Experiences and Decision Making for Bone Marrow Transplant in Sickle Cell Disease: Patients and Caregivers Perspective

Background:Bone marrow transplantation (BMT) is currently the only curative treatment option available for patients with sickle cell disease (SCD). There are very limited data available on the patients and caregivers perspective on decision making for BMT and their overall experiences with the BMT process. The objective of this study was to determine patients and caregivers knowledge of BMT, the factors that influenced families to consider BMT as a treatment option and their experiences during and after transplantation.

Methods:At Children's Healthcare of Atlanta, we conducted 22 qualitative interviews involving patient-caregiver dyads using a semi-structured interview guide. The questions focused on patient and caregiver decision-making and their experience with bone marrow transplant for SCD. All interviews were recorded and transcribed verbatim. Transcripts were then double-coded. Content analysis was performed using NVivo 10.

Results: Eleven patients (n=6; Females) and 11 caregivers (n=10, Mothers) participated. At the time of interview, patient's age range was 11-32 years with a median value of 20 years; at the time of transplant, patient's age range was 6 to 17 years with a median value of 11 years. Time from transplant ranged from 0-17 years with a median value of 2 years. Analysis revealed that most patients' and caregivers' were presented with BMT as a treatment option based on the severity of patient's clinical situations. Six participants, patients and caregivers combined, described minimal knowledge and understanding of the BMT process and/or the associated risks/benefits involved with transplant before consultation with the BMT team. Both patients and caregivers reported that the decision to move forward with BMT was strongly influenced by the progression of sickle cell related complications. All patients were either suffering from chronic pain or had been on chronic transfusions for several years. Given the effects of BMT on the entire family, 7 patients and 5 caregivers reported the need to involve the entire family in the decision-making process. Other factors that played a significant role in the decision-making process for patients and caregivers included: information and education received from the BMT team, having an HLA-matched sibling or unrelated donor, and spirituality. A few patients and caregivers stated that hearing from other families would have provided them a more realistic understanding of the transplant process and further aided in their decision-making. Of the 7 patients and caregivers asked about fertility issues and its impact on transplant decision making, 3 patients, and 3 caregivers conveyed that it was the "least of their concerns" and did not feel the need to proceed with fertility preservation. One caregiver voiced concerns about fertility issues, but after consultation with a fertility specialist decided to proceed with BMT. About 95% of patients and caregivers described transplant day as a "re-birth day" or "new birthday". Most patients conveyed that having to deal with chemotherapy-related side effects, such as hair loss, the need for nasogastric tubes, and being isolated from family and friends was all "worth it" because they no longer have sickle cell disease. For 8 caregivers, prayers and family support played a prominent role in helping to cope with the fears and stress involved with BMT. Eight patients indicated that prayers and distractions such as watching movies, playing video games and doing school work helped with coping. Despite having to deal with the arduous transplant process and transplant-related complications, patients and caregivers alike were satisfied with their decision and expressed no decisional regret.While this study included some patients who had severe complications including chronic GVHD, we did not interview caregivers of any patient who had died following BMT.

Conclusion: Decision making for bone marrow transplant as a treatment option for those with sickle cell disease is a complex process. Most patients and caregivers have limited knowledge about bone marrow transplant being a curative treatment for sickle cell disease, and the need to educate patients and families is crucial. Gaining an understanding of factors that influence decision making for this population and their experiences with the transplant process will generate crucial insights that will impact the care of future patients and research studies.