TY - JOUR
T1 - A complication risk score to evaluate clinical severity of thalassaemia syndromes
AU - Vitrano, Angela
AU - Meloni, Antonella
AU - Addario Pollina, Walter
AU - Karimi, Mehran
AU - El-Beshlawy, Amal
AU - Hajipour, Mahmoud
AU - Di Marco, Vito
AU - Hussain Ansari, Saqib
AU - Filosa, Aldo
AU - Ricchi, Paolo
AU - Ceci, Adriana
AU - Daar, Shahina
AU - Titi Singer, Sylvia
AU - Naserullah, Zaki A.
AU - Pepe, Alessia
AU - Scondotto, Salvatore
AU - Dardanoni, Gabriella
AU - Bonifazi, Fedele
AU - Vichinsky, Elliott
AU - Maggio, Aurelio
N1 - Publisher Copyright:
© 2020 British Society for Haematology and John Wiley & Sons Ltd
PY - 2021/2
Y1 - 2021/2
N2 - The thalassaemia syndromes (TS) show different phenotype severity. Developing a reliable, practical and global tool to determine disease severity and tailor treatment would be of great value. Overall, 7910 patients were analysed with the aim of constructing a complication risk score (CoRS) to evaluate the probability of developing one or more complications. Nine independent variables were included in the investigation as predictors. Logistic regression models were used for Group A [transfusion-dependent thalassaemia (TDT)], Group B [transfused non-TDT (NTDT)] and Group C (non-transfused NTDT). Statistically significant predictors included age (years), haemoglobin levels, hepatic transaminases [alanine aminotransferase (ALT) and aspartate aminotransferase] and left-ventricular ejection fraction (LVEF) for Group A; age (years), age at first chelation (months), ALT and LVEF for Group B; and age (years), mean serum ferritin (SF) levels and LVEF for Group C. The area under the receiver operating characteristic curve was 84·5%, 82·1% and 80·0% for Groups A, Group B and Group C respectively, suggesting the models had good discrimination. Finally, the CoRS for each group was categorised into four risk classes (low, intermediate, high, and very high) using the centiles of its distribution. In conclusion, we have developed a CoRS for TS that can assist physicians in prospectively tailoring patients’ treatment.
AB - The thalassaemia syndromes (TS) show different phenotype severity. Developing a reliable, practical and global tool to determine disease severity and tailor treatment would be of great value. Overall, 7910 patients were analysed with the aim of constructing a complication risk score (CoRS) to evaluate the probability of developing one or more complications. Nine independent variables were included in the investigation as predictors. Logistic regression models were used for Group A [transfusion-dependent thalassaemia (TDT)], Group B [transfused non-TDT (NTDT)] and Group C (non-transfused NTDT). Statistically significant predictors included age (years), haemoglobin levels, hepatic transaminases [alanine aminotransferase (ALT) and aspartate aminotransferase] and left-ventricular ejection fraction (LVEF) for Group A; age (years), age at first chelation (months), ALT and LVEF for Group B; and age (years), mean serum ferritin (SF) levels and LVEF for Group C. The area under the receiver operating characteristic curve was 84·5%, 82·1% and 80·0% for Groups A, Group B and Group C respectively, suggesting the models had good discrimination. Finally, the CoRS for each group was categorised into four risk classes (low, intermediate, high, and very high) using the centiles of its distribution. In conclusion, we have developed a CoRS for TS that can assist physicians in prospectively tailoring patients’ treatment.
KW - complications
KW - prognostic model
KW - risk score
KW - thalassaemia
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U2 - 10.1111/bjh.17203
DO - 10.1111/bjh.17203
M3 - Article
C2 - 33216983
AN - SCOPUS:85096701576
SN - 0007-1048
VL - 192
SP - 626
EP - 633
JO - British Journal of Haematology
JF - British Journal of Haematology
IS - 3
ER -