High-dose chemotherapy (HDCT) with auto-SCT in children with atypical teratoid/rhabdoid tumors (AT/RT): a report from the European Rhabdoid Registry (EU-RHAB)

Benesch, Martin; Bartelheim, K.; Fleischhack, Gudrun; Gruhn, Bernd; Schlegel, Paul G.; Witt, Olaf; Stachel, Klaus-Daniel; Hauch, Holger; Urban, Christian; Quehenberger, Franz; Massimino, Maura; Pietsch, Torsten; Hasselblatt, Martin; Giangaspero, Felice; Kordes, Uwe; Schneppenheim, Reinhard; Hauser, Péter; Klingebiel, T.; Frühwald, Michael C.

doi:10.1038/bmt.2013.208

Cited by 65 publications

(58 citation statements)

References 21 publications

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“…Eight patients received high-dose chemotherapy (HDCT) with autologous stem cell transplantation following induction chemotherapy at the treating physicians discretion. Results for these eight patients have partly been reported [10]. Inclusion criteria were diagnosis of an AT/RT as confirmed by negative INI1 staining and typical neuropathological features as confirmed by a reference neuropathologist, age below 18 years of age and the presence of informed consent by the legal guardians to contribute patient-associated data.…”

Section: Methodsmentioning

confidence: 99%

Improved 6‐year overall survival in AT/RT – results of the registry study Rhabdoid 2007

et al. 2016

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Atypical teratoid rhabdoid tumors (AT/RT) are characterized by mutations and subsequent inactivation of SMARCB1 (INI1, hSNF5), a predilection for very young children and an unfavorable outcome. The European Registry for rhabdoid tumors (EU-RHAB) was established to generate a common European database and to establish a standardized treatment regimen as the basis for phase I/II trials. Thus, genetic analyses, neuropathologic and radiologic diagnoses, and a consensus treatment regimen were prospectively evaluated. From 2005 to 2009, 31 patients with AT/RT from four countries were recruited into the registry study Rhabdoid 2007 and treated with systemic and intraventricular chemotherapy. Eight patients received high-dose chemotherapy, 23 radiotherapy, and 17 maintenance therapy. Reference evaluations were performed in 64% (genetic analyses, FISH, MLPA, sequencing) up to 97% (neuropathology, INI1 stain). Germ-line mutations (GLM) were detected in 6/21 patients. Prolonged overall survival was associated with age above 3 years, radiotherapy and achievement of a complete remission. 6-year overall and event-free survival rates were 46% (±0.10) and 45% (±0.09), respectively. Serious adverse events and one treatmentrelated death due to insufficiency of a ventriculo peritoneal shunt (VP-shunt) and consecutive herniation were noted. Acquisition of standardized data including reference diagnosis and a standard treatment schedule improved data quality Cancer MedicineOpen Access 1766

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Section: Methodsmentioning

confidence: 99%

Improved 6‐year overall survival in AT/RT – results of the registry study Rhabdoid 2007

et al. 2016

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“…Progression-free survival and overall survival at 2 years are 53% and 70%, respectively. 9 We present probably the youngest Asian patient of primary lumbar AT/RT with initial presentation of gradual sitting imbalance, food refusal and urinary retention. However, rapid recurrence of tumor was noted in 3 months after complete resection of tumor and first course of intensive chemotherapy.…”

