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Article: Non-Malignant Cerebrospinal Fluid Ascites in a Patient with Atypical Teratoid Rhabdoid Tumor

Upadhyaya, Santhosh A. / Klimo Jr., Paul / Robinson, Giles W.

2017 Volume 40, Issue 4, Page(s) 216–219

Abstract: Background: The development of symptomatic ascites from a ventriculoperitoneal shunt is rare in children without prior intra-abdominal pathology. Here we report a case of symptomatic ascites in a young child with a ventriculoperitoneal shunt and ... ...

Institution	Department of Oncology, Division of Neuro-Oncology, St. Jude Children's Research Hospital, Memphis, TN, USA Department of Neurosurgery, LeBonheur Children's Hospital, Memphis, TN, USA Semmes-Murphey Neurologic & Spine Institute, Memphis, TN, USA
Abstract	Background: The development of symptomatic ascites from a ventriculoperitoneal shunt is rare in children without prior intra-abdominal pathology. Here we report a case of symptomatic ascites in a young child with a ventriculoperitoneal shunt and metastatic atypical teratoid rhabdoid tumor. Case Report: This 15-month-old boy developed symptomatic ascites while undergoing chemotherapy for his malignant brain tumor. Diagnostic work up to identify the etiology of this ascites yielded a non-malignant, sterile transudate, refractory to repeated paracentesis. The ascites finally resolved with the conversion of the ventriculoperitoneal shunt to a ventriculoatrial shunt. Conclusion: This case report highlights the need to be suspicious of non-malignant cerebrospinal fluid ascites as a cause of peritoneal fluid accumulation even in children with malignant primary brain tumor. In this setting, conversion of the shunt from peritoneal to atrial drainage can rapidly alleviate the morbidity associated with this condition.
Keywords	Ventriculoperitoneal shunt ; Ventriculoatrial shunt ; Non-malignant CSF ascites ; Atypical teratoid rhabdoid tumor
Language	English
Publishing date	2017-03-21
Publisher	S. Karger GmbH
Publishing place	Freiburg, Germany
Document type	Article
Note	Novel Insights from Clinical Practice
ZDB-ID	2760274-6
ISSN	2296-5262 ; 2296-5270
ISSN (online)	2296-5262
ISSN	2296-5270
DOI	10.1159/000458441
Database	Karger publisher's database

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Article: Big Data Research in Pediatric Neurosurgery: Content, Statistical Output, and Bibliometric Analysis

Oravec, Chesney S. / Motiwala, Mustafa / Reed, Kevin / Jones, Tamekia L. / Klimo Jr., Paul

Pediatric Neurosurgery

2019 Volume 54, Issue 2, Page(s) 85–97

Abstract: Background/Aims: We sought to describe pediatric “big data” publications since 2000, their statistical output, and clinical implications. Methods: We searched 4 major North American neurosurgical journals for articles utilizing non-neurosurgery-specific ... ...

