Extra-cranial Malignant Rhabdoid Tumor in Children: A Single Institute Experience
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Purpose Malignant rhabdoid tumor (MRT) is a rare and highly aggressive tumor that affects youngchildren. Due to its extreme rarity, most of the available data are based on retrospectivecase series. To add to the current knowledge of this disease, we reviewed the patientstreated for extra-cranial MRT in our institute. Materials and MethodsA retrospective medical record review was conducted on children treated for pathologicallyconfirmed extra-cranial MRT at Seoul National University Children’s Hospital betweenJanuary 2003 and May 2013. ResultsEleven patients (7 boys, 4 girls) were diagnosed with extra-cranial MRT at a median age of9 months old. INI1 staining was important in the pathological confirmation. Six patients(55%) had renal MRT and five (45%) had soft tissue MRT. Five patients (45%) had metastasesat diagnosis. All patients underwent chemotherapy, eight patients (73%) underwentsurgery, six patients (55%) received therapeutic radiotherapy, and four patients (36%)underwent high dose chemotherapy with autologous stem cell rescue (HDCT/ASCR) withmelphalan, etoposide, and carboplatin. Five patients (45%) died of disease followingprogression (n=3) or relapse (n=2), however, there was no treatment related mortality. Theoverall survival of the cohort was 53.0% and the event-free survival was 54.5% with amedian follow-up duration of 17.8 months (range, 2.3 to 112.3 months). ConclusionExtra-cranial MRT is still a highly aggressive tumor in young children. However, the improvedsurvival of our cohort is promising and HDCT/ASCR with melphalan, etoposide, and carboplatinmay be a promising treatment option.
Abstract
Purpose Malignant rhabdoid tumor (MRT) is a rare and highly aggressive tumor that affects youngchildren. Due to its extreme rarity, most of the available data are based on retrospectivecase series. To add to the current knowledge of this disease, we reviewed the patientstreated for extra-cranial MRT in our institute. Materials and MethodsA retrospective medical record review was conducted on children treated for pathologicallyconfirmed extra-cranial MRT at Seoul National University Children’s Hospital betweenJanuary 2003 and May 2013. ResultsEleven patients (7 boys, 4 girls) were diagnosed with extra-cranial MRT at a median age of9 months old. INI1 staining was important in the pathological confirmation. Six patients(55%) had renal MRT and five (45%) had soft tissue MRT. Five patients (45%) had metastasesat diagnosis. All patients underwent chemotherapy, eight patients (73%) underwentsurgery, six patients (55%) received therapeutic radiotherapy, and four patients (36%)underwent high dose chemotherapy with autologous stem cell rescue (HDCT/ASCR) withmelphalan, etoposide, and carboplatin. Five patients (45%) died of disease followingprogression (n=3) or relapse (n=2), however, there was no treatment related mortality. Theoverall survival of the cohort was 53.0% and the event-free survival was 54.5% with amedian follow-up duration of 17.8 months (range, 2.3 to 112.3 months). ConclusionExtra-cranial MRT is still a highly aggressive tumor in young children. However, the improvedsurvival of our cohort is promising and HDCT/ASCR with melphalan, etoposide, and carboplatinmay be a promising treatment option.
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