Treatment of Nephroblastomatosis (Diffuse Hyperplastic Perilobar Nephroblastomatosis)

Preoperative chemotherapy.[3]

Renal-sparing surgery. Given the high incidence of bilaterality and subsequent Wilms tumors, renal-sparing surgery may be indicated.[1]

The Children’s Oncology Group AREN0534 (NCT00945009) study was the first clinical trial to prospectively enroll and uniformly treat patients with diffuse hyperplastic perilobar nephroblastomatosis. Patients were enrolled after having central radiological review. Diffuse hyperplastic perilobar nephroblastomatosis was defined as the cortical surface of the kidney being composed of hyperplastic rests, with the entire nephrogenic zone involved, and with a thick rind capping all of one or both kidneys. Patients were treated with induction chemotherapy consisting of vincristine and dactinomycin (regimen EE-4A) and had cross-sectional imaging at weeks 6 and 12. Therapy could be continued if the patient's disease was stable or decreasing. Maximum treatment duration was 19 weeks.[3][Level of evidence: C1]

The Children’s Oncology Group AREN0534 (NCT00945009) study was the first clinical trial to prospectively enroll and uniformly treat patients with diffuse hyperplastic perilobar nephroblastomatosis. Patients were enrolled after having central radiological review. Diffuse hyperplastic perilobar nephroblastomatosis was defined as the cortical surface of the kidney being composed of hyperplastic rests, with the entire nephrogenic zone involved, and with a thick rind capping all of one or both kidneys. Patients were treated with induction chemotherapy consisting of vincristine and dactinomycin (regimen EE-4A) and had cross-sectional imaging at weeks 6 and 12. Therapy could be continued if the patient's disease was stable or decreasing. Maximum treatment duration was 19 weeks.[3][Level of evidence: C1]

Nine patients were enrolled, and the median age was 10 months. One patient was not evaluable based on radiological criteria for diffuse hyperplastic perilobar nephroblastomatosis. Eight patients had confirmed bilateral diffuse hyperplastic perilobar nephroblastomatosis and started chemotherapy without tissue diagnosis. One patient had an upfront partial nephrectomy that was found to have diffuse hyperplastic perilobar nephroblastomatosis in the specimen and was subsequently treated with EE-4A. All patients were alive at a median follow-up of 6.6 years (range, 4.5–9.1 years).

The Children’s Oncology Group AREN0534 (NCT00945009) study was the first clinical trial to prospectively enroll and uniformly treat patients with diffuse hyperplastic perilobar nephroblastomatosis. Patients were enrolled after having central radiological review. Diffuse hyperplastic perilobar nephroblastomatosis was defined as the cortical surface of the kidney being composed of hyperplastic rests, with the entire nephrogenic zone involved, and with a thick rind capping all of one or both kidneys. Patients were treated with induction chemotherapy consisting of vincristine and dactinomycin (regimen EE-4A) and had cross-sectional imaging at weeks 6 and 12. Therapy could be continued if the patient's disease was stable or decreasing. Maximum treatment duration was 19 weeks.[3][Level of evidence: C1]

No patients were anephric. Fourteen of the 16 kidneys were functioning (87.5%).

The Children’s Oncology Group AREN0534 (NCT00945009) study was the first clinical trial to prospectively enroll and uniformly treat patients with diffuse hyperplastic perilobar nephroblastomatosis. Patients were enrolled after having central radiological review. Diffuse hyperplastic perilobar nephroblastomatosis was defined as the cortical surface of the kidney being composed of hyperplastic rests, with the entire nephrogenic zone involved, and with a thick rind capping all of one or both kidneys. Patients were treated with induction chemotherapy consisting of vincristine and dactinomycin (regimen EE-4A) and had cross-sectional imaging at weeks 6 and 12. Therapy could be continued if the patient's disease was stable or decreasing. Maximum treatment duration was 19 weeks.[3][Level of evidence: C1]

Three of eight patients did not develop Wilms tumor. One of these patients had a complete radiographic response, one had a few sclerotic lesions at the end of therapy, and one had significant response but still had radiographic abnormalities.

