Development of a Thalassemia International Prognostic Scoring System (TIPSS).

Angela Vitrano, Khaled M Musallam, Antonella Meloni, Mehran Karimi, Shahina Daar, Paolo Ricchi, Silvia Costantini, Efthymia Vlachaki, Vito Di Marco, Amal El-Beshlawy, Mahmoud Hajipour, Saqib Hussain Ansari, Aldo Filosa, Adriana Ceci, Sylvia Titi Singer, Zaki A Naserullah, Alessia Pepe, Filippo Cademartiri, Sebastiano Addario Pollina, Salvatore Scondotto, Gabriella Dardanoni, Fedele Bonifazi, Vijay G Sankaran, Elliott Vichinsky, Ali T Taher, Aurelio Maggio

Journal: Blood cells, molecules & diseases 2023;99():102710

PMID: 36463683

Full Text on Elsevier Science View on PubMed

Abstract

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.

Address: Campus of Haematology Franco and Piera Cutino, AOOR Villa Sofia-V. Cervello, Palermo, Italy.; Thalassemia Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates.; Department of Radiology, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy.; Haematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran.; Department of Haematology, College of Medicine and Health Sciences, Sultan Qaboos University, Oman; Wallenberg Research Centre, Stellenbosch Institute for Advanced Study, Stellenbosch University, Stellenbosch, South Africa.; Rare Blood Cell Disease Unit, "Cardarelli" Hospital, Naples, Italy.; Thalassaemia Unit, Ippokratio University Hospital, Thessaloniki, Greece.; Sezione di Gastroenterologia e Epatologia, Dipartimento Biomedico di Medicina Interna e Specialistica, University of Palermo, Palermo, Italy.; Department of Pediatric Haematology, Faculty of Medicine, Cairo University, Cairo, Egypt.; Pediatric Gastroenterology, Hepatology and Nutrition Research Center, Research Institute for Children's Health, Shahid Beheshti University of Medical Sciences, Tehran, Iran.; Children's Hospital Karachi (CHK), Karachi, Pakistan.; Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus, Valenzano (BA), Italy.; Division of Hematology-Oncology, Department of Pediatrics, University of California San Francisco, UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.; Dammam Maternity and Child Hospital, Dammam, Saudi Arabia.; Institute of Radiology, Department of Medicine, University of Padua, Italy.; D.A.S.O.E, Regione Siciliana, Palermo, Italy.; Division of Hematology/Oncology, Boston Children's Hospital and Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA; Broad Institute of MIT and Harvard, Cambridge, MA, USA; Harvard Stem Cell Institute, Cambridge, MA, USA.; Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.; Campus of Haematology Franco and Piera Cutino, AOOR Villa Sofia-V. Cervello, Palermo, Italy. Electronic address: [email protected].

DOI: 10.1016/j.bcmd.2022.102710

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MeSH Terms: Humans, Prognosis, Retrospective Studies, Portasystemic Shunt, Transjugular Intrahepatic, beta-Thalassemia, Thalassemia