Hoping for a normal life: Decision-making on hematopoietic stem cell transplantation by patients with a hemoglobinopathy and their caregivers

Background: To provide insight into the perspectives of children and young adults with transfusion-dependent thalassemia and sickle cell disease and their caregivers regarding the decision for hematopoietic stem cell transplantation (HSCT). Procedure: A qualitative longitudinal multicenter study. Data collection consisted of 40 audio-recorded conversations between physicians and families and 77 interviews with patients and/or caregivers related to 27 unique cases, collected at different time points throughout the decision-making process. Results: Conversations and interviews revealed “hoping for a normal life” as an overarching theme, consisting of four main topics: (i) “Building a frame of reference” refers to a process where patients or families try to obtain comprehensive information on HSCT and translate this to their situation to decide. (ii) “Balancing between loss and benefit” reports the process of considering the advantages and disadvantages of continuing with supportive care to treat their disease versus choosing HSCT. (iii) “Experiencing the impact of HSCT” describes the impactfull experience of the HSCT period by those who chose HSCT. (iv) “Balancing again” refers to reflecting on the decision made. Conclusions: The hope for a normal life guided the decision-making process, described as a constant balance between the impact of the disease and HSCT. A structured approach to explore patients’ and caregivers’ perspectives on HSCT decision-making is needed, where specifically discussing the impact of the disease and hope for a normal life need to be integrated in the process.