{"entity":{"id":"wilms-tumor","kind":"cancer","name":"Wilms tumour (nephroblastoma)","aka":[],"tldr":"Wilms tumour is a kidney cancer of young children and one of paediatric oncology's success stories: surgery plus a few months of chemotherapy cures about nine in ten. Today's trials aim to give the lowest-risk children almost no chemotherapy while finding the few with aggressive biology.","summary":"Wilms tumour is an embryonal kidney cancer arising from nephrogenic rests, associated with WT1, CTNNB1, WTX, TP53 (anaplastic), and microRNA-processing gene (DROSHA, DGCR8) mutations, and with predisposition syndromes (WAGR, Denys-Drash, Beckwith-Wiedemann). Two cooperative-group philosophies coexist: COG (upfront nephrectomy, then risk-stratified chemotherapy using stage, histology, 1p/16q loss of heterozygosity and 1q gain) and SIOP (pre-operative vincristine-actinomycin then nephrectomy, with post-operative therapy by histologic response and stage; UMBRELLA protocol).\n\nFavourable-histology stage I-II disease is treated with vincristine and actinomycin D (EE-4A) or, for very low-risk stage I tumours in children under 2 with tumours <550 g, surgery alone; stage III-IV adds doxorubicin and flank/whole-lung radiotherapy, with lung irradiation omitted in rapid complete responders without 1p/16q LOH (AREN0533). Diffuse anaplastic tumours need intensive regimen UH-1/UH-2 with carboplatin, cyclophosphamide and etoposide; bilateral tumours receive neoadjuvant chemotherapy and nephron-sparing surgery. Relapse is treated by risk group (ICE regimens, high-dose chemotherapy in some). Survivorship issues include cardiotoxicity, renal function, second cancers and fertility (radiation).","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Wilms%27_tumor","links":[{"label":"NCI PDQ: Wilms tumour","url":"https://www.cancer.gov/types/kidney/patient/wilms-treatment-pdq"},{"label":"SIOP-RTSG UMBRELLA protocol (Nat Rev Urol 2017)","url":"https://doi.org/10.1038/nrurol.2017.163"},{"label":"Children's Oncology Group","url":"https://childrensoncologygroup.org/"}],"tags":["gap-fill","paediatric"],"related":["paediatric-oncology-roadmap","neuroblastoma","hepatoblastoma","retinoblastoma","pleuropulmonary-blastoma"],"cancers":[],"sections":[],"technologies":["cytotoxic-chemotherapy","imrt-igrt","robotic-surgery","germline-testing","survivorship-care-plan","ultrasound"],"targets":["tp53"],"drugs":["vincristine","dactinomycin","doxorubicin","cyclophosphamide","carboplatin","etoposide","ifosfamide"],"companies":["childrens-oncology-group"],"institutions":[],"pathways":["wnt","p53-cell-cycle"],"terms":["germline-vs-somatic","nephrectomy","wilms-risk-markers"],"trials":["ccss","aren0532"],"people":["jeffrey-dome"],"bottlenecks":[],"keyPapers":["paper-van-den-heuvel-eibrink-nat-rev-urol"],"journals":["journal-of-pediatric-hematology-oncology","pediatric-hematology-and-oncology"],"dependsOn":[],"notes":[],"group":"paediatric","burden":"The most common childhood kidney cancer: ~1 in 10,000 children, ~650 cases per year in the US, peak age 3-4; overall survival ~90% in high-income countries versus ~50% or lower in much of Africa.","subtypes":["Favourable histology (~90%)","Diffuse anaplastic (TP53-mutant, ~5-10%)","Focal anaplastic","Bilateral Wilms (stage V, ~5%)","Syndromic Wilms (WAGR, Denys-Drash, Beckwith-Wiedemann)","Non-Wilms renal tumours (clear cell sarcoma, rhabdoid, renal cell) are managed separately"],"biomarkers":["Stage (COG or SIOP post-chemotherapy)","Histology: