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Wilms tumour (nephroblastoma)

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.

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).

State of the art today

  • 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.
Show survival figures (2)

Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

  • Survival ~90% with steadily less therapy: surgery-only for the youngest lowest-risk children, radiation omitted for lung responders.
  • 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).
Who it affects
Show survival figures (1)

Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

  • 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.

Where the cases are

Cases by country · GLOBOCAN 2022
All countries →

No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.

Standard of care

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Very low risk (stage I FH, <2 years, <550 g)

Nephrectomy alone with close surveillance (AREN0532).

COG AREN0532 (JCO 2019)
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.

COG AREN0532; SIOP-RTSG UMBRELLA
VincristineDactinomycin (actinomycin D)
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).

COG AREN0533 (JCO 2018)
VincristineDactinomycin (actinomycin D)DoxorubicinIMRT / IGRT (modern external beam)
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.

COG AREN0321
CyclophosphamideCarboplatinEtoposideDoxorubicinIfosfamide

Subtypes & biomarkers

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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 clinicians test

Target prevalence in this cancer

History

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  1. 1899Max Wilms's monograph on mixed tumours of the kidney
  2. 1956Actinomycin D active in Wilms tumour (Farber)

    Combined with surgery and radiation, cure rates begin to climb.

    Dactinomycin (actinomycin D)
  3. 1969National Wilms Tumor Study (NWTS-1) begins

    Cooperative-group model that raised survival from ~30% to ~90% over five trials.

  4. 1990WT1 cloned (Call, Gessler)

    First Wilms tumour gene; also links to WAGR and Denys-Drash.

  5. 20051p/16q LOH predicts relapse (NWTS-5, Grundy)
  6. 2018AREN0533: lung radiotherapy omitted for rapid complete responders
    IMRT / IGRT (modern external beam)
  7. 2019AREN0532: surgery alone confirmed for very-low-risk stage I
    Active surveillance

Pipeline

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Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo
Not mapped hereActinomycin antibiotic (transcription inhibitor)
Dactinomycin (actinomycin D) · Cosmegen

Open problems

  • Diffuse anaplastic and relapsed disease: survival ~50% or lower.
  • Global inequity: Wilms is curable, yet most children with it worldwide die.
  • Late effects of doxorubicin and radiation in 90% survivors.
  • Bilateral disease: preserving kidney function.

Trials

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Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Wilms tumour (nephroblastoma)
condition: Wilms tumor
Open on ClinicalTrials.gov →

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Expert centres

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Where the expertise is

Centres linked to this cancer in OnCo

Seeking a second opinion: ask your oncologist for a referral to a high-volume centre; most accept records and pathology by mail or telehealth. In the US, use the NCI's Find a Cancer Center tool or the nonprofit Cancer Commons, which navigates options for advanced cancers at no cost.

Questions to ask

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Bring to your appointment

Questions to ask your oncologist about Wilms tumour

Generated from this cancer's standard of care, biomarkers, and pipeline · 16 questions

Newly diagnosed

  1. What is my exact diagnosis, stage, and grade, and which tests established them?
    Why: Everything else follows from an accurate stage and subtype.
  2. Which biomarkers have been tested on my tumour (for example Stage, Histology: anaplasia; SIOP risk group after pre-op chemotherapy, 1p and 16q loss of heterozygosity, 1q gain, TP53 mutation), and what were the results?
    Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
  3. Which subtype is my cancer, and does that change the recommended treatment?
    Why: Recognised subtypes for this cancer include Favourable histology, Diffuse anaplastic, Focal anaplastic.
  4. Is germline (inherited) genetic testing recommended for me or my family?
    Why: Inherited variants can change treatment and matter for relatives.

Very low risk (stage I FH, <2 years, <550 g)

  1. For my situation (very low risk (stage i fh, <2 years, <550 g)), which of the standard options do you recommend and why?
    Why: Guideline options include: Nephrectomy alone with close surveillance (AREN0532).

