This study is the first nationwide survey of the caregiver burden of adult patients with β-thalassemia major in mainland China, with a focus on high-incidence areas. In this study, the caregiver burden of adult patients with β-thalassemia major was predominantly moderate and severe(38.00 ± 17.02), similar to the findings of studies on caregiver burden in children with β-thalassemia in India and Pakistan[19, 26], and higher than those in some studies on adult patients with cancer (34.2 ± 16.4)[27], but lower than some studies on adult patients undergoing hematopoietic stem cell transplantation (45.6 ± 13.3)[28].
The mean age of patients with β-thalassemia major in this study was only 24.7 ± 5.6 years, probably because they usually had poor QoL, short life expectancy, and high mortality; thus, there were few elderly patients[29]. This finding was also consistent with the literature reporting that the highest life expectancy of patients with β-thalassemia major in Guangxi, China, was only 28 years[10]. However, most patients with β-thalassemia major in the United Kingdom live at least until their mid-forties. This may be because about half of the patients in the UK with β-thalassemia major are treated with iron chelation therapy through daily intramuscular injection of desferrioxamine, administered nightly as an 8–12 h subcutaneous infusion, for at least 5 nights per week. This became standard management in 1982 and has been shown to improve survival[9].
Among the patients in this study, most were unemployed, possibly because blood transfusion therapy, iron chelation therapy, and other related treatments require 3–5 days per month for patients with thalassemia; more importantly, adult patients with β-thalassemia major often experience pain, fatigue, and have poor QoL. Moreover, some adult patients are turned away from work units[20].
Regarding comorbidities and treatment, most adult patients with β-thalassemia major had comorbidities, and approximately three-fifths had not experienced interruptions in blood transfusion or iron chelation therapy. Some experts suggested that adult patients with β-thalassemia major can survive for a long time with the combination of regular blood transfusion and iron chelation therapies; however, frequent blood transfusions lead to iron overload, which is also one of the main reasons for patient complications[30]. In addition, regarding the social support level for patients with β-thalassemia, we observed medium-level scores on the SSRS and its subdimensions. Compared to patients with other diseases, complications associated with blood transfusion and iron chelation therapies can lead to physical problems such as facial bone deformities, growth retardation, and psychological problems[31, 32], resulting in a lack of social acceptance. Therefore, patients may be unwilling to tell others about their experiences to receive help. Other studies have also shown that adult patients with β-thalassemia have less social support, and that family is the most important source, while friends are the least important source of social support[33].
Among the caregivers, more than 70% were married women, aged around 50 years old, and more than three-fifths cared for patients without help from others, and also needed to take care of others besides adult patients with β-thalassemia major. Studies conducted in Rawalpindi and Greece reported similar results [26, 34]. Males are expected to be breadwinners in familial and societal contexts, and because of their unique physical and psychological characteristics, females are regarded as better caregivers than males. Females can not only maintain a good rehabilitation environment but also coordinate conflicts [35, 36]. Therefore, females are left to care for sick children at home, and they also follow the traditional labor division in households.
We found that patients who had interrupted their blood transfusion therapy were positively associated with caregiver burden; that is to say, patients who interrupted their blood transfusion therapy had a larger burden on caregivers. This was consistent with the results of patient and caregiver burden of transfusion-dependent β-thalassemia[37]. One possible explanation for this phenomenon is that adult patients exceed the best age for hematopoietic stem cell transplantation, which is the only cure for patients with thalassemia; thus, they are subject to lifelong blood transfusion[38]. However, lifelong blood transfusion can lead to iron overload and increase the risk of death. Some experts have pointed out that only long-term blood transfusion therapy combined with iron chelator therapy can allow adult patients with β-thalassemia major to survive for a long time[39]. Although interrupting blood transfusion therapy can save some medical costs in the short-term, once complications arise and the condition worsens, surgical treatment is required, and patients may lose their labor capacity. In addition, the cost of follow-up treatment greatly increases the financial burden on the family, causing the caregiver to experience both physical and mental pressures[40].
We also observed a positive association between caregivers who needed to take care of others and the caregiver burden. This result is consistent with those of another study on the burden among caregivers of patients with acute leukemia and epilepsy[41]. Most caregivers of adult patients with thalassemia are > 50 years of age; thus, they are in the middle-aged or elderly stage of the disease-prone period. Caregivers also need to take care of other people besides the patients, such as their own parents, children, and grandchildren. The long-term doubling of time, money, and energy consumption greatly increases the caregiver burden, which, in turn, affects their physical and mental health and may lead to anxiety, depression, and other emotions[42], thus further increasing the caregiver burden.
In addition, we observed a negative association between married caregiver status and caregiver burden, which is consistent with the results related to caregiver burden in patients undergoing hemodialysis[43]. This may be because the family function of married caregivers is better; caregivers can obtain more resources from family members, such as psychological and financial support, to cope with the patient's disease, and the family can also help the patient build confidence in the recovery, thereby reducing caregiver burden.
In their study on the caregiver burden of patients with thalassemia, Gok et al.[17] reported that caregiver education level did not influence their level of stress, a finding consistent with that in the present study. Moreover, in this study, patient education level and employment status were not significant determinants, which was similar to research on caregiver burden among mothers of children undergoing treatment for acute lymphocytic leukemia (ALL) [41]. However, a study on caregiver burden in patients undergoing hemodialysis showed decreasing caregiver burden with increasing patient education level [44]. Therefore, these determinants require consideration when seeking to alleviate caregiver burden.
This study contributes to the literature in several ways. First, most studies related to thalassemia in mainland China are pathological or clinical treatment-related, and less attention has been paid to the determinants of caregiver burden, which were emphasized in the present study. Second, existing studies on the caregiver burden of patients with thalassemia have mainly focused on pediatric patients, ignoring adult patients who have relatively worse QoL, higher mortality, and more serious complications due to long-term irregular treatment. This study attempted to address this gap. Finally, the findings of this study provide a reference to identify caregivers with a higher burden among those caring for patients with β-thalassemia major.
This study has several limitations. First, due to the difficulty in recruiting samples, the sample size in some provinces was relatively small, which limited the representativeness and generalizability of the conclusions. Second, this study was based on a cross-sectional design; therefore, it could only test the associations among variables simultaneously, rather than the causal relationships. Therefore, future large-scale (e. g., adult patients with β-thalassemia intermediate and their caregivers) and prospective cohort studies should be considered.