the boost to the tumour bed was safe; cutting the dose to the whole brain and spine in young children was not, so 23.4 Gy remains the floor for most.

A risk-adapted Wilms tumour trial: children whose lung metastases vanished after six weeks of chemotherapy were spared lung radiation, while those with stubborn nodules or a high-risk chromosome pattern got stronger chemotherapy and did better than in the past.

Craniopharyngioma is a benign but destructive brain tumour growing from embryonic remnants beside the pituitary gland and hypothalamus. Surgery, or limited surgery plus radiotherapy, cures most people, but the price can be lifelong hormone deficiency and severe obesity. The adult (papillary) form carries a BRAF mutation and shrinks markedly with BRAF and MEK inhibitors, its first drug treatment.

Children started surviving cancer in the 1960s, and within a decade the doctors who had cured them began writing down what the cure had cost. The phrase that stuck, from a 1975 paper by Giulio D'Angio, is that cure is not enough.

London paediatric neuro-oncologist who led the trial showing that dabrafenib and trametinib work in children with BRAF V600-mutant high-grade glioma.

Diffuse midline glioma grows through the brainstem and cannot be removed surgically. A single change in a histone protein (H3 K27M) rewires how the tumour reads its DNA. Radiotherapy was long the only help; in 2025 the first drug aimed at this tumour, dordaviprone (ONC201), was approved after durable shrinkage in some patients, and GD2 CAR-T cells have produced striking early responses.

Cancer in the years when education and first jobs happen costs more than the time taken. In the largest cohort, 23 per cent of childhood cancer survivors had used special education services against 8 per cent of siblings, and survivors of several cancers were less likely to finish high school. The important finding is that where the educational support was given, the gap closed.

Ependymomas grow from the cells lining the fluid spaces of the brain and spinal cord, mostly in children under five. Removing the whole tumour followed by focused radiotherapy controls most cases; molecular groups defined in 2021 behave differently, with posterior fossa group A relapsing often, and there is no approved drug.

The largest osteosarcoma trial ever run, across four cooperative groups on two continents. Neither adding interferon for good responders nor adding ifosfamide and etoposide for poor responders improved outcomes, so three-drug MAP chemotherapy remained the standard and the field turned to new biology.

Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded.

Male survivors were about half as likely as their brothers to father a child, and the causes are specific: testicular radiotherapy above 7.5 gray, and high cumulative cyclophosphamide, ifosfamide, procarbazine or cisplatin. A young man with none of those was no less likely than his brother. Sperm banking works; tissue banking before puberty has produced no births.

Most female survivors treated with chemotherapy and no radiotherapy to the pelvis or brain can become pregnant: the large cohort that asked found chemotherapy-specific effects were few. The exceptions are busulfan, high-dose lomustine, pelvic and cranial radiotherapy and transplant conditioning. Before puberty, freezing ovarian tissue is the only option.

FIREFLY-1 showed that a once-weekly pill, tovorafenib, shrinks most relapsed childhood low-grade gliomas driven by BRAF changes, including the common KIAA1549-BRAF fusion that older BRAF drugs could not treat safely. It led to the first approval of a drug for this disease.

Germ cell tumours arise from the cells meant to become eggs or sperm and can appear in the gonads, lower back, chest or brain. They are among the most curable childhood cancers because they respond to cisplatin chemotherapy and release blood markers that make monitoring easy. The work now is to cure with less: surgery alone for low-risk tumours, gentler platinum drugs, and protecting hearing.

Memphis paediatric neuro-oncologist who led the study showing that the Hedgehog inhibitor vismodegib helps only adults and older children whose medulloblastoma belongs to the SHH subgroup, an early example of subgroup-directed brain tumour therapy.

Group 3 and group 4 medulloblastoma are the two commonest forms of this cerebellar brain tumour and the ones without a druggable driver. Group 3 strikes young children, often with extra copies of MYC and spread through the spinal fluid; group 4 affects older boys. Both get surgery, craniospinal radiotherapy and chemotherapy; trials showed the radiation dose cannot be cut for young children.

Radiotherapy that reaches the pituitary stops the growth hormone signal, and radiotherapy to the spine stops the spine growing. In a Dutch cohort of 573 survivors, 8.9 per cent ended up more than two standard deviations below mean adult height, the largest losses after total body irradiation and craniospinal radiotherapy. Replacement restores some height.

H3 K27M is a single change in a histone that defines diffuse midline glioma, including the brain-stem tumour DIPG. In 2025 dordaviprone became the first drug approved for tumours carrying it.

Hearing loss in a child still learning to speak and read costs more than the same loss in an adult. In the St Jude Lifetime Cohort, severe hearing impairment affected 34.9 per cent of platinum-treated survivors and 38.3 per cent of those irradiated at the cochlea, against 8.8 per cent of unexposed survivors, and tracked deficits in reasoning, fluency and mathematics.

Hepatoblastoma is a childhood liver cancer, mostly of toddlers, cured in most standard-risk cases with cisplatin chemotherapy and surgery, including liver transplant when the tumour cannot be cut out. Sodium thiosulfate given after cisplatin halves the permanent hearing loss cisplatin causes, and became the first approved otoprotectant in 2022.

