- •We describe a disease severity scoring system for adult and pediatric NTDT patients.
- •Consensus of expert opinion was used to establish the scoring system.
- •20 parameters were selected for inclusion with an extra six for pediatric patients.
- •The system may be developed into a practical tool for widespread clinical use.
Abbreviations:ALT (alanine aminotransferase), dw (dry weight), ELISA (enzyme-linked immunosorbent assay), eGFR (estimated glomerular filtration rate), Hb (hemoglobin), LIC (liver iron concentration), LVEF (left ventricular ejection fraction), MRI (magnetic resonance imaging), N/A (not available), MCID (minimum clinically important difference), NTDT (non-transfusion-dependent thalassemia), NYHA (New York Heart Association), PHT (pulmonary hypertension), QoL (quality of life), RNA (ribonucleic acid), SF (serum ferritin), TRV (tricuspid regurgitation jet velocity), ULN (upper limit of normal)
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