anaplasia; SIOP risk group after pre-op chemotherapy (blastemal-type = high risk)","1p and 16q loss of heterozygosity (COG)","1q gain","TP53 mutation (anaplastic)","Tumour weight and age (surgery-only eligibility)","Germline WT1 / 11p15 testing"],"standardOfCare":[{"setting":"Very low risk (stage I FH, <2 years, <550 g)","approach":"Nephrectomy alone with close surveillance (AREN0532).","refs":["active-surveillance"],"guideline":{"version":"COG AREN0532 (JCO 2019)","url":"https://doi.org/10.1200/JCO.18.02102"}},{"setting":"Stage I-II favourable histology","approach":"Nephrectomy then vincristine + actinomycin D for 18 weeks (EE-4A), or SIOP pre-op VA ×4 weeks then stage-adapted post-op therapy.","refs":["vincristine","dactinomycin"],"guideline":{"version":"COG AREN0532; SIOP-RTSG UMBRELLA"}},{"setting":"Stage III-IV favourable histology","approach":"Vincristine, actinomycin D, doxorubicin (DD-4A) for 24 weeks; flank/abdominal radiotherapy for stage III; whole-lung radiotherapy for lung metastases not in rapid complete response (AREN0533).","refs":["vincristine","dactinomycin","doxorubicin","imrt-igrt"],"guideline":{"version":"COG AREN0533 (JCO 2018)"}},{"setting":"Diffuse anaplastic or relapsed","approach":"Intensive UH-1/UH-2 (vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide) with radiotherapy; relapse: ICE-type regimens, surgery, RT, high-dose therapy or trials.","refs":["cyclophosphamide","carboplatin","etoposide","doxorubicin","ifosfamide"],"guideline":{"version":"COG AREN0321"}}],"stateOfArt":["Survival ~90% with steadily less therapy: surgery-only for the youngest lowest-risk children, radiation omitted for lung responders.","Molecular markers (1p/16q LOH, 1q gain) already stratify therapy, a rare achievement in paediatric solid tumours.","Two cooperative strategies (COG vs SIOP) reach similar outcomes, giving the field a natural experiment.","The largest gap is geographic: mortality in sub-Saharan Africa is several-fold higher, driven by late presentation, abandonment and supportive-care shortfalls (SIOP PODC adapted regimens)."],"history":[{"year":1899,"title":"Max Wilms's monograph on mixed tumours of the kidney","refs":[]},{"year":1956,"title":"Actinomycin D active in Wilms tumour (Farber)","note":"Combined with surgery and radiation, cure rates begin to climb.","refs":["dactinomycin"]},{"year":1969,"title":"National Wilms Tumor Study (NWTS-1) begins","note":"Cooperative-group model that raised survival from ~30% to ~90% over five trials.","refs":[]},{"year":1990,"title":"WT1 cloned (Call, Gessler)","note":"First Wilms tumour gene; also links to WAGR and Denys-Drash.","refs":[]},{"year":2005,"title":"1p/16q LOH predicts relapse (NWTS-5, Grundy)","refs":[]},{"year":2018,"title":"AREN0533: lung radiotherapy omitted for rapid complete responders","refs":["imrt-igrt"]},{"year":2019,"title":"AREN0532: surgery alone confirmed for very-low-risk stage I","refs":["active-surveillance"]}],"pipeline":["vincristine","dactinomycin"],"openProblems":["Diffuse anaplastic and relapsed disease: survival ~50% or lower.","Global inequity: Wilms is curable, yet most children with it worldwide lack access to the treatment that cures it; adapted regimens (SIOP PODC) are the response.","Late effects of doxorubicin and radiation in 90% survivors.","Bilateral disease: preserving kidney function."],"parent":"childhood-cancers"},"route":"/cancers/wilms-tumor/","neighbours":{"roadmap":[{"id":"paediatric-oncology-roadmap","kind":"roadmap","name":"Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first","route":"/roadmaps/paediatric-oncology-roadmap/"}],"cancer":[{"id":"atrt","kind":"cancer","name":"Atypical