# Rare cancers of childhood (NCI PDQ umbrella)

Source: https://onco.cc/cancers/rare-childhood-cancers/  
OnCo record `rare-childhood-cancers` (Cancer). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Some childhood cancers are so rare that no single hospital sees enough to learn from. The NCI groups them together: heart tumours, airway papillomas, cancers of the thyroid, adrenal, nose and throat, melanoma and carcinomas more typical of adults. The answer has been international registries and expert networks that pool every case, so treatment guidance exists even without trials.

## Summary

The NCI PDQ summary on rare cancers of childhood collects tumours that fall outside the common paediatric disease groups: head and neck carcinomas (nasopharyngeal carcinoma, salivary, laryngeal, thyroid, oesthesioneuroblastoma, NUT carcinoma), thoracic tumours (pleuropulmonary blastoma, tracheobronchial tumours, thymoma, cardiac tumours, mesothelioma), abdominal tumours (adrenocortical carcinoma, gastrointestinal carcinomas, carcinoid, pancreatic tumours, pancreatoblastoma), genital and urinary tumours (bladder, testicular non-germ cell, ovarian epithelial), skin cancers (melanoma, basal and squamous cell carcinoma), and multiple endocrine neoplasia syndromes. Where OnCo has a dedicated record (nasopharyngeal, thyroid, adrenocortical, melanoma, pleuropulmonary blastoma, NUT carcinoma, sinonasal, pheochromocytoma-paraganglioma, multiple endocrine neoplasia) this page points to it; this record covers the residue and the organisational response.

Two entities on the NCI list have no other home. Childhood cardiac tumours are mostly benign rhabdomyomas associated with tuberous sclerosis complex, which regress spontaneously and, when obstructive, respond to mTOR inhibitors (everolimus or sirolimus) rather than surgery; fibromas, myxomas and rare sarcomas are treated surgically. Recurrent respiratory (laryngeal) papillomatosis is a benign HPV 6/11 disease acquired at birth that causes hoarseness and airway obstruction, requires repeated debulking, rarely transforms to squamous carcinoma, and is being prevented by HPV vaccination of mothers and reduced by systemic bevacizumab in refractory cases; the nonavalent vaccine as adjuvant therapy is also studied.

The defining feature of these tumours is that randomised trials are impossible, so the field built other tools: the COG Rare Tumors committee and its registry, the European Cooperative Study Group for Paediatric Rare Tumours (EXPeRT) and its consensus recommendations, the Pediatric MATCH molecular-assignment platform that accepts any refractory solid tumour, and the practice of borrowing adult protocols with paediatric dose adjustments. Adult-type carcinomas in children are often linked to germline predisposition (TP53, DICER1, mismatch repair, APC) and trigger genetic testing.

## Fields

- Kind: Cancer
- Last checked: 2026-09-10
- Also known as: Childhood cardiac tumours; Laryngeal papillomatosis; Recurrent respiratory papillomatosis; Childhood melanoma; Childhood carcinomas; Very rare tumours of childhood
- Tags: nci-coverage; paediatric; umbrella
- Group: paediatric
- Burden: Individually vanishingly rare, but together the tumours grouped by the NCI as rare cancers of childhood account for roughly one in ten childhood cancers, and their share rises in adolescence (NCI PDQ).
- Subtypes: Childhood cardiac tumours (rhabdomyoma with tuberous sclerosis, fibroma, myxoma, sarcoma); Recurrent respiratory papillomatosis (HPV 6/11); Childhood melanoma and non-melanoma skin cancer; Childhood head and neck carcinomas (nasopharyngeal, salivary, laryngeal, oesthesioneuroblastoma, NUT carcinoma); Childhood gastrointestinal and pancreatic carcinomas (including pancreatoblastoma); Childhood thoracic tumours (pleuropulmonary blastoma, thymoma, mesothelioma); Childhood endocrine tumours (thyroid, adrenocortical, pheochromocytoma, MEN syndromes)
- Biomarkers: Germline predisposition testing (TP53, DICER1, mismatch repair, APC, RET, SDHx); Tuberous sclerosis complex (TSC1/TSC2) in cardiac rhabdomyoma; HPV 6/11 typing in respiratory papillomatosis; Tumour-specific adult markers borrowed by site

## Sections of this record

The page is a hub with ten sections in reading order; large sections have their own page. The same plan as JSON: https://onco.cc/api/v1/cancers/rare-childhood-cancers/sections.json

