title: Development of a new disease severity scoring system for patients with non-transfusion-dependent thalassemia.
abstract: BACKGROUND
  Patients with non-transfusion-dependent thalassemia (NTDT) present with a spectrum of disease severities. Since there are multiple pathophysiologies in such patients, tailoring treatment remains essential. Therefore, one simple, reliable tool would be beneficial to assess disease severity and tailor therapy, particularly for internal medicine specialists who may treat a variety of NTDT patients with a multitude of complications. This would allow for standardization of assessments leading to timely interventions and prevention of complications.
  
  
  METHODS
  A working group of NTDT experts was formed to develop a new disease severity scoring system for adult and pediatric patients with NTDT, based on parameters considered to be most pertinent in defining disease severity.
  
  
  RESULTS
  20 parameters were selected for inclusion in the disease severity scoring system. An additional six parameters, largely related to growth and development, were selected specifically for pediatric patients (≤ 16 years of age). Consensus of expert opinion was used to establish the selected methods of assessment for each parameter, based on feasibility and availability of technology, cost containment, and avoidance of patient risk.
  
  
  CONCLUSIONS
  We propose that this new disease severity scoring system for adult and pediatric NTDT patients could be developed into a practical tool for widespread clinical use.
authors.affiliation: Universita di Milano, Ca Granda Foundation IRCCS, Milan, Italy. Electronic address: maria.cappellini@unimi.it.
authors.forename: M Domenica
authors.initials: MD
authors.lastname: Cappellini
authors.affiliation: University College London, London, United Kingdom.
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
authors.affiliation: Universita di Milano, Ca Granda Foundation IRCCS, Milan, Italy; American University of Beirut, Beirut, Lebanon.
authors.forename: Khaled M
authors.initials: KM
authors.lastname: Musallam
authors.affiliation: First Department of Pediatrics, University of Athens, Athens, Greece.
authors.forename: Antonis
authors.initials: A
authors.lastname: Kattamis
authors.affiliation: Department of Pediatrics and Thalassemia Center, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: Vip
authors.initials: V
authors.lastname: Viprakasit
authors.affiliation: Ospedale Regionale Microcitemie, Dipartimento Scienze Biomediche e Biotechnologie, Università di Cagliari, Cagliari, Italy.
authors.forename: Renzo
authors.initials: R
authors.lastname: Galanello
authors.affiliation: American University of Beirut, Beirut, Lebanon.
authors.forename: Ali T
authors.initials: AT
authors.lastname: Taher
mesh_terms: D000293:Adolescent; D000328:Adult; D000410:Alanine Transaminase / Q000097:blood*; D001145:Arrhythmias, Cardiac / Q000453:epidemiology; D001663:Bilirubin / Q000097:blood*; D001851:Bone Diseases, Metabolic / Q000453:epidemiology; D002648:Child; D002763:Cholecystectomy; D002764:Cholecystitis / Q000453:epidemiology; D002769:Cholelithiasis / Q000453:epidemiology; D015897:Comorbidity; D006333:Heart Failure / Q000453:epidemiology; D006454:Hemoglobins / Q000378:metabolism*; D006509:Hepatitis B / Q000453:epidemiology; D006526:Hepatitis C / Q000453:epidemiology; D006801:Humans; D006976:Hypertension, Pulmonary / Q000453:epidemiology; D007501:Iron / Q000097:blood*; D007871:Leg Ulcer / Q000453:epidemiology; D008103:Liver Cirrhosis / Q000453:epidemiology; D010024:Osteoporosis / Q000453:epidemiology; D010976:Platelet Count; D012720:Severity of Illness Index; D013163:Splenomegaly / Q000453:epidemiology; D013318:Stroke Volume; D013927:Thrombosis / Q000453:epidemiology; D017086:beta-Thalassemia / Q000097:blood* / Q000453:epidemiology
chemical_list: D006454:Hemoglobins; D007501:Iron; D000410:Alanine Transaminase; D001663:Bilirubin
keywords: Disease severity; Non-transfusion-dependent thalassemia; Scoring