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 who received high-dose Carboplatin, Thiotepa according to the protocol {type:entrez-protein,attrs:{text:CCG99703,term_id:384066493,term_text:CCG99703}}CCG99703 between 1998-2012. Data on pathology, molecular subgrouping, chemotherapeutic, radiation, ototoxicity, neurognitive evaluations and survival were collected. RESULTS: There were 53(30 males) patients diagnosed at a median age of 24 months(2.9-63.2). Twenty(37.7%) had metastatic disease and 35(66%) underwent gross total resection(GTR). Seventeen(32.1%) had nodular desmoplastic(ND) subtype. Twenty two(56.4%) out the 39 patients with molecular subtyping belong to the SHH group. Three patients received intrathecal chemotherapy, 6 received HD MTX during induction and 7 underwent maintenance chemotherapy. Seventeen patients received radiation, including only 9(16.9%) in an adjuvant setting. Complete continuous remission(CCR) after induction and consolidation were respectively 73.6% and 77.4 %. Two patients died of treatment related toxicity. Forty-two patients are alive at a median follow-up of 3.7 years from diagnosis with a projected 5-year PFS and OS of respectively 69.2%(±7) and 76.1%(±6.5). ND histology, SHH subgroup and CCR were significantly associated better PFS, but only CCR post induction remained significant for better OS. Non irradiated children had a better PFS compare to those who received radiation(5y PFS 84.6% versus 42.2%p = 0.007). Severe ototoxicity(≥ Brock grade 3) was present in 13.2% of 31 evaluable patients. Thirteen required hearing support. Neurocognitive assessements were available in 24 survivors(57%). Mean FSIQ for the cohort was 91(range 67-119). CONCLUSION: Young children with MB treated with this strategy have a very encouraging OS (76.4 %) while only 17% of the patients who received adjuvant radiation. Although the ototoxicity was significant, neurocognitive profile of the survivors appears to be within normal range.
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