Institution	College of Medicine, University of Tennessee Health Science Center, Memphis, Tennessee, USA Departments of Pediatrics and Preventive Medicine, University of Tennessee Health Science Center, Memphis, Tennessee, USA Children’s Foundation Research Institute, Le Bonheur Children’s Hospital, Memphis, Tennessee, USA Department of Neurosurgery, University of Tennessee Health Science Center, Memphis, Tennessee, USA Semmes Murphey, Memphis, Tennessee, USA Le Bonheur Children’s Hospital, Memphis, Tennessee, USA
Abstract	Background/Aims: We sought to describe pediatric “big data” publications since 2000, their statistical output, and clinical implications. Methods: We searched 4 major North American neurosurgical journals for articles utilizing non-neurosurgery-specific databases for clinical pediatric neurosurgery research. Articles were analyzed for descriptive and statistical information. We analyzed effect sizes (ESs), confidence intervals (CIs), and p values for clinical relevance. A bibliometric analysis was performed using several key citation metrics. Results: We identified 74 articles, which constituted 1.7% of all pediatric articles (n = 4,436) published, with an exponential increase after 2013 (53/74, 72%). The Healthcare Cost and Utilization Project (HCUP) databases were most frequently utilized (n = 33); hydrocephalus (n = 19) was the most common study topic. The statistical output (n = 49 studies with 464 ESs, 456 CIs, and 389 p values) demonstrated that the majority of the ESs (253/464, 55%) were categorized as small; half or more of the CI spread (CIS) values and p values were high (274/456, 60%) and very strong (195/389, 50%), respectively. Associations with a combination of medium-to-large ESs (i.e., magnitude of difference), medium-to-high CISs (i.e., precision), and strong-to-very strong p values comprised only 20% (75/381) of the reported ESs. The total number of citations for the 74 articles was 1,115 (range per article, 0–129), with the median number of citations per article being 8.5. Four studies had > 50 citations, and 2 of them had > 100 citations. The calculated h-index was 16, h-core citations were 718, the e-index was 21.5, and the Google i10-index was 34. Conclusions: There has been a dramatic increase in the use of “big data” in the pediatric neurosurgical literature. Reported associations that may, as a group, be of greatest interest to practitioners represented only 20% of the total output from these publications. Citations were weighted towards a few highly cited publications.
Keywords	Administrative databases ; Big data ; Outcomes ; Pediatric neurosurgery
Language	English
Publishing date	2019-02-20
Publisher	S. Karger AG
Publishing place	Basel, Switzerland
Document type	Article
Note	Original Paper
ZDB-ID	1091757-3
ISSN	1423-0305 ; 1016-2291
ISSN (online)	1423-0305
ISSN	1016-2291
DOI	10.1159/000495790
Database	Karger publisher's database

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Zs.A 1133: Show issues

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Article: Emergent and Urgent Craniotomies in Pediatric Patients: Resource Utilization and Cost Analysis

Ajmera, Sonia / Motiwala, Mustafa / Lingo, Ryan / Khan, Nickalus R. / Smith, Lydia J. / Giles, Kim / Vaughn, Brandy / Klimo Jr., Paul

Pediatric Neurosurgery

2019 Volume 54, Issue 5, Page(s) 301–309

Abstract: Background: Pediatric neurosurgeons are occasionally tasked with performing surgery expeditiously to preserve a child’s neurologic faculties and life. Objective: This study examines the etiologies, outcomes, and costs for urgent or emergent craniotomies ... ...

Institution	College of Medicine, University of Tennessee Health Science Center, Memphis, Tennessee, USA Neurological & Spine Institute, Savannah, Georgia, USA Department of Neurosurgery, University of Tennessee Health Science Center, Memphis, Tennessee, USA Le Bonheur Children’s Hospital, Memphis, Tennessee, USA Semmes Murphey, Memphis, Tennessee, USA
Abstract	Background: Pediatric neurosurgeons are occasionally tasked with performing surgery expeditiously to preserve a child’s neurologic faculties and life. Objective: This study examines the etiologies, outcomes, and costs for urgent or emergent craniotomies at a Level I Pediatric Trauma center over a 7-year time period. Methods: A retrospective review was conducted for each patient who underwent an emergent or urgent craniotomy within 24 hours of presentation between January 2010 and April 2017. Demographic, clinical, and surgical details were recorded for a total of 48 variables. Any readmission within 90 days was analyzed. Hospital charges for each admission and readmission were collected and adjusted for inflation to October 2018 values. Results: Among the 223 children who underwent urgent or emergent craniotomies, the majority were admitted for traumatic injuries (n = 163, 73.1%). The most common traumatic mechanism was fall (n = 51, 22.9%), and the most common non-traumatic cause was tumor (n = 21, 9.4%). Overall, craniotomies were typically performed for hematoma evacuation of one type or combination (n = 115, 51.6%) during off-peak times (n = 178, 79.8%). Seventy-seven (34.5%) subjects experienced 1 or more postoperative events, 22 of whom returned to the operating room. There were 13 (5.8%) and 33 (14.8%) readmissions within 30 days and 90 days of discharge, respectively. Non-trauma patients (compared with trauma patients) and polytrauma (compared with isolated head injury) had greater healthcare needs, resulting in higher charges. Conclusion: Most urgent or emergent pediatric craniotomies were performed for the treatment of traumatic injuries involving hematoma evacuation, but non-traumatic patients were more complex requiring greater resources.
Keywords	Emergent/urgent craniotomy ; Pediatric trauma ; Costs ; Resources
Language	English
Publishing date	2019-08-09
Publisher	S. Karger AG
Publishing place	Basel, Switzerland
Document type	Article
Note	Research Article
ZDB-ID	1091757-3
ISSN	1423-0305 ; 1016-2291
ISSN (online)	1423-0305
ISSN	1016-2291
DOI	10.1159/000501042
Database	Karger publisher's database