Section: Discussionmentioning

confidence: 99%

Atypical teratoid/rhabdoid tumor of lumbar spine in a toddler child

Jaw

Chiou

et al. 2017

Spinal Cord Ser Cases

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BACKGROUND: Atypical teratoid/rhabdoid tumor (AT/RT) is a rare pediatric central nervous system malignancy with poor outcome. AT/RT is infrequently located in the spinal cord. CLINICAL PRESENTATION: A 16-month-old boy presented with progressive urinary retention and weakness of the lower extremities. Magnetic resonance imaging of the spine revealed an intradural extramedullary mass occupying the spinal canal at the level of T10-L3. The tumor was successfully resected by using neuroendoscopy. Histopathology demonstrated rhabdoid cells with eccentric nuclei and eosinophilic cytoplasmic hyaline inclusions. Immunohistologically, the tumor cells showed positive for epithelial membrane antigen, vimentin and neuron-specific enolase, and negative for integrase interactor 1. After surgery, the patient showed significant improvement in sitting and other neurological signs but presenting with flaccid neurogenic bladder. Intrathecal chemotherapy under European Rhabdoid Registry (EU-RHAB) protocol with Doxorubicin, Carboplatin, Etoposide, Ifosfamide, Vincristine, Cyclophosphamide and Actinomycin-D was given. However, recurrent intradural extramedullary tumor at the level of T11-L2 developed in 3 months. CONCLUSION: We report a young Asian case of AT/RT in thoracolumbar spine with recurrent tumor shortly after complete surgical resection of the tumor. (2017) Spinal Cord Series and Cases INTRODUCTIONAtypical teratoid/rhabdoid tumor (AT/RT) is a rare, highly aggressive and lethal neoplasm of the central nervous system (CNS) that usually occurs in very young children and infants. It may also occur in other anatomic sites, such as the kidneys, liver, abdomen or soft tissues. For CNS AT/RT, the posterior cranial fossa is the most frequent and well-studied location. 1 The prevalence of pediatric spinal AT/RT is rare, with a total of 38 cases in the literature between 1987 and 2015. 2 To our knowledge, only three cases are reported as presenting in the lumbar area. [2][3][4] We herein report a case of lumbar intradural extramedullary AT/RT in an 1-year 4-month-old boy with review of the literature.

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“…В одной из них на когорте из 19 детей были по-казаны 2-летная ОВ 50 ± 12 %, 2-летняя БСВ -29 ± 11 %. Однако стоит отметить, что 11 пациентов после проведения трансплантации гемопоэтических ство-ловых клеток погибли от прогрессирования процесса с медианой наблюдения 14 мес [4]. В другой работе, также посвященной пациентам с АТРО, показано отсутствие статистической разницы между группами больных, получавших стандартные курсы ХТ (n = 11) и HDCT (n = 13).…”

Section: результаты лечения злокачественных рабдоидных опухолейunclassified

“…Ча-стота встречаемости варьирует от 0,19 до 1,4 случая на 1 000 000 детей в год у пациентов до 15 лет с медиа-ной возраста на момент постановки диагноза от 16 до 24 мес [1][2][3][4]. Соотношение мальчики:девочки 0,86-1,2:1 [2,4]. В основе ЗРО, за исключением единичных случаев, лежит инактивирующая мута-ция гена-супрессора опухолевого роста SMARCB1.…”

Section: Introductionunclassified

“…Локальный контроль в объеме полного удаления опухоли и лучевой терапии (ЛТ) у части пациентов способствует достижению длительной бессобытийной выживаемости (БСВ) [5,6,9], тогда как роль высоко-дозной химиотерапии (ХТ) с последующей аутоло-гичной трансплантацией гемопоэтических стволовых клеток (ауто-ТГСК) остается дискутабельной [4]. Не-обходимо исследование новых методов лечения ЗРО [10] и анализ их результатов для улучшения выживае-мости пациентов с данной нозологией.…”

Section: Introductionunclassified

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Malignant rhabdoid tumors of soft tissues in children. Literature review

Телешова¹

2017

Ross. ž. det. gematol. onkol.

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High-dose chemotherapy (HDCT) with auto-SCT in children with atypical teratoid/rhabdoid tumors (AT/RT): a report from the European Rhabdoid Registry (EU-RHAB)

Cited by 65 publications

References 21 publications

Improved 6‐year overall survival in AT/RT – results of the registry study Rhabdoid 2007

Improved 6‐year overall survival in AT/RT – results of the registry study Rhabdoid 2007

Atypical teratoid/rhabdoid tumor of lumbar spine in a toddler child

Malignant rhabdoid tumors of soft tissues in children. Literature review

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