The Children’s Oncology Group AREN0534 (NCT00945009) study was the first clinical trial to prospectively enroll and uniformly treat patients with diffuse hyperplastic perilobar nephroblastomatosis. Patients were enrolled after having central radiological review. Diffuse hyperplastic perilobar nephroblastomatosis was defined as the cortical surface of the kidney being composed of hyperplastic rests, with the entire nephrogenic zone involved, and with a thick rind capping all of one or both kidneys. Patients were treated with induction chemotherapy consisting of vincristine and dactinomycin (regimen EE-4A) and had cross-sectional imaging at weeks 6 and 12. Therapy could be continued if the patient's disease was stable or decreasing. Maximum treatment duration was 19 weeks.[3][Level of evidence: C1]

Five of eight patients had Wilms tumor at some point in their disease: one at diagnosis, one during therapy, and three after therapy (at 2 months and again at 4 months, 6 months, and 8 months). Only one of the patients developed a Wilms tumor with anaplastic histology.

The Children’s Oncology Group AREN0534 (NCT00945009) study was the first clinical trial to prospectively enroll and uniformly treat patients with diffuse hyperplastic perilobar nephroblastomatosis. Patients were enrolled after having central radiological review. Diffuse hyperplastic perilobar nephroblastomatosis was defined as the cortical surface of the kidney being composed of hyperplastic rests, with the entire nephrogenic zone involved, and with a thick rind capping all of one or both kidneys. Patients were treated with induction chemotherapy consisting of vincristine and dactinomycin (regimen EE-4A) and had cross-sectional imaging at weeks 6 and 12. Therapy could be continued if the patient's disease was stable or decreasing. Maximum treatment duration was 19 weeks.[3][Level of evidence: C1]

A 19-week course of EE-4A prevents some patients from developing Wilms tumor (three of eight), but it is not fully effective. The ideal duration of therapy remains unknown.

In a retrospective series of 52 patients with diffuse hyperplastic perilobar nephroblastomatosis (46 with confirmed diffuse hyperplastic perilobar nephroblastomatosis), 33 patients were treated with chemotherapy and/or radiation therapy initially, 16 patients underwent unilateral nephrectomy followed by adjuvant therapy, and 3 patients were observed only.[1]

In a retrospective series of 52 patients with diffuse hyperplastic perilobar nephroblastomatosis (46 with confirmed diffuse hyperplastic perilobar nephroblastomatosis), 33 patients were treated with chemotherapy and/or radiation therapy initially, 16 patients underwent unilateral nephrectomy followed by adjuvant therapy, and 3 patients were observed only.[1]

A total of 24 patients developed Wilms tumor (including the 3 patients who were observed only), at a median of 30 months.

In a retrospective series of 52 patients with diffuse hyperplastic perilobar nephroblastomatosis (46 with confirmed diffuse hyperplastic perilobar nephroblastomatosis), 33 patients were treated with chemotherapy and/or radiation therapy initially, 16 patients underwent unilateral nephrectomy followed by adjuvant therapy, and 3 patients were observed only.[1]

Eighteen of the 33 patients who received chemotherapy alone developed a Wilms tumor.

In a retrospective series of 52 patients with diffuse hyperplastic perilobar nephroblastomatosis (46 with confirmed diffuse hyperplastic perilobar nephroblastomatosis), 33 patients were treated with chemotherapy and/or radiation therapy initially, 16 patients underwent unilateral nephrectomy followed by adjuvant therapy, and 3 patients were observed only.[1]

Of 16 patients who underwent a nephrectomy and adjuvant therapy, 3 developed Wilms tumor, despite the fact that 14 of 16 patients had bilateral disease.

In a retrospective series of 52 patients with diffuse hyperplastic perilobar nephroblastomatosis (46 with confirmed diffuse hyperplastic perilobar nephroblastomatosis), 33 patients were treated with chemotherapy and/or radiation therapy initially, 16 patients underwent unilateral nephrectomy followed by adjuvant therapy, and 3 patients were observed only.[1]

Thirty-three percent of the patients who developed Wilms tumor had anaplastic Wilms tumor at some time during their course, probably as a result of selection of chemotherapy-resistant tumors; thus, early detection is critical.