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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

1
Department of Pediatric Oncology, University Children's Hospital, Tuebingen, Germany.
2
Department of Pediatric Oncology, University of Saarland, Homburg, Germany.
3
Departments of Pediatric Hematology and Oncology, and Stem Cell Transplantation Program, University Children's Hospital, University of Wuerzburg, Wuerzburg, Germany.
4
Department of Pediatric Oncology, University Hospital Frankfurt, University of Frankfurt, Frankfurt, Germany.
5
Pediatric Oncology and Hematology, University Hospital Heidelberg, Heidelberg, Germany.
6
Department of Pediatric Hematology and Oncology, University Hospital Jena, Jena Germany.

Abstract

BACKGROUND:

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.

PROCEDURE:

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.

RESULTS:

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).

CONCLUSION:

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.

PMID:
25431868
DOI:
10.1055/s-0034-1390504
[Indexed for MEDLINE]
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