The diagnostic approach includes obtaining a thorough history for key diagnostic symptoms such as a painless, unilateral upper abdominal/flank mass, congenital syndromes, and congenital urogenital anomalies.[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
Abdominal ultrasound is the initial test of choice to establish the diagnosis, and computed tomography (CT) or magnetic resonance imaging (MRI) of abdomen and pelvis are used to stage the tumor and plan further therapy. Definitive diagnosis of suspected Wilms tumor is based on histology of tumor following surgical resection (nephrectomy) or biopsy (if tumor is unresectable). Metastatic disease should be ruled out on CT chest (or chest x-ray in resource-limited areas) and abdominal/pelvis CT or MRI.
History
Family history of Wilms tumor and presence of any congenital urogenital anomalies should be documented.[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
Specific phenotypic anomalies that are associated with overgrowth or nonovergrowth syndromes should be excluded.[17]PDQ Pediatric Treatment Editorial Board. Wilms tumor and other childhood kidney tumors treatment (PDQ®): health professional version. 2022 Dec 23. In: PDQ cancer information summaries [internet]. Bethesda, MD: National Cancer Institute; 2002.
https://www.cancer.gov/types/kidney/hp/wilms-treatment-pdq#_1
http://www.ncbi.nlm.nih.gov/pubmed/26389282?tool=bestpractice.com
For example, hyperinsulinemic hypoglycemia, which may be transient or persistent, occurs in 50% of children with Beckwith-Wiedemann syndrome during the neonatal period and infancy; therefore, if this syndrome is suspected, birth history for hypoglycemia should be noted.[35]Green DM, Breslow NE, Beckwith JB, et al. Screening of children with hemihypertrophy, aniridia, and Beckwith-Wiedemann syndrome in patients with Wilms tumor: a report from the National Wilms Tumor Study. Med Pediatr Oncol. 1993;21(3):188-92.
http://www.ncbi.nlm.nih.gov/pubmed/8095320?tool=bestpractice.com
Wilms tumor is more common in black and white children compared with Asian children, and most commonly occurs in the first 5 years of life.[1]Nakata K, Colombet M, Stiller CA, et al. Incidence of childhood renal tumours: an international population-based study. Int J Cancer. 2020 Dec 15;147(12):3313-27.
https://onlinelibrary.wiley.com/doi/10.1002/ijc.33147
http://www.ncbi.nlm.nih.gov/pubmed/32902866?tool=bestpractice.com
[2]Szychot E, Apps J, Pritchard-Jones K. Wilms' tumor: biology, diagnosis and treatment. Transl Pediatr. 2014 Jan;3(1):12-24.
https://tp.amegroups.com/article/view/3228/html
http://www.ncbi.nlm.nih.gov/pubmed/26835318?tool=bestpractice.com
The tumor most commonly presents as a painless, unilateral upper abdominal/flank mass.[2]Szychot E, Apps J, Pritchard-Jones K. Wilms' tumor: biology, diagnosis and treatment. Transl Pediatr. 2014 Jan;3(1):12-24.
https://tp.amegroups.com/article/view/3228/html
http://www.ncbi.nlm.nih.gov/pubmed/26835318?tool=bestpractice.com
Bilateral tumors are uncommon and occur in around 10% of patients; they may present simultaneously in both kidneys (synchronous) or may involve one kidney followed by the other (metachronous).[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
[5]Charlton J, Irtan S, Bergeron C, et al. Bilateral Wilms tumour: a review of clinical and molecular features. Expert Rev Mol Med. 2017 Jul 18;19:e8.
https://www.cambridge.org/core/journals/expert-reviews-in-molecular-medicine/article/bilateral-wilms-tumour-a-review-of-clinical-and-molecular-features/B4C8FBEE8C2C189D7D739B9BD64F1AA8
http://www.ncbi.nlm.nih.gov/pubmed/28716159?tool=bestpractice.com
Other clinical features, such as pallor, abdominal pain, fever, hematuria (visible or nonvisible), poor appetite, and weight loss may be present.[2]Szychot E, Apps J, Pritchard-Jones K. Wilms' tumor: biology, diagnosis and treatment. Transl Pediatr. 2014 Jan;3(1):12-24.
