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Long-term health-related quality of life in patients with β-thalassemia after unrelated hematopoietic stem cell transplantation

Bone Marrow Transplantation volume 57, pages 1833–1836 (2022)Cite this article

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In western nations, regular iron chelation therapy and optimization of erythrocyte transfusion support have improved the life expectancy of patients with β-Thalassemia Major (β-TM) born in the last few decades, approaching that of healthy peers [1]. Nevertheless, the curative role of hematopoietic stem cell transplantation (HSCT) in β-TM still remains very relevant [2]. An international consensus recommends that HSCT be offered as soon as possible before developing iron overload and iron-related tissue damage in patients with an available HLA identical sibling donor [3]. However, less than 30% of patients can find a sibling donor within the family, depending on nationality and ethnic group. Lacking a sibling donor, a matched unrelated HSCT represents a valid alternative [4, 5]. However, this procedure is an option to be performed in centers with appropriate expertise [3, 4]. Among different parameters used to evaluate the success of the transplant, health-related quality of life (HRQoL) plays a key role. Very little data is available on HRQoL of sibling HSCT and, to our best knowledge, only one study reported HRQoL outcomes in β-TM patients transplanted from unrelated donors [6]. Moreover, no direct comparisons on HRQoL are available between patients who underwent siblings or matched unrelated HSCT.

The main objective of our study was to analyze long-term HRQoL in matched unrelated HSCT in β-TM carried out in centers with high expertise in the procedure. The secondary objective was to compare HRQoL between unrelated and sibling HSCT.

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Fig. 1: SF-36 questionnaire scores in transplanted patients from sibling or unrelated donors.

Data availability

The data that support the findings of this study are available from the corresponding author, GC, upon reasonable request

References

  1. Taher AT, Musallam KM, Cappellini MD. β-Thalassemias. N Engl J Med. 2021;384:727–43.

  2. Mulas O, Mola B, Caocci G, La, Nasa G. Conditioning regimens in patients with β-thalassemia who underwent hematopoietic stem cell transplantation: a scoping review. J Clin Med. 2022;11:907.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  3. Angelucci E, Matthes-Martin S, Baronciani D, Bernaudin F, Bonanomi S, Cappellini MD, et al. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel. Haematologica. 2014;99:811–20.

    Article  PubMed  PubMed Central  Google Scholar 

  4. Shenoy S, Angelucci E, Arnold SD, Baker KS, 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. 2017;23:552–61.

    Article  PubMed  Google Scholar 

  5. Caocci G, Orofino MG, Vacca A, Piroddi A, Piras E, Addari MC, et al. Long-term survival of beta thalassemia major patients treated with hematopoietic stem cell transplantation compared with survival with conventional treatment. Am J Hematol. 2017;92:1303–10.

    Article  CAS  PubMed  Google Scholar 

  6. Caocci G, Pisu S, Argiolu F, Giardini C, Locatelli F, Vacca A, et al. Decision-making in adult thalassemia patients undergoing unrelated bone marrow transplantation: quality of life, communication and ethical issues. Bone Marrow Transplant. 2006;37:165–9.

    Article  CAS  PubMed  Google Scholar 

  7. Bahar B, Gholibeikian S, Aghdami N, Heshmat R, Safavifar F, Samiee S. Quality of life in beta thalassaemia major at least six months post-allogeneic stem cell transplantation in comparison to the same non-transplanted patients. Bone Marrow Transplant. 2005;35:S268.

    Google Scholar 

  8. Caocci G, Efficace F, Ciotti F, Roncarolo MG, Vacca A, Piras E, et al. Prospective assessment of health-related quality of life in pediatric patients with beta-thalassemia following hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2011;17:861–6.

    Article  PubMed  Google Scholar 

  9. Caocci G, Vacca A, Piras E, Serreli V, Dessi C, Marcias M, et al. Return to normal life after hematopoietic stem cell transplantation for thalassemia: a study of patients transplanted from matched sibling donors. Bone Marrow Transplant. 2016;51:1640–1.