teratoid/rhabdoid tumour (ATRT)","route":"/cancers/atrt/"},{"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":"hepatoblastoma","kind":"cancer","name":"Hepatoblastoma","route":"/cancers/hepatoblastoma/"},{"id":"hyperparathyroidism-jaw-tumour-syndrome","kind":"cancer","name":"Hyperparathyroidism-jaw tumour syndrome (CDC73-related parathyroid carcinoma)","route":"/cancers/hyperparathyroidism-jaw-tumour-syndrome/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"},{"id":"retinoblastoma","kind":"cancer","name":"Retinoblastoma","route":"/cancers/retinoblastoma/"},{"id":"tfe3-rearranged-renal-cell-carcinoma","kind":"cancer","name":"TFE3-rearranged (translocation) renal cell carcinoma","route":"/cancers/tfe3-rearranged-renal-cell-carcinoma/"}],"technology":[{"id":"active-surveillance","kind":"technology","name":"Active surveillance","route":"/technologies/active-surveillance/"},{"id":"rejuv-paed-breast-after-chest-radiotherapy","kind":"technology","name":"Breast cancer after chest radiotherapy in childhood, and the screening that follows","route":"/technologies/rejuv-paed-breast-after-chest-radiotherapy/"},{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"},{"id":"germline-testing","kind":"technology","name":"Germline (hereditary) testing","route":"/technologies/germline-testing/"},{"id":"global-oncology-access","kind":"technology","name":"Global oncology and access in low- and middle-income countries","route":"/technologies/global-oncology-access/"},{"id":"rejuv-paed-chronic-disease-burden","kind":"technology","name":"How much illness childhood cancer survivors carry, and at what age","route":"/technologies/rejuv-paed-chronic-disease-burden/"},{"id":"imrt-igrt","kind":"technology","name":"IMRT / IGRT (modern external beam)","route":"/technologies/imrt-igrt/"},{"id":"rejuv-paed-kidneys","kind":"technology","name":"Kidneys after cisplatin, ifosfamide, radiotherapy and nephrectomy in childhood","route":"/technologies/rejuv-paed-kidneys/"},{"id":"rejuv-paed-late-mortality","kind":"technology","name":"Late deaths after childhood cancer, what causes them, and the proof that gentler treatment worked","route":"/technologies/rejuv-paed-late-mortality/"},{"id":"rejuv-paed-uk-long-term-follow-up","kind":"technology","name":"Long-term follow-up in the United Kingdom: what a survivor is actually offered","route":"/technologies/rejuv-paed-uk-long-term-follow-up/"},{"id":"robotic-surgery","kind":"technology","name":"Robotic & minimally invasive surgery","route":"/technologies/robotic-surgery/"},{"id":"rejuv-paed-second-cancers","kind":"technology","name":"Second cancers after childhood cancer: the risk by treatment, and why it is falling","route":"/technologies/rejuv-paed-second-cancers/"},{"id":"survivorship-care-plan","kind":"technology","name":"Survivorship care and late-effects surveillance","route":"/technologies/survivorship-care-plan/"},{"id":"rejuv-paed-cog-ltfu-guidelines","kind":"technology","name":"The Children's Oncology Group Long-Term Follow-Up Guidelines","route":"/technologies/rejuv-paed-cog-ltfu-guidelines/"},{"id":"rejuv-paed-heart","kind":"technology","name":"The heart after anthracyclines and chest radiotherapy in