title: Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study.
abstract: Despite recent advances in understanding the pathophysiologic mechanisms behind the thalassemia intermedia (TI) phenotype, data on the effects of treatment are deficient. To provide such data, we evaluated 584 TI patients for the associations between patient and disease characteristics, treatment received, and the rate of complications. The most common disease-related complications were osteoporosis, extramedullary hematopoeisis (EMH), hypogonadism, and cholelithiasis, followed by thrombosis, pulmonary hypertension (PHT), abnormal liver function, and leg ulcers. Hypothyroidism, heart failure, and diabetes mellitus were less frequently observed. On multivariate analysis, older age and splenectomy were independently associated with an increased risk of most disease-related complications. Transfusion therapy was protective for thrombosis, EMH, PHT, heart failure, cholelithiasis, and leg ulcers. However, transfusion therapy was associated with an increased risk of endocrinopathy. Iron chelation therapy was in turn protective for endocrinopathy and PHT. Hydroxyurea treatment was associated with an increased risk of hypogonadism yet was protective for EMH, PHT, leg ulcers, hypothyroidism, and osteoporosis. Attention should be paid to the impact of age on complications in TI, and the beneficial role of splenectomy deserves revisiting. This study provides evidence that calls for prospective evaluation of the roles of transfusion, iron chelation, and hydroxyurea therapy in TI patients.
authors.affiliation: Department of Internal Medicine, Hematology-Oncology Division, American University of Beirut Medical Center, Beirut, Lebanon. ataher@aub.edu.lb
authors.forename: Ali T
authors.initials: AT
authors.lastname: Taher
authors.forename: Khaled M
authors.initials: KM
authors.lastname: Musallam
authors.forename: Mehran
authors.initials: M
authors.lastname: Karimi
authors.forename: Amal
authors.initials: A
authors.lastname: El-Beshlawy
authors.forename: Khawla
authors.initials: K
authors.lastname: Belhoul
authors.forename: Shahina
authors.initials: S
authors.lastname: Daar
authors.forename: Mohamed-SalahEldin
authors.initials: MS
authors.lastname: Saned
authors.forename: Abdul-Hamid
authors.initials: AH
authors.lastname: El-Chafic
authors.forename: Maria R
authors.initials: MR
authors.lastname: Fasulo
authors.forename: Maria D
authors.initials: MD
authors.lastname: Cappellini
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D002675:Child, Preschool; D019353:Endemic Diseases / Q000517:prevention & control* / Q000706:statistics & numerical data; D004700:Endocrine System Diseases / Q000453:epidemiology / Q000517:prevention & control; D005260:Female; D006411:Hematopoiesis, Extramedullary; D006801:Humans; D006976:Hypertension, Pulmonary / Q000453:epidemiology / Q000517:prevention & control; D008107:Liver Diseases / Q000453:epidemiology / Q000517:prevention & control; D008297:Male; D008875:Middle Aged; D011364:Professional Practice / Q000592:standards; D012189:Retrospective Studies; D013789:Thalassemia / Q000150:complications* / Q000453:epidemiology* / Q000517:prevention & control / Q000628:therapy*; D013927:Thrombosis / Q000453:epidemiology / Q000517:prevention & control; D055815:Young Adult