Patient and family experience with chronic transfusion therapy for sickle cell disease: A qualitative study

Abstract

Background: 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. Methods: Fifteen 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. Results: Four 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. Conclusions: CTT 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.

Barriers and Facilitators to Chronic Red Cell Transfusion Therapy in Pediatric Sickle Cell Anemia

“…Caregivers described fear of initiating CRCT due to concerns for infection and fear of medical events and reactions during transfusions while providers specifically described concerns for alloimmunization and iron overload. Previous studies with caregivers of children undergoing CRCT similarly found that logistical challenges, venous access, and fear and worry about CRCT were predominant negative themes, in addition to chelation therapy (Boateng et al, 2013; Hawkins et al, 2020). Chelation therapy was not noted as a barrier in the current study by caregiver participants (even among those with extensive CRCT experience).…”

“…Previous studies have noted the importance of improving health literacy by providing accurate, disease-specific education about CRCT. In particular, information about likely complications and the role of families in the potential management of complications, such as alloimmunization or iron overload, is particularly important (Hawkins et al, 2020; Yee et al, 2019).…”

Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

“…Raising a child with SCD is associated with a myriad of psychological stressors for primary caregivers 27 . Families experience stress from the burden of SCD treatments, such as CTT, regular clinic visits, and the financial hardship from missed workdays 27,28 . Taking care of their child through the pain crises and other severe SCD‐related complications may cause the greatest distress for primary caregivers 27 .…”

“… 27 Families experience stress from the burden of SCD treatments, such as CTT, regular clinic visits, and the financial hardship from missed workdays. 27 , 28 Taking care of their child through the pain crises and other severe SCD-related complications may cause the greatest distress for primary caregivers. 27 Primary caregivers cope in a variety of ways, such as joining support groups as well as engaging in health information–seeking behavior (HISB).…”

Assessing Patient Preferences for Treatment Options for Pediatric Sickle Cell Disease: A Critical Review of Quantitative and Qualitative Studies

“… 37 Creary et al conducted a semi-structured interview of caregivers’ of the patients who were offered HU treatment, including those who agreed and those who refused HU, to understand their preferences regarding HU. 38 Caregivers identified the drug’s side effect profile, especially when HU is perceived as a chemotherapy drug, the uncertainty in disease progression, and inability to perceive the severity of SCD in their child as factors swaying them away from the medicine. They also found that even caregivers who chose HU for their children reported being fearful about initiating it.…”