title: Clinicopathological and radiological study of Egyptian β-thalassemia intermedia and β-thalassemia major patients: relation to complications and response to therapy.
abstract: The clinico epidemiological characteristics, frequency of complications, and response to various therapeutic modalities in 80 Egyptian β-thalassemia intermedia (β-TI) patients were compared with 70 β-thalassemia major (β-TM) patients. β-Thalassemia intermedia patients had a higher incidence of left atrium dilatation, right ventricular dilatation and pulmonary hypertension, whereas, β-TM patients showed a higher incidence of left ventricular (LV) dilatation, restrictive LV filling and impaired LV contractility, with an overall higher incidence of heart disease (p <0.001). Short stature, delayed puberty, osteoporosis, bone fractures, diabetes mellitus and viral hepatitis was frequently observed in β-TM patients compared with β-TI patients (p <0.05). Administration of hydroxyurea (HU) alone was associated with significant improvement in hematological parameters and quality of life for β-TI patients. In conclusion, the risk of complications still burdens the life of Egyptian thalassemia patients and their frequency varies between β-TI and β-TM. We provide evidence that calls for the use of HU in β-TI patients.
authors.affiliation: Department of Pediatric, Faculty of Medicine, Ain Shams University, Cairo, Egypt.
authors.forename: Galila M
authors.initials: GM
authors.lastname: Mokhtar
authors.forename: Azza A G
authors.initials: AA
authors.lastname: Tantawy
authors.forename: Amira A M
authors.initials: AA
authors.lastname: Adly
authors.forename: Eman A R
authors.initials: EA
authors.lastname: Ismail
mesh_terms: D015502:Absorptiometry, Photon; D000293:Adolescent; D000328:Adult; D000986:Antisickling Agents / Q000627:therapeutic use; D015913:Chelation Therapy / Q000379:methods; D002648:Child; D002675:Child, Preschool; D003676:Deferoxamine / Q000627:therapeutic use; D004452:Echocardiography; D004534:Egypt; D005260:Female; D005500:Follow-Up Studies; D006331:Heart Diseases / Q000175:diagnosis / Q000473:pathology / Q000503:physiopathology; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D006976:Hypertension, Pulmonary / Q000150:complications / Q000503:physiopathology; D008297:Male; D010024:Osteoporosis / Q000150:complications / Q000175:diagnosis; D017063:Outcome Assessment, Health Care; D011788:Quality of Life; D017262:Siderophores / Q000627:therapeutic use; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications* / Q000188:drug therapy* / Q000473:pathology
chemical_list: D000986:Antisickling Agents; D017262:Siderophores; D003676:Deferoxamine; D006918:Hydroxyurea