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Curr Med Res Opin. 2017 Aug;33(8):1525-1533. doi: 10.1080/03007995.2017.1326890. Epub 2017 Jun 7.

Pattern of complications and burden of disease in patients affected by beta thalassemia major.

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a Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus , Valenzano ( BA ), Italy.
b Fondazione Salvatore Maugeri , Pavia , Italy.
c Consorzio per Valutazioni Biologiche e Farmacologiche , Pavia , Italy.
d Dipartimento di Biomedicina e oncologia umana Sezione pediatrica , Università "A. Moro" di Bari , Italy.
e Fondazione "Leonardo Giambrone" per la Guarigione dalla Thalassemia , Castelvolturno ( CE ), Italy.
f Fondazione G. Monasterio C.N.R. , Regione Toscana , Pisa , Italy.
g Azienda Ospedaliera Padova Centro Leucemie Oncoematologia Pediatrica , Padova , Italy.
h Azienda Ospedaliera Universitaria Consorziale Policlinico di Bari, Unità Operativa di Pediatria Generale e Specialistica "Federico Vecchio" Bari , Italy.
i Azienda Ospedaliera "A. Cardarelli" UOS Talassemia Pediatrica e Emoglobinopatie Pediatriche , Napoli , Italy.
j Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello , Palermo , Italy.



Despite the correct application of blood transfusions and chelation treatments, beta thalassemia patients have many complications. Systematic population analyses on types and frequency of these complications are very few. The aim of this study is to characterize the complications, their risk factors and their clinical and economic impact.


Complications at baseline and events occurring during one observational year were analyzed in 272 patients aged >12 years. Risk factors were analyzed through chi-squared and unpaired t tests. Logistic regression was applied to perform the risk factors multivariate analysis.


A total of 554 complications (1-6 per patient) affected 82.3% of patients. Cardiac complications were less represented than expected. Musculoskeletal diseases were the most represented complications followed by hepatic, sexual and endocrine diseases. Splenectomized patients, born before 1970 and aged >40 years, starting iron chelation therapy when aged >4 years or after receiving more than 20 blood transfusions, presented a significantly higher number of complications. A total of 885 adverse events requiring 34125 additional medical services occurred in 1 year. Of these, 34.9% were related to treatments and 65.1% to other causes. Event numbers, additional medical interventions and cost increased progressively in patients affected by one or more complication compared to patients with no complications.


The pattern of complications changes according to birth cohort and differentiates older from younger patients. The burden of the disease and its costs increase after the onset of the first complication, therefore prevention of complications is fundamental in these patients.


Thalassemia; adverse reactions; complications; cost of the disease; risk factors

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