Trial reportPediatric neurology2015
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.
Trial report in Pediatric neurology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 86 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
86 citing papers in PubMed, 1 synthesis or guideline pooled it, 161 citations in OpenAlex.
- Desmoplastic nodular medulloblastoma in young children: a management dilemma.Neuro-oncology · 2018Pooled it
- Phase 3 randomized trial of high-dose methotrexate for young children with high-risk embryonal brain tumors: A report from the Children's Oncology Group.Neuro-oncology · 2025Trial
- Intensive Multimodality Therapy for Extraocular Retinoblastoma: A Children's Oncology Group Trial (ARET0321).Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2022Trial
- Vorinostat and isotretinoin with chemotherapy in young children with embryonal brain tumors: A report from the Pediatric Brain Tumor Consortium (PBTC-026).Neuro-oncology · 2022Trial
- Excellent outcome of young children with nodular desmoplastic medulloblastoma treated on "Head Start" III: a multi-institutional, prospective clinical trial.Neuro-oncology · 2020Trial
- Phase II Study of Nonmetastatic Desmoplastic Medulloblastoma in Children Younger Than 4 Years of Age: A Report of the Children's Oncology Group (ACNS1221).Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2020Trial
- Risk-adapted therapy for young children with medulloblastoma (SJYC07): therapeutic and molecular outcomes from a multicentre, phase 2 trial.The Lancet. Oncology · 2018Trial
- Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma.Neuro-oncology · 2017Trial
- Recurrent C19MC-altered embryonal tumors with multilayered rosettes: outcomes of stereotactic radiosurgery.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Article
- Real-world outcomes of radiotherapy timing in infants with medulloblastoma by molecular subtype: a single-center retrospective cohort study.Journal of neuro-oncology · 2026Article
- High-dose chemotherapy with autologous stem cell rescue in children under 5 years of age with central nervous system embryonal tumors: results from a prospective cohort in an upper-middle-income country.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Article
- Pediatric medulloblastoma.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Review
- Therapy and biomarker dependent progression-free survival in infant sonic hedgehog medulloblastoma: a multi-national retrospective cohort study.EClinicalMedicine · 2026Article
- Radiation-sparing management of WNT medulloblastoma of early childhood with high dose chemotherapy: In response to Gupta et al.Journal of neuro-oncology · 2026Article
- Craniospinal irradiation plus tumor-bed boost with standard adjuvant chemotherapy OR high-dose chemotherapy plus autologous stem cell transplant as a radiation-sparing approach in WNT medulloblastoma of early childhood: Fire OR Ice?Journal of neuro-oncology · 2026Article
- High-dose chemotherapy and craniospinal irradiation-sparing approach for WNT medulloblastoma of early childhood.Journal of neuro-oncology · 2026Article
- Report from the National Pediatric Cancer Foundation - infantile glioma and other non-embryonal central nervous system tumors: evolving molecular advances and current treatment landscape.Frontiers in oncology · 2026Review
- Pineal Tumors and Pineal Region Tumors.Advances and technical standards in neurosurgery · 2026Review
- Clinical, toxicity and long-term neurocognitive outcomes of first-line intensive chemotherapy in infant medulloblastoma: a single-center cohort study following AIEOP recommendations.Journal of neuro-oncology · 2025Article
- Outcomes of Infants and Young Children With CNS Embryonal Tumors Using Pre-Irradiation Chemotherapy: A Decade Long Experience.Cancer medicine · 2025Article
26 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors at 11 institutions in 1 country.
Funding
Abstract
backgroundThe primary goals of the Children's Cancer Group 99703 study were to assess the feasibility and tolerability of-as well as the response rate to-a novel dose-intensive chemotherapy regimen.
methodsBetween March 1998 and October 2004, 92 eligible patients were enrolled. Following biopsy/resection, patients received three identical cycles of Induction chemotherapy (vincristine, cyclophosphamide, etoposide, and cisplatin) administered every 21-28 days. Patients without tumor progression then received three consolidation cycles of marrow-ablative chemotherapy (thiotepa and carboplatin) followed by autologous hematopoietic cell rescue.
resultsThe maximum tolerated dose of thiotepa was 10 mg/kg/day × 2 days per cycle. The toxic mortality rate was zero during induction and 2.6% during consolidation. Centrally evaluated response rates to induction and consolidation in evaluable patients with residual tumor were 73.3% and 66.7%, respectively. Disease progression rates on induction and consolidation were 4%. Five-year event-free survival and overall survival were 43.9 ± 5.2% and 63.6 ± 5% respectively. Gross total resection versus less than gross total resection were the only significant outcome comparisons: 5-year maximum tolerated dose and overall survival of 54.4 ± 7% versus 28.9 ± 7% (P = 0.0065) and 75.9 ± 8% versus 48.7 ± 8% (P = 0.0034), respectively. The 5-year maximum tolerated dose for localized (M0) versus metastatic (M1+) medulloblastoma was 67.5 ± 9.5% versus 30 ± 14.5% (P = 0.007). The 5-year maximum tolerated dose and overall survival for desmoplastic medulloblastoma patients versus other medulloblastoma were 78.6 ± 11% versus 50.5 ± 12% (P = 0.038) and 85.7 ± 9.4% versus 60.6 ± 11.6% (P = 0.046), respectively.
conclusionsThis phase I dose-escalation study of marrow-ablative thiotepa regimen determined a maximum tolerated dose that had acceptable toxicity. Overall survival data justify this strategy for current Children's Oncology Group studies.
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