title: Children with relapsed or refractory nephroblastoma: favorable long-term survival after high-dose chemotherapy and autologous stem cell transplantation.
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.
  
  
  METHODS
  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).
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Pediatric Oncology, University Children's Hospital, Tuebingen, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Illhardt
authors.affiliation: Department of Pediatric Oncology, University of Saarland, Homburg, Germany.
authors.forename: M
authors.initials: M
authors.lastname: Ebinger
authors.affiliation: Department of Pediatric Oncology, University Children's Hospital, Tuebingen, Germany.
authors.forename: C P
authors.initials: CP
authors.lastname: Schwarze
authors.affiliation: Department of Pediatric Oncology, University Children's Hospital, Tuebingen, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Feuchtinger
authors.affiliation: Department of Pediatric Oncology, University of Saarland, Homburg, Germany.
authors.forename: R
authors.initials: R
authors.lastname: Furtwängler
authors.affiliation: Departments of Pediatric Hematology and Oncology, and Stem Cell Transplantation Program, University Children's Hospital, University of Wuerzburg, Wuerzburg, Germany.
authors.forename: P G
authors.initials: PG
authors.lastname: Schlegel
authors.affiliation: Department of Pediatric Oncology, University Hospital Frankfurt, University of Frankfurt, Frankfurt, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Klingebiel
authors.affiliation: Pediatric Oncology and Hematology, University Hospital Heidelberg, Heidelberg, Germany.
authors.forename: J
authors.initials: J
authors.lastname: Greil
authors.affiliation: Department of Pediatric Hematology and Oncology, University Hospital Jena, Jena Germany.
authors.forename: J F
authors.initials: JF
authors.lastname: Beck
authors.affiliation: Department of Pediatric Oncology, University Children's Hospital, Tuebingen, Germany.
authors.forename: R
authors.initials: R
authors.lastname: Handgretinger
authors.affiliation: Department of Pediatric Oncology, University Children's Hospital, Tuebingen, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Lang
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003609:Dactinomycin / Q000008:administration & dosage / Q000009:adverse effects; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D004317:Doxorubicin / Q000008:administration & dosage / Q000009:adverse effects; D005047:Etoposide / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007680:Kidney Neoplasms / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000009:adverse effects; D009364:Neoplasm Recurrence, Local / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D019172:Transplantation Conditioning; D014750:Vincristine / Q000008:administration & dosage / Q000009:adverse effects; D009396:Wilms Tumor / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology
chemical_list: D003609:Dactinomycin; D014750:Vincristine; D005047:Etoposide; D004317:Doxorubicin; D016190:Carboplatin; D008558:Melphalan