N J Perera1, N S Lau, S Mathews, C Waite, P J Ho, I D Caterson. 1. Department of Endocrinology and Institute of Haematology, Royal Prince Alfred Hospital and Institute of Obesity Nutrition and Exercise, University of Sydney, Sydney, New South Wales, Australia. nimalie.perera@email.cs.nsw.gov.au
Abstract
BACKGROUND: Thalassaemia major is a common and serious medical problem worldwide that is associated with a range of complications, including effects on multiple endocrine pathways. Minimizing or preventing comorbidities is important for these individuals who need life-long multidisciplinary care and treatment. However, there are limited overviews of the endocrine complications associated with this illness, nor any consensus regarding management guidelines. METHOD: A retrospective cohort analysis of β-thalassaemia patients attending an ambulatory transfusion clinic at Royal Prince Alfred Hospital was conducted from June 2008. RESULTS: All of our subjects (n=29) had at least one endocrinopathy present with 16 patients (55%) having three or more (≥3) endocrinopathies. Hypogonadism was the most prevalent followed by osteoporosis and growth failure (less than 3rd centile) with a frequency of 16/29 (55%), 14/29 (48%) and 10/29 (35%) patients respectively. Those with more endocrinopathies (≥3) had a longer duration of transfusion therapy when compared with those with fewer endocrinopathies. CONCLUSION: A summary of our clinical guidelines, which have been used to monitor and manage these complications, is presented along with a discussion on the results and pathophysiology of the associated endocrinopathies.
BACKGROUND: Thalassaemia major is a common and serious medical problem worldwide that is associated with a range of complications, including effects on multiple endocrine pathways. Minimizing or preventing comorbidities is important for these individuals who need life-long multidisciplinary care and treatment. However, there are limited overviews of the endocrine complications associated with this illness, nor any consensus regarding management guidelines. METHOD: A retrospective cohort analysis of β-thalassaemia patients attending an ambulatory transfusion clinic at Royal Prince Alfred Hospital was conducted from June 2008. RESULTS: All of our subjects (n=29) had at least one endocrinopathy present with 16 patients (55%) having three or more (≥3) endocrinopathies. Hypogonadism was the most prevalent followed by osteoporosis and growth failure (less than 3rd centile) with a frequency of 16/29 (55%), 14/29 (48%) and 10/29 (35%) patients respectively. Those with more endocrinopathies (≥3) had a longer duration of transfusion therapy when compared with those with fewer endocrinopathies. CONCLUSION: A summary of our clinical guidelines, which have been used to monitor and manage these complications, is presented along with a discussion on the results and pathophysiology of the associated endocrinopathies.
Authors: Robert W Grady; Renzo Galanello; Rachel E Randolph; Dorothy A Kleinert; Carlo Dessi; Patricia J Giardina Journal: Haematologica Date: 2012-08-08 Impact factor: 9.941
Authors: Mohamad H Qari; Yasser Wali; Muneer H Albagshi; Mohammad Alshahrani; Azzah Alzahrani; Ibrahim A Alhijji; Abdulkareem Almomen; Abdullah Aljefri; Hussain H Al Saeed; Shaker Abdullah; Ahmad Al Rustumani; Khoutir Mahour; Shaker A Mousa Journal: Orphanet J Rare Dis Date: 2013-09-17 Impact factor: 4.123