https://tp.amegroups.com/article/view/3228/html
http://www.ncbi.nlm.nih.gov/pubmed/26835318?tool=bestpractice.com
[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
[17]PDQ Pediatric Treatment Editorial Board. Wilms tumor and other childhood kidney tumors treatment (PDQ®): health professional version. 2022 Dec 23. In: PDQ cancer information summaries [internet]. Bethesda, MD: National Cancer Institute; 2002.
https://www.cancer.gov/types/kidney/hp/wilms-treatment-pdq#_1
http://www.ncbi.nlm.nih.gov/pubmed/26389282?tool=bestpractice.com
Shortness of breath may be associated with anemia or lung metastasis.
Very rarely, Wilms tumor occurs in extrarenal locations.[6]Shojaeian R, Hiradfar M, Sharifabad PS, et al. Extrarenal Wilms’ tumor: challenges in diagnosis, embryology, treatment and prognosis. In: van den Heuvel-Eibrink MM, ed. Wilms tumor [internet]. Brisbane: Codon Publications; 2016 Mar: chapter 6.
https://www.ncbi.nlm.nih.gov/books/NBK373353
http://www.ncbi.nlm.nih.gov/pubmed/27512762?tool=bestpractice.com
The presentation (rapidly growing painless mass) is usually similar to the classic Wilms tumor, but signs and symptoms are unique to the location of the tumor.
Physical exam
The location and extent of the abdominal mass should be documented. The mass is usually retroperitoneal ("ballotable"), and does not move with respirations.[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
It is smooth, firm to touch, and nontender. The patient may have abdominal distention. Genitalia should be examined for any congenital urogenital anomalies (i.e., hypospadias, atypical genitalia, or cryptorchidism).[16]Narod SA, Hawkins MM, Robertson CM, et al. Congenital anomalies and childhood cancer in Great Britain. Am J Hum Genet. 1997 Mar;60(3):474-85.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1712528
http://www.ncbi.nlm.nih.gov/pubmed/9042906?tool=bestpractice.com
[29]Breslow NE, Beckwith JB. Epidemiological features of Wilms' tumor: results of the National Wilms' Tumor Study. J Natl Cancer Inst. 1982 Mar;68(3):429-36.
http://www.ncbi.nlm.nih.gov/pubmed/6278194?tool=bestpractice.com
Presence of a varicocele in supine position may be associated with tumor extension into the inferior vena cava or the renal vein.[36]Idowu BM, Tanimola AG. Wilm's tumor presenting with scrotal varicocele in an 11-month-old boy. Indian J Radiol Imaging. 2018 Apr-Jun;28(2):247-9.
https://www.thieme-connect.com/products/ejournals/abstract/10.4103/ijri.IJRI_279_17
http://www.ncbi.nlm.nih.gov/pubmed/30050251?tool=bestpractice.com
Hypertension is present in approximately 25% of patients, and is secondary to compression of renal vasculature, or due to renin hypersecretion.[4]Davidoff AM. Wilms tumor. Adv Pediatr. 2012;59(1):247-67.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3589819
http://www.ncbi.nlm.nih.gov/pubmed/22789581?tool=bestpractice.com
[37]Maas MH, Cransberg K, van Grotel M, et al. Renin-induced hypertension in Wilms tumor patients. Pediatr Blood Cancer. 2007 May;48(5):500-3.
