Feasibility and Outcomes of Craniospinal Irradiation and Concurrent Daily Carboplatin in Children and Adults With High-risk Central Nervous System Tumours

Aims

High-risk medulloblastoma, pineoblastoma, and other embryonal central nervous system (CNS) tumours are associated with a poor prognosis. Children's Oncology Group trial CCG-99701 demonstrated concomitant daily carboplatin during radiotherapy, followed by adjuvant chemotherapy, to be a promising strategy for treating these malignancies in children. In this case series, we describe treatment feasibility, toxicities, and outcomes for children and adults from a heterogeneous real-world population, treated with this regimen for high-risk CNS tumours.

Materials and methods

Data from clinical records were collected on surgery, radiotherapy, chemotherapy, acute toxicities, supportive treatment, and survival for all patients treated with the regimen between January 2012 and June 2018.

Results

Twenty-seven patients (13 children and 14 adults) received chemoradiotherapy. Seven patients (26%) were rested for one or more radiotherapy fractions due to medical or anaesthetic issues, and 4 patients (15%) had an extended overall treatment time. Grade 4 haematological toxicity was common, but no grade 4 non-haematological toxicities were observed. Twenty-two patients (81%) required haematological support. Two patients discontinued adjuvant chemotherapy early due to toxicity. Two-year overall survival (OS) in paediatric and adult patients was 85% and 46%, respectively, and 5-year OS was 69% and 38%, respectively. Progression-free survival (PFS) was significantly longer in paediatric patients than in adults (median PFS not reached and 0.9 years, respectively; hazard ratio: 0.26; 95% confidence interval [CI]: 0.09 to 0.75; P = 0.01). There was no significant difference in OS between paediatric and adult patients (median OS not reached and 1.3 years, respectively; hazard ratio: 0.43; 95% CI: 0.14 to 1.29; P = 0.12).

Conclusion

The treatment regimen was feasible for paediatric and adult patients but was associated with significant toxicity. The survival outcomes in paediatric patients were favourable, consistent with published data. Due to the close clinical supervision required, this regimen should ideally be delivered in centres with experience in managing paediatric or teenage and young adult high-risk CNS tumours.