Stage I-II favourable histology

  1. For my situation (stage i-ii favourable histology), which of the standard options do you recommend and why?
    Why: Guideline options include: Nephrectomy then vincristine + actinomycin D for 18 weeks (EE-4A), or SIOP pre-op VA ×4 weeks then stage-adapted post-op therapy.
  2. Am I a candidate for Vincristine, Dactinomycin (actinomycin D), and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Stage III-IV favourable histology

  1. For my situation (stage iii-iv favourable histology), which of the standard options do you recommend and why?
    Why: Guideline options include: 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).
  2. Am I a candidate for Vincristine, Dactinomycin (actinomycin D), Doxorubicin, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Diffuse anaplastic or relapsed

  1. For my situation (diffuse anaplastic or relapsed), which of the standard options do you recommend and why?
    Why: Guideline options include: Intensive UH-1/UH-2 (vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide) with radiotherapy; relapse: ICE-type regimens, surgery, RT, high-dose therapy or trials.
  2. Am I a candidate for Cyclophosphamide, Carboplatin, Etoposide or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Any stage

  1. Are there clinical trials I could join, for example of Vincristine, Dactinomycin (actinomycin D)?
    Why: Trials are how the next standard of care is set; asking early keeps options open.
  2. 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.
  3. 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.
  4. I read that “Diffuse anaplastic and relapsed disease: survival ~50% or lower”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.
  5. I read that “Global inequity: Wilms is curable, yet most children with it worldwide die”. 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.

Everything relevant

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Direct links plus the targets, companies, and technologies of this cancer's products.

technologies

15
Active surveillanceAllogeneic stem cell transplantationAutologous stem cell transplant (high-dose therapy)CAR-T cell therapyCardio-oncologyCytotoxic chemotherapyGermline (hereditary) testingGlobal oncology and access in low- and middle-income countriesIMRT / IGRT (modern external beam)Isolated limb perfusion and infusionPlatinum agentsRobotic & minimally invasive surgerySurvivorship care and late-effects surveillanceTopoisomerase-I inhibitors (and ADC payloads)Ultrasound

targets

2
KMT2A (MLL) rearrangementTP53

drugs

7
ApprovedCytotoxic chemotherapy (platinum)
Carboplatin
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
Not mapped hereActinomycin antibiotic (transcription inhibitor)
Dactinomycin (actinomycin D) · Cosmegen
ApprovedCytotoxic chemotherapy (anthracycline)
Doxorubicin · Adriamycin
Not mapped hereTopoisomerase II inhibitor (podophyllotoxin derivative)
Etoposide · VePesid / Etopophos / Toposar
ApprovedCytotoxic chemotherapy (alkylating agent)
Ifosfamide · Ifex
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo

institutions

1
Children's Oncology Group (COG)

pathways

2
p53 / RB / cell-cycle checkpointWnt / β-catenin

terms

1
Germline vs somatic mutations

collections

2
Alex's Lemonade Stand Foundation (ALSF)St. Baldrick's Foundation

people

1
Sidney Farber

Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

Query for this cancer: (TITLE:"Wilms tumour" OR ABSTRACT:"Wilms tumour" OR TITLE:"nephroblastoma" OR ABSTRACT:"nephroblastoma") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Wilms tumour (nephroblastoma), not a curated reading list.

Connected

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Pages like this

not linked directly; found by shared links

technologies

8
Active surveillanceCytotoxic chemotherapyGermline (hereditary) testingGlobal oncology and access in low- and middle-income countriesIMRT / IGRT (modern external beam)Robotic & minimally invasive surgerySurvivorship care and late-effects surveillanceUltrasound

targets

1
TP53

drugs

7
ApprovedCytotoxic chemotherapy (platinum)
Carboplatin
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
Not mapped hereActinomycin antibiotic (transcription inhibitor)
Dactinomycin (actinomycin D) · Cosmegen
ApprovedCytotoxic chemotherapy (anthracycline)
Doxorubicin · Adriamycin
Not mapped hereTopoisomerase II inhibitor (podophyllotoxin derivative)
Etoposide · VePesid / Etopophos / Toposar
ApprovedCytotoxic chemotherapy (alkylating agent)
Ifosfamide · Ifex
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo

institutions

1
Children's Oncology Group (COG)

pathways

2
p53 / RB / cell-cycle checkpointWnt / β-catenin

terms

1
Germline vs somatic mutations

collections

2
Alex's Lemonade Stand Foundation (ALSF)St. Baldrick's Foundation

people

1
Sidney Farber