Send to

Choose Destination
Klin Padiatr. 2014 Nov;226(6-7):351-6. doi: 10.1055/s-0034-1390504. Epub 2014 Nov 28.

Children with relapsed or refractory nephroblastoma: favorable long-term survival after high-dose chemotherapy and autologous stem cell transplantation.

Author information

Department of Pediatric Oncology, University Children's Hospital, Tuebingen, Germany.
Department of Pediatric Oncology, University of Saarland, Homburg, Germany.
Departments of Pediatric Hematology and Oncology, and Stem Cell Transplantation Program, University Children's Hospital, University of Wuerzburg, Wuerzburg, Germany.
Department of Pediatric Oncology, University Hospital Frankfurt, University of Frankfurt, Frankfurt, Germany.
Pediatric Oncology and Hematology, University Hospital Heidelberg, Heidelberg, Germany.
Department of Pediatric Hematology and Oncology, University Hospital Jena, Jena Germany.



High-dose chemotherapy (HDC) with autologous stem-cell rescue (ASCR) is a treatment option for pediatric patients with relapsed nephroblastoma. We present long term results of 9 patients treated between 1993 and 2013 at our center.


Reinduction therapy was carried out according to GPOH and SIOP recommendations. The conditioning regimen consisted of carboplatin (1 200 mg/m²), etoposide (800 mg/m² or 40 mg/kg) and melphalan (180 mg/m²). Purging of the grafts with immunomagnetic CD34 positive selection was performed in 5 patients.


8 of 9 Patients (90%) are alive without evidence of disease after a median follow-up of 8.5 years. Leukocyte engraftment occurred after a median of 10 days (range 8-12). Median numbers of 667/µl CD3+, 329/µl CD4+, 369/µl CD8+T cells and 949/µl B cells were reached after 180 days. No negative impact of CD34 selection was observed. No transplantation-related death occurred. Acute toxicity comprised mucositis III°-IV° in all and veno-occlusive disease in one patient. Long term effects probably related to treatment occurred in 3/7 evaluable patients and comprised hearing impairment, reduced renal phosphate reabsorption, mild creatinine elevation and hypothyroidism (n=1, each).


Thus, in our experience HDC with ASCR is an effective treatment of recurrent or refractory nephroblastoma with acceptable side effects. However, a randomized trial proving its efficiency with a high level of evidence is needed.

[Indexed for MEDLINE]
Free full text

Supplemental Content

Full text links

Icon for Georg Thieme Verlag Stuttgart, New York
Loading ...
Support Center