Long-term Effect of Hematopoietic Stem Cell Transplantation on the Quality of Life of Patients with ß-thalassemia Major in Guangxi, China - A Cross-sectional Study.

OBJECTIVE: The purpose of our study was to compare the quality of life (QOL) of patients with hematopoietic stem cell transplantation (HSCT) for more than 2 years for ß -thalassemia major (ß-TM) with that of ß-TM patients with conventional therapy (blood infusion and iron chelation) and that of the general population. METHODS: This was a cross-sectional comparative study on the QOL of 225 ß-TM patients treated with blood transfusion and iron chelation therapy, 133 ß-TM patients who had undergone HSCT or 270 ageand sex-matched healthy individuals from Guangxi, China. Child-self and parent-proxy reports of the PedsQL 4.0 Generic Core Scales were used to prospectively evaluate QOL. RESULTS: The incidence of acute GVHD was 14.3% (grade III-IV in 4.5% of patients), and that of chronic GVHD was 3.8%. This was lower than that of previous studies since the inclusion of anti-thymocyte globulin (ATG). Patients who underwent transplantation from a voluntary donor had higher QOL scores and lower rates of acute GVHD, chronic GVHD and comorbidities than those receiving stem cell sources from an HLA mismatched related donor (haploidentical donor). Transplants with PBSCs or UCBT, PBSCT+BMT, BMT, or BMT+UCBT as stem cell sources did not have any impact on QOL. The QOL of ß-TM patients was very similar to that of the general population. More complications (P<0.001), shorter post-transplantation time (P<0.001), and older age at HSCT (P=0.01) were associated with poorer child QOL (P=0.020). Additional analyses investigating QOL of ß-TM patients receiving conventional treatment with ß-TM revealed poorer outcomes than the cohort of transplanted patients. CONCLUSION: ß-TM patients can be cured by HSCT and regain QOL as good as that of the general population. ß-TM patients are suggested to undergo HSCT as soon as possible to avoid complications related to iron overload and blood infusion.

Quality of Life in Patients with ß-thalassemia Major: Short-term and Long-term Effects After Haematopoietic Stem Cell Transplantation.

BACKGROUND: Allogeneic haematopoietic stem cell transplantation (ALLO-HSCT) is a potentially curative approach to treat ß-thalassemia major (ß-TM). OBJECTIVE AND METHODS: To assess the quality of life (QOL) of patients with ß-TM after ALLO-HSCT, we searched PubMed, Embase, Web of Science, and MEDLINE for articles on the quality of life (QOL) of patients with ß-TM from 1 Feb 2020 to 31 Mar 2020. RESULTS: Our review revealed that the QOL of patients with ß-TM after ALLO-HSCT from a sibling donor is higher than that of patients that received blood infusion and iron-chelating therapy. Survivors of ALLO-HSCT have a QOL as good as that of a healthy population and the ability to return to normal life. However, studies thus far are limited to investigations with a few patients with ß-TM who received ALLO-HSCT of the bone marrow (BM) from a sibling donor or related donor. Graft vs. host disease, patient age, gender, sexual desire, health condition, psychological state, financial and employment stress, and social support contributed to a worse QOL after ALLO-HSCT. Medicine usage, physical therapy, and psychological intervention may help improve the decline in QOL related to ALLO-HSCT in patients with ß-TM. CONCLUSION: Doctors and nurses must focus on implementing medicine usage, physical therapy, and psychological intervention to improve the decline in QOL related to ALLO-HSCT.