MB-35 * VERY ENCOURAGING LONG-TERM SURVIVAL AND NEUROCOGNITIVE OUTCOME OF YOUNG CHILDREN TREATED FOR MEDULLOBLASTOMA WITH SEQUENTIAL HIGH DOSE CHEMOTHERAPY
Bibliographic record
Abstract
BACKGROUND: High dose chemotherapy strategies were developed to avoid craniospinal irradiation and prevent unacceptable neurotoxicity in young children. However, long-term outcome, including neurocognitive outcome of this approach has not been widely described. METHODS: This retrospective study collected data from 7 institutions, on young children with medulloblastoma treated with high-dose Carboplatin, Thiotepa according to the protocol CCG99703 between 1998-2012. Data on pathology, molecular subgrouping, chemotherapeutic, radiation, ototoxicity, neurocognitive evaluations and survival were collected. RESULTS: The cohort included 53(30 males) patients diagnosed at a median age of 24 months (2.9-63.2). Twenty (37.7%) were metastatic at diagnosis and 35(66%) underwent gross total resection(GTR). Seventeen patients (32.1%) had nodular desmoplastic (ND) histology. SHH subgroup accounted for 55.8% of the 43 patients tested (including 8/24 classic medulloblastoma). Seventeen (32.1%) patients received radiation, including 9 (16.9%) in an adjuvant setting. The 5 y PFS and OS for the entire cohort was 69.6% (±6.9%) and 76.1%(±6.5) respectively. The 5 y PFS for the patients with classic MB treated without adjuvant RT was 58.5%(±11.5%). GTR, ND histology, SHH subgroup, and continuous complete remission(CCR) were significantly associated with better PFS, but only the SHH subgroup, and CCR post consolidation remained significant for better OS, whereas macroscopic metastasis(M2/3) was associated with worst survival. Adjuvant radiation was not associated with improved outcome. Fifteen(45.5% of tested patients) patients had severe ototoxicity(≥ Chan 2b) including 13 who required hearing support. Neurocognitive assessments were available in 24 surviving patients (57%). Mean FSIQ for the cohort was 91.6 (52-119). CONCLUSION: Young children with medulloblastoma treated with this strategy have a very encouraging OS(76.1%) with < 20% patients who received adjuvant radiation. For classic medulloblastoma patients, this strategy shows an encouraging PFS of 58% without adjuvant radiation. Although ototoxicity is significant, neurocognitive profile of the survivors appears to be within the normal range.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".