Feasibility of Proton Beam Therapy for Infants with Brain Tumours: Experiences from the Prospective KiProReg Registry Study

Jazmati, Danny; Steinmeier, Theresa; Khalil, Diana; Frisch, Sabine; Peters, Sarah; Schleithoff, Stefanie Schulze; Bäumer, Christian; Rutkowski, Stefan; Frühwald, Michael C.; Blase, C.; Tippelt, Stephan; Timmermann, Beate

doi:10.1016/j.clon.2021.03.006

Cited by 10 publications

(6 citation statements)

References 40 publications

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“…A recent study from Germany evaluated the treatment of infants with tumors of the central nervous system (1 patient with PB) with Proton Beam Therapy (PBT) and concluded that PBT is feasible for very young children with central nervous system tumors in the short term. 25 None of our patients received PBT, it is not available in our country yet.…”

Section: Discussionmentioning

confidence: 95%

Clinical characteristics, late effects and outcomes in pineoblastomas in children: a single center experience

Görgün¹,

Koç²,

Kebudi³

et al. 2021

Turk J Pediatr

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Pineoblastoma (PB) is a rare embryonal tumor of the pineal gland. 1 Histologically, PB are classified as WHO grade IV tumors. 2,3 While they typically appear radiographically as focal enhancing mass, PB can also be locally invasive and spread outside the pineal region through the subarachnoid space. 4 In this study, we assessed the demographics, treatment, late effects and outcome of PB patients who were diagnosed and treated in the Istanbul University Oncology Institute (IUOI) during 1990-2012. Material and MethodsDuring 1990-2012, 516 children with brain tumors were diagnosed and treated in the IUOI. Among these patients six (1.16 %) were diagnosed with PB. Demographics, age of diagnosis, first complaint, tumor region, diagnosis type, seeding metastasis to the spinal axis or cerebrospinal fluid (CSF), treatment and survival of these patients were evaluated retrospectively from patient records. All patients had cranial and spinal axis MRI with contrast at diagnosis and during follow-up.

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Section: Discussionmentioning

confidence: 95%

Clinical characteristics, late effects and outcomes in pineoblastomas in children: a single center experience

Görgün¹,

Koç²,

Kebudi³

et al. 2021

Turk J Pediatr

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“…Schuermann et al also presented low rates of acute treatment toxicity also considering concomitant chemotherapy in an overlapping cohort [ 34 ]. Previous publications on the KiProReg study regarding infants also found PBT to be a part of a complex multimodal treatment, but not being the main reason for acute toxicity and treatment interruptions [ 14 ].…”

Section: Discussionmentioning

confidence: 99%

“…Over time, more data have evolved, supporting low toxicity after PBT [ 10 , 11 , 12 , 13 ]. Even in very young children, recent studies have demonstrated good feasibility [ 14 ].…”

Section: Introductionmentioning

confidence: 99%

Proton Beam Therapy for Pediatric Tumors of the Central Nervous System—Experiences of Clinical Outcome and Feasibility from the KiProReg Study

Peters

Frisch²,

Stock

et al. 2022

Cancers

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As radiotherapy is an important part of the treatment in a variety of pediatric tumors of the central nervous system (CNS), proton beam therapy (PBT) plays an evolving role due to its potential benefits attributable to the unique dose distribution, with the possibility to deliver high doses to the target volume while sparing surrounding tissue. Children receiving PBT for an intracranial tumor between August 2013 and October 2017 were enrolled in the prospective registry study KiProReg. Patient’s clinical data including treatment, outcome, and follow-up were analyzed using descriptive statistics, Kaplan–Meier, and Cox regression analysis. Adverse events were scored according to the Common Terminology Criteria for Adverse Events (CTCAE) 4.0 before, during, and after PBT. Written reports of follow-up imaging were screened for newly emerged evidence of imaging changes, according to a list of predefined keywords for the first 14 months after PBT. Two hundred and ninety-four patients were enrolled in this study. The 3-year overall survival of the whole cohort was 82.7%, 3-year progression-free survival was 67.3%, and 3-year local control was 79.5%. Seventeen patients developed grade 3 adverse events of the CNS during long-term follow-up (new adverse event n = 7; deterioration n = 10). Two patients developed vision loss (CTCAE 4°). This analysis demonstrates good general outcomes after PBT.

