High-dose treatment for malignant rhabdoid tumor of the kidney: No evidence for improved survival-The Gesellschaft für Pädiatrische Onkologie und Hämatologie (GPOH) experience

Pediatr Blood Cancer. 2018 Jan;65(1). doi: 10.1002/pbc.26746. Epub 2017 Aug 26.


Background: Malignant rhabdoid tumor of the kidney (MRTK) is the most aggressive childhood renal tumor with overall survival (OS) rates ranging from 22% to 42%. Whether high-dose chemotherapy with autologous stem-cell transplantation (HDSCT) in an intensive first-line treatment offers additional benefit is an ongoing discussion.

Methods: A retrospective analysis of all 58 patients with MRTK from Austria, Switzerland, and Germany treated in the framework of consecutive, prospective renal/rhabdoid tumor studies SIOP9/GPO, SIOP93-01/GPOH (where SIOP is International Society of Pediatric Oncology and GPOH is German Society of Pediatric Oncology and Hematology), SIOP2001/GPOH, and European Rhabdoid Tumor Registry from 1991 to 2014.

Results: Median age at diagnosis was 11 months. Fifty percent of patients had metastases or multifocal disease at diagnosis (Stage IV). Local stage distribution was as follows: not done/I/II/III-1/6/11/40. Fifteen (26%) patients underwent upfront surgery. Thirty-seven (64%) patients achieved a complete remission, 17 (29%) relapsed, 34 (59%) died of disease progression, and two (3%) died of treatment-related complication. Mean time to the first event was 3.5 months. Two-year EFS/OS (where EFS is event-free survival) for the whole group was 37 ± 6%/38 ± 6%. Metastases/multifocal disease, younger age, and local stage III were associated with significantly inferior survival. Eleven (19%) patients underwent HDSCT (carboplatin + thiotepa, n = 6; carboplatin + etoposide + melphalan, n = 4; others, n = 1); 2-year OS in this group was 60 ± 15% compared to 34 ± 8% in the non-HDSCT group (P = 0.064). However, the time needed from radiologic to histologic diagnosis, stem-cell harvest, and HDSCT must also be taken into account to avoid selection bias by excluding the highest risk group with early progression (<90 days). Thus, 2-year EFS only for patients without progression until day 90 was 60 ± 16% consolidated by HDSCT compared to 62 ± 11% without (P = 0.8).

Conclusion: Our retrospective analysis suggests comparable outcomes for patients with and without HDSCT, if adjusted for early disease progression.

Keywords: GPOH; INI1-negative tumor; SIOP; childhood kidney tumor; high-risk nephroblastoma; myeloablative therapy; rhabdoid tumor.

Publication types

  • Clinical Trial
  • Multicenter Study

MeSH terms

  • Adolescent
  • Age Factors
  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage*
  • Child
  • Child, Preschool
  • Dactinomycin / administration & dosage
  • Disease-Free Survival
  • Female
  • Humans
  • Infant
  • Infant, Newborn
  • Kidney Neoplasms / drug therapy*
  • Kidney Neoplasms / mortality*
  • Male
  • Prospective Studies
  • Registries*
  • Rhabdoid Tumor / drug therapy*
  • Rhabdoid Tumor / mortality*
  • Survival Rate
  • Vincristine / administration & dosage


  • Dactinomycin
  • Vincristine

Supplementary concepts

  • SIOP protocol