Abstract
BackgroundThere is a limited understanding of the patient and family experience of Chronic Transfusion Therapy (CTT) for prevention of complications of Sickle Cell Disease (SCD). We sought to understand patient and family experience with CTT using qualitative methods.MethodsFifteen parents of children < 18 years old and nine children 12–18 years old with SCD who were receiving CTT for > 1 year were interviewed using a semi-structured interview format, and interviews were analyzed using open coding methods.ResultsFour themes created a narrative of the patient and family experience of CTT: 1) Burden of CTT, 2) Coping with CTT, 3) Perceived benefits and risks of CTT, and 4) Decision making regarding CTT. Participants reported substantial burden of CTT, including the impact of CTT on daily life and family, distress about venous access, burden of chelation therapy, and anxiety about CTT complications. Participants described how they coped with CTT. Participants reported increased energy, decreased pain, fewer hospitalizations, and stroke prevention with CTT, but also recognized complications of CTT, though awareness was limited in adolescents. Parents described sharing in the informed decision-making process with their healthcare provider about CTT, but adolescent patient participants reported that they were not involved in this process.ConclusionsCTT is associated with significant patient and family burden. Support from family, healthcare providers and school may help individuals cope with some of this burden. These findings provide the basis for future studies to identify strategies to mitigate the burden of CTT and improve the patient experience with this therapy. Future studies should also systematically assess patient knowledge about the key components of CTT and chelation using quantitative assessments.
Highlights
There is a limited understanding of the patient and family experience of Chronic Transfusion Therapy (CTT) for prevention of complications of Sickle Cell Disease (SCD)
Parents of children with SCD receiving CTT report improved Health-Related Quality of Life (HR-QOL) [11] with better physical functioning, less bodily pain, and improved overall health when compared to parents of children not on CTT [11]
This study is complementary to, and extends the findings from previous survey based studies of HRQOL in children receiving CTT for SCD [11, 17]. The findings of this qualitative study are consistent with a prior qualitative study by Stegenga et al, who interviewed 10 pediatric patients to describe the impact of CTT on school and identified perceived benefits of CTT as stroke prevention and improved energy [18]
Summary
There is a limited understanding of the patient and family experience of Chronic Transfusion Therapy (CTT) for prevention of complications of Sickle Cell Disease (SCD). We sought to understand patient and family experience with CTT using qualitative methods. Chronic transfusion therapy (CTT) reduces or prevents sickle cell disease (SCD)-related complications. CTT is associated with complications such as transfusional iron overload [7], red cell alloimmunization [8], transfusion reactions, and the potential for infectious disease transmission [9, 10]. While survey-based methods measure HR-QOL and allow for quantitative comparisons, they provide a limited understanding of the entirety of the patient and family experience of CTT in SCD. Qualitative research methods [14] allow for a rich, in-depth description of the patient experience, and can fill the knowledge gap in understanding patient and family experience
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