# Wilms tumour (nephroblastoma)

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

## TL;DR

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).

Favourable-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).

## Fields

- Kind: Cancer
- Last checked: 2026-09-08
- Tags: gap-fill; paediatric
- 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

## 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/wilms-tumor/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/wilms-tumor/#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/wilms-tumor/#what-it-is [6 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/wilms-tumor/#finding-it [7 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/wilms-tumor/#treating-it [4 settings, 3 decisions 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/wilms-tumor/#evidence [3 trials, 1 key paper, 7 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/wilms-tumor/#science [10 targets, 2 pathways]
- 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/wilms-tumor/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/wilms-tumor/#living-with-it [16 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/wilms-tumor/coming/ [7 medicines, 3 trials, 4 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/wilms-tumor/data/ [53 connected records]

## Standard of care

- Very low risk (stage I FH, <2 years, <550 g): Nephrectomy alone with close surveillance (AREN0532). ([Active surveillance](https://onco.cc/technologies/active-surveillance/))
- Stage I-II favourable histology: Nephrectomy then vincristine + actinomycin D for 18 weeks (EE-4A), or SIOP pre-op VA ×4 weeks then stage-adapted post-op therapy. ([Vincristine](https://onco.cc/drugs/vincristine/), [Dactinomycin (actinomycin D)](https://onco.cc/drugs/dactinomycin/))
- Stage III-IV favourable histology: 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). ([Vincristine](https://onco.cc/drugs/vincristine/), [Dactinomycin (actinomycin D)](https://onco.cc/drugs/dactinomycin/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [IMRT / IGRT (modern external beam)](https://onco.cc/technologies/imrt-igrt/))
- Diffuse anaplastic or relapsed: Intensive UH-1/UH-2 (vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide) with radiotherapy; relapse: ICE-type regimens, surgery, RT, high-dose therapy or trials. ([Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Carboplatin](https://onco.cc/drugs/carboplatin/), [Etoposide](https://onco.cc/drugs/etoposide/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [Ifosfamide](https://onco.cc/drugs/ifosfamide/))

## State of the art

- 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).

## Open problems

- 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.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Wilms%27_tumor
- NCI PDQ: Wilms tumour: https://www.cancer.gov/types/kidney/patient/wilms-treatment-pdq
- SIOP-RTSG UMBRELLA protocol (Nat Rev Urol 2017): https://doi.org/10.1038/nrurol.2017.163
- Children's Oncology Group: https://childrensoncologygroup.org/

## Connected records

- roadmaps: [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)
- cancers: [Atypical teratoid/rhabdoid tumour (ATRT)](https://onco.cc/cancers/atrt/), [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/), [Hepatoblastoma](https://onco.cc/cancers/hepatoblastoma/), [Hyperparathyroidism-jaw tumour syndrome (CDC73-related parathyroid carcinoma)](https://onco.cc/cancers/hyperparathyroidism-jaw-tumour-syndrome/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/), [Retinoblastoma](https://onco.cc/cancers/retinoblastoma/), [TFE3-rearranged (translocation) renal cell carcinoma](https://onco.cc/cancers/tfe3-rearranged-renal-cell-carcinoma/)
- technologies: [Active surveillance](https://onco.cc/technologies/active-surveillance/), [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [Germline (hereditary) testing](https://onco.cc/technologies/germline-testing/), [Global oncology and access in low- and middle-income countries](https://onco.cc/technologies/global-oncology-access/), [IMRT / IGRT (modern external beam)](https://onco.cc/technologies/imrt-igrt/), [Robotic & minimally invasive surgery](https://onco.cc/technologies/robotic-surgery/), [Survivorship care and late-effects surveillance](https://onco.cc/technologies/survivorship-care-plan/), [Ultrasound](https://onco.cc/technologies/ultrasound/)
- targets: [AMER1](https://onco.cc/targets/amer1/), [CTR9](https://onco.cc/targets/ctr9/), [DGCR8](https://onco.cc/targets/dgcr8/), [MLLT1](https://onco.cc/targets/mllt1/), [REST](https://onco.cc/targets/rest/), [SIX1](https://onco.cc/targets/six1/), [TP53](https://onco.cc/targets/tp53/), [TRIM28](https://onco.cc/targets/trim28/), [WT1](https://onco.cc/targets/wt1/)
- drugs: [Carboplatin](https://onco.cc/drugs/carboplatin/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Dactinomycin (actinomycin D)](https://onco.cc/drugs/dactinomycin/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [Etoposide](https://onco.cc/drugs/etoposide/), [Ifosfamide](https://onco.cc/drugs/ifosfamide/), [Vincristine](https://onco.cc/drugs/vincristine/)
- companies: [Children's Oncology Group (COG)](https://onco.cc/companies/childrens-oncology-group/)
- pathways: [p53 / RB / cell-cycle checkpoint](https://onco.cc/pathways/p53-cell-cycle/), [Wnt / β-catenin](https://onco.cc/pathways/wnt/)
- terms: [Germline vs somatic mutations](https://onco.cc/terms/germline-vs-somatic/), [Nephrectomy](https://onco.cc/terms/nephrectomy/), [Wilms tumour risk markers (anaplasia, 1p/16q loss, 1q gain, SIOP and COG risk groups)](https://onco.cc/terms/wilms-risk-markers/)
- trials: [Childhood Cancer Survivor Study (CCSS)](https://onco.cc/trials/ccss/), [COG AREN0532](https://onco.cc/trials/aren0532/), [COG AREN0533](https://onco.cc/trials/aren0533/)
- people: [Jeffrey S. Dome](https://onco.cc/people/jeffrey-dome/), [Sidney Farber](https://onco.cc/people/sidney-farber/)
- key papers: [Position paper: Rationale for the treatment of Wilms tumour in the UMBRELLA SIOP-RTSG 2016 protocol](https://onco.cc/key-papers/paper-van-den-heuvel-eibrink-nat-rev-urol/)
- journals: [Journal of pediatric hematology/oncology](https://onco.cc/journals/journal-of-pediatric-hematology-oncology/), [Pediatric hematology and oncology](https://onco.cc/journals/pediatric-hematology-and-oncology/)
- collections: [Alex's Lemonade Stand Foundation (ALSF)](https://onco.cc/collections/alexs-lemonade-stand/), [St. Baldrick's Foundation](https://onco.cc/collections/st-baldricks/)
- institutions: [Children's National Hospital](https://onco.cc/institutions/childrens-national-hospital/)

---
JSON: https://onco.cc/api/v1/entities/wilms-tumor.json