TY - JOUR
T1 - Development of a Thalassemia International Prognostic Scoring System (TIPSS)
AU - International Working Group on Thalassemia (IWG-THAL)
AU - Vitrano, Angela
AU - Musallam, Khaled M.
AU - Meloni, Antonella
AU - Karimi, Mehran
AU - Daar, Shahina
AU - Ricchi, Paolo
AU - Costantini, Silvia
AU - Vlachaki, Efthymia
AU - Di Marco, Vito
AU - El-Beshlawy, Amal
AU - Hajipour, Mahmoud
AU - Ansari, Saqib Hussain
AU - Filosa, Aldo
AU - Ceci, Adriana
AU - Singer, Sylvia Titi
AU - Naserullah, Zaki A.
AU - Pepe, Alessia
AU - Cademartiri, Filippo
AU - Pollina, Sebastiano Addario
AU - Scondotto, Salvatore
AU - Dardanoni, Gabriella
AU - Bonifazi, Fedele
AU - Sankaran, Vijay G.
AU - Vichinsky, Elliott
AU - Taher, Ali T.
AU - Maggio, Aurelio
N1 - Publisher Copyright:
© 2022
PY - 2023/3/1
Y1 - 2023/3/1
N2 - A prognostic scoring system that can differentiate β-thalassemia patients based on mortality risk is lacking. We analysed data from 3145 β-thalassemia patients followed through a retrospective cohort design for the outcome of death. An a priori list of prognostic variables was collected. β Coefficients from a multivariate cox regression model were used from a development dataset (n = 2516) to construct a formula for a Thalassemia International Prognostic Scoring System (TIPSS) which was subsequently applied to a validation dataset (n = 629). The median duration of observation was 10.0 years. The TIPSS score formula was constructed as exp (1.4 × heart disease + 0.9 × liver disease + 0.9 × diabetes + 0.9 × sepsis + 0.6 × alanine aminotransferase ≥42 IU/L + 0.6 × hemoglobin ≤9 g/dL + 0.4 × serum ferritin ≥1850 ng/mL). TIPSS score thresholds of greatest differentiation were assigned as <2.0 (low-risk), 2.0 to <5.0 (intermediate-risk), and ≥5.0 (high-risk). The TIPSS score was a good predictor for the outcome of death in the validation dataset (AUC: 0.722, 95%CI: 0.641–0.804) and survival was significantly different between patients in the three risk categories (P < 0.001). Compared to low-risk patients, the hazard ratio for death was 2.778 (95%CI: 1.335–5.780) in patients with intermediate-risk and 6.431 (95%CI: 3.151–13.128) in patients with high-risk. This study provides a novel tool to support mortality risk categorization for patients with β-thalassemia that could help management and research decisions.
AB - A prognostic scoring system that can differentiate β-thalassemia patients based on mortality risk is lacking. We analysed data from 3145 β-thalassemia patients followed through a retrospective cohort design for the outcome of death. An a priori list of prognostic variables was collected. β Coefficients from a multivariate cox regression model were used from a development dataset (n = 2516) to construct a formula for a Thalassemia International Prognostic Scoring System (TIPSS) which was subsequently applied to a validation dataset (n = 629). The median duration of observation was 10.0 years. The TIPSS score formula was constructed as exp (1.4 × heart disease + 0.9 × liver disease + 0.9 × diabetes + 0.9 × sepsis + 0.6 × alanine aminotransferase ≥42 IU/L + 0.6 × hemoglobin ≤9 g/dL + 0.4 × serum ferritin ≥1850 ng/mL). TIPSS score thresholds of greatest differentiation were assigned as <2.0 (low-risk), 2.0 to <5.0 (intermediate-risk), and ≥5.0 (high-risk). The TIPSS score was a good predictor for the outcome of death in the validation dataset (AUC: 0.722, 95%CI: 0.641–0.804) and survival was significantly different between patients in the three risk categories (P < 0.001). Compared to low-risk patients, the hazard ratio for death was 2.778 (95%CI: 1.335–5.780) in patients with intermediate-risk and 6.431 (95%CI: 3.151–13.128) in patients with high-risk. This study provides a novel tool to support mortality risk categorization for patients with β-thalassemia that could help management and research decisions.
KW - Management
KW - Mortality
KW - Outcomes
KW - Prognosis
KW - Survival
KW - Thalassemia
KW - Portasystemic Shunt, Transjugular Intrahepatic/adverse effects
KW - Humans
KW - Retrospective Studies
KW - beta-Thalassemia/complications
UR - http://www.scopus.com/inward/record.url?scp=85143487929&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85143487929&partnerID=8YFLogxK
UR - https://www.mendeley.com/catalogue/78b1e607-34fa-3371-802f-d581f58a1b67/
U2 - 10.1016/j.bcmd.2022.102710
DO - 10.1016/j.bcmd.2022.102710
M3 - Review article
C2 - 36463683
AN - SCOPUS:85143487929
SN - 1079-9796
VL - 99
JO - Blood Cells, Molecules, and Diseases
JF - Blood Cells, Molecules, and Diseases
M1 - 102710
ER -