- Overview (on the hub): The TL;DR, the family this cancer belongs to, the organ, who gets it and what the state of the art is. https://onco.cc/cancers/rare-childhood-cancers/#overview [4 state-of-the-art points]
- What it is (on the hub): Anatomy, the subtypes and how they differ, how it is staged, and where advanced disease spreads. https://onco.cc/cancers/rare-childhood-cancers/#what-it-is [7 subtypes]
- Finding it (on the hub): How it shows itself, how it is confirmed, what screening exists, and the biomarkers clinicians test for. https://onco.cc/cancers/rare-childhood-cancers/#finding-it [4 biomarkers]
- Treating it (on the hub): The standard of care by setting, the medicines, surgery and radiotherapy named in it, and the regimens behind them. https://onco.cc/cancers/rare-childhood-cancers/#treating-it [3 settings, 1 decision with options]
- Evidence (on the hub): Trials recruiting now, the landmark trials, the key papers and what they mean, the latest literature, and the milestones year by year. https://onco.cc/cancers/rare-childhood-cancers/#evidence [4 trials, 5 milestones]
- The science (on the hub): The molecular landscape: the targets and how often each appears, the pathways, the mechanics stages and the preclinical models. https://onco.cc/cancers/rare-childhood-cancers/#science [3 targets]
- Where you are (own page): Cases by country, the UK and NHS pathway and other country lenses, and the expert centres with trials on record. https://onco.cc/cancers/rare-childhood-cancers/where-you-are/ [1 centre]
- Living with it (on the hub): The decisions you may face, the aids that walk through them, the warnings on record, the first sixty days and the questions to ask. https://onco.cc/cancers/rare-childhood-cancers/#living-with-it [15 questions, 6 red cards]
- What is coming (own page): Everything in development, the open problems and what is being done about them, the roadmaps, and what changed on this record. https://onco.cc/cancers/rare-childhood-cancers/coming/ [4 medicines, 3 trials, 3 open problems]
- Data (own page): Every connected record, the notes, the JSON, Markdown and RDF twins, and where the record came from and when it was checked. https://onco.cc/cancers/rare-childhood-cancers/data/ [39 connected records]

## Standard of care

- Cardiac rhabdomyoma with tuberous sclerosis: Observation for spontaneous regression; mTOR inhibitor (everolimus or sirolimus) for haemodynamically significant tumours; surgery reserved for obstruction unresponsive to medical therapy. ([Everolimus](https://onco.cc/drugs/everolimus/))
- Recurrent respiratory papillomatosis: Repeated microdebrider or laser debulking to maintain the airway; systemic bevacizumab for severe refractory disease; HPV vaccination (prevention through maternal vaccination; adjuvant use under study). ([Bevacizumab](https://onco.cc/drugs/bevacizumab/), [Nonavalent HPV vaccine](https://onco.cc/drugs/gardasil-9/), [HPV & HBV vaccination](https://onco.cc/technologies/hpv-vaccine/))
- Adult-type carcinoma in a child: Treat per the adult site-specific standard with paediatric dose adjustment, enrol in the COG or EXPeRT rare tumour registry, and test for germline predisposition. ([Germline (hereditary) testing](https://onco.cc/technologies/germline-testing/), [NCI-COG Pediatric MATCH (APEC1621)](https://onco.cc/trials/pediatric-match/))

## State of the art

- Registries and consensus networks (COG Rare Tumors, EXPeRT) supply evidence where trials cannot; their recommendations are now the reference for most of these tumours.
- mTOR inhibition has replaced surgery for most obstructive cardiac rhabdomyomas in tuberous sclerosis.
- HPV vaccination is beginning to reduce respiratory papillomatosis, and bevacizumab controls the most severe cases.
- Germline testing is routine for adult-type cancers in children because a high proportion are hereditary.

