Experiences and Decision Making in Hematopoietic Stem Cell Transplant in Sickle Cell Disease: Patients' and Caregivers' Perspectives

Abstract

Sickle cell disease (SCD) is one of the most commonly inherited hemoglobin disorders that has a significant impact on quality of life, increased childhood morbidity, and premature mortality. Currently, hematopoietic stem cell transplant (HSCT) is the only treatment with a curative intent. The objective of this study was to determine patients' and caregivers' knowledge of HSCT, the factors influencing the decision to pursue HSCT, their experiences, and the impact of a successful HSCT on their daily living. At Children's Healthcare of Atlanta, we conducted a qualitative study using a semistructured interview guide of patient-caregiver dyads and 2 focus-group sessions of adult long-term survivors of HSCT to elicit key factors in decision making, their experiences with HSCT, and the impact of HSCT. Interviews and focus-group sessions were recorded and transcribed verbatim. Transcripts were coded and analyzed for emerging themes using NVivo 10.0. We enrolled 11 patient-caregiver dyads (n = 6, female patients; n = 10, mothers) in the qualitative interviews and 2 focus groups with 5 (n = 2, females) and 7 (n = 3, females) participants in each group, respectively. Our analysis revealed 3 prominent themes: (1) factors and concerns influencing HSCT decision making; (2) HSCT experiences; and (3) impact of HSCT on daily life. Participants reported that progression of disease-related complications and availability of a matched donor strongly influenced the decision to pursue HSCT. Although patients and caregivers had to deal with the arduous process of HSCT and transplant-related morbidities, participants were satisfied with their decision and expressed no decisional regrets. Decision making for HSCT for patients with SCD is a complex process. Understanding the key influential factors in decision making and the impact HSCT has on these patients and their families will generate crucial insights that can guide the care of future patients and research studies.

The experience of adults with sickle cell disease and their HLA‐matched adult sibling donors after allogeneic hematopoietic stem cell transplantation

“…Unlike cancer, those with SCD did not know a pain‐free life before HSCT. Our results parallel those of Khemani et al () revealing that most recipients were pleased with the HSCT outcome with fewer SCD symptoms and the potential for a longer life.…”

“…Our results show that recipients and donors were satisfied with their decisions and provide insights into the underlying decision‐making process and experiences of HSCT (Khemani et al, ). The signing of the consent form represented the endpoint of a complex decision‐making process that included input from a network of people.…”

“…Our results show that recipients and donors were satisfied with their decisions and provide insights into the underlying decisionmaking process and experiences of HSCT (Khemani et al, 2017).…”

“…The results of this qualitative study conducted out of one Midwest transplant centre provide rich description and insights into the complex nature of the HSCT experience for both recipients and donors. Like the Khemani et al () study finding, the power of the decline in the recipient's health accompanied by increasing pain crises and hospitalizations over time strongly motivated both recipients and donors. Each expected that HSCT would "cure" the SCD.…”

“…Because HSCT is a newer therapy used in adults with SCD, few studies report on recipient and/or sibling donor experiences, decision‐making processes, or the impact of HSCT on their lives. A recent qualitative study of patients and their primary caregivers and long‐term survivors at a children's hospital found that having a matched‐donor, perceived SCD burden and disease progression and family support significantly influenced the decision to undergo a HSCT (Khemani et al., ). Although participants indicated that HSCT was a difficult process, they expressed satisfaction with their decision and no regrets to pursue HSCT.…”