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: 2.39). 07/2009; 52(7):888-90. DOI: 10.1002/pbc.21958
Source: PubMed


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.

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    ABSTRACT: Malignant rhabdoid tumours (MRT) and their central nervous system (CNS) counterparts atypical teratoid/rhabdoid tumours (ATRT) are rare, highly aggressive malignant neoplasms of childhood. Although there are isolated reports of long-term survival with intensive, multimodal therapy, outcomes are generally poor. We conducted a retrospective review of all patients diagnosed with MRT/ATRT at Great Ormond Street Hospital over the 20 years from 1989 to 2009. All cases were subjected to expert pathological review including INI-1 immunostaining. In a final cohort of 34 cases, overall survival was 17.4%, with median survival 10.1 months. Outcome in patients aged <3 years was significantly worse (median survival 6.2 months vs. 19.2 months). Data demonstrated a statistically significant benefit of radiotherapy (median survival 14.9 months vs. 6.6 months), although this analysis is confounded by the impact of patient age. There were four long-term survivors (>30 months), all of whom received chemotherapy with or without surgical resection or radiotherapy. In the present study, immunohistochemistry revealed no significant staining for either c-Erb or c-Met in any case, suggesting that targeting these molecules is unlikely to be of benefit in treating MRT/ATRT. In view of poor outcomes, there is a clear need for new treatment strategies and the identification of novel molecular targets for MRT/ATRT.
    No preview · Article · Jan 2010 · Pediatric Blood & Cancer
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    ABSTRACT: Rhabdoid tumours are rare aggressive tumours of infancy. The definition classically relies on a characteristic morphology and the inactivation of the hSNF5/INI1 tumour suppressor gene. This entity includes central nervous system tumours (ATRT), renal tumours (RTK) and soft-part tumours. Their rarity and morphological pleomorphism make the diagnosis often challenging. However, the recently introduced immunohistochemistry with anti-INI1 (anti-SMARCB1) antibody is a very useful diagnostic tool. Deletions at the 22q11.2 locus and mutations in hSNF5/INI1 sequence must be investigated in order to confirm the diagnosis and to give insights on a presumable germline mutation. Indeed, a predisposition may be found in up to 30% of cases. The treatment is based on aggressive chemotherapy, surgery and irradiation. The prognosis remains poor and the survival rate is below 30%, whatever the anatomic location. Understanding the role of hSNF5/INI1 within the SWI-SNF complex for the epigenetic regulation of transcription might drive the future targeted therapies.
    No preview · Article · Jan 2010 · Bulletin du cancer
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    ABSTRACT: Survival data of malignant rhabdoid tumour of the kidney (MRTK) registered in SIOP trials, advocating preoperative chemotherapy, are not available. To evaluate characteristics, response and survival of MRTK patients registered in recent SIOP protocols. An evaluation of all MRTK patients treated from 1993 to 2005 (SIOP trials 93-01 and 2001) was performed. Data were obtained from study specific case record forms and entered centrally in a database. Hundred and seven patients were identified (57 male), with a median age at diagnosis of 13 months (interquartile range 6-27 months), and a median follow-up time of 60 months. Left and right kidneys were equally affected. Tumour stage distribution was stage I (6%), stage II (22%), stage III (43%), stage IV (22%) and stage V (3%). Stage IV patients included 17 with pulmonary metastasis (8 lung-only) and 12 with multiple organ metastases (bone, brain and liver). Primary surgery was the upfront treatment approach in 22/107 patients (21%), by which 19 patients reached a complete remission (CR). Median difference in tumour volume before and after preoperative chemotherapy was 69 ml (interquartile range: 4.5-158.0, P < 0.0001), indicating marked chemosensitivity. The 5-year event-free survival (EFS) of the total group was 22% (95% CI: 15-33) and overall survival 26% (95% CI: 18-37). Most events (86%) occurred within the first 2 years after diagnosis. Younger age at diagnosis was an important adverse prognostic factors for survival. In contrast, tumour volume at diagnosis, nor volume reduction was associated with outcome. MRTK has a poor outcome especially in young and advanced-stage disease patients. Neither tumour volume at diagnosis, nor pre-operative chemosensitivity are prognostic factors for survival.
    Full-text · Article · May 2011 · Pediatric Blood & Cancer
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