http://www.ncbi.nlm.nih.gov/pubmed/16794999?tool=bestpractice.com
Hepatomegaly may indicate metastatic disease.[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
Intracardiac extension of Wilms tumor is rare.[10]Abdullah Y, Karpelowsky J, Davidson A, et al. Management of nine cases of Wilms' tumour with intracardiac extension - a single centre experience. J Pediatr Surg. 2013 Feb;48(2):394-9.
http://www.ncbi.nlm.nih.gov/pubmed/23414872?tool=bestpractice.com
Phenotypic abnormalities that may be characteristic of Wilms tumor-associated syndromes should be documented.[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
Rarely, children may present with a paraneoplastic syndrome that affects the central and peripheral nervous system (e.g., generalized weakness, fatigue, ptosis, hypokinesis, dysarthria, urinary retention, facial diplegia, ophthalmoplegia, and autonomic dysfunction).[9]Petersen CL, Hemker BG, Jacobson RD, et al. Wilms tumor presenting with lambert-eaton myasthenic syndrome. J Pediatr Hematol Oncol. 2013 May;35(4):267-70.
http://www.ncbi.nlm.nih.gov/pubmed/23612377?tool=bestpractice.com
Laboratory investigations
Renal and hepatic function, complete blood count, urinalysis, serum protein/albumin, and coagulation studies should be ordered, although they are not required for diagnosis.
Imaging
Initial studies are aimed at establishing renal origin and extent of the mass. Abdominal ultrasonography is the recommended first-line test for establishing presumptive diagnosis and is usually adequate for this purpose.[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
[38]van der Beek JN, Artunduaga M, Schenk JP, et al. Similarities and controversies in imaging of pediatric renal tumors: A SIOP-RTSG and COG collaboration. Pediatr Blood Cancer. 2022 Nov 9;e30080.
http://www.ncbi.nlm.nih.gov/pubmed/36349564?tool=bestpractice.com
Typical finding is a large echogenic, heterogenous, unilateral, mainly solid (although small areas of cystic changes may be seen) intrarenal mass. If Wilms tumor is suspected on ultrasound, the patient should be promptly referred to a major pediatric cancer center where further imaging will be planned.
Either CT or MRI of the abdomen and pelvis should be obtained for locoregional staging by evaluating the contralateral kidney for synchronous disease and determining the size and number of ipsilateral masses, presence of lymphadenopathy, signs of possible rupture, infiltration into adjacent organs, presence and extent of tumor thrombus, and presence of metastatic disease to organs such as the liver.[38]van der Beek JN, Artunduaga M, Schenk JP, et al. Similarities and controversies in imaging of pediatric renal tumors: A SIOP-RTSG and COG collaboration. Pediatr Blood Cancer. 2022 Nov 9;e30080.
http://www.ncbi.nlm.nih.gov/pubmed/36349564?tool=bestpractice.com
The International Society of Paediatric Oncology (SIOP) protocol as well as the Children's Oncology Group (COG) protocol use chest CT for detection of lung lesions at diagnosis.[38]van der Beek JN, Artunduaga M, Schenk JP, et al. Similarities and controversies in imaging of pediatric renal tumors: A SIOP-RTSG and COG collaboration. Pediatr Blood Cancer. 2022 Nov 9;e30080.
http://www.ncbi.nlm.nih.gov/pubmed/36349564?tool=bestpractice.com
In resource-limited regions, chest x-ray can be used to identify lung metastasis; however, plain radiography may miss smaller pulmonary lesions (typically <1cm).[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
The role of positron emission tomography scan in the staging and assessment of response is not established.[39]Provenzi M, Saettini F, Conter V, et al. Is there a role for FDG-PET for the assessment of treatment efficacy in Wilms' tumor? A case report and literature review. Pediatr Hematol Oncol. 2013 Oct;30(7):633-9.
http://www.ncbi.nlm.nih.gov/pubmed/24050763?tool=bestpractice.com
[Figure caption and citation for the preceding image starts]: Wilms tumor: MRI findingsUHRAD.com; used with permission [Citation ends].
