Overview
Nephroblastoma, commonly known as Wilms tumor, is the most common malignant kidney tumor of childhood. It arises from embryonal renal tissue (metanephric blastema) and typically presents as an abdominal mass, often discovered incidentally by a parent or during a routine examination. The tumor primarily affects the kidneys but can spread to the lungs, liver, and rarely to other sites. Key clinical features include a palpable abdominal or flank mass, abdominal pain, hematuria (blood in the urine), fever, and occasionally hypertension. Some children may also present with aniridia, genitourinary anomalies, or hemihypertrophy, which are features associated with predisposition syndromes such as WAGR syndrome (Wilms tumor, aniridia, genitourinary anomalies, and intellectual disability), Denys-Drash syndrome, and Beckwith-Wiedemann syndrome. The molecular pathogenesis of nephroblastoma involves several genes, most notably WT1 (located on chromosome 11p13) and WT2 (at 11p15.5), as well as other loci including WTX (on the X chromosome) and mutations in CTNNB1. Approximately 1-2% of cases are familial, while the vast majority are sporadic. Bilateral tumors occur in about 5-10% of cases and are more frequently associated with germline genetic alterations. Treatment of nephroblastoma is one of the great success stories of pediatric oncology. Current management involves a multimodal approach including surgical resection (nephrectomy), chemotherapy, and in some cases radiation therapy. Treatment protocols differ between North America (Children's Oncology Group, which favors upfront surgery) and Europe (SIOP, which uses preoperative chemotherapy). Overall survival rates now exceed 90% for localized disease with favorable histology. Prognosis is less favorable for tumors with anaplastic (unfavorable) histology or advanced-stage disease, though outcomes continue to improve with refined risk stratification and treatment protocols.
Also known as:
Clinical phenotype terms— hover any for plain English:
Variable
Can be inherited in different ways depending on the underlying gene
Childhood
Begins in childhood, roughly ages 1 to 12
FDA & Trial Timeline
5 eventsChildren's Oncology Group — PHASE3
Memorial Sloan Kettering Cancer Center — PHASE2
Centre Oscar Lambret — PHASE1, PHASE2
St. Jude Children's Research Hospital — PHASE2
French Africa Pediatric Oncology Group
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Nephroblastoma.
5 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Nephroblastoma.
Community
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Start the conversation →Latest news about Nephroblastoma
Disease timeline:
New recruiting trial: A Study Using Risk Factors to Determine Treatment for Children With Favorable Histology Wilms Tumors (FHWT)
A new clinical trial is recruiting patients for Nephroblastoma
New recruiting trial: CUE-102 in Recurrent Glioblastoma
A new clinical trial is recruiting patients for Nephroblastoma
Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Nephroblastoma
What is Nephroblastoma?
Nephroblastoma, commonly known as Wilms tumor, is the most common malignant kidney tumor of childhood. It arises from embryonal renal tissue (metanephric blastema) and typically presents as an abdominal mass, often discovered incidentally by a parent or during a routine examination. The tumor primarily affects the kidneys but can spread to the lungs, liver, and rarely to other sites. Key clinical features include a palpable abdominal or flank mass, abdominal pain, hematuria (blood in the urine), fever, and occasionally hypertension. Some children may also present with aniridia, genitourinary a
At what age does Nephroblastoma typically begin?
Typical onset of Nephroblastoma is childhood. Age of onset can vary across affected individuals.
Are there clinical trials for Nephroblastoma?
Yes — 5 recruiting clinical trials are currently listed for Nephroblastoma on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Nephroblastoma?
24 specialists and care centers treating Nephroblastoma are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.