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Article: Potentially useful outcome measures for clinical research in pediatric neurosurgery.

Klimo, Paul / Kestle, John R W

Journal of neurosurgery

2005 Volume 103, Issue 3 Suppl, Page(s) 207–212

Abstract: The choice of outcome (or outcomes) and their measurement are critical for a sound clinical trial. Surgeons have traditionally measured simple outcomes such as death, duration of survival, or tumor recurrence but have recently developed more ... ...

Abstract	The choice of outcome (or outcomes) and their measurement are critical for a sound clinical trial. Surgeons have traditionally measured simple outcomes such as death, duration of survival, or tumor recurrence but have recently developed more sophisticated measures of the effect of an intervention. Many outcome measures require a lengthy maturation process, which includes a determination of the instrument's validity, reliability, and sensitivity; thus, using established instruments rather than creating new ones is recommended. The authors illustrate several guidelines for the determination of appropriate outcome measures by using examples from their experience and describe several outcome measures that can be used in pediatric neurosurgery. These include general outcome measures such as the Pediatric Evaluation of Disability Inventory and the Functional Independence Measure for Children, which measure physical function and independence in chronically ill and disabled children as well as disease-specific measures for hydrocephalus (Hydrocephalus Outcome Questionnaire), cerebral palsy (gross motor function and performance measures), head injury (Pediatric Cerebral Performance Category and Children's Coma Scale), and oncology (Pediatric Cancer Quality-of-Life Inventory).
MeSH term(s)	Activities of Daily Living ; Child ; Child, Preschool ; Disabled Children/classification ; Guidelines as Topic ; Health Status ; Humans ; Infant ; Infant, Newborn ; Neurosurgical Procedures/standards ; Outcome Assessment (Health Care) ; Pediatrics/standards ; Reproducibility of Results ; Sensitivity and Specificity ; Surveys and Questionnaires ; Treatment Outcome
Language	English
Publishing date	2005-09
Publishing country	United States
Document type	Journal Article
ZDB-ID	3089-2
ISSN	1933-0693 ; 0022-3085
ISSN (online)	1933-0693
ISSN	0022-3085
DOI	10.3171/ped.2005.103.3.0207
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Article: Traumatic atlanto-occipital dislocation in children.

Astur, Nelson / Sawyer, Jeffrey R / Klimo, Paul / Kelly, Derek M / Muhlbauer, Michael / Warner, William C

The Journal of the American Academy of Orthopaedic Surgeons

2014 Volume 22, Issue 5, Page(s) 274–282

Abstract: Although once considered an invariably fatal injury, improvements in diagnosis and management have made atlanto-occipital dislocation (AOD) a survivable injury. MRI is the preferred imaging modality; occasionally, flexion/extension/distraction ... ...

