| 总结: | The aim of this thesis was to generate evidence to improve childhood cancer health outcomes and resource use in Egypt. Childhood cancer is an urgent priority in Egypt, owing to the large numbers of children with cancer, limited resources, and inferior survival. Therefore, there is a need to improve childhood cancer health outcomes (survival) with optimal resource use. The thesis generates three types of evidence using hybrid research methods: (1) Real-world evidence through conducting observational cohort studies in a local setting in Egypt (Chapters 3–5); (2) systematic evidence from the literature through conducting a systematic review study (Chapter 6); and (3) qualitative evidence from experts’ opinions in the local context through conducting a qualitative interview study (Chapter 7). In Chapter 3, I determined the survival rates and temporal trends in survival for 14,808 children with cancer treated at Children’s Cancer Hospital Egypt (CCHE) between 2007 and 2017, and identified cancers with inferior survival compared to international benchmarks. In Chapter 4, I determined childhood cancer hospital resource use and associated costs for 8,886 children with cancer treated between 2013 and 2017, and identified cancers with the highest resource use/costs. In Chapter 5, I estimated treatment cost-effectiveness at CCHE and found that it was not cost-effective for children with relapsed/refractory acute leukaemia. Accordingly, In Chapter 6, I conducted a systematic review on costs/cost-effectiveness of treatment for relapsed/refractory paediatric acute leukaemia. In Chapter 7, I conducted interviews to determine the clinicians’ perceptions of barriers and facilitators to implementing cost-effective evidence-based treatment at CCHE. Overall, I have reported that the 5-year overall survival rate for children with cancer at CCHE was 72.1%, where 12 (out of 18) cancer types had inferior survival compared to international benchmarks, and that children with acute myeloid leukaemia had the highest costs/resource use. Also, I found that treatment of relapsed/refractory acute leukemia was not cost-effective at CCHE, and the systematic review findings have shown that CAR–T cell therapy was a cost-effective treatment option, besides other novel therapies. I have identified four themes of barriers and facilitators to implementing cost-effective evidence-based treatment at CCHE. In conclusion, the thesis provides evidence-based recommendations for improving childhood cancer survival, resource use/costs, and treatment cost-effectiveness, addressing the implementation gaps at CCHE/Egypt. In Chapter 8, I discuss the implications of my thesis, and I propose a context-specific conceptual framework of evidence synthesis to improve childhood cancer health outcomes and resource use in Egypt.
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