Page 204 - 2021_09-Haematologica-web
P. 204

Letters to the Editor
Pharmaceutical Industry and Novartis; ATT has been or is a consultant for Novartis, Celgene Corp (Bristol Myers Squibb), Vifor Pharma, Silence Therapeutics and Ionis Pharmaceuticals; and received research funding from Novartis, Celgene Corp (Bristol Myers Squibb), La Jolla Pharmaceutical Company, Roche, Protagonist Therapeutics and Agios Pharmaceuticals; AM has been or is a member of advisory boards for Novartis, Celgene Corp (Bristol Meyers Squibb) and Bluebird Bio. The remaining authors have no conflicts of interest to disclose.
Contributions: KMM, AV, AM: study design; AV, AM, SAP, MK, AE-B, MH, VDM, SHA, AF, PR, AC, SD, EV, STS, ZAN, EV: data collection; KMM, AV: data analysis; KMM, AV, AM: manu- script drafting; data interpretation and manuscript review for intellectual content: all authors. Final approval for submission: all authors.
Acknowledgments: the authors would like to thank all patients for agreeing to participate in this study. The support of the Foundation Franco and Piera Cutino is appreciated.
Data-sharing: data were collected and stored on the IHR electronic platform (www.sanitasicilia.eu/IWG), and are available from the corre- sponding author upon request.
References
1. Musallam KM, Rivella S, Vichinsky E, Rachmilewitz EA. Non-trans- fusion-dependent thalassemias. Haematologica. 2013;98(6):833- 844.
2. Musallam KM, Cappellini MD, Taher AT. Variations in hemoglobin level and morbidity burden in non-transfusion-dependent beta-tha- lassemia. Ann Hematol. 2021;100(7):1903-1905.
3. Musallam KM, Cappellini MD, Daar S, et al. Serum ferritin level and morbidity risk in transfusion-independent patients with beta-tha- lassemia intermedia: the ORIENT study. Haematologica. 2014; 99(11):e218-221.
4. 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.
5. 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.
6. Taher AT, Musallam KM, Karimi M, et al. Overview on practices in thalassemia intermedia management aiming for lowering complica- tion rates across a region of endemicity: the OPTIMAL CARE study. Blood. 2010;115(10):1886-1892.
7. Taher A, Vichinsky E, Musallam K, Cappellini MD, Viprakasit V. Guidelines for the Management of non transfusion dependent tha- lassaemia (NTDT). Nicosia, Cyprus: Thalassaemia International Federation; 2013. PMID: 24672826
8. Taher AT, Porter J, Viprakasit V, et al. Deferasirox reduces iron over- load significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-con- trolled study. Blood. 2012;120(5):970-977.
9.Calvaruso G, Vitrano A, Di Maggio R, et al. Deferiprone versus deferoxamine in thalassemia intermedia: Results from a 5-year long- term Italian multicenter randomized clinical trial. Am J Hematol. 2015;90(7):634-638.
10.Ricchi P, Meloni A, Pistoia L, et al. Longitudinal follow-up of patients with thalassaemia intermedia who started transfusion ther- apy in adulthood: a cohort study. Br J Haematol. 2020;191(1):107- 114.
11. Musallam KM, Rivella S, Taher AT. Management of non-transfu- sion-dependent beta-thalassemia (NTDT): The next 5 years. Am J Hematol. 2021;96(3):E57-E59.
12. Rivella S. beta-thalassemias: paradigmatic diseases for scientific dis- coveries and development of innovative therapies. Haematologica. 2015;100(4):418-430.
13.Moukhadder HM, Halawi R, Cappellini MD, Taher AT. Hepatocellular carcinoma as an emerging morbidity in the tha- lassemia syndromes: a comprehensive review. Cancer. 2017; 123(5):751-758.
14. Borgna-Pignatti C, Garani MC, Forni GL, et al. Hepatocellular carci- noma in thalassaemia: an update of the Italian Registry. Br J Haematol. 2014;167(1):121-126.
15. Musallam KM, Motta I, Salvatori M, et al. Longitudinal changes in serum ferritin levels correlate with measures of hepatic stiffness in transfusion-independent patients with beta-thalassemia intermedia. Blood Cells Mol Dis. 2012;49(3-4):136-139.
2492
haematologica | 2021; 106(9)


































































































   202   203   204   205   206