Abstract	Although once considered an invariably fatal injury, improvements in diagnosis and management have made atlanto-occipital dislocation (AOD) a survivable injury. MRI is the preferred imaging modality; occasionally, flexion/extension/distraction fluoroscopy may be required to determine craniovertebral stability. Early surgical stabilization is recommended for all children with AOD. Early occipitocervical fusion using screws in combination with a rod or plate, or sublaminar wires with a contoured rod, coupled with autograft bone, provide immediate stabilization and a high fusion rate. Halo immobilization and traction are contraindicated in the management of AOD in children because of the risk of displacement of the injured occipitocervical joint. Postoperative hydrocephalus is frequent and should be suspected when neurologic decline occurs after fixation. Nearly half of children who survive AOD will have residual neurologic deficits.
MeSH term(s)	Atlanto-Occipital Joint/diagnostic imaging ; Atlanto-Occipital Joint/injuries ; Child ; Child, Preschool ; External Fixators ; Humans ; Hydrocephalus/etiology ; Infant ; Joint Dislocations/complications ; Joint Dislocations/diagnosis ; Joint Dislocations/surgery ; Magnetic Resonance Imaging ; Prognosis ; Radiography ; Spinal Fusion
Language	English
Publishing date	2014-05
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	1200524-1
ISSN	1940-5480 ; 1067-151X
ISSN (online)	1940-5480
ISSN	1067-151X
DOI	10.5435/JAAOS-22-05-274
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Article ; Online: Traumatic atlanto-occipital dislocation in children: evaluation, treatment, and outcomes.

Astur, Nelson / Klimo, Paul / Sawyer, Jeffrey R / Kelly, Derek M / Muhlbauer, Michael S / Warner, William C

The Journal of bone and joint surgery. American volume

2013 Volume 95, Issue 24, Page(s) e194(1–8)

Abstract: Background: Advancements in emergency care and diagnostic methods have increased the number of children who survive atlanto-occipital dislocation. We report our experience with one of the largest series of pediatric patients with atlanto-occipital ... ...

Abstract	Background: Advancements in emergency care and diagnostic methods have increased the number of children who survive atlanto-occipital dislocation. We report our experience with one of the largest series of pediatric patients with atlanto-occipital dislocation. Methods: Patients ranging in age from newborn to sixteen years old who had a diagnosis of atlanto-occipital dislocation from 1991 through 2011 were identified. Inclusion criteria were complete radiographic and clinical records and duration of follow-up of at least six months. Basic patient characteristics, mechanism of injury, associated injuries, neurological impairment, surgical treatment and type of implant used for fixation, complications, and clinical and radiographic outcomes were recorded. Results: The fourteen patients who were included (seven male and seven female) had a mean age of 5.2 years at the time of injury. An automobile accident in which the victim was a passenger was the most common mechanism of injury. Twelve patients had associated injuries, with a brain injury in eleven of them, and nearly half sustained a spinal cord injury. According to the Traynelis classification system, eight patients had a type-II (longitudinal) atlanto-occipital dislocation, five had a type-I (anterior) dislocation, and one had a type-III (posterior) dislocation. All patients had posterior occipitocervical fusion with internal fixation. The mean duration of follow-up was 75.4 months. The most common postoperative complication was hydrocephalus, which occurred in four patients. Spinal fusion occurred in all patients by four to six months postoperatively. At the time of the most recent follow-up evaluation, half of the patients had neurological impairment. Conclusions: More patients with atlanto-occipital dislocation now survive the initial trauma, although most have associated injuries and many have neurological impairment. Our preferred management is early occipitocervical fusion and stabilization. If there is neurological decline after spinal fixation, obstructive hydrocephalus should be suspected.
MeSH term(s)	Accidents, Traffic ; Adolescent ; Atlanto-Occipital Joint/injuries ; Brain Injuries/complications ; Brain Injuries/diagnosis ; Brain Injuries/surgery ; Child ; Child, Preschool ; Female ; Humans ; Infant ; Infant, Newborn ; Joint Dislocations/complications ; Joint Dislocations/diagnostic imaging ; Joint Dislocations/surgery ; Male ; Radiography ; Spinal Cord Injuries/complications ; Spinal Cord Injuries/diagnosis ; Spinal Cord Injuries/surgery ; Spinal Fusion ; Treatment Outcome
Language	English
Publishing date	2013-12-18
Publishing country	United States
Document type	Journal Article
ZDB-ID	220625-0
ISSN	1535-1386 ; 0021-9355
ISSN (online)	1535-1386
ISSN	0021-9355
DOI	10.2106/JBJS.L.01295
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Article ; Online: Phase II study of alisertib as a single agent for treating recurrent or progressive atypical teratoid/rhabdoid tumor.