childhood","route":"/technologies/rejuv-paed-heart/"},{"id":"ultrasound","kind":"technology","name":"Ultrasound","route":"/technologies/ultrasound/"}],"target":[{"id":"amer1","kind":"target","name":"AMER1","route":"/targets/amer1/"},{"id":"ctr9","kind":"target","name":"CTR9","route":"/targets/ctr9/"},{"id":"dgcr8","kind":"target","name":"DGCR8","route":"/targets/dgcr8/"},{"id":"mllt1","kind":"target","name":"MLLT1","route":"/targets/mllt1/"},{"id":"rest","kind":"target","name":"REST","route":"/targets/rest/"},{"id":"six1","kind":"target","name":"SIX1","route":"/targets/six1/"},{"id":"tp53","kind":"target","name":"TP53","route":"/targets/tp53/"},{"id":"trim28","kind":"target","name":"TRIM28","route":"/targets/trim28/"},{"id":"wt1","kind":"target","name":"WT1","route":"/targets/wt1/"}],"drug":[{"id":"carboplatin","kind":"drug","name":"Carboplatin","route":"/drugs/carboplatin/"},{"id":"cyclophosphamide","kind":"drug","name":"Cyclophosphamide","route":"/drugs/cyclophosphamide/"},{"id":"dactinomycin","kind":"drug","name":"Dactinomycin (actinomycin D)","route":"/drugs/dactinomycin/"},{"id":"doxorubicin","kind":"drug","name":"Doxorubicin","route":"/drugs/doxorubicin/"},{"id":"etoposide","kind":"drug","name":"Etoposide","route":"/drugs/etoposide/"},{"id":"ifosfamide","kind":"drug","name":"Ifosfamide","route":"/drugs/ifosfamide/"},{"id":"vincristine","kind":"drug","name":"Vincristine","route":"/drugs/vincristine/"}],"company":[{"id":"childrens-oncology-group","kind":"company","name":"Children's Oncology Group (COG)","route":"/companies/childrens-oncology-group/"}],"pathway":[{"id":"p53-cell-cycle","kind":"pathway","name":"p53 / RB / cell-cycle checkpoint","route":"/pathways/p53-cell-cycle/"},{"id":"wnt","kind":"pathway","name":"Wnt / β-catenin","route":"/pathways/wnt/"}],"term":[{"id":"germline-vs-somatic","kind":"term","name":"Germline vs somatic mutations","route":"/terms/germline-vs-somatic/"},{"id":"nephrectomy","kind":"term","name":"Nephrectomy","route":"/terms/nephrectomy/"},{"id":"wilms-risk-markers","kind":"term","name":"Wilms tumour risk markers (anaplasia, 1p/16q loss, 1q gain, SIOP and COG risk groups)","route":"/terms/wilms-risk-markers/"}],"trial":[{"id":"ccss","kind":"trial","name":"Childhood Cancer Survivor Study (CCSS)","route":"/trials/ccss/"},{"id":"aren0532","kind":"trial","name":"COG AREN0532","route":"/trials/aren0532/"},{"id":"aren0533","kind":"trial","name":"COG AREN0533","route":"/trials/aren0533/"}],"person":[{"id":"jeffrey-dome","kind":"person","name":"Jeffrey S. Dome","route":"/people/jeffrey-dome/"},{"id":"sidney-farber","kind":"person","name":"Sidney Farber","route":"/people/sidney-farber/"}],"paper":[{"id":"paper-van-den-heuvel-eibrink-nat-rev-urol","kind":"paper","name":"Position paper: Rationale for the treatment of Wilms tumour in the UMBRELLA SIOP-RTSG 2016 protocol","route":"/key-papers/paper-van-den-heuvel-eibrink-nat-rev-urol/"}],"journal":[{"id":"journal-of-pediatric-hematology-oncology","kind":"journal","name":"Journal of pediatric hematology/oncology","route":"/journals/journal-of-pediatric-hematology-oncology/"},{"id":"pediatric-hematology-and-oncology","kind":"journal","name":"Pediatric hematology and oncology","route":"/journals/pediatric-hematology-and-oncology/"}],"collection":[{"id":"alexs-lemonade-stand","kind":"collection","name":"Alex's Lemonade Stand Foundation (ALSF)","route":"/collections/alexs-lemonade-stand/"},{"id":"st-baldricks","kind":"collection","name":"St. Baldrick's Foundation","route":"/collections/st-baldricks/"}],"institution":[{"id":"childrens-national-hospital","kind":"institution","name":"Children's National Hospital","route":"/institutions/childrens-national-hospital/"}]}}