{"entity":{"id":"rare-childhood-cancers","kind":"cancer","name":"Rare cancers of childhood (NCI PDQ umbrella)","aka":["Childhood cardiac tumours","Laryngeal papillomatosis","Recurrent respiratory papillomatosis","Childhood melanoma","Childhood carcinomas","Very rare tumours of childhood"],"tldr":"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.\n\nTwo 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.\n\nThe 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.","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Childhood_cancer","links":[{"label":"NCI PDQ: rare cancers of childhood","url":"https://www.cancer.gov/types/childhood-cancers/patient/rare-childhood-cancers-pdq"},{"label":"NCI PDQ: childhood cardiac tumours","url":"https://www.cancer.gov/types/cardiac/patient-child-cardiac-treatment-pdq"},{"label":"EXPeRT: European Cooperative Study Group for Paediatric Rare Tumours","url":"https://www.raretumors-children.eu/"}],"tags":["nci-coverage","paediatric","umbrella"],"related":["nasopharyngeal","thyroid","adrenocortical","melanoma","pleuropulmonary-blastoma","nut-carcinoma","sinonasal","pheochromocytoma-paraganglioma","multiple-endocrine-neoplasia","race-for-children-act","paediatric-germ-cell-tumours","retinoblastoma","hepatoblastoma"],"cancers":[],"sections":[],"technologies":["hpv-vaccine","germline-testing","cancer-registries-surveillance"],"targets":[],"drugs":["everolimus","bevacizumab","gardasil-9"],"companies":["shanghai-fosun-pharmaceutical-industrial-development","shanghai-kechow-pharma","childrens-oncology-group","cclg"],"institutions":["siop-europe"],"pathways":[],"terms":["hereditary-cancer-syndromes","aya-oncology"],"trials":["pediatric-match","nct07407803"],"people":[],"bottlenecks":["b-rare-cancers","b-trial-enrolment"],"keyPapers":[],"journals":["journal-of-pediatric-hematology-oncology","journal-of-pediatric-hematology-oncology-nursing","pediatric-hematology-and-oncology"],"dependsOn":[],"notes":[],"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"],"standardOfCare":[{"setting":"Cardiac rhabdomyoma with tuberous sclerosis","approach":"Observation for spontaneous regression; mTOR inhibitor (everolimus or sirolimus) for haemodynamically significant tumours; surgery reserved for obstruction unresponsive to medical therapy.","refs":["everolimus"],"guideline":{"version":"NCI PDQ: childhood cardiac tumours","url":"https://www.cancer.gov/types/cardiac/patient-child-cardiac-treatment-pdq"}},{"setting":"Recurrent respiratory papillomatosis","approach":"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).","refs":["bevacizumab","gardasil-9","hpv-vaccine"],"guideline":{"version":"NCI PDQ: laryngeal papillomatosis (rare cancers of childhood)","url":"https://www.cancer.gov/types/childhood-cancers/patient/rare-childhood-cancers-pdq"}},{"setting":"Adult-type carcinoma in a child","approach":"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.","refs":["germline-testing","pediatric-match"],"guideline":{"version":"EXPeRT consensus recommendations; NCI PDQ","url":"https://www.cancer.gov/types/childhood-cancers/patient/rare-childhood-cancers-pdq"}}],"stateOfArt":["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."],"history":[{"year":2000,"title":"COG Rare Tumors committee established","note":"Creates a registry and protocols for infrequent tumours.","refs":[]},{"year":2008,"title":"EXPeRT founded","note":"European Cooperative Study Group for Paediatric Rare Tumours pools national registries (Italy, Germany, France, Poland, UK).","refs":[]},{"year":2012,"title":"Everolimus regresses cardiac rhabdomyoma in tuberous sclerosis","note":"Case series lead to medical management replacing surgery.","refs":["everolimus"]},{"year":2017,"title":"Pediatric MATCH opens","note":"Molecular assignment platform for any refractory childhood solid tumour.","refs":["pediatric-match"]},{"year":2020,"title":"Systemic bevacizumab for severe respiratory papillomatosis reported","refs":["bevacizumab"]}],"pipeline":["pediatric-match","everolimus","gardasil-9","tq-b3234"],"openProblems":["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."],"parent":"childhood-cancers"},"route":"/cancers/rare-childhood-cancers/","neighbours":{"cancer":[{"id":"adrenocortical","kind":"cancer","name":"Adrenocortical carcinoma","route":"/cancers/adrenocortical/"},{"id":"childhood-cancers","kind":"cancer","name":"Childhood cancers (all types)","route":"/cancers/childhood-cancers/"},{"id":"pleuropulmonary-blastoma","kind":"cancer","name":"Childhood lung and airway tumours (pleuropulmonary blastoma, tracheobronchial tumours)","route":"/cancers/pleuropulmonary-blastoma/"},{"id":"paediatric-germ-cell-tumours","kind":"cancer","name":"Germ cell tumours of childhood and adolescence (extracranial and CNS)","route":"/cancers/paediatric-germ-cell-tumours/"},{"id":"hepatoblastoma","kind":"cancer","name":"Hepatoblastoma","route":"/cancers/hepatoblastoma/"},{"id":"melanoma","kind":"cancer","name":"Melanoma","route":"/cancers/melanoma/"},{"id":"multiple-endocrine-neoplasia","kind":"cancer","name":"Multiple endocrine neoplasia syndromes (MEN1, MEN2, MEN4)","route":"/cancers/multiple-endocrine-neoplasia/"},{"id":"sinonasal","kind":"cancer","name":"Nasal cavity and paranasal sinus cancers (including esthesioneuroblastoma)","route":"/cancers/sinonasal/"},{"id":"nasopharyngeal","kind":"cancer","name":"Nasopharyngeal carcinoma","route":"/cancers/nasopharyngeal/"},{"id":"nut-carcinoma","kind":"cancer","name":"NUT carcinoma (midline carcinoma with NUTM1 rearrangement)","route":"/cancers/nut-carcinoma/"},{"id":"pancreatoblastoma","kind":"cancer","name":"Pancreatoblastoma","route":"/cancers/pancreatoblastoma/"},{"id":"pheochromocytoma-paraganglioma","kind":"cancer","name":"Pheochromocytoma and paraganglioma (PPGL)","route":"/cancers/pheochromocytoma-paraganglioma/"},{"id":"retinoblastoma","kind":"cancer","name":"Retinoblastoma","route":"/cancers/retinoblastoma/"},{"id":"thyroid","kind":"cancer","name":"Thyroid cancer","route":"/cancers/thyroid/"}],"term":[{"id":"aya-oncology","kind":"term","name":"Adolescent and young adult (AYA) oncology","route":"/terms/aya-oncology/"},{"id":"hereditary-cancer-syndromes","kind":"term","name":"Hereditary cancer syndromes","route":"/terms/hereditary-cancer-syndromes/"},{"id":"race-for-children-act","kind":"term","name":"RACE for Children Act","route":"/terms/race-for-children-act/"},{"id":"sdh-deficiency","kind":"term","name":"SDH deficiency (SDHB immunohistochemistry loss)","route":"/terms/sdh-deficiency/"}],"technology":[{"id":"cancer-registries-surveillance","kind":"technology","name":"Cancer registries and population surveillance","route":"/technologies/cancer-registries-surveillance/"},{"id":"germline-testing","kind":"technology","name":"Germline (hereditary) testing","route":"/technologies/germline-testing/"},{"id":"hpv-vaccine","kind":"technology","name":"HPV & HBV vaccination","route":"/technologies/hpv-vaccine/"}],"drug":[{"id":"bevacizumab","kind":"drug","name":"Bevacizumab","route":"/drugs/bevacizumab/"},{"id":"everolimus","kind":"drug","name":"Everolimus","route":"/drugs/everolimus/"},{"id":"gardasil-9","kind":"drug","name":"Nonavalent HPV vaccine","route":"/drugs/gardasil-9/"},{"id":"tq-b3234","kind":"drug","name":"TQ-B3234","route":"/drugs/tq-b3234/"}],"company":[{"id":"cclg","kind":"company","name":"Children's Cancer and Leukaemia Group","route":"/companies/cclg/"},{"id":"childrens-oncology-group","kind":"company","name":"Children's Oncology Group (COG)","route":"/companies/childrens-oncology-group/"},{"id":"precigen","kind":"company","name":"Precigen","route":"/companies/precigen/"},{"id":"shanghai-fosun-pharmaceutical-industrial-development","kind":"company","name":"Shanghai Fosun Pharmaceutical Industrial Development","route":"/companies/shanghai-fosun-pharmaceutical-industrial-development/"},{"id":"shanghai-kechow-pharma","kind":"company","name":"Shanghai Kechow Pharma","route":"/companies/shanghai-kechow-pharma/"}],"institution":[{"id":"siop-europe","kind":"institution","name":"SIOP Europe (European Society for Paediatric Oncology)","route":"/institutions/siop-europe/"}],"trial":[{"id":"nct04724980","kind":"trial","name":"Adjuvant PRGN-2012 in Adult Patients With Recurrent Respiratory Papillomatosis","route":"/trials/nct04724980/"},{"id":"determine-trial","kind":"trial","name":"DETERMINE","route":"/trials/determine-trial/"},{"id":"nct07407803","kind":"trial","name":"Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas","route":"/trials/nct07407803/"},{"id":"pediatric-match","kind":"trial","name":"NCI-COG Pediatric MATCH (APEC1621)","route":"/trials/pediatric-match/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"},{"id":"b-trial-enrolment","kind":"bottleneck","name":"Trials enrol too few, too slowly","route":"/bottlenecks/b-trial-enrolment/"}],"journal":[{"id":"journal-of-pediatric-hematology-oncology","kind":"journal","name":"Journal of pediatric hematology/oncology","route":"/journals/journal-of-pediatric-hematology-oncology/"},{"id":"journal-of-pediatric-hematology-oncology-nursing","kind":"journal","name":"Journal of pediatric hematology/oncology nursing","route":"/journals/journal-of-pediatric-hematology-oncology-nursing/"},{"id":"pediatric-hematology-and-oncology","kind":"journal","name":"Pediatric hematology and oncology","route":"/journals/pediatric-hematology-and-oncology/"}]}}