Title:Long-term Effect of Hematopoietic Stem Cell Transplantation on the
Quality of Life of Patients with β-thalassemia Major in Guangxi, China - A
Cross-sectional Study
Volume: 18
Issue: 3
Author(s): Lu Zhai, Yuhua Liu, Rongrui Huo, Zhaofang Pan, Yaqun Zhang, Zhi Li, Fang Li, Jing Fan*Wei Wei*
Affiliation:
- Department of Stem Cell Transplantation, the First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China
- Department of Stem Cell Transplantation, the First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China
- Department of Geriatrics Gastroenterology Division, the First Affiliated Hospital of Guangxi Medical
University, Nanning, 530021, China
Keywords:
Haploidentical hematopoietic stem cell transplantation, unrelated hematopoietic stem cell transplantation, blood infusion, iron chelation, β-thalassemia major, quality of life.
Abstract:
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 β -thalassemia major (β-TM)
with that of β-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 β-TM patients treated with
blood transfusion and iron chelation therapy, 133 β-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
β-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 β-TM patients receiving conventional treatment
with β-TM revealed poorer outcomes than the cohort of transplanted patients.
Conclusion: β-TM patients can be cured by HSCT and regain QOL as good as that of the general population.
β-TM patients are suggested to undergo HSCT as soon as possible to avoid complications related to
iron overload and blood infusion.
