Endocrine Complications in Children with Transfusion Dependent Thalassemia
Article Sidebar
Main Article Content
Abstract
Background: Thalassemia is a common inherited disorder that results in anemia. Regular blood transfusion and adequate iron chelation increase life expectancy. However, endocrine complications have been more common and contribute to morbidity in these patients.
Objective: To determine the endocrine complications in transfusion dependent thalassemia (TDT) patients and to evaluate the factors affecting these complications
Methods: A retrospective cohort study was conducted. TDT patients aged 1 to 15 years, who were followed up at Surin Hospital between 1 January 2018 to 31 December 2019 were reviewed.
Results: 46 TDT patients, aged between 1.8 to 14.8 years, were evaluated. The endocrine complications were short stature 15.2 % and low bone mineral density (BMD) 39.1 %. Hypothyroidism, delayed puberty and diabetes mellitus or impaired fasting glucose were not found. Factors associated with low BMD were iron chelation (OR 10.13, 95% CI: 1.11-92.42, p= 0.040), age (OR 1.13, 95% CI: 1.18-1.80, p<0.001) and duration of disease (OR 1.28, 95% CI: 1.02-1.59, p=0.029). There was no significant factor associated with short stature.
Conclusion: Endocrine complications in TDT patients can be found during the childhood period and increase with age. Therefore, regular endocrine evaluation is necessary for early diagnosis and proper management of associated complications to improve the quality of life of these patients.
Keywords: Transfusion dependent thalassemia, endocrine complications, short stature, low bone mineral density
Article Details
References
สถาบันสุขภาพแห่งชาติมหาราชินี. แนวทางการวินิจฉัยและการรักษาโลหิตจางธาลัสซีเมีย: พีเอลีฟวิ่ง; 2557.
Amid A, Saliba AN, Taher AT, Klaassen RJ. Thalassaemia in children: from quality of care to quality of life. Arch Dis Child 2015;100(11):1051-7.
Fucharoen S, Winichagoon P. Haemoglobinopathies in southeast Asia. Indian J Med Res 2011;134(4):498-506.
Cappellini M-D, Cohen A, Porter J, Taher A, Viprakasit V. Guidelines for the management of transfusion dependent thalassaemia (TDT): Thalassaemia International Federation Nicosia, Cyprus; 2014.
Borgna-Pignatti C, Cappellini MD, De Stefano P, Del Vecchio GC, Forni GL, Gamberini MR, et al. Survival and complications in thalassemia. Ann N Y Acad Sci 2005;1054:40-7.
Borgna-Pignatti C, Rugolotto S, De Stefano P, Zhao H, Cappellini MD, Del Vecchio GC, et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica 2004;89(10):1187-93.
Teawtrakul N, Jetsrisuparb A, Pongudom S, Sirijerachai C, Chansung K, Wanitpongpun C, et al. Epidemiologic study of major complications in adolescent and adult patients with thalassemia in Northeastern Thailand: the E-SAAN study phase I. Hematology 2018;23(1):55-60.
Tufekci O, Atabay B, Turker M, Yilmaz Bengoa S, Gozmen S, Karapinar T, et al. Evaluation of the Clinical and Laboratory Characteristics of Previously Followed-up Thalassemia Intermedia Patients to Provide Them Better Care in the Future. J Pediatr Hematol Oncol 2017;39(6):440-4.
Borgna-Pignatti C, Gamberini MR. Complications of thalassemia major and their treatment. Expert Rev Hematol 2011;4(3):353-66.
De Sanctis V, Soliman AT, Elsedfy H, Skordis N, Kattamis C, Angastiniotis M, et al. Growth and endocrine disorders in thalassemia: The international network on endocrine complications in thalassemia (I-CET) position statement and guidelines. Indian J Endocrinol Metab 2013;17(1):8-18.
Isik P, Yarali N, Tavil B, Demirel F, Karacam GB, Sac RU, et al. Endocrinopathies in Turkish children with Beta thalassemia major: results from a single center study. Pediatr Hematol Oncol 2014;31(7):607-15.
Merchant RH, Shirodkar A, Ahmed J. Evaluation of growth, puberty and endocrine dysfunctions in relation to iron overload in multi transfused Indian thalassemia patients. Indian J Pediatr 2011;78(6):679-83.
Shamshirsaz AA, Bekheirnia MR, Kamgar M, Pourzahedgilani N, Bouzari N, Habibzadeh M, et al. Metabolic and endocrinologic complications in beta-thalassemia major: a multicenter study in Tehran. BMC Endocr Disord 2003;3(1):4.
Sharma R, Seth A, Chandra J, Gohain S, Kapoor S, Singh P, et al. Endocrinopathies in adolescents with thalassaemia major receiving oral iron chelation therapy. Paediatr Int Child Health 2016;36(1):22-7.