Upadhyaya, Santhosh A / Campagne, Olivia / Billups, Catherine A / Orr, Brent A / Onar-Thomas, Arzu / Tatevossian, Ruth G / Mostafavi, Roya / Myers, Jason R / Vinitsky, Anna / Moreira, Daniel C / Lindsay, Holly B / Kilburn, Lindsay / Baxter, Patricia / Smith, Amy / Crawford, John R / Partap, Sonia / Bendel, Anne E / Aguilera, Dolly G / Nichols, Kim E /

Rampersaud, Evadnie / Ellison, David W / Klimo, Paul / Patay, Zoltan / Robinson, Giles W / Broniscer, Alberto / Stewart, Clinton F / Wetmore, Cynthia / Gajjar, Amar

Neuro-oncology

2022 Volume 25, Issue 2, Page(s) 386–397

Abstract: Background: Recurrent atypical teratoid/rhabdoid tumor (AT/RT) is, most often, a fatal pediatric malignancy with limited curative options.: Methods: We conducted a phase II study of Aurora kinase A inhibitor alisertib in patients aged <22 years with ... ...

Abstract	Background: Recurrent atypical teratoid/rhabdoid tumor (AT/RT) is, most often, a fatal pediatric malignancy with limited curative options. Methods: We conducted a phase II study of Aurora kinase A inhibitor alisertib in patients aged <22 years with recurrent AT/RT. Patients received alisertib once daily (80 mg/m2 as enteric-coated tablets or 60 mg/m2 as liquid formulation) on Days 1-7 of a 21-day cycle until progressive disease (PD) occurred. Alisertib plasma concentrations were measured in cycle 1 on Days 1 (single dose) and 7 (steady state) and analyzed with noncompartmental pharmacokinetics. Trial efficacy end point was ≥10 participants with stable disease (SD) or better at 12 weeks. Results: SD (n = 8) and partial response (PR) (n = 1) were observed among 30 evaluable patients. Progression-free survival (PFS) was 30.0% ± 7.9% at 6 months and 13.3% ± 5.6% at 1 year. One-year overall survival (OS) was 36.7% ± 8.4%. Two patients continued treatment for >12 months. PFS did not differ by AT/RT molecular groups. Neutropenia was the most common adverse effect (n = 23/30, 77%). The 22 patients who received liquid formulation had a higher mean maximum concentration (Cmax) of 10.1 ± 3.0 µM and faster time to Cmax (Tmax = 1.2 ± 0.7 h) than those who received tablets (Cmax = 5.7 ± 2.4 µM, Tmax = 3.4 ± 1.4 h). Conclusions: Although the study did not meet predetermined efficacy end point, single-agent alisertib was well tolerated by children with recurrent AT/RT, and SD or PR was observed in approximately a third of the patients.
MeSH term(s)	Child ; Humans ; Antineoplastic Agents/therapeutic use ; Rhabdoid Tumor/drug therapy ; Azepines/therapeutic use ; Pyrimidines/therapeutic use ; Central Nervous System Neoplasms/drug therapy ; Aurora Kinase A ; Protein Kinase Inhibitors/therapeutic use ; Protein Kinase Inhibitors/adverse effects
Chemical Substances	Antineoplastic Agents ; MLN 8237 ; Azepines ; Pyrimidines ; Aurora Kinase A (EC 2.7.11.1) ; Protein Kinase Inhibitors
Language	English
Publishing date	2022-06-02
Publishing country	England
Document type	Clinical Trial, Phase II ; Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	2028601-6
ISSN	1523-5866 ; 1522-8517
ISSN (online)	1523-5866
ISSN	1522-8517
DOI	10.1093/neuonc/noac151
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Zs.A 5307: Show issues

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Article ; Online: Serial assessment of measurable residual disease in medulloblastoma liquid biopsies.

