Abstract
Bone marrow transplant (BMT) is a potentially curative treatment for sickle cell disease (SCD). Patient and caregiver attitudes toward BMT for SCD and the willingness to accept risks of BMT vary, but these attitudes are not well understood. To understand patient and caregiver perceptions of and attitudes toward BMT for SCD and decision-making about BMT. Qualitative study of interview transcripts from a convenience sample. Transcripts were from adults with SCD and caregivers of patients with SCD recruited from national and regional SCD conferences, symposia, and sickle cell clinics in 2 cities. Interview transcripts were used from the needs assessment phase to develop a patient-decision aid in 2013 to 2014 (group 1) and from the baseline point in 2015 to 2016 (group 2) of the parent trial, a randomized clinical trial of adults and caregivers of patients with SCD to evaluate the effectiveness of a patient decision aid. Participant perspectives on decision-making regarding BMT for SCD. Fifty-seven transcripts from adults with SCD and 50 transcripts from caregivers of patients with SCD were included. Median (interquartile range [IQR]) age of adults with SCD was 34 (21-50) years in group 1 and 30 (23-38) years in group 2. The median (IQR) age of caregivers was 42.5 (31-52) years in group 1 and 41 (35-46.5) years in group 2. Most transcripts from adults with SCD (75.0% in group 1 and 72.4% in group 2) and caregivers of patients with SCD (76.7% in group 1 and 85.0% in group 2) were from female participants. Bone marrow transplant was perceived as a treatment option associated with serious risks. Reported attitudes toward BMT occurred on a continuum ranging from unfavorable to favorable. Participants reported serious decisional dilemma regarding BMT for SCD. Most participants expressed interest in learning about BMT or curative treatments. This qualitative study found a continuum in attitudes toward BMT for SCD and highlights the complexity of decision-making in BMT for SCD. Patients and families with SCD expressed an interest in learning about BMT. Future prospective studies of patient decision-making regarding BMT, especially in the context of emerging curative and novel disease-modifying therapies for SCD, are warranted.
Highlights
Most transcripts from adults with sickle cell disease (SCD) (75.0% in group 1 and 72.4% in group 2) and caregivers of patients with SCD (76.7% in group 1 and 85.0% in group 2) were from female participants
Most participants expressed interest in learning about Bone marrow transplant (BMT) or curative treatments. This qualitative study found a continuum in attitudes toward BMT for SCD and highlights the complexity of decision-making in BMT for SCD
Patients and families with SCD expressed an interest in learning about BMT
Summary
Sickle cell disease (SCD), a chronic multisystem disorder, affects approximately 100 000 people in the US1 and is associated with morbidity, poor health-related quality of life, and premature mortality. Bone marrow transplant (BMT) is a potential curative therapy. Factors that may be associated with uptake of BMT include availability of a donor, perceived risk of morbidity and mortality from BMT, knowledge of SCD9 including long-term complications, access to BMT clinical trial information, mistrust of medical professionals, awareness of BMT as a curative option, costs associated with BMT, variability in physician philosophy regarding BMT, and patient and family attitudes toward BMT. While adult patients with SCD, adolescents, and parents of children with SCD17 are willing to accept some risk of BMT-associated morbidity and mortality for the possibility of a cure in a hypothetical scenario, some families with children with sickle cell anemia warranting chronic transfusions decline BMT, even when they have a matched donor.13We sought to understand the perspectives of patients and caregivers toward BMT through qualitative interviews, which have the potential to provide in-depth insights. This study is a theoryinformed secondary analysis of a subset of interview transcripts from a parent study, a study to develop a web-based patient decision aid for therapeutic options in SCD and evaluate its effectiveness in a randomized clinical trial.. Factors that may be associated with uptake of BMT include availability of a donor, perceived risk of morbidity and mortality from BMT, knowledge of SCD9 including long-term complications, access to BMT clinical trial information, mistrust of medical professionals, awareness of BMT as a curative option, costs associated with BMT, variability in physician philosophy regarding BMT, and patient and family attitudes toward BMT.. While adult patients with SCD, adolescents, and parents of children with SCD17 are willing to accept some risk of BMT-associated morbidity and mortality for the possibility of a cure in a hypothetical scenario, some families with children with sickle cell anemia warranting chronic transfusions decline BMT, even when they have a matched donor..
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
