Changes in the Quality of Life of Adults with Sickle Cell Disease following Allogeneic Stem Cell Transplantation: A Mixed-Methods, Prospective Cohort Study
Recent advances in conditioning regimens have rendered nonmyeloablative allogeneic hematopoietic stem cell transplantation (HSCT) a viable curative treatment for adults with sickle cell disease (SCD). However, prospective studies comparing pre- and post-transplant patient-reported quality of life (QoL) outcomes are lacking.
To test the hypothesis that patient-reported QoL improves after allogeneic HSCT in adult patients with SCD, we conducted a prospective cohort study employing a mixed-methods approach at the Amsterdam UMC, the Netherlands. This study included patients with SCD aged 18 years or older, and involved both longitudinal quantitative and qualitative data collection. A set of 9 Patient-Reported Outcomes Measurement Information System (PROMIS®) measures was completed before and 6, 12, and 18 months post-HSCT. We compared PROMIS® T-scores between the different time points using the Mann-Whitney U test, and against reference values from the general population using a one-sided t-test (IBM SPSS Statistics 28, two-sided p-value <0.05 considered significant). Semi-structured interviews conducted before and after transplantation were thematically analyzed using MAXQDA software.
Seventeen patients (7 females, 10 males; median age 26 years) with SCD (82% HbSS genotype) underwent matched sibling (9) or haploidentical (8) donor transplantation. All patients engrafted successfully.
At baseline, patients’ T-scores were significantly worse compared to the general population for Physical Function (p=<0.001), Sleep Disturbance (p=0.007), Fatigue (p=<0.001), Anxiety (p=0.011), the Ability to Participate Social Roles and Activities (p=0.009), and Satisfaction with Social Roles and Activities (p=0.028). Thereafter, all mean PROMIS® T-scores showed a clinically meaningful improvement of ≥3 points on the T-score scale during the post-transplantation period. At the last follow-up (+18 months), mean T-scores were comparable to the general population regarding Fatigue (p=0.33), Sleep disturbance (p=0.56), and Anxiety (p=0.54). The T-scores for Pain Interference (p=0.01), Physical Function (p<0.01), Anger (p=0.01), Depression (p=0.03), Ability to participate in social roles and activities (p<0.01), and Satisfaction with social roles and activities (p<0.01) even surpassed those of the general population. Thematic analysis of the before and after interviews revealed that the effects of HSCT on mental health can be complex, with a significant number of participants requiring professional psychological assistance. These effects included difficulties with adjustment, processing the psychological aftermath of SCD, and identity conflicts. While most patients were satisfied with the post-transplant situation, transplant-related toxicities, such as reduced fertility or facial acne due to sirolimus, seemed to negatively impact the mental health of transplanted patients.
In conclusion, we present a unique prospective, mixed-methods QoL evaluation of adult transplant recipients with SCD, showing significant improvement in patient-reported QoL outcomes. The effects of curative therapies on mental health can be complex and warrant professional psychosocial support early in the process.