Exploring Maintenance Therapy in Pediatric Embryonal Tumor With Multilayered Rosettes

被引：0

作者：

Ahmed, Mohamed M. ^{[1
]}

Carceller, Fernando ^{[1
,2
]}

Bridges, Leslie R. ^{[3
]}

Mallucci, Conor ^{[4
]}

Singh, Navneet ^{[5
]}

Vaidya, Sucheta ^{[1
,2
]}

机构：

[1] Royal Marsden NHS Fdn Trust, Children & Young Peoples Unit, London, England

[2] Inst Canc Res, Div Clin Studies, London, England

[3] St Georges Univ Hosp NHS Fdn Trust, Dept Cellular Pathol, London, England

[4] Alder Hey Childrens Hosp NHS Fdn Trust, Dept Neurosurg, Liverpool, England

[5] St Georges NHS Fdn Trust, Dept Neurosurg, London, England

来源：

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY | 2025年 / 47卷 / 05期

关键词：

maintenance; CNS; ETMR; C19MC; intrathecal topotecan; cis-retinoic acid; valproate; HEAD-START-II; HIGH-DOSE CHEMOTHERAPY; THAN; 3; YEARS; INTENSIVE CHEMOTHERAPY; INTRATHECAL TOPOTECAN; YOUNG-CHILDREN; CELL RESCUE; MYELOABLATIVE CHEMOTHERAPY; CLINICAL-TRIAL; BRAIN-TUMORS;

D O I：

10.1097/MPH.0000000000003033

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Embryonal tumors with multilayered rosettes (ETMR) represent a distinct entity characterized by aggressive behavior. Historical retrospective analyses have documented dire overall survival rates ranging from 0% to 14% at 1 year. However, a contemporary report by Khan and colleagues shows overall survival rates reaching 29% at 2 years and 27% at 4 years. We present the case of an 18-month-old girl diagnosed with ETMR, confirmed by chromosome 19 microRNA cluster amplification following initial presentation with focal seizures. The patient underwent a combination of surgical interventions, high-dose chemotherapy with stem cell rescue, and proton therapy, achieving a disease-free status after completing standard treatment. Subsequently, a 12-month maintenance regimen comprising intrathecal topotecan, oral sodium valproate, and oral cis-retinoic acid was administered. The maintenance therapy was well tolerated, with manageable adverse effects. The patient remains progression-free for 32 months postmaintenance therapy (50 months from initial presentation). This study explores the feasibility and safety profile of maintenance therapy in ETMR. Future studies may explore this approach to determine its efficacy in children with ETMR.

引用

页码：242 / 245

页数：4

共 43 条

[1]

Bell E, 2022, NEURO-ONCOLOGY, V24, P15

[2] Phase I clinical trial of intrathecal topotecan in patients with neoplastic meningitis [J].

Blaney, SM ;

Heideman, R ;

Berg, S ;

Adamson, P ;

Gillespie, A ;

Geyer, JR ;

Packer, R ;

Matthay, K ;

Jaeckle, K ;

Cole, D ;

Kuttesch, N ;

Poplack, DG ;

Balis, FM .

JOURNAL OF CLINICAL ONCOLOGY, 2003, 21 (01) :143-147

[3] Embryonal tumor with multilayered rosettes: Diagnostic tools update and review of the literature [J].

Ceccom, Johnatan ;

Bourdeaut, Franck ;

Loukh, Najat ;

Rigau, Valerie ;

Milin, Serge ;

Takin, Romulus ;

Richer, Wilfrid ;

Uro-Coste, Emmanuelle ;

Couturier, Jerome ;

Bertozzi, Anne Isabelle ;

Delattre, Olivier ;

Delisle, Marie Bernadette .

CLINICAL NEUROPATHOLOGY, 2014, 33 (01) :15-22

[4] Embryonal tumor with multilayered rosettes: Overview of diagnosis and therapy [J].

Chadda, Karan R. ;

Solano-Paez, Palma ;

Khan, Sara ;

Llempen-Lopez, Mercedes ;

Phyu, Poe ;

Horan, Gail ;

Trotman, Jamie ;

Tarpey, Patrick ;

Erker, Craig ;

Lindsay, Holly ;

Addy, Dilys ;

Jacques, Thomas S. ;

Allinson, Kieren ;

Pizer, Barry ;

Huang, Annie ;

Murray, Matthew J. .

NEURO-ONCOLOGY ADVANCES, 2023, 5 (01)

[5] Intrathecal chemotherapy delivered by a lumbar-thecal catheter in metastatic medulloblastoma: a case illustration [J].

Clayton, J. ;

Vloeberghs, M. ;

Jaspan, T. ;

Walker, D. ;

MacArthur, D. ;

Grundy, R. .

ACTA NEUROCHIRURGICA, 2008, 150 (07) :709-712

[6] Pilot Study of Intensive Chemotherapy With Peripheral Hematopoietic Cell Support for Children Less Than 3 Years of Age With Malignant Brain Tumors, the CCG-99703 Phase I/II Study. A Report From the Children's Oncology Group [J].

Cohen, Bruce H. ;

Geyer, J. Russell ;

Miller, Douglas C. ;

Curran, John G. ;

Zhou, Tianni ;

Holmes, Emi ;

Ingles, Sue Ann ;

Dunkel, Ira J. ;

Hilden, Joanne ;

Packer, Roger J. ;

Pollack, Ian F. ;

Gajjar, Amar ;

Finlay, Jonathan L. .

PEDIATRIC NEUROLOGY, 2015, 53 (01) :31-46

[7] Epileptogenesis and oncogenesis: An antineoplastic role for antiepileptic drugs in brain tumours? [J].

Cucchiara, Federico ;

Pasqualetti, Francesco ;

Giorg, Filippo Sean ;

Danesi, Romano ;

Bocci, Guido .

PHARMACOLOGICAL RESEARCH, 2020, 156

[8]

Dhall G., head start

[9]

Dhall G., 2024, Neuro Oncol, V26, P0

[10] Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the "Head Start" I and II protocols [J].

Dhall, Girish ;

Grodman, Howard ;

Ji, Lingyun ;

Sands, Stephen ;

Gardner, Sharon ;

Dunkel, Ira J. ;

McCowage, Geoffrey B. ;

Diez, Blanca ;

Allen, Jeffrey C. ;

Gopalan, Anjali ;

Cornelius, Albert S. ;

Termuhlen, Amanda ;

Abromowitch, Minnie ;

Sposto, Richard ;

Finlay, Jonathan L. .

PEDIATRIC BLOOD & CANCER, 2008, 50 (06) :1169-1175

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