Nephroblastoma, also known as Wilms tumor, is a malignant renal embryonic mixed tumor. The tumor may originate from the metanephric blastema, and its occurrence may be related to genetic factors, appearing familial in some cases. Some cases are observed to coexist with other malformations, and reports indicate gene deletions on the short arm of chromosome 11 in this disease. The incidence of this disease ranks first among pediatric abdominal tumors, predominantly affecting infants and young children. In recent years, treatment outcomes have significantly improved, with survival rates reaching 80-90%.

bubble_chart Clinical Manifestations

1. The most common presentation is an abdominal mass, often incidentally discovered as a smooth, solid, moderately firm, non-tender mass in one hypochondrium, with a few cases being bilateral. Some patients may exhibit tenderness, suggesting possible tumor rupture through the membrane. Enlargement of the tumor can lead to abdominal pain, dyspnea, loss of appetite, and weight loss.
2. Involvement of the renal pelvis may result in hematuria, seen in one-third of cases. Hypertension occurs in half of the cases and is associated with elevated renin levels.
3. Metastasis or large tumors may present with anemia, weight loss, cachexia, fever, etc.

bubble_chart Auxiliary Examination

1. Blood picture: Anemia may be present, or there may be an increase in red blood cells.
2. Urinalysis: Microscopic hematuria may be detected.
3. Elevated levels of erythropoietin in the blood that do not decrease after surgery suggest distant metastasis.
4. Imaging studies: Intravenous pyelography may show displacement, deformation, or destruction of the renal pelvis and calyces on the affected side, or it may not be visualized. Ultrasound may reveal irregular tumor margins and unevenly distributed echo spots within the tumor; pathological examination of tumor cells can be performed via ultrasound-guided puncture. CT can show an enlarged kidney shadow, with uneven density within the tumor and unclear boundaries with the renal parenchyma.
5. Histopathological examination: The tumor tissue consists of blastemal, stromal, and epithelial components. Over 90% of cases are of the favorable histology type. Anaplastic, clear cell, and rhabdoid types are associated with unfavorable prognosis.

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bubble_chart Diagnosis

1. Stage I: The tumor is localized and can be completely resected.
2. Stage II: Regional tumor (with local spread or infiltration beyond the renal membrane, but can be completely resected).
3. Stage III: Residual regional tumor foci and/or regional lymph node involvement and/or tumor spread to surrounding tissues.
4. Stage IV: Distant metastasis (commonly to the lungs and liver).

bubble_chart Treatment Measures

Modern treatment includes comprehensive therapy with close coordination of surgery, radiotherapy, and chemotherapy.

I. Indications for Surgical Treatment

All cases diagnosed with Wilms' tumor without metastasis should undergo complete surgical resection as early as possible. If distant metastasis has occurred but the primary follicular tumor can still be resected, it should be removed whenever possible. For patients with large tumors, preoperative chemotherapy or radiotherapy may be administered to shrink the tumor, followed by delayed surgery to facilitate tumor removal.

II. Radiotherapy

Preoperative radiotherapy is used for large tumors that are difficult to resect surgically. Generally, 1–2 weeks of radiotherapy can shrink the tumor. Postoperative radiotherapy usually begins within 2 weeks after surgery, once the wound has healed. The radiation field is determined by the tumor stage, primarily targeting the tumor bed and the main stirred pulse adjacent lymph nodes. The total radiation dose should be determined based on age, stage, and pathological type.

III. Chemotherapy

1. Preoperative chemotherapy aims to shrink the tumor for easier surgical removal. Options include actinomycin D (15 μg/(kg·d), IV, for 5 days, and vincristine (VCR) (1.5 mg/m², IV, on day 1). Combination chemotherapy with both drugs is preferred.
2. Postoperative Chemotherapy: Postoperative chemotherapy may follow one of the three regimens (A, B, or C) below: **Favorable Histology:**
   1. - **Stage I:** Regimen A: ACTD (actinomycin D) is administered preoperatively on day 6 and postoperatively at weeks 5, 13, and 24. VCR is given weekly starting on day 7, then on days 1 and 5 during ACTD courses. Radiotherapy is not required.
   2. - **Stage II:** Regimen B: The first 10 weeks are the same as Stage I, followed by ACTD and VCR at weeks 13, 22, 31, 40, 49, and 58. Radiotherapy is not required.
   3. - **Stages III–IV:** Regimen C: ACTD is given preoperatively on day 6 and postoperatively at weeks 13, 26, 36, 52, and 63. VCR is administered weekly starting on day 7 and continued on days 1 and 5 during ACTD courses. Adriamycin (ADM) is given at weeks 6, 19, 22, 45, and 58. Postoperative radiotherapy begins within 10 days after surgery: 10 Gy to the abdomen for Stage III and 12 Gy to the abdomen and lungs for Stage IV.
   **Unfavorable Histology:** Anaplastic Stage I follows the same regimen as favorable Stage I. Anaplastic Stages II–IV and clear cell sarcoma Stages I–IV follow the same regimen as favorable Stages III–IV. Adding cyclophosphamide (CTX) to the regimen for anaplastic types may improve efficacy. Rhabdoid tumors still have poor outcomes with modern regimens. With the above chemotherapy regimens, the 4-year survival rate can reach 77–97%.
3. **Treatment for Recurrent Cases:** - For recurrence without prior use of Regimen C, Regimen C may be applied. - For recurrence after Regimen C, the following regimens may be attempted:
   1. - **VP-16 + CTX:** VP-16 (100 mg/m², IV, for 5 days) + CTX (800–1000 mg/m², IV, on day 1), repeated every 3 weeks.
   2. - **VP-16 + CDDP:** VP-16 (125 mg/m²·d, IV, for 5 days, infused over 30 minutes in potassium-containing saline, protected from light) + CDDP (75 mg/m², once, infused over 8 hours in 100 mL/m² of potassium-containing glucose and half-normal saline, protected from light).
   - This regimen requires strong antiemetics, appropriate use of mannitol or diuretics, and monitoring for hearing and renal damage. Renal function should be checked before each cycle. If creatinine clearance is below 60 mL/(min·1.73 m²) or other glomerular damage is present, the regimen should be discontinued.

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IV. Treatment for Bilateral Wilms' Tumor

Approximately 5% of cases are bilateral, mostly Stage I with a favorable prognosis. One side may undergo nephrectomy, while the other undergoes tumor resection only, preserving the adrenal gland and at least half of the kidney. Postoperative radiotherapy and chemotherapy are then administered.

bubble_chart Differentiation

It should be differentiated from other retroperitoneal masses, such as neuroblastoma, teratoma, and hydronephrosis.