An interim analysis of the national treatment guideline for paediatric germ cell tumours and sex cord stromal tumours by the South African Association of Paediatric Haematology Oncology

Abstract

Background: The South African Association of Paediatric Haematology Oncology (SAAPHO) developed a national treatment guideline for children with benign and malignant extracranial germ cell tumours (MEGCTs) and sex cord stromal tumours (SCSTs), named MEGCT-2016, and prospectively enrolled them into an observational study with standard-risk and high-risk treatment arms. Aim: To assess the adherence to the national treatment guideline, MEGCT-2016, and to evaluate the progress of patients at three years of enrollment. Setting: South African Paediatric oncology units (POUs). Methods: Patients eligible for the MEGCT-2016 study were enrolled at nine POUs. Patients were treated with surgery alone for limited-stage disease or surgery and chemotherapy for advanced disease. Patient, treatment-related, and outcome data were captured in a REDCap™ database. The Kaplan–Meier procedure was used to calculate the 3-year overall survival (OS). Toxicity data and audiology and glomerular filtration rate (GFR) testing practices are described and compared to historical cohorts. Results: A total of 63 record entries were analysed: 31 patients had MEGCTs, 26 benign EGCTs (19 mature teratomas and 7 immature teratomas) and 6 patients had SCSTs. The 3-year OS was improved in children with limited-stage MEGCTs (100% for Stage 1, Stage 2 and Stage 3 disease) compared to retrospective cohorts. Stage 4 patients had a less favourable 3-year OS (37.5%) compared to historical cohorts. Audiology and GFR assessments were markedly improved. Treatment-related morbidity was almost entirely restricted to advanced-stage patients following intensive chemotherapy. Conclusion: Prospective national treatment guideline implementation is feasible in South African POUs and has improved practice and outcomes compared to historical cohorts. The interim analysis has allowed modification of the treatment algorithm for patients with Stage 4 disease to improve outcomes in this group. Contribution: National paediatric cancer disease-specific treatment guidelines improves clinical practice and childhood cancer outcomes and creates a foundation from which to launch paediatric clinical trials in South African POUs.