Long-Term Survival After Autologous Peripheral Stem Cell Transplantation in Two Patients With Malignant Rhabdoid Tumor of the Kidney

Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
Pediatric Blood & Cancer (Impact Factor: 2.56). 07/2009; 52(7):888-90. DOI: 10.1002/pbc.21958
Source: PubMed

ABSTRACT A 5-month-old male with stage II malignant rhabdoid tumor of the kidney (MRTK) and a 24-month-old male with stage III MRTK were treated with surgical resection of tumors and chemotherapy of alternating ICE (ifosfamide, carboplatin, and etoposide) and VDC (vincristine, doxorubicin, and cyclophosphamide), followed by high-dose chemotherapy using etoposide, carboplatin, and melphalan with autologous hematopoietic stem cell transplantation (SCT). Two patients have been alive without any evidence of disease for 30 and 37 months after diagnosis, respectively, and require no medication. Consolidation with SCT should be further studies for selected patients with high-risk MRTK.

  • [Show abstract] [Hide abstract]
    ABSTRACT: Extracranial rhabdoid tumours are rare, and often occur in infants. Although the kidney is the most common site, they can occur anywhere in the body. Most contain a biallelic inactivating mutation in SMARCB1, which is part of the chromatin remodelling complex SWI/SNF, and functions as a classic tumour suppressor gene. Despite multimodal therapy, outcome in rhabdoid tumours remains poor with only 31% of patients surviving to 1 year. The young age of patients limits use of radiotherapy, which, along with age, is an important prognostic factor. Because the tumours are rare, no standard therapeutic pathway exists, and no randomised trials have examined the role of new therapeutic approaches. Improved understanding of the biology and role of SMARCB1 has enabled identification of new targets for small molecule inhibitors to combine with chemotherapy backbones that we might establish from the current EpSSG and COG studies.
    The Lancet Oncology 07/2013; 14(8):e329-36. DOI:10.1016/S1470-2045(13)70088-3 · 24.73 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Rhabdoid tumors are rare but highly aggressive tumors with a predilection for infants and young children. The majority of these tumors harbor biallelic mutations in SMARCB1/INI1/hSNF5. Rather rare cases with mutations in other SWI/SNF core members such as BRG1 are on record. Rhabdoid tumors have only recently been registered and treated according to specifically designed treatment recommendations and in the framework of clinical trials. Within the last decade, prognosis has improved significantly but at least 50% of patients still relapse and subsequently almost inevitably succumb to their disease. This review summarizes past and current clinical approaches and presents an overview of the rationales for targeted therapy with potential for future clinical treatment trials for rhabdoid tumors.
    Pediatric Hematology and Oncology 07/2013; DOI:10.3109/08880018.2013.791737 · 0.96 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Background Extracranial malignant rhabdoid tumor (MRT) is a rare pediatric cancer with a poor prognosis. The kidney is the most common site. Isolated reports have shown improvements in patient survival, but no specific treatment regimen has shown efficacy over others.ProcedureRetrospective review of patients diagnosed with extracranial MRT at Children's Hospital Los Angeles between 1983 and 2012.ResultsThe median age at presentation for the 21 patients was 13 months (range, 0–108 months). Ten patients had renal primary tumors. The median time to progression was 4 months (range, 0.4–7 months). The 5-year event free survival (EFS) and overall survival (OS) of the entire cohort was 38 ± 10.6%. After 2002, patients diagnosed with extracranial MRT were administered a chemotherapy regimen of vincristine, doxorubicin and high dose cyclophosphamide (VDC). The OS for the patients diagnosed before and after 2002 were 20 ± 12% and 54 ± 15%, respectively. Of the 13 patients who received VDC containing regimen, eight patients achieved a complete radiological remission; five of these patients are long-term survivors. Four patients who received autologous bone marrow transplantation were alive at last follow-up. All patients with unresectable primary tumors died. Patients who had disease progression or relapse did not survive.Conclusions Patients with extracranial MRT have a poor prognosis. Treatment with high dose alkylator therapy followed by consolidation with high dose chemotherapy and autologous bone marrow transplant for those patients in radiographic complete remission appears to have a beneficial effect on survival. Pediatr Blood Cancer © 2014 Wiley Periodicals, Inc.
    Pediatric Blood & Cancer 08/2014; 61(8). DOI:10.1002/pbc.25093 · 2.56 Impact Factor