Systematic Review and Meta-Analysis of Health-Related Quality of Life in Patients with β-Thalassemia Underwent Hematopoietic Stem Cell Transplantation

Abstract: Background: β-Thalassemia major (β-TM) represents one of the most important hemoglobinopathies worldwide. Remarkable improvements have been achieved in supportive therapy based on blood transfusions and iron chelation, and nowadays, this approach is capable of assuring a long life in these patients in industrialized countries. The only curative treatment is represented by hematopoietic stem cell transplantation (HSCT). However, this treatment may be burdened by deterioration in the Health-Related Quality of Life (HRQoL). This paper aimed to evaluate the role of HRQoL in transplanted β-TM patients with a systematic review and meta-analysis. Methods: PubMed database, Web of Science, and Scopus were systematically searched for studies published between January 1st, 2000 to September 2020. The following terms were entered in the database queries: β-thalassemia, HRQoL, and HSCT. The study was carried out according to the Preferred Reporting Items for Systematic and Meta-analyses (PRISMA) statement. Results: We identified a total of 33 potential studies. Among these, 10 were finally considered in the systematic review and 5 in the meta-analysis. Overall, good scores in the principal domains of HRQoL were reported by transplanted patients. These data were confirmed by results of meta-analysis that showed significant difference between transplanted and β-TM patients treated with conventional therapy in the physical and emotional dimension, with a medium effect size [d=0.65, 95% CI (0.29-1.02), z = 3.52, p =0.0004, I2 =75%; and d=0.59, 95% CI (0.43-0.76), z = 6.99, p <0.00001,I 2 =0%, respectively]. Conclusion: HRQoL is generally good in β-TM transplanted patients and may significantly contribute in deciding whether or not to transplant a β-TM patient treated with conventional therapy.