Extracranial malignant rhabdoid tumors in childhood

Abstract

BACKGROUND.Extracranial malignant rhabdoid tumor (MRT) is a rare, aggressive, pediatric malignancy with a historically poor outcome. Recent efforts to intensify treatment for MRT have resulted in isolated reports of long‐term survival.METHODS.The authors conducted a retrospective review of consecutive patients with MRT at Childrens Hospital Los Angeles over the 20 years from 1983 to 2003.RESULTS.Fourteen children were diagnosed with MRT over the 20‐year study period. The median age at presentation was 22.5 months (range, 0.5–108 months). Five patients had renal primary tumors, and 9 patients had extrarenal tumors. Eleven of 14 patients had stage III or IV disease at diagnosis. Five patients (35.7%) were long‐term survivors. The time to disease progression was rapid (mean, 3.6 months). There were no recurrences or deaths beyond 10 months after diagnosis. All survivors received multimodal therapy, including both chemotherapy and surgery with or without radiation. In addition, 2 patients received high‐dose chemotherapy with hematopoietic stem cell rescue (HSCT) after neoadjuvant chemotherapy and local tumor control. Both of those patients were long‐term survivors. There were no survivors after disease recurrence or progression.CONCLUSIONS.Patients with localized disease and complete surgical resection were most likely to survive long‐term. Consolidation with HSCT may benefit selected patients with advanced disease stage. International collaboration and further understanding of the biology of this disease is necessary to improve the survival of children with MRT. Cancer 2007. © 2007 American Cancer Society.

Dual Targeting of PDGFRα and FGFR1 Displays Synergistic Efficacy in Malignant Rhabdoid Tumors

“…MRTs are often lethal within the first year of diagnosis and many patients are refractory to standard chemotherapy (Madigan et al., 2007). There is thus an urgent need for new effective therapies.…”

“…MRTs are highly aggressive, and, despite intensive multimodal therapy, prognosis remains dismal with many children not surviving beyond 12 months (Madigan et al., 2007). SMARCB1 mutation is the sole driver of disease, and MRTs and AT/RTs lack additional gene amplifications or deletions and demonstrate low rates of mutations (Chun et al., 2016, Johann et al., 2016, Lee et al., 2012).…”

Malignant Extra Renal Rhabdoid Tumour Presenting as Central Airway Obstruction

“…The incidence of ERRT was reported as 0.15 per million children <15 years [1]. The central nervous system is the most frequent site of ERRT; however, it is reported in various sites of body [2]. Extra renal rhabdoid tumors have been described in the liver, brain, tongue, neck, chest, heart, pelvis, extremities, and several other sites.…”

“…MRTs were initially identified as a sarcomatous variant of the Wilms tumour but were subsequently demonstrated to have characteristic pathological features. In recent years, occurrence of rhabdoid tumours has been reported at a number of extra renal sites (extra renal malignant rhabdoid tumour) [2]. According to the best of our knowledge, this is the first reported case of malignant rhabdoid tumour presenting as tracheal growth and central airway obstruction.…”

A Rapid Progression of Disease After Surgical Excision of a Malignant Rhabdoid Tumor of the Bladder

“…In a retrospective study of University of California, Los Angeles’ 20-year history with pediatric extracranial MRTs, 5 of the 14 patients with extracranial MRTs were long-term survivors (range, 26–104 months). 13 Complete surgical resection correlated positively with survival. 13 All 5 long-term survivors underwent surgery for local control with 4 of the 5 survivors having complete resection.…”