## Open problems

- Evidence remains observational for almost every entity; international registries and adult-paediatric joint protocols are the response.
- Adolescents with adult-type carcinomas fall between paediatric and adult services; AYA programmes and the RACE for Children Act (which requires paediatric evaluation of adult cancer drugs) aim to close the gap.
- Access to molecular profiling and matched therapy for rare tumours outside major centres.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Childhood_cancer
- NCI PDQ: rare cancers of childhood: https://www.cancer.gov/types/childhood-cancers/patient/rare-childhood-cancers-pdq
- NCI PDQ: childhood cardiac tumours: https://www.cancer.gov/types/cardiac/patient-child-cardiac-treatment-pdq
- EXPeRT: European Cooperative Study Group for Paediatric Rare Tumours: https://www.raretumors-children.eu/

## Connected records

- cancers: [Adrenocortical carcinoma](https://onco.cc/cancers/adrenocortical/), [Childhood cancers (all types)](https://onco.cc/cancers/childhood-cancers/), [Childhood lung and airway tumours (pleuropulmonary blastoma, tracheobronchial tumours)](https://onco.cc/cancers/pleuropulmonary-blastoma/), [Germ cell tumours of childhood and adolescence (extracranial and CNS)](https://onco.cc/cancers/paediatric-germ-cell-tumours/), [Hepatoblastoma](https://onco.cc/cancers/hepatoblastoma/), [Melanoma](https://onco.cc/cancers/melanoma/), [Multiple endocrine neoplasia syndromes (MEN1, MEN2, MEN4)](https://onco.cc/cancers/multiple-endocrine-neoplasia/), [Nasal cavity and paranasal sinus cancers (including esthesioneuroblastoma)](https://onco.cc/cancers/sinonasal/), [Nasopharyngeal carcinoma](https://onco.cc/cancers/nasopharyngeal/), [NUT carcinoma (midline carcinoma with NUTM1 rearrangement)](https://onco.cc/cancers/nut-carcinoma/), [Pancreatoblastoma](https://onco.cc/cancers/pancreatoblastoma/), [Pheochromocytoma and paraganglioma (PPGL)](https://onco.cc/cancers/pheochromocytoma-paraganglioma/), [Retinoblastoma](https://onco.cc/cancers/retinoblastoma/), [Thyroid cancer](https://onco.cc/cancers/thyroid/)
- terms: [Adolescent and young adult (AYA) oncology](https://onco.cc/terms/aya-oncology/), [Hereditary cancer syndromes](https://onco.cc/terms/hereditary-cancer-syndromes/), [RACE for Children Act](https://onco.cc/terms/race-for-children-act/), [SDH deficiency (SDHB immunohistochemistry loss)](https://onco.cc/terms/sdh-deficiency/)
- technologies: [Cancer registries and population surveillance](https://onco.cc/technologies/cancer-registries-surveillance/), [Germline (hereditary) testing](https://onco.cc/technologies/germline-testing/), [HPV & HBV vaccination](https://onco.cc/technologies/hpv-vaccine/)
- drugs: [Bevacizumab](https://onco.cc/drugs/bevacizumab/), [Everolimus](https://onco.cc/drugs/everolimus/), [Nonavalent HPV vaccine](https://onco.cc/drugs/gardasil-9/), [TQ-B3234](https://onco.cc/drugs/tq-b3234/)
- companies: [Children's Cancer and Leukaemia Group](https://onco.cc/companies/cclg/), [Children's Oncology Group (COG)](https://onco.cc/companies/childrens-oncology-group/), [Precigen](https://onco.cc/companies/precigen/), [Shanghai Fosun Pharmaceutical Industrial Development](https://onco.cc/companies/shanghai-fosun-pharmaceutical-industrial-development/), [Shanghai Kechow Pharma](https://onco.cc/companies/shanghai-kechow-pharma/)
- institutions: [SIOP Europe (European Society for Paediatric Oncology)](https://onco.cc/institutions/siop-europe/)
- trials: [Adjuvant PRGN-2012 in Adult Patients With Recurrent Respiratory Papillomatosis](https://onco.cc/trials/nct04724980/), [DETERMINE](https://onco.cc/trials/determine-trial/), [Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas](https://onco.cc/trials/nct07407803/), [NCI-COG Pediatric MATCH (APEC1621)](https://onco.cc/trials/pediatric-match/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/), [Trials enrol too few, too slowly](https://onco.cc/bottlenecks/b-trial-enrolment/)
- journals: [Journal of pediatric hematology/oncology](https://onco.cc/journals/journal-of-pediatric-hematology-oncology/), [Journal of pediatric hematology/oncology nursing](https://onco.cc/journals/journal-of-pediatric-hematology-oncology-nursing/), [Pediatric hematology and oncology](https://onco.cc/journals/pediatric-hematology-and-oncology/)

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JSON: https://onco.cc/api/v1/entities/rare-childhood-cancers.json