Tumor histology
Definitive diagnosis is based on histology of tumor following surgical resection (nephrectomy) or biopsy (if tumor is unresectable).[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
An open biopsy with a minimum of 10-12 cores is required in the COG protocol if a tumor is unresectable.[3]Spreafico F, Fernandez CV, Brok J, et al. Wilms tumour. Nat Rev Dis Primers. 2021 Oct 14;7(1):75.
https://www.nature.com/articles/s41572-021-00308-8
http://www.ncbi.nlm.nih.gov/pubmed/34650095?tool=bestpractice.com
[40]Lopyan NM, Ehrlich PF. Surgical management of Wilms tumor (nephroblastoma) and renal cell carcinoma in children and young adults. Surg Oncol Clin N Am. 2021 Apr;30(2):305-23.
http://www.ncbi.nlm.nih.gov/pubmed/33706902?tool=bestpractice.com
In the SIOP UMBRELLA protocol, pretreatment biopsy is not routinely recommended.[41]Vujanić GM, Gessler M, Ooms AHAG, et al. The UMBRELLA SIOP-RTSG 2016 Wilms tumour pathology and molecular biology protocol. Nat Rev Urol. 2018 Nov;15(11):693-701.
https://www.nature.com/articles/s41585-018-0100-3
http://www.ncbi.nlm.nih.gov/pubmed/30310143?tool=bestpractice.com
Percutaneous cutting needle biopsy (tru-cut biopsy) can be considered in patients whose tumors are suspected not to be Wilms tumor, such as young children with stage IV disease and in children >10 years of age, as the frequency of non-Wilms renal tumors is increased in these age groups.[41]Vujanić GM, Gessler M, Ooms AHAG, et al. The UMBRELLA SIOP-RTSG 2016 Wilms tumour pathology and molecular biology protocol. Nat Rev Urol. 2018 Nov;15(11):693-701.
https://www.nature.com/articles/s41585-018-0100-3
http://www.ncbi.nlm.nih.gov/pubmed/30310143?tool=bestpractice.com
[42]van den Heuvel-Eibrink MM, van Tinteren H, Rehorst H, et al. Malignant rhabdoid tumours of the kidney (MRTKs), registered on recent SIOP protocols from 1993 to 2005: a report of the SIOP renal tumour study group. Pediatr Blood Cancer. 2011 May;56(5):733-7.
http://www.ncbi.nlm.nih.gov/pubmed/21370404?tool=bestpractice.com
Genetic testing
Molecular genetic testing is a useful diagnostic tool for the identification of phenotypic syndromes that may be associated with Wilms tumor. Specifically, patients with isolated Wilms tumor, or those with Wilms tumor and associated anomalies, may be tested for deletions of WT1 and other contiguous genes such as PAX6 by fluorescent in situ hybridization (FISH). Patients with clinical features suggestive of Beckwith-Wiedemann syndrome may be tested for a duplication of 11p15.5 by high-resolution chromosomal analysis and FISH.[43]Diller L, Ghahremani M, Morgan J, et al. Constitutional WT1 mutations in Wilms tumor patients. J Clin Oncol. 1998 Nov;16(11):3634-40.
http://www.ncbi.nlm.nih.gov/pubmed/9817285?tool=bestpractice.com
Loss of heterozygosity (LOH) studies for 16q and 1p are performed on freshly frozen tumor tissue.[44]Wittmann S, Zirn B, Alkassar M, et al. Loss of 11q and 16q in Wilms tumors is associated with anaplasia, tumor recurrence, and poor prognosis. Genes Chromosomes Cancer. 2007 Feb;46(2):163-70.
http://www.ncbi.nlm.nih.gov/pubmed/17099873?tool=bestpractice.com
In every child with Wilms tumor, biobanking of tumor material (fresh frozen tumor tissue) and healthy kidney tissue should be done to perform research studies helping to define new risk factors and targets for better treatment options.