TriangleHEALTH PROFESSIONAL · SOURCE READING
HSCT
Source: Wilms Tumor and Other Childhood Kidney Tumors Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 15, 2025 · Captured 2026-09-09.
Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.
High-dose chemotherapy followed by autologous HSCT has been used for recurrent high-risk patients.[311,312]; [313,314][Level of evidence C1]
Source links and citations
Evidence (HSCT):
Investigators used the European Blood and Marrow Transplantation Registry to examine the outcomes of children with Wilms tumor (n = 69) who received high-dose chemotherapy with autologous HSCT as consolidation during first or second remission. Different high-dose chemotherapy regimens were used, containing either melphalan (n = 34) or thiotepa (n = 14).[315][Level of evidence C1]
Source links and citations
Investigators used the European Blood and Marrow Transplantation Registry to examine the outcomes of children with Wilms tumor (n = 69) who received high-dose chemotherapy with autologous HSCT as consolidation during first or second remission. Different high-dose chemotherapy regimens were used, containing either melphalan (n = 34) or thiotepa (n = 14).[315][Level of evidence C1]
The 5-year OS and EFS probabilities were 0.67 (± 0.06) and 0.63 (± 0.06), respectively (median observation time, 7.8 years).
Investigators used the European Blood and Marrow Transplantation Registry to examine the outcomes of children with Wilms tumor (n = 69) who received high-dose chemotherapy with autologous HSCT as consolidation during first or second remission. Different high-dose chemotherapy regimens were used, containing either melphalan (n = 34) or thiotepa (n = 14).[315][Level of evidence C1]
Children who underwent transplant in first remission had 5-year EFS and OS probabilities of 0.69 and 0.72, respectively.
Investigators used the European Blood and Marrow Transplantation Registry to examine the outcomes of children with Wilms tumor (n = 69) who received high-dose chemotherapy with autologous HSCT as consolidation during first or second remission. Different high-dose chemotherapy regimens were used, containing either melphalan (n = 34) or thiotepa (n = 14).[315][Level of evidence C1]
The use of melphalan alone for high-dose chemotherapy led to noninferior survival rates, compared with other drugs or drug combinations, and to better engraftment, compared with thiotepa-containing regimens.
Similar outcome results were reported in a series of 54 patients with Wilms tumor who received high-dose chemotherapy with autologous stem cell rescue (first-line therapy, n = 13; recurrence therapy, n = 41).[314][Level of evidence C1]
Source links and citations
Similar outcome results were reported in a series of 54 patients with Wilms tumor who received high-dose chemotherapy with autologous stem cell rescue (first-line therapy, n = 13; recurrence therapy, n = 41).[314][Level of evidence C1]
For patients treated at the time of recurrence, the 5-year EFS and OS rates were 57% and 69%, respectively (median follow-up, 7 years).
The outcomes of 253 patients with relapsed Wilms tumor who received high-dose chemotherapy followed by autologous HSCT between 1990 and 2013 were reported to and reviewed by the Center for International Blood and Marrow Transplantation Research.[316]
Source links and citations
The outcomes of 253 patients with relapsed Wilms tumor who received high-dose chemotherapy followed by autologous HSCT between 1990 and 2013 were reported to and reviewed by the Center for International Blood and Marrow Transplantation Research.[316]
The 5-year estimate for EFS was 36%, and the 5-year estimate for OS was 45%.
The outcomes of 253 patients with relapsed Wilms tumor who received high-dose chemotherapy followed by autologous HSCT between 1990 and 2013 were reported to and reviewed by the Center for International Blood and Marrow Transplantation Research.[316]
Relapse of primary disease was the cause of death in 81% of the population.
No randomized trials comparing chemotherapy and transplant have been reported, and case series suffer from selection bias.
Patients in whom such salvage attempts fail should be offered treatment on phase I or phase II clinical trials.
Publication references
Read the original reference and check its publication notices.
Preserved source evidence · Not medical advice