Rare cancers of childhood (NCI PDQ umbrella)
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.
Overview
The NCI PDQ summary on rare cancers of childhood collects tumours that fall outside the major 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.
State of the art today
- 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.
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).
- HPV & HBV vaccinationStandard of care
- AI in radiologyEstablished
- cfDNA fragmentomicsEstablished
- Colorectal cancer screening (colonoscopy, FIT, stool DNA, blood)Standard of care
- DNA methylation profilingEstablished
- HCC surveillance in cirrhosis (ultrasound + AFP)Standard of care
- A 28-day national pathway for people with a positive multi-cancer blood test
- A breath test to rule out cancer in people with vague symptoms
- A cancer blood test for older people arriving at A&E with unexplained symptoms
- A legislated, publicly reported 28-day standard from urgent referral to diagnosis
- A live national dashboard of stage at diagnosis as the scorecard for early detection
- A single 'cancer check at 60' appointment bundling all screening tests
Background: Alpha-fetoprotein (AFP), Barrett's oesophagus, CA 19-9, Early detection, Faecal immunochemical test (FIT). Also on OnCo: Symptoms and red flags · Early detection roadmap.
Where the cases are
No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.
Observation for spontaneous regression; mTOR inhibitor (everolimus or sirolimus) for haemodynamically significant tumours; surgery reserved for obstruction unresponsive to medical therapy.
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).
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.
Subtypes & biomarkers
top- 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)
- 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
Target prevalence in this cancer
- 2000COG Rare Tumors committee established
Creates a registry and protocols for infrequent tumours.
- 2008EXPeRT founded
European Cooperative Study Group for Paediatric Rare Tumours pools national registries (Italy, Germany, France, Poland, UK).
- 2012Everolimus regresses cardiac rhabdomyoma in tuberous sclerosis
Case series lead to medical management replacing surgery.
- 2017Pediatric MATCH opens
Molecular assignment platform for any refractory childhood solid tumour.
- 2020Systemic bevacizumab for severe respiratory papillomatosis reported
Open problems, and what is being done about each
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.
and how the field plans to fix it →What is being done about thisCost and accessAvailable now- HPV & HBV vaccinationStandard of care
- ImatinibApproved
- Trastuzumab biosimilarsApproved
In trials- Gefitinib vs gefitinib plus pemetrexed-carboplatin in EGFR-mutant lung cancer (Tata Memorial)Positive
- IARC India HPV vaccine dose study (one, two or three doses)Positive
- IMAGINE (varnimcabtagene autoleucel, Immuneel)Positive
- Low-dose nivolumab plus metronomic chemotherapy (Tata Memorial)Positive
- Low-dose olanzapine for cancer anorexia (Tata Memorial)Positive
- METRO PLUS (Tata Memorial Centre, Varanasi)Positive
Ideas and roadmaps- 90-day reliance approval for cancer drugs cleared by two stringent regulators
- A cheap old tablet to restore appetite
- A combination pricing rule so two-drug regimens are not priced as two monopolies
- A coordinated reserve and shared schedule for the world's medical isotope reactors
- A dedicated global financing window for cancer, modelled on the Global Fund
- A delinked market-entry reward paid by payers when a repurposed generic wins approval
Background: Accelerated approval, Biosimilar, Real-world evidence. Also on OnCo: Financial help and assistance navigator · Coverage by country · HTA decisions.
