Browsing by Author "Van Heerden, Jaques"
Now showing 1 - 2 of 2
Results Per Page
Sort Options
- ItemThe management and outcomes of neuroblastoma in South Africa(Stellenbosch : Stellenbosch University, 2021-03) Van Heerden, Jaques; Kruger, Mariana; Stellenbosch University. Faculty of Medicine and Health Sciences. Dept. of Paediatrics and Child Health.ENGLISH ABSTRACT: Neuroblastoma (NB) is the second most diagnosed childhood solid tumour in high-income countries (HIC), but the incidence has not accurately been described in low- and middle-income countries (LMICs). The diagnostic difficulty, with limited treatment modalities, contributes to poor outcomes in LMICs. This PhD dissertation investigates the management of NB in South Africa with the aim to develop the first prospective national neuroblastoma treatment protocol/clinical trial to improve overall survival (OS).Using the South African Cancer Study Group’s Tumour Registry data, between 2000 and 2016 the incidence of NB in South Africa was found to be between 1.74 to 2.6 cases/million children, which waslower than the 10.5 cases per million children reported in HICs. South Africa had a higher number of patients with high-risk (HR) tumours (75.6%), mainly due to advanced disease (70%). The 2-year OS was excellent for very low risk (VLR) (94.1%) and low risk (LR) disease (81.6%), while acceptable for intermediate risk (IR) disease (66.7%) but poor for HR disease (27.6%) (p<0.001, 95% CI). Limitations in risk stratification included the low number of tumours tested for MYCN (38.4%), with more than half being MYCN-amplified (54%), and no other NB related genetic characteristics. Several treatment protocols were used in the different paediatric oncology units in South Africa during the study period (2000-2014) and the OPEC/OJEC (carboplatin, cisplatin, etoposide, cyclophosphamide and vincristine) induction chemotherapy regimen proved to be the least toxic with better metastatic remission rates for HR-NB. Ferritin had predictive value for complete metastatic remission rate, while LDH had predictive value for two-year OS and were found to be suitable tumour markers to use as surrogates for sophisticated genetic testing and mIBG-scans in the context of limited resources. Age at diagnosis, specifically the 18-month cut-point value, remained a significant prognostic factor, similar to HICs.Due to limited access to autologous stem cell transplants, the role of surgery and radiotherapy in the management of HR disease were investigated and found to significantly improve five-year OS with surgery and marginally with radiotherapy (p<0.001, 95% CI). Furthermore, the disparities in neuroblastoma health care provision in the different provinces in South Africa was found to exist and should be addressed to ensure equitable health care provision for all children as per the South African Constitution. The implementation of the newly developed national NB single arm clinical trial in South Africa, adjusted to available national resources, was a complex process with major navigational bureaucratic challenges. Yet the process might serve as a guideline for similar processes in LMICs. The recruitment of patients into the national NB clinical trial proved to be difficult due to both the COVID-19 pandemic and reluctance to recruit advanced stage patients into clinical trials. However, with careful investigation and in collaborative spirit, rare diseases such as neuroblastoma in South Africa could be managed in national management protocols, aimed at improving overall survival and cure.
- ItemManagement of neuroblastoma in limited-resource settings(Baishideng Publishing Group Inc., 2020) Van Heerden, Jaques; Kruger, MarianaBACKGROUND: Neuroblastoma (NB) is a heterogeneous disease with variable outcomes among countries. Little is known about NB in low- and middle-income countries (LMICs). AIM: The aim of this review was to evaluate regional management protocols and challenges in treating NB in paediatric oncology units in LMICs compared to high-income countries (HICs). METHODS: PubMed, Global Health, Embase, SciELO, African Index Medicus and Google Scholar were searched for publications with keywords pertaining to NB, LMICs and outcomes. Only English language manuscripts and abstracts were included. A descriptive review was done, and tables illustrating the findings were constructed. RESULTS: Limited information beyond single-institution experiences regarding NB outcomes in LMICs was available. The disease characteristics varied among countries for the following variables: sex, age at presentation, MYCN amplification, stage and outcome. LMICs were found to be burdened with a higher percentage of stage 4 and high-risk NB compared to HICs. Implementation of evidence-based treatment protocols was still a barrier to care. Many socioeconomic variables also influenced the diagnosis, management and follow-up of patients with NB. CONCLUSION Patients presented at a later age with more advanced disease in LMICs. Management was limited by the lack of resources and genetic studies for improved NB classification. Further research is needed to develop modified diagnostic and treatment protocols for LMICs in the face of limited resources.