Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2023 Dec 10;19(Suppl-1):e174501792301031.
doi: 10.2174/17450179-v17-e211208-2021-HT2-1910-4. eCollection 2023.

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

Affiliations

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

Olga Mulas et al. Clin Pract Epidemiol Ment Health. .

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, I2=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.

Keywords: Allogenic hematopoietic stem cell transplantation; HRQoL; Immunogenetic; Iron chelation therapy; Transplant; β-Thalassemia major.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflict of interest, financial or otherwise.

Figures

Fig. (1)
Fig. (1)
PRISMA 2009 Flow Diagram.
Fig. (2)
Fig. (2)
°patients aged < 18 years, * patients aged ≥12 years.

Similar articles

Cited by

References

    1. Caocci G., La Nasa G., d’Aloja E., Vacca A., Piras E., Pintor M., Demontis R., Pisu S. Ethical issues of unrelated hematopoietic stem cell transplantation in adult thalassemia patients. BMC Med. Ethics. 2011;12(1):4. doi: 10.1186/1472-6939-12-4. - DOI - PMC - PubMed
    1. Nisbet-Brown E., Olivieri N.F., Giardina P.J., Grady R.W., Neufeld E.J., Séchaud R., Krebs-Brown A.J., Anderson J.R., Alberti D., Sizer K.C., Nathan D.G. Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial. Lancet. 2003;361(9369):1597–1602. doi: 10.1016/S0140-6736(03)13309-0. - DOI - PubMed
    1. Ladis V., Chouliaras G., Berdoukas V., Chatziliami A., Fragodimitri C., Karabatsos F., Youssef J., Kattamis A., Karagiorga-Lagana M. Survival in a large cohort of Greek patients with transfusion-dependent beta thalassaemia and mortality ratios compared to the general population. Eur. J. Haematol. 2011;86(4):332–338. doi: 10.1111/j.1600-0609.2011.01582.x. - DOI - PubMed
    1. Porter J. Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy. Hematol Am Soc Hematol Educ Program. 2018;30(1):361–70. doi: 10.1182/asheducation-2018.1.361. - DOI - PMC - PubMed
    1. Shenoy S., Angelucci E., Arnold S.D., Baker K.S., Bhatia M., Bresters D., et al. Current results and future research priorities in late effects after hematopoietic stem cell transplantation for children with sickle cell disease and thalassemia: a consensus statement from the second pediatric blood and marrow transplant consortium international conference on late effects after pediatric hematopoietic stem cell transplantation. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2017;23(4):552–61. - PubMed

LinkOut - more resources