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Landmark trials in OnCo
Expert centres
topCentres linked to this cancer in OnCo
- Catalan Institute of Oncology (ICO)L'Hospitalet de Llobregat, ESvia HPV & HBV vaccination, Germline (hereditary) testing
- Children's Oncology Group (COG)Monrovia, CA, USvia this cancer, NCI-COG Pediatric MATCH (APEC1621)
- A.C. Camargo Cancer CenterSão Paulo, BRvia Germline (hereditary) testing
- All India Institute of Medical Sciences, New DelhiNew Delhi, INvia HPV & HBV vaccination
- Cancer Institute (WIA), AdyarChennai, INvia HPV & HBV vaccination
- via HPV & HBV vaccination
- Children's Cancer and Leukaemia GroupLeicester, GBvia this cancer
- Children's Hospital of PhiladelphiaPhiladelphia, PA, USvia Germline (hereditary) testing
- Department of Biotechnology, Government of IndiaNew Delhi, INvia HPV & HBV vaccination
- Dharmais National Cancer CenterJakarta, IDvia HPV & HBV vaccination
- Edinburgh Cancer Centre / CRUK Scotland CentreEdinburgh, GBvia Germline (hereditary) testing
- European Cancer OrganisationBrussels, BEvia HPV & HBV vaccination
- via Germline (hereditary) testing
- Gates FoundationSeattle, WA, USvia HPV & HBV vaccination
- German Cancer Research Center (DKFZ)Heidelberg, DEvia HPV & HBV vaccination
- via Germline (hereditary) testing
- Groote Schuur Hospital / University of Cape TownCape Town, ZAvia Germline (hereditary) testing
- Hadassah Medical CenterJerusalem, ILvia Germline (hereditary) testing
- via Germline (hereditary) testing
- Hospital de Amor (Barretos Cancer Hospital)Barretos, BRvia HPV & HBV vaccination
- Hospital de Clínicas de Porto AlegrePorto Alegre, BRvia Germline (hereditary) testing
- Hospital Universitari i Politècnic La FeValencia, ESvia Germline (hereditary) testing
- via Germline (hereditary) testing
- via Germline (hereditary) testing
- via HPV & HBV vaccination
- Innovative Therapies for Children with Cancer (ITCC)Villejuif, FRvia NCI-COG Pediatric MATCH (APEC1621)
- Institut National d'Oncologie, RabatRabat, MAvia HPV & HBV vaccination
- Institut PasteurParis, FRvia HPV & HBV vaccination
- Institute of Oncology LjubljanaLjubljana, SIvia HPV & HBV vaccination
- Instituto Nacional de Câncer (INCA)Rio de Janeiro, BRvia HPV & HBV vaccination
- via HPV & HBV vaccination
- Instituto Nacional de Cancerología (Mexico)Mexico City, MXvia HPV & HBV vaccination
- via HPV & HBV vaccination
- via Germline (hereditary) testing
- Kaiser Permanente Division of ResearchOakland, CA, USvia HPV & HBV vaccination
- Kenyatta National HospitalNairobi, KEvia HPV & HBV vaccination
- via Germline (hereditary) testing
- King Hussein Cancer CenterAmman, JOvia Germline (hereditary) testing
- Korle Bu Teaching HospitalAccra, GHvia Germline (hereditary) testing
- Lagos University Teaching HospitalLagos, NGvia Germline (hereditary) testing
- Leiden University Medical CenterLeiden, NLvia HPV & HBV vaccination
- via HPV & HBV vaccination
- MovemberMelbourne, AUvia Germline (hereditary) testing
- National Cancer Institute (NIH)Bethesda, MD, USvia NCI-COG Pediatric MATCH (APEC1621)
- National Taiwan University HospitalTaipei, TWvia HPV & HBV vaccination
- NCI Center for Cancer Research (intramural programme)Bethesda, MD, USvia HPV & HBV vaccination
- Ocean Road Cancer InstituteDar es Salaam, TZvia HPV & HBV vaccination
- Peking Union Medical College HospitalBeijing, CNvia Germline (hereditary) testing
- Philippine General HospitalManila, PHvia HPV & HBV vaccination
- QIMR Berghofer Medical Research InstituteBrisbane, AUvia Germline (hereditary) testing
- Ramathibodi Hospital, Mahidol UniversityBangkok, THvia Germline (hereditary) testing
- Shaare Zedek Medical CenterJerusalem, ILvia Germline (hereditary) testing
- Shizuoka Cancer CenterNagaizumi, Shizuoka, JPvia Germline (hereditary) testing
- via this cancer
- via Germline (hereditary) testing
- Society of Gynecologic OncologyChicago, IL, USvia HPV & HBV vaccination
- The Hospital for Sick Children (SickKids)Toronto, ON, CAvia Germline (hereditary) testing
- Tohoku University HospitalSendai, JPvia Germline (hereditary) testing
- via HPV & HBV vaccination
- Uganda Cancer InstituteKampala, UGvia HPV & HBV vaccination
- University of Malaya Medical CentreKuala Lumpur, MYvia Germline (hereditary) testing
- via HPV & HBV vaccination
- via HPV & HBV vaccination
Questions to ask
topQuestions to ask your oncologist about Rare cancers of childhood
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Why: Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example Germline predisposition testing, Tuberous sclerosis complexin cardiac rhabdomyoma, HPV 6/11 typing in respiratory papillomatosis, Tumour-specific adult markers borrowed by site), and what were the results?Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Why: Recognised subtypes for this cancer include Childhood cardiac tumours, Recurrent respiratory papillomatosis, Childhood melanoma and non-melanoma skin cancer.