    Article  CAS  PubMed  Google Scholar 

  10. Cheuk DKL, Mok ASP, Lee ACW, Chiang AKS, Ha SY, Lau YL, et al. Quality of life in patients with transfusion-dependent thalassemia after hematopoietic SCT. Bone Marrow Transplant. 2008;42:319–27.

    Article  CAS  PubMed  Google Scholar 

  11. Kelly MJ, Pennarola BW, Rodday AM, Parsons SK. on behalf of the Journeys to Recovery Study, the HSCT-CHESSTM Study. Health-related quality of life (HRQL) in children with sickle cell disease and thalassemia following hematopoietic stem cell transplant (HSCT). Pediatr Blood Cancer. 2012;59:725–31.

    Article  PubMed  Google Scholar 

  12. La Nasa G, Caocci G, Efficace F, Dessì C, Vacca A, Piras E, et al. Long-term health-related quality of life evaluated more than 20 years after hematopoietic stem cell transplantation for thalassemia. Blood. 2013;122:2262–70.

    Article  PubMed  Google Scholar 

  13. Uygun V, Tayfun F, Akcan M, Karasu GT, Kupesiz A, Hazar V, et al. Quality of life assessment in hematopoietic stem cell transplantation performed on thalassemia major patients. Pediatr Hematol Oncol. 2012;29:461–71.

    Article  PubMed  Google Scholar 

  14. Patel S, Swaminathan VV, Mythili V, Venkatadesikalu M, Sivasankaran M, Jayaraman D, et al. Quality matters—hematopoietic stem cell transplantation versus transfusion and chelation in thalassemia major. Indian Pediatr. 2018;55:1056–8.

    Article  PubMed  Google Scholar 

  15. Javanbakht M, Keshtkaran A, Shabaninejad H, Karami H, Zakerinia M, Delavari S. Comparison of blood transfusion plus chelation therapy and bone marrow transplantation in patients with beta-thalassemia: application of SF-36, EQ-5D, and visual analogue scale measures. Int J Health Policy Manag. 2015;4:733–40.

    Article  PubMed  PubMed Central  Google Scholar 

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Acknowledgements

We thank Dr. Emanuela Morelli and Dr. Luigi Marras for collecting web data and supporting the analysis of data

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Author notes

  1. These authors contributed equally: Olga Mulas, Giovanni Caocci

Authors and Affiliations

  1. Department of Medical Sciences and Public Health, University of Cagliari, Cagliari, Italy

    Olga Mulas, Giovanni Caocci & Giorgio La Nasa

  2. Hematology Unit, Businco Hospital, Cagliari, Italy

    Olga Mulas, Giovanni Caocci, Eugenia Piras, Clara Targhetta, Veronica Frau, Adriana Vacca & Giorgio La Nasa

  3. Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases Data Center, Rome, Italy

    Fabio Efficace

  4. Pediatric Clinic, Thalassemia and Rare Diseases, Pediatric Hospital “Microcitemico A. Cao”, Cagliari, Italy

    Susanna Barella

  5. Bone Marrow Transplant Center, Pediatric Hospital “Microcitemico A. Cao”, Cagliari, Italy

    Antonio Piroddi & Maria Grazia Orofino

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  1. Olga Mulas
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Contributions

OM, GC conceptualized and designed the study; OM, GC, EP, CT, VF, SB, AP, MGO, AV, GLN treated patients, collected and assembled the data; OM and FE performed the statistical analysis; OM, GC wrote the paper; OM, GC, FE, EP, CT, VF, SB, AP, MGO, AV, GLN were responsible for the final approval of the paper.

Corresponding author

Correspondence to Giovanni Caocci.

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Competing interests

FE: consultancy fees from Amgen, Abbvie, Janssen, and Novartis. Research support (Institution) from Abbvie, Amgen, Novartis, all unrelated to this work. The other authors have no competing interests to disclose.

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Mulas, O., Caocci, G., Efficace, F. et al. Long-term health-related quality of life in patients with β-thalassemia after unrelated hematopoietic stem cell transplantation. Bone Marrow Transplant 57, 1833–1836 (2022). https://doi.org/10.1038/s41409-022-01823-2

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