High‐dose chemotherapy with autologous stem cell transplantation in infants and young children with ependymoma: A 10‐year experience with the Head Start II protocol

Rajagopal, Revathi; Foo, Jen Chun; Jawin, Vida; Qaddoumi, Ibrahim; Bouffet, Éric

doi:10.1111/petr.13421

Cited by 6 publications

(4 citation statements)

References 9 publications

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“…Ten-year OS and PFS decrease further to 50 ± 5% and 29 ± 5%, respectively 1 . The use of chemotherapeutic agents for EPN has been extensively studied for decades, however, survival benefit remains controversial 6 , 7 . In-depth understanding of the biology of EPN recurrence is needed.…”

Section: Introductionmentioning

confidence: 99%

Epigenetic Alterations of Repeated Relapses in Patient-matched Childhood Ependymomas

Zhao

Zhang

et al. 2022

Nat Commun

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Recurrence is frequent in pediatric ependymoma (EPN). Our longitudinal integrated analysis of 30 patient-matched repeated relapses (3.67 ± 1.76 times) over 13 years (5.8 ± 3.8) reveals stable molecular subtypes (RELA and PFA) and convergent DNA methylation reprogramming during serial relapses accompanied by increased orthotopic patient derived xenograft (PDX) (13/27) formation in the late recurrences. A set of differentially methylated CpGs (DMCs) and DNA methylation regions (DMRs) are found to persist in primary and relapse tumors (potential driver DMCs) and are acquired exclusively in the relapses (potential booster DMCs). Integrating with RNAseq reveals differentially expressed genes regulated by potential driver DMRs (CACNA1H, SLC12A7, RARA in RELA and HSPB8, GMPR, ITGB4 in PFA) and potential booster DMRs (PLEKHG1 in RELA and NOTCH, EPHA2, SUFU, FOXJ1 in PFA tumors). DMCs predicators of relapse are also identified in the primary tumors. This study provides a high-resolution epigenetic roadmap of serial EPN relapses and 13 orthotopic PDX models to facilitate biological and preclinical studies.

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Section: Introductionmentioning

confidence: 99%

Epigenetic Alterations of Repeated Relapses in Patient-matched Childhood Ependymomas

Zhao

Zhang

et al. 2022

Nat Commun

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“…6 Concurrently, the Head Start I and II results were reported on infant ependymomas treated with five cycles of intensive induction chemotherapy utilizing high-dose methotrexate and consolidation with one cycle of AuHSCR. 8,15 In one study, CSI therapy was applied only for those with less than complete response after consolidation. 8 There were five metastatic patients in the 29 ependymoma cohort, four of whom achieved a complete response after chemotherapy induction.…”

Section: Management and Outcomes In Radiation-sparing Approachesmentioning

confidence: 99%

“…Start II approach without methotrexate, two children <3 years died before completion of induction chemotherapy (one for tumor progression and the other for sepsis). 15 Subsequently, Head Start III pursued a similar strategy for infant ependymoma. 16 There are additional radiation sparing strategies reported on infant ependymomas, although the characteristics and outcomes of metastatic patients in the cohorts were not reported.…”

Section: Management and Outcomes In Radiation-sparing Approachesmentioning

confidence: 99%

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Infantile metastatic ependymoma with a novel molecular profile and favorable outcome to intensive chemotherapy without irradiation: Case‐based review

Schieffer

et al. 2022

Genes Chromosomes & Cancer

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Ependymal tumors are the third most common brain tumor under 14 years old. Even though metastatic disease is a rare event, it affects mostly young children and carries an adverse prognosis. The factors associated with dissemination and the best treatment approach have not yet been established and there is limited published data on how to manage metastatic disease, especially in patients under 3 years of age. We provide a review of the literature on clinical characteristics and radiation‐sparing treatments for metastatic ependymoma in children under 3 years of age treated. The majority (73%) of the identified cases were above 12 months old and had the PF as the primary site at diagnosis. Chemotherapy‐based approaches, in different regimens, were used with radiation reserved for progression or relapse. The prognosis varied among the studies, with an average of 50%–58% overall survival. This study also describes the case of a 7‐month‐old boy with metastatic posterior fossa (PF) ependymoma, for whom we identified a novel SPECC1L‐RAF1 gene fusion using a patient‐centric comprehensive molecular profiling protocol. The patient was successfully treated with intensive induction chemotherapy followed by high‐dose chemotherapy and autologous hematopoietic progenitor cell rescue (AuHSCR). Currently, the patient is in continuous remission 5 years after his diagnosis, without radiation therapy. The understanding of the available therapeutic approaches may assist physicians in their management of such patients. This report also opens the perspective of newly identified molecular alterations in metastatic ependymomas that might drive more chemo‐sensitive tumors.

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High-Dose Chemotherapy/Stem Cell Transplantation (HDSCT)

Schechter,

Rassekh

2024

Pediatric Neuro-Oncology

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High‐dose chemotherapy with autologous stem cell transplantation in infants and young children with ependymoma: A 10‐year experience with the Head Start II protocol

Cited by 6 publications

References 9 publications

Epigenetic Alterations of Repeated Relapses in Patient-matched Childhood Ependymomas

Epigenetic Alterations of Repeated Relapses in Patient-matched Childhood Ependymomas

Infantile metastatic ependymoma with a novel molecular profile and favorable outcome to intensive chemotherapy without irradiation: Case‐based review

High-Dose Chemotherapy/Stem Cell Transplantation (HDSCT)

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