What Does It Mean to Be Cured?: Perspectives from Individuals Living with Sickle Cell Disease.
TL;DR
A critical divergence between biomedical framings and patient perceptions of cure is revealed, and aligning technological descriptions with lived experience will be essential to prevent misplaced expectations, strengthen trust, and support more socially integrated approaches to clinical translation.
Abstract
Background
Hematopoietic stem cell transplantation (HSCT) and gene therapies for sickle cell disease (SCD) are frequently described as cures in scientific literature, clinical spaces, and media reports. Yet, little empirical research has examined how individuals living with SCD perceive the meaning of "cure".
Methods
Over a 24-month period, ethnographic observations were conducted at a single large clinical research site alongside semi-structured interviews with 25 participants (23 individuals living with SCD and 2 caregivers of children with SCD). Participants represented diverse treatment trajectories, including those who did and did not undergo HSCT or gene therapy. Adult participants ranged in age from 18 to 41, and the children of the caregiver participants were under 12 years of age. Interviews were analyzed using reflexive and abductive thematic and narrative analytic approaches.
Results
Four themes characterized participants' perceptions of cure: (1) cure as symptom management and eradication; (2) cure as freedom from social and structural burdens; (3) cure as identity disruption and continuity; and (4) cure as tradeoff. These themes were further organized by three cross-cutting analytic dimensions - temporality, contestation, and embodiment - that shaped how participants articulated cure across clinical and social contexts.
Conclusion
These findings reveal a critical divergence between biomedical framings and patient perceptions of cure. As cell and gene therapies expand into clinical practice and public discourse, aligning technological descriptions with lived experience will be essential to prevent misplaced expectations, strengthen trust, and support more socially integrated approaches to clinical translation.