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“…Still, a significant risk of distant treatment failures remains, ie, outside the former field of radiotherapy. 69 , 70 , 72 , 73 …”

Section: Clinical Management Of Atypical Teratoid Rhabdoid Tumors – C...mentioning

confidence: 99%

“…65 Studies by Yamasaki et al and Yang et al indicate that early vs late RT does not affect the treatment success in children below 3 years. 58,66,[68][69][70][71] Current evidence indicates that proton beam therapy (PBT) with doses equivalent to photons may achieve comparable results in terms of local tumor control and overall survival while potentially saving patients from toxicities, ie, symptomatic radiation necrosis. Still, a significant risk of distant treatment failures remains, ie, outside the former field of radiotherapy.…”

Section: Multimodal Treatment Approaches In the Management Of Atrtmentioning

confidence: 99%

Current Molecular and Clinical Landscape of ATRT – The Link to Future Therapies

Gastberger,

Fincke,

Mucha

et al. 2023

CMAR

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ATRT is a highly aggressive and rare pediatric CNS tumor of very young children. Its genetic hallmark is bi-allelic inactivation of SMARCB1 encoding INI1. Rarely SMARCA4 encoding BRG1 is affected. Up to 30% are associated with constitutional heterozygous pathogenic variants in one of the two genes, giving rise to the Rhabdoid-Tumor-Predisposition-Syndromes (RTPS) 1 and 2. Characteristic DNA methylation profiles distinguish ATRT from other SMARCB1 -deficient entities. Three distinct subtypes ATRT-MYC, -TYR, and -SHH are on record. ATRT-SHH may be further divided into the subgroups ATRT-SHH1A, -SHH1B, and -SHH2. The cure of ATRT remains challenging, notwithstanding an increasing understanding of molecular pathomechanisms and genetic background. The implementation of multimodal institutional treatment protocols has improved prognosis. Regardless of treatment approaches, clinical risk factors such as age, metastases, and DNA methylation subtype affect survival probability. We provide a critical appraisal of current conventional multimodal regimens and emerging targeted treatment approaches investigated in clinical trials and entity-specific registries. Intense treatment approaches featuring radiotherapy (RT) and high-dose chemotherapy (HDCT) face the difficulty of balancing tumor control and treatment-related toxicity. Current approaches focus on minimizing radiation fields by proton beam therapy or to withhold RT in HDCT-only approaches. Still, a 40–75% relapse rate upon first-line treatment reveals the need for novel treatment strategies in primary and even more in recurrent/refractory (r/r) disease. Among targeted treatments, immune checkpoint inhibitors and epigenetically active agents appear most promising. Success remains limited in single agent approaches. We hypothesize that mechanism-informed combination therapy will enhance response, as the low mutational burden of ATRT may contribute to acquiring resistance to single targeted agents. As DNA methylation group-specific gene expression profiles appear to influence response to distinct agents, the future treatment of ATRT should respect clinical and biological heterogeneity in risk group adjusted treatment protocols.

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Feasibility of Proton Beam Therapy for Infants with Brain Tumours: Experiences from the Prospective KiProReg Registry Study

Cited by 10 publications

References 40 publications

Clinical characteristics, late effects and outcomes in pineoblastomas in children: a single center experience

Clinical characteristics, late effects and outcomes in pineoblastomas in children: a single center experience

Proton Beam Therapy for Pediatric Tumors of the Central Nervous System—Experiences of Clinical Outcome and Feasibility from the KiProReg Study

Current Molecular and Clinical Landscape of ATRT – The Link to Future Therapies

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