An International Health Repository (IHR) protocol was approved on May 25th, 2017 by the Italian Ethical Committee (EudraCT Code Numbers 2017-004457-17 and 143AOR2017). Subsequently, an International Working Group (IWG) on Thalassemia was formed and it met in Palermo, Italy, on September 15th and 16th, 2017. Indicators of phenotype severity thought to be most pertinent in defining disease severity were shared among the IWG based on their expertise as well as on expert opinion reported on literature. These data were collected and stored on IHR platform (www.sanitasicilia.eu/IWG ).In order to define the prognostic score (PS) a retrospective multicenter case-control study was carried ahead. Overall, clinical findings of 7910 patients who attended the IWG centres from 1976 to 2018 were collected .we propose that the severity scoring system described here could be developed into a practical tool for widespread clinical application, not only to evaluate patient status and classify disease subgroups, but also to inform treatment decisions and monitor patient progress in response to therapy. Finally, the use of this scoring system may help to select appropriate candidate for innovative treatment.
Development of a Severity Score System for Thalassemia Syndromes
Scondotto S;
2019-01-01
Abstract
An International Health Repository (IHR) protocol was approved on May 25th, 2017 by the Italian Ethical Committee (EudraCT Code Numbers 2017-004457-17 and 143AOR2017). Subsequently, an International Working Group (IWG) on Thalassemia was formed and it met in Palermo, Italy, on September 15th and 16th, 2017. Indicators of phenotype severity thought to be most pertinent in defining disease severity were shared among the IWG based on their expertise as well as on expert opinion reported on literature. These data were collected and stored on IHR platform (www.sanitasicilia.eu/IWG ).In order to define the prognostic score (PS) a retrospective multicenter case-control study was carried ahead. Overall, clinical findings of 7910 patients who attended the IWG centres from 1976 to 2018 were collected .we propose that the severity scoring system described here could be developed into a practical tool for widespread clinical application, not only to evaluate patient status and classify disease subgroups, but also to inform treatment decisions and monitor patient progress in response to therapy. Finally, the use of this scoring system may help to select appropriate candidate for innovative treatment.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
