Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia

Khaled M Musallam¹, Angela Vitrano², Antonella Meloni³, Sebastiano Addario Pollina⁴, Mehran Karimi⁵, Amal El-Beshlawy⁶, Mahmoud Hajipour⁷, Vito Di Marco⁸, Saqib Hussain Ansari⁹, Aldo Filosa¹⁰, Paolo Ricchi¹⁰, Adriana Ceci¹¹, Shahina Daar¹², Efthymia Vlachaki¹³, Sylvia Titi Singer¹⁴, Zaki A Naserullah¹⁵, Alessia Pepe³, Salvatore Scondotto⁴, Gabriella Dardanoni⁴, Fedele Bonifazi¹¹, Vijay G Sankaran¹⁶, Elliott Vichinsky¹⁴, Ali T Taher¹⁷, Aurelio Maggio²

Affiliations

¹ Thalassemia Center, Burjeel Medical City, Abu Dhabi, UAE
² Campus of Haematology Franco and Piera Cutino, AOOR Villa Sofia-V. Cervello, Palermo.
³ MRI Unit, Fondazione G. Monasterio CNR-Regione Toscana, Pisa.
⁴ D.A.S.O.E, Regione Siciliana, Palermo.
⁵ Haematology Research Center, Shiraz University of Medical Sciences, Shiraz.
⁶ Department of Pediatric Haematology, Faculty of Medicine, Cairo University, Cairo.
⁷ Pediatric Gastroenterology, Hepatology and Nutrition Research Center, Research Institute for Children's Health, Shahid Beheshti University of Medical Sciences, Tehran.
⁸ Department of Promozione della Salute, Materno Infantile, Medicina Interna e Specialistica di Eccellenza (PROMISE), University of Palermo, Palermo, Italy
⁹ Department of Pediatric Haematology and Molecular Medicine, National Institute of Blood Diseases and Bone Marrow Transplantation, Karachi.
¹⁰ Rare Blood Cell Disease Unit, "Cardarelli" Hospital, Naples.
¹¹ Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus, Valenzano (BA).
¹² Department of Haematology, College of Medicine and Health Sciences, Sultan Qaboos University, Sultanate of Oman; Wallenberg Research Centre, Stellenbosch Institute for Advanced Study, Stellenbosch University, Stellenbosch.
¹³ Thalassaemia Unit, Ippokratio University Hospital, Thessaloniki.
¹⁴ Division of Hematology-Oncology, Department of Pediatrics, University of California San Francisco, UCSF Benioff Children's Hospital Oakland, Oakland, CA.
¹⁵ Dammam Maternity and Child Hospital, Dammam.
¹⁶ Division of Hematology/Oncology, Boston Children's Hospital and Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Harvard Stem Cell Institute, Cambridge, MA.
¹⁷ Department of Internal Medicine, American University of Beirut Medical Center, Beirut.

PMID: 33882642
PMCID: PMC8409024
DOI: 10.3324/haematol.2021.278684

Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia

Khaled M Musallam et al. Haematologica. 2021.

. 2021 Sep 1;106(9):2489-2492.

doi: 10.3324/haematol.2021.278684.

Authors

Affiliations

¹ Thalassemia Center, Burjeel Medical City, Abu Dhabi, UAE
² Campus of Haematology Franco and Piera Cutino, AOOR Villa Sofia-V. Cervello, Palermo.
³ MRI Unit, Fondazione G. Monasterio CNR-Regione Toscana, Pisa.
⁴ D.A.S.O.E, Regione Siciliana, Palermo.
⁵ Haematology Research Center, Shiraz University of Medical Sciences, Shiraz.
⁶ Department of Pediatric Haematology, Faculty of Medicine, Cairo University, Cairo.
⁷ Pediatric Gastroenterology, Hepatology and Nutrition Research Center, Research Institute for Children's Health, Shahid Beheshti University of Medical Sciences, Tehran.
⁸ Department of Promozione della Salute, Materno Infantile, Medicina Interna e Specialistica di Eccellenza (PROMISE), University of Palermo, Palermo, Italy
⁹ Department of Pediatric Haematology and Molecular Medicine, National Institute of Blood Diseases and Bone Marrow Transplantation, Karachi.
¹⁰ Rare Blood Cell Disease Unit, "Cardarelli" Hospital, Naples.
¹¹ Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus, Valenzano (BA).
¹² Department of Haematology, College of Medicine and Health Sciences, Sultan Qaboos University, Sultanate of Oman; Wallenberg Research Centre, Stellenbosch Institute for Advanced Study, Stellenbosch University, Stellenbosch.
¹³ Thalassaemia Unit, Ippokratio University Hospital, Thessaloniki.
¹⁴ Division of Hematology-Oncology, Department of Pediatrics, University of California San Francisco, UCSF Benioff Children's Hospital Oakland, Oakland, CA.
¹⁵ Dammam Maternity and Child Hospital, Dammam.
¹⁶ Division of Hematology/Oncology, Boston Children's Hospital and Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Harvard Stem Cell Institute, Cambridge, MA.
¹⁷ Department of Internal Medicine, American University of Beirut Medical Center, Beirut.

PMID: 33882642
PMCID: PMC8409024
DOI: 10.3324/haematol.2021.278684

No abstract available

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Figures

**Figure 1.**
**Kaplan-Meier survival curves.** (A) all-cause mortality, (B) all-cause mortality according to regular transfusion therapy status, (C) mortality from cardiovascular disease according to regular transfusion therapy status, and (D) mortality from hepatic disease according to iron chelation therapy status.

See this image and copyright information in PMC

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References

1. Musallam KM, Rivella S, Vichinsky E, Rachmilewitz EA. Non-transfusion- dependent thalassemias. Haematologica. 2013;98(6):833-844. - PMC - PubMed
1. Musallam KM, Cappellini MD, Taher AT. Variations in hemoglobin level and morbidity burden in non-transfusion-dependent beta-thalassemia. Ann Hematol. 2021;100(7):1903-1905. - PubMed
1. Musallam KM, Cappellini MD, Daar S, et al. . Serum ferritin level and morbidity risk in transfusion-independent patients with beta-thalassemia intermedia: the ORIENT study. Haematologica. 2014; 99(11):e218-221. - PMC - PubMed
1. Musallam KM, Cappellini MD, Wood JC, et al. . Elevated liver iron concentration is a marker of increased morbidity in patients with beta thalassemia intermedia. Haematologica. 2011;96(11):1605-1612. - PMC - PubMed
1. Vitrano A, Calvaruso G, Lai E, et al. . The era of comparable life expectancy between thalassaemia major and intermedia: Is it time to revisit the major-intermedia dichotomy? Br J Haematol. 2017; 176(1):124-130. - PubMed

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Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia

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Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia

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