Developing an International Prognostic Scoring System for Thalassemia: A New Tool for Revisiting Classification of Thalassemia

Β-thalassemia is classified into three phenotypes, depending on the severity of symptoms: 1) thalassemia major (TM); 2) thalassemia Intermedia (TI); 3) β trait or thalassemia minor (Tm). Recent data suggest that the survival of well-treated patients with TM is now similar to that of those with TI. The current guidelines categorising beta thalassemia patients into either TM or TI were put in place over 30yrs ago. With the current state of safe transfusion and better chelation, maintaining these guidelines may prevent patients from achieving a better quality of life. In addition, a retrospective study on 4943 subjects suggest that, even in patients defined as Tm, there is an increase in morbidity (cirrhosis, kidney disease, etc.) compared with the non-thalassemia population.

Primary Aim

The primary aim of this project is retrospectively re-classification Thalassemia Syndromes towards a classification including a “continuum” of the same disease divided in stages according with the severity of phenotype based on the following main outcomes obtained from “scoring severity system” published by Cappellini et al., 20151:

If the rationale of the study is confirmed, some of the patients with TM, TI and Tm will be found to be in the same Stage, independently of the diagnosis at onset (i.e subjects with TM in same Stage of TI, subjects with Tm in same stage of TI). This will allow a new classification based on Thalassemia Syndromes divided in Stages or Class of Risk.

Secondary Aims

  •  To assess the difference in survival between Thalassemia Major versus Intermedia
  •  To assess the impact of the main prognostic factors influencing the risk for complications (See below for factors)
  •  To assess the above outcomes on patients from Oman (Oman study) including analyses resulting in 25 yr morbidity/ mortality of single cohort
Project leader(s):
  • Shahina Daar (Department of Hematology, Sulatan Qaboos University)

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