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Combining deliberative engagement with qualitative research to assess patient and caregiver perspectives on curative therapies for sickle cell disease in Africa

Bukini, D.; Mashaka, J.; Rifai, A.; Kanza, C.; Kassim, K.; Chanzi, R.; Maingu, D.; Luoga, F.; Lema, W.; Konteh, F.; Alimohamed, M. Z.; Amin, E.; Spector, J.; Makani, J.

2025-07-30 public and global health
10.1101/2025.07.30.25332426 medRxiv
Show abstract

The vast majority of individuals impacted by sickle cell disease (SCD) live in Africa, where access to potentially curative therapies such as hematopoietic stem cell transplant and gene therapies have been severely limited until now. As part of a broad effort to help prepare scientific, healthcare, and patient communities in Tanzania for the introduction of advanced SCD therapies, we sought to understand perspectives relating to curative options for SCD held by patients and their families. To achieve this, innovative qualitative research methodology was required given the highly variable level of baseline knowledge among patient communities regarding curative treatments for SCD. We therefore adopted an approach of deliberative engagement followed by traditional qualitative methods that facilitated successful identification of participants, development of research tools, and, ultimately, elicitation of valuable insights that enabled in-depth analyses. Our experience demonstrates that, when exploring perspectives relating to complex medical procedures in this population, deliberative engagement substantially enhances the outputs of qualitative research.

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