Abstract
Background: Individuals living with transfusion-dependent β-thalassemia (TDT) experience reduced health-related quality of life (HRQoL) due to fatigue and chronic pain, which cause disruptions to daily life. Currently, limited qualitative data exist that describe these impacts.
Objective: This study aimed to examine the ways in which symptoms and current treatments of TDT impact HRQoL, to holistically describe the humanistic burden of TDT, and to identify the unmet needs of individuals living with TDT.
Methods: Adults (aged ≥18 years) with TDT and caregivers of adolescents (aged 12‒17 years) with TDT participated in semi-structured one-on-one virtual interviews and focus group discussions. Interviews were conducted in the United States and United Kingdom and lasted approximately 60 minutes. After transcription, the interviews were analyzed thematically using a framework approach.
Results: A total of 10 interviews/focus group discussions (six interviews and four focus group discussions) were conducted with 14 adults with TDT and two caregivers of adolescents with TDT. Framework analysis revealed five themes describing HRQoL (negative impacts on daily activities, social life, family life, work and education, and psychological wellbeing) and three themes describing the lived experience of TDT (impact of red blood cell transfusions and iron chelation therapy, treatment, and stigma). Physical, psychological, and treatment-related factors contributed to negative impacts on daily activities, social and family life, and work and education. Concerns about reduced lifespan, relationships and family planning, and financial independence were detrimental to participants’ mental wellbeing. Participants reported having high resilience to the many physical and psychological challenges of living with TDT. A lack of TDT-specific knowledge among healthcare professionals, particularly regarding chronic pain associated with the disease, left some participants feeling ignored or undermined. Additionally, many participants experienced stigma and were reluctant to disclose their disease to others.
Conclusions: Individuals living with TDT experience substantial negative impacts on HRQoL that disrupt their daily lives, disruptions which are intensified by inadequate healthcare interactions, demanding treatment schedules, and stigma. Our study highlights the unmet needs of individuals living with TDT, especially for alternative treatments that reduce or eliminate the need for red blood cell transfusions and iron chelation therapy.
Objective: This study aimed to examine the ways in which symptoms and current treatments of TDT impact HRQoL, to holistically describe the humanistic burden of TDT, and to identify the unmet needs of individuals living with TDT.
Methods: Adults (aged ≥18 years) with TDT and caregivers of adolescents (aged 12‒17 years) with TDT participated in semi-structured one-on-one virtual interviews and focus group discussions. Interviews were conducted in the United States and United Kingdom and lasted approximately 60 minutes. After transcription, the interviews were analyzed thematically using a framework approach.
Results: A total of 10 interviews/focus group discussions (six interviews and four focus group discussions) were conducted with 14 adults with TDT and two caregivers of adolescents with TDT. Framework analysis revealed five themes describing HRQoL (negative impacts on daily activities, social life, family life, work and education, and psychological wellbeing) and three themes describing the lived experience of TDT (impact of red blood cell transfusions and iron chelation therapy, treatment, and stigma). Physical, psychological, and treatment-related factors contributed to negative impacts on daily activities, social and family life, and work and education. Concerns about reduced lifespan, relationships and family planning, and financial independence were detrimental to participants’ mental wellbeing. Participants reported having high resilience to the many physical and psychological challenges of living with TDT. A lack of TDT-specific knowledge among healthcare professionals, particularly regarding chronic pain associated with the disease, left some participants feeling ignored or undermined. Additionally, many participants experienced stigma and were reluctant to disclose their disease to others.
Conclusions: Individuals living with TDT experience substantial negative impacts on HRQoL that disrupt their daily lives, disruptions which are intensified by inadequate healthcare interactions, demanding treatment schedules, and stigma. Our study highlights the unmet needs of individuals living with TDT, especially for alternative treatments that reduce or eliminate the need for red blood cell transfusions and iron chelation therapy.
| Original language | English |
|---|---|
| Journal | The Patient - Patient-Centred Outcomes Research |
| Early online date | 26 Mar 2024 |
| DOIs | |
| Publication status | E-pub ahead of print - 26 Mar 2024 |
Keywords
- transfusion-dependent β-thalassemia
- hemoglobinopathy
- health-related quality of life
- fatigue
- chronic pain
- qualitative analysis