Liu, Anthony P Y / Smith, Kyle S / Kumar, Rahul / Paul, Leena / Bihannic, Laure / Lin, Tong / Maass, Kendra K / Pajtler, Kristian W / Chintagumpala, Murali / Su, Jack M / Bouffet, Eric / Fisher, Michael J / Gururangan, Sridharan / Cohn, Richard / Hassall, Tim / Hansford, Jordan R / Klimo, Paul / Boop, Frederick A / Stewart, Clinton F /

Harreld, Julie H / Merchant, Thomas E / Tatevossian, Ruth G / Neale, Geoffrey / Lear, Matthew / Klco, Jeffery M / Orr, Brent A / Ellison, David W / Gilbertson, Richard J / Onar-Thomas, Arzu / Gajjar, Amar / Robinson, Giles W / Northcott, Paul A

Cancer cell

2021 Volume 39, Issue 11, Page(s) 1519–1530.e4

Abstract: Nearly one-third of children with medulloblastoma, a malignant embryonal tumor of the cerebellum, succumb to their disease. Conventional response monitoring by imaging and cerebrospinal fluid (CSF) cytology remains challenging, and a marker for ... ...

Abstract	Nearly one-third of children with medulloblastoma, a malignant embryonal tumor of the cerebellum, succumb to their disease. Conventional response monitoring by imaging and cerebrospinal fluid (CSF) cytology remains challenging, and a marker for measurable residual disease (MRD) is lacking. Here, we show the clinical utility of CSF-derived cell-free DNA (cfDNA) as a biomarker of MRD in serial samples collected from children with medulloblastoma (123 patients, 476 samples) enrolled on a prospective trial. Using low-coverage whole-genome sequencing, tumor-associated copy-number variations in CSF-derived cfDNA are investigated as an MRD surrogate. MRD is detected at baseline in 85% and 54% of patients with metastatic and localized disease, respectively. The number of MRD-positive patients declines with therapy, yet those with persistent MRD have significantly higher risk of progression. Importantly, MRD detection precedes radiographic progression in half who relapse. Our findings advocate for the prospective assessment of CSF-derived liquid biopsies in future trials for medulloblastoma.
MeSH term(s)	Biomarkers, Tumor/cerebrospinal fluid ; Biomarkers, Tumor/genetics ; Cell-Free Nucleic Acids/cerebrospinal fluid ; Cerebellar Neoplasms/cerebrospinal fluid ; Cerebellar Neoplasms/diagnosis ; Cerebellar Neoplasms/genetics ; Child ; Chromosomal Instability ; DNA Copy Number Variations ; Disease Progression ; Female ; Humans ; Liquid Biopsy ; Male ; Medulloblastoma/cerebrospinal fluid ; Medulloblastoma/diagnosis ; Medulloblastoma/genetics ; Neoplasm, Residual ; Prospective Studies ; Whole Genome Sequencing/methods
Chemical Substances	Biomarkers, Tumor ; Cell-Free Nucleic Acids
Language	English
Publishing date	2021-10-21
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	2078448-X
ISSN	1878-3686 ; 1535-6108
ISSN (online)	1878-3686
ISSN	1535-6108
DOI	10.1016/j.ccell.2021.09.012
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Zs.A 5650: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
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Article ; Online: Relevance of Molecular Groups in Children with Newly Diagnosed Atypical Teratoid Rhabdoid Tumor: Results from Prospective St. Jude Multi-institutional Trials.

Upadhyaya, Santhosh A / Robinson, Giles W / Onar-Thomas, Arzu / Orr, Brent A / Johann, Pascal / Wu, Gang / Billups, Catherine A / Tatevossian, Ruth G / Dhanda, Sandeep Kumar / Srinivasan, Ashok / Broniscer, Alberto / Qaddoumi, Ibrahim / Vinitsky, Anna / Armstrong, Gregory T / Bendel, Anne E / Hassall, Tim / Partap, Sonia / Fisher, Paul G / Crawford, John R /

Chintagumpala, Murali / Bouffet, Eric / Gururangan, Sridharan / Mostafavi, Roya / Sanders, Robert P / Klimo, Paul / Patay, Zoltan / Indelicato, Daniel J / Nichols, Kim E / Boop, Frederick A / Merchant, Thomas E / Kool, Marcel / Ellison, David W / Gajjar, Amar

Clinical cancer research : an official journal of the American Association for Cancer Research

2021 Volume 27, Issue 10, Page(s) 2879–2889

Abstract: Purpose: Report relevance of molecular groups to clinicopathologic features, germline : Materials and methods: Seventy-four participants with newly diagnosed ATRT were treated in two trials: infants (SJYC07: age < 3 years; : Results: Twenty-four ... ...