High-risk childhood leukaemia means a child aged ten or over, a very high white cell count, T-cell disease, spread to the brain or testes, or adverse genetics, and it is treated with longer and more intensive chemotherapy. Most children are still cured; the T-cell form gained the drug nelarabine after the AALL0434 trial, and cranial radiotherapy has been dropped for almost everyone.

High-risk neuroblastoma has spread widely in a child over 18 months old or carries extra copies of the MYCN gene. Treatment lasts about 18 months and uses every tool: chemotherapy, surgery, high-dose chemotherapy with stem cell rescue, radiotherapy, and the anti-GD2 antibody dinutuximab, which raised survival in ANBL0032; eflornithine, given afterwards, was approved in 2023 to lower relapse.

The international society of doctors and scientists who study histiocytic disorders; its LCH trials, run since the 1990s, set the worldwide standard for treating Langerhans cell histiocytosis in children.

Young people often say their cancer took a long time to diagnose, and the research agrees that time to diagnosis varies widely by tumour type and age. What the research does not support is a single number: a systematic review found the studies used different definitions and skewed data that could not be combined, so no meta-analysis was possible.

The figures, with the cohort and the age attached, because they are misquoted more than any others. On self-report at a mean age of 26, 62.3 per cent of survivors had a chronic condition. On clinical testing, the cumulative prevalence of any chronic condition by age 45 was 95.5 per cent, and by age 50 a survivor had 17.1 conditions against 9.2 in matched controls.

Leukaemia diagnosed in the first year of life is a different disease from leukaemia in older children: most cases carry a broken KMT2A gene and respond poorly to chemotherapy, and fewer than half of infants were cured for twenty years. One course of the immune drug blinatumomab after induction raised two-year disease-free survival from about half to over 80 percent in a pilot study.

IMT is a rare tumour, grouped with the sarcomas, of spindle cells mixed with inflammatory cells, most often in the lung or abdomen of children and young adults. Surgery cures most, and about half carry an ALK gene fusion, so the ALK-blocking pill crizotinib is approved for those that cannot be removed, one of the first targeted approvals for a childhood solid tumour.

Dutch paediatric oncologist who led the study showing that adding one course of blinatumomab to chemotherapy sharply improves survival for babies with KMT2A-rearranged leukaemia.

Europe's network of children's hospitals that run the first trials of new cancer drugs in children, so that European children can access experimental medicines close to home.

Adding the antibody rituximab to intensive chemotherapy in children with high-risk Burkitt and related lymphomas cut treatment failures by about two-thirds, making an already curable disease more so. It is the model of a joint European-North American children's cancer trial.

Intermediate-risk neuroblastoma sits between the tumours that go away on their own and the high-risk disease that needs everything. A few cycles of moderate chemotherapy followed by surgery cure most children, and trials have spent twenty years showing how few cycles are enough.

Three countries wrote three different sets of follow-up rules for the same survivors, and the rules disagreed about who to screen, how and how often. Since 2010 this group has been settling those disagreements one organ at a time, in public, with the evidence graded and the disagreement named.

Paris paediatric neuro-oncologist who led HERBY, the trial that showed adding bevacizumab does not help children with high-grade glioma, and who leads European trials in diffuse midline glioma.

Led the COG renal tumour committee that used biology to tailor Wilms tumour treatment.

Neuroblastoma geneticist whose lab found the ALK mutations and immunotherapy targets now in paediatric trials.

Leads the COG Hodgkin lymphoma committee and the trials bringing brentuximab and nivolumab to children with the disease.

A Cochrane review of 61 studies found reported rates of kidney damage after childhood cancer treatment ranging from nought to 84 per cent, which is a statement about the literature rather than about kidneys. On systematic clinical testing of one large cohort, kidney dysfunction was present in 5 per cent, among the least common of the organ problems measured.

Paediatric oncologist who chaired COG AALL0434, the largest trial ever run in childhood T-cell leukaemia, which showed that adding nelarabine improves disease-free survival.

Langerhans cell histiocytosis is a disorder in which a small group of immune cells with a faulty growth signal (most often a BRAF mutation) pile up in bone, skin, pituitary or organs. It ranges from a single bone lesion that heals after biopsy to a life-threatening disease of infants. A year of gentle chemotherapy cures most children, and BRAF or MEK inhibitors rescue those with resistant disease.

Five-year survivors still die earlier than their peers, but much less than they did. Fifteen-year mortality among American five-year survivors fell from 12.4 per cent for children treated in the early 1970s to 6.0 per cent for those treated in the 1990s, and the fall tracks the radiotherapy and anthracycline that were taken out of the protocols.

The Histiocyte Society's third international trial showed that treating multisystem Langerhans cell histiocytosis for a full year, rather than six months, roughly halves the chance of the disease coming back, while adding methotrexate added nothing but toxicity.

Britain sorts survivors into three levels of follow-up by how intensive their treatment was: a postal or telephone review at one end, a specialist late-effects clinic at the other. A Scottish cohort applied the levels retrospectively and found they worked: late effects affected 11.6 per cent of level one survivors and 65.2 per cent of level three.