- Is germline (inherited) genetic testing recommended for me or my family?Why: Inherited variants can change treatment and matter for relatives.
Cardiac rhabdomyoma with tuberous sclerosis
- For my situation (cardiac rhabdomyoma with tuberous sclerosis), which of the standard options do you recommend and why?Why: Guideline options include: Observation for spontaneous regression; mTOR inhibitor (everolimus or sirolimus) for haemodynamically significant tumours; surgery reserved for obstruction unresponsive to medical therapy.
- Am I a candidate for Everolimus, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Recurrent respiratory papillomatosis
- For my situation (recurrent respiratory papillomatosis), which of the standard options do you recommend and why?Why: Guideline options include: 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).
- Am I a candidate for Bevacizumab, Nonavalent HPV vaccine, and what side effects should I expect?Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
Adult-type carcinoma in a child
- For my situation (adult-type carcinoma in a child), which of the standard options do you recommend and why?Why: Guideline options include: 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.
- How do the results of NCI-COG Pediatric MATCH (APEC1621) apply to someone like me?Why: Trial populations differ from individual patients; ask how closely you match.
Any stage
- Are there clinical trials I could join, for example of NCI-COG Pediatric MATCH (APEC1621), Everolimus, Nonavalent HPV vaccine?Why: Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Why: Supportive care improves quality of life and helps patients complete treatment.
- I read that “Evidence remains observational for almost every entity; international registries and adult-paediatric joint protocols are the response”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
- I read that “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”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.
Direct links plus the targets, companies, and technologies of this cancer's products.
technologies
6targets
3drugs
3companies
4institutions
3terms
3trials
1bottlenecks
2Latest papers
topQuery for this cancer: (TITLE:"Rare cancers of childhood" OR ABSTRACT:"Rare cancers of childhood" OR TITLE:"NCI PDQ umbrella" OR ABSTRACT:"NCI PDQ umbrella" OR TITLE:"Childhood cardiac tumours" OR ABSTRACT:"Childhood cardiac tumours" OR TITLE:"Laryngeal papillomatosis" OR ABSTRACT:"Laryngeal papillomatosis" OR TITLE:"Recurrent respiratory papillomatosis" OR ABSTRACT:"Recurrent respiratory papillomatosis" OR TITLE:"Childhood melanoma" OR ABSTRACT:"Childhood melanoma") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Rare cancers of childhood (NCI PDQ umbrella), not a curated reading list.
Pages like this
not linked directly; found by shared links- InstitutionInnovative Therapies for Children with Cancer (ITCC)
Shares RACE for Children Act, NCI-COG Pediatric MATCH (APEC1621), SIOP Europe – European Society for Paediatric Oncology, Trials enrol too few, too slowly and the tags nci-coverage, paediatric.
- InstitutionACCELERATE
Shares RACE for Children Act, SIOP Europe – European Society for Paediatric Oncology, Rare and paediatric cancers without markets and the tags nci-coverage, paediatric.
- CancerPaediatric low-grade glioma
Shares Children's Cancer and Leukaemia Group, RACE for Children Act, NCI-COG Pediatric MATCH (APEC1621), SIOP Europe – European Society for Paediatric Oncology and the tags nci-coverage, paediatric.
- CancerAtypical teratoid/rhabdoid tumour (ATRT)
Shares NCI-COG Pediatric MATCH (APEC1621), SIOP Europe – European Society for Paediatric Oncology, Hereditary cancer syndromes, Children's Oncology Group (COG) and the tags nci-coverage, paediatric.
- InstitutionHistiocyte Society
Shares Rare and paediatric cancers without markets and the tags nci-coverage, paediatric.
- CancerDiffuse midline glioma, H3 K27-altered (including DIPG)
Shares RACE for Children Act, NCI-COG Pediatric MATCH (APEC1621), SIOP Europe – European Society for Paediatric Oncology, Children's Oncology Group (COG) and the tags nci-coverage, paediatric.
- CancerEpendymoma
Shares NCI-COG Pediatric MATCH (APEC1621), SIOP Europe – European Society for Paediatric Oncology, Children's Oncology Group (COG), Rare and paediatric cancers without markets and the tags nci-coverage, paediatric.
- TrialACTION
Shares RACE for Children Act, Rare and paediatric cancers without markets and the tags nci-coverage, paediatric.