Abstract	Purpose: Report relevance of molecular groups to clinicopathologic features, germline Materials and methods: Seventy-four participants with newly diagnosed ATRT were treated in two trials: infants (SJYC07: age < 3 years; Results: Twenty-four participants (32%) were alive at time of analysis at a median follow-up of 8.4 years (range, 3.1-14.1 years). Methylation profiling classified 64 ATRTs as TYR ( Conclusions: Among infants, those with ATRT-TYR had the best OS. ATRT-SHH was associated with metastases and consequently with inferior outcomes. Children with nonmetastatic ATRT benefit from postoperative CSI and adjuvant chemotherapy.
MeSH term(s)	Antineoplastic Combined Chemotherapy Protocols/adverse effects ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use ; Biomarkers, Tumor ; Child ; Child, Preschool ; DNA Copy Number Variations ; DNA Methylation ; Diagnosis, Differential ; Disease Management ; Disease Susceptibility ; Female ; Germ-Line Mutation ; Humans ; Infant ; Male ; Mutation ; Prognosis ; Rhabdoid Tumor/diagnosis ; Rhabdoid Tumor/etiology ; Rhabdoid Tumor/mortality ; Rhabdoid Tumor/therapy ; SMARCB1 Protein/genetics ; Teratoma/diagnosis ; Teratoma/etiology ; Teratoma/mortality ; Teratoma/therapy ; Treatment Outcome
Chemical Substances	Biomarkers, Tumor ; SMARCB1 Protein ; SMARCB1 protein, human
Language	English
Publishing date	2021-03-18
Publishing country	United States
Document type	Clinical Trial, Phase II ; Journal Article ; Multicenter Study ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	1225457-5
ISSN	1557-3265 ; 1078-0432
ISSN (online)	1557-3265
ISSN	1078-0432
DOI	10.1158/1078-0432.CCR-20-4731
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Zs.A 4223: Show issues

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Article: Clinical trials and evidence-based medicine for metastatic spine disease.

Klimo, Paul / Kestle, John R W / Schmidt, Meic H

Neurosurgery clinics of North America

2004 Volume 15, Issue 4, Page(s) 549–564

Abstract: Treatment of metastatic epidural spinal disease has undergone significant changes over the last 20 years. No longer is indiscriminate decompressive laminectomy offered as the only surgical treatment. It carries all the risks associated with an invasive ... ...

Abstract	Treatment of metastatic epidural spinal disease has undergone significant changes over the last 20 years. No longer is indiscriminate decompressive laminectomy offered as the only surgical treatment. It carries all the risks associated with an invasive procedure and offers the patient little benefit unless it is used to remove disease isolated to the posterior elements. The existing literature suggests that surgery that frees the spinal cord at the site of compression in addition to reconstructing and stabilizing the spinal column is more effective at preserving and regaining neural function, notably ambulatory function and sphincter function, than conventional radiotherapy. It is also highly effective in relieving pain. The preliminary results ofa recent RCT provide the first class I evidence to support a reversal in the current philosophy of primary treatment for many patients with meta-static disease. Conventional radiotherapy has a clearly defined role as adjuvant therapy and as primary therapy in those who are unable to tolerate or benefit significantly from surgery. The role of nonconventional radiation therapy, such as IMRT and SRS, remains to be elucidated.
MeSH term(s)	Clinical Trials as Topic ; Decompression, Surgical ; Evidence-Based Medicine ; Humans ; Orthopedic Procedures ; Spinal Cord Compression/etiology ; Spinal Cord Compression/therapy ; Spinal Neoplasms/secondary ; Spinal Neoplasms/therapy ; Treatment Outcome
Language	English
Publishing date	2004-10
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	1196855-2
ISSN	1558-1349 ; 1042-3680
ISSN (online)	1558-1349
ISSN	1042-3680
DOI	10.1016/j.nec.2004.04.016
Database	MEDical Literature Analysis and Retrieval System OnLINE

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