Belhoul KM, Bakir ML, Saned MS, Kadhim AM, Musallam KM, Taher AT. Serum ferritin levels and endocrinopathy in medically treated patients with beta thalassemia major. Ann Hematol 2012;91(7):1107-14.
Shalitin S, Carmi D, Weintrob N, Phillip M, Miskin H, Kornreich L, et al. Serum ferritin level as a predictor of impaired growth and puberty in thalassemia major patients. Eur J Haematol 2005;74(2):93-100.
Tanner JM, Whitehouse RH. Clinical longitudinal standards for height, weight, height velocity, weight velocity, and stages of puberty. Arch Dis Child 1976;51(3):170-9.
Prader A. Testicular size: assessment and clinical importance. Triangle 1966;7(6):240-3.
กรมอนามัย กระทรวงสาธารณสุข. เกณฑ์อ้างอิงน้ำหนัก ส่วนสูง เครื่องชี้วัดภาวะโภชนาการของประชาชนไทย อายุ 1 วัน - 19 ปี 2542. นนทบุรี : กรมอนามัย กระทรวงสาธารณสุข; 2542.
Cunningham MJ, Macklin EA, Neufeld EJ, Cohen AR, Thalassemia Clinical Research N. Complications of beta-thalassemia major in North America. Blood 2004;104(1):34-9.
De Sanctis V, Elsedfy H, Soliman AT, Elhakim IZ, Soliman NA, Elalaily R, et al. Endocrine profile of beta-thalassemia major patients followed from childhood to advanced adulthood in a tertiary care center. Indian J Endocrinol Metab 2016;20(4):451-9.
Altincik A, Akin M. Prevalence of Endocrinopathies in Turkish Children With beta-Thalassemia Major: A Single-Center Study. J Pediatr Hematol Oncol 2016;38(5):389-93.
Soliman AT, Khalafallah H, Ashour R. Growth and factors affecting it in thalassemia major. Hemoglobin. 2009;33 Suppl 1:S116-26.
Vogiatzi MG, Macklin EA, Trachtenberg FL, Fung EB, Cheung AM, Vichinsky E, et al. Differences in the prevalence of growth, endocrine and vitamin D abnormalities among the various thalassaemia syndromes in North America. Br J Haematol 2009;146(5):546-56.
Soliman AT, elZalabany M, Amer M, Ansari BM. Growth and pubertal development in transfusion-dependent children and adolescents with thalassaemia major and sickle cell disease: a comparative study. J Trop Pediatr 1999;45(1):23-30.
Tan KA, Lum SH, Yahya A, Krishnan S, Jalaludin MY, Lee WS. Prevalence of growth and endocrine disorders in Malaysian children with transfusion-dependent thalassaemia. Singapore Med J. 2019;60(6):303-8.
Nokeaingtong K, Charoenkwan P, Silvilairat S, Saekho S, Pongprot Y, Dejkhamron P. A Longitudinal Study of Growth and Relation With Anemia and Iron Overload in Pediatric Patients With Transfusion-dependent Thalassemia. J Pediatr Hematol Oncol 2016;38(6):457-62.
Najafipour F, Aliasgarzadeh A, Aghamohamadzadeh N, Bahrami A, Mobasri M, Niafar M, et al. A cross-sectional study of metabolic and endocrine complications in beta-thalassemia major. Ann Saudi Med 2008;28(5):361-6.
Low LC. Growth, puberty and endocrine function in beta-thalassaemia major. J Pediatr Endocrinol Metab 1997;10(2):175-84.
Delvecchio M, Cavallo L. Growth and endocrine function in thalassemia major in childhood and adolescence. J Endocrinol Invest 2010;33(1):61-8.
De Sanctis V, Katz M, Vullo C, Bagni B, Ughi M, Wonke B. Effect of different treatment regimes on linear growth and final height in beta-thalassaemia major. Clin Endocrinol (Oxf) 1994;40(6):791-8.
Vogiatzi MG, Macklin EA, Fung EB, Cheung AM, Vichinsky E, Olivieri N, et al. Bone disease in thalassemia: a frequent and still unresolved problem. J Bone Miner Res 2009;24(3):543-57.
Vogiatzi MG, Autio KA, Schneider R, Giardina PJ. Low bone mass in prepubertal children with thalassemia major: insights into the pathogenesis of low bone mass in thalassemia. J Pediatr Endocrinol Metab 2004;17(10):1415-21.
Shamshirsaz AA, Bekheirnia MR, Kamgar M, Pakbaz Z, Tabatabaie SM, Bouzari N, et al. Bone mineral density in Iranian adolescents and young adults with beta-thalassemia major. Pediatr Hematol Oncol 2007;24(7):469-79.
