Body Composition in Pediatric Patients With Thalassemia Major

Официальное название

Body Composition in Pediatric Patients With Thalassemia Major Attending Assiut University Children's Hospital

Обзор

To assess body composition (fat mass, lean mass, and bone mass) in pediatric patients with beta thalassemia major using appropriate tools (e.g.,Bioelectrical impedance analysis (BIA)). To evaluate the association between body composition and clinical parameters such as transfusion frequency, nutritional status, serum ferritin levels, endocrine complications.

Подробное описание

Thalassemias are severe inherited anemias characterized by microcytic, hypochromic, and short-lived red blood cells due to defective hemoglobin synthesis(1) β-thalassemias are a heterogeneous group of hereditary hemoglobinopathies characterized by defects in the β-globin chain of hemoglobin and belong to autosomal recessive disorders(2) Three clinical and hematological conditions of increasing severity are recognized and include beta-thalassemia carrier state, thalassemia intermedia, and thalassemia major (3, 4).

Regular blood transfusion is a vital treatment in β-thalassemia major patients to prevent the consequences of anemia. Iron overloading is frequently observed in β-thalassemia major patients with transfusion therapy . Excessive iron can cause multiple organ damage (5, 6, 7, 8).

Patients with β-thalassemia major (BTM) suffer from several patterns of growth retardation including short stature, underweight, wasting, and decreased bone density(9, 10).

Impaired nutritional status has been commonly observed in patients with thalassemia major. Specific nutritional deficiencies, such as vitamin D deficiency, are also often observed due to increased losses of micronutrients or increased endogenous requirements (11).

Patients suffering from thalassemia major have impaired body composition, especially whole-body lean mass and bone mineral density(12, 13). Body composition is influenced by many parameters including age, gender, endocrine system status, nutrition, and exercise (14). All the above parameters are impaired in patients with thalassemia major in addition to hyperactivity of bone marrow, iron overload, and diminished bone mineral density (15, 16).

Patients with thalassemia tend to have increased adiposity(17, 18). These alterations in body composition are more frequently seen in thalassemic patients compared to the general population and are related to the high prevalence of sarcopenic obesity (19).

Bioelectrical impedance analysis (BIA), a technique using electrical properties to analyze body composition. Using BIA provides a more accurate and non-invasive assessment of muscle mass(20).

Body composition in pediatric patients with beta\_thalassemia major has not been well studied, especially in Egypt.There is a lack of sufficient data addressing changes in muscle mass, fat distribution, and their metabolic consequences in this group of patients.

This study will provide new insights into body composition profile of thalassemic children in Assisut University Children's Hospital.The results are expected to fill an important gap in knowledge, support early detection of abnormalities, and contribute to better strategies for management and prevention of long\_term complications.

Первичные конечные точки

• Body composition parameters (Fat Mass, Fat Free Mass) [Срок оценки: Assessed at baseline and every 6 months for up to 12 months.]

Вторичные конечные точки (1)

• Association between transfusion frequency, nutritional status, serum ferritin, endocrine complications and body composition. [Срок оценки: Measured at baseline and every 6 months for up to 12 months]

Критерии участия

Критерии включения

• Children aged 5-18 years
• Diagnosed with beta thalassemia major
• Receiving regular transfusions
• Informed consent/assent provided.

Критерии исключения

• Presence of other chronic systemic diseases
• On medications that significantly affect body composition (e.g., steroids)
• Severe physical or cognitive disability interfering with assessment

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Другое

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Публикации

• Kuriyan R. Body composition techniques. Indian J Med Res. 2018 Nov;148(5):648-658. doi: 10.4103/ijmr.IJMR_1777_18. PMID 30666990
• Visser M, van Venrooij LM, Vulperhorst L, de Vos R, Wisselink W, van Leeuwen PA, de Mol BA. Sarcopenic obesity is associated with adverse clinical outcome after cardiac surgery. Nutr Metab Cardiovasc Dis. 2013 Jun;23(6):511-8. doi: 10.1016/j.numecd.2011.12.001. Epub 2012 Mar 6. PMID 22397879
• Wong P, Fuller PJ, Gillespie MT, Kartsogiannis V, Milat F, Bowden DK, Strauss BJ. The effect of gonadal status on body composition and bone mineral density in transfusion-dependent thalassemia. Osteoporos Int. 2014 Feb;25(2):597-604. doi: 10.1007/s00198-013-2454-y. Epub 2013 Aug 1. PMID 23903954
• Fung EB, Xu Y, Kwiatkowski JL, Vogiatzi MG, Neufeld E, Olivieri N, Vichinsky EP, Giardina PJ; Thalassemia Clinical Research Network. Relationship between chronic transfusion therapy and body composition in subjects with thalassemia. J Pediatr. 2010 Oct;157(4):641-7, 647.e1-2. doi: 10.1016/j.jpeds.2010.04.064. Epub 2010 Jun 12. PMID 20547400
• Katz S, Weinerman S. Osteoporosis and gastrointestinal disease. Gastroenterol Hepatol (N Y). 2010 Aug;6(8):506-17. PMID 20978554
• Casale M, Citarella S, Filosa A, De Michele E, Palmieri F, Ragozzino A, Amendola G, Pugliese U, Tartaglione I, Della Rocca F, Cinque P, Nobili B, Perrotta S. Endocrine function and bone disease during long-term chelation therapy with deferasirox in patients with beta-thalassemia major. Am J Hematol. 2014 Dec;89(12):1102-6. doi: 10.1002/ajh.23844. Epub 2014 Sep 26. PMID 25197009
• De Sanctis V, Soliman AT, Elsedfy H, Skordis N, Kattamis C, Angastiniotis M, Karimi M, Yassin MA, El Awwa A, Stoeva I, Raiola G, Galati MC, Bedair EM, Fiscina B, El Kholy M. 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 Jan;17(1):8-18. doi: 10.4103/2230-82 PMID 23776848
• Vlychou M, Alexiou E, Thriskos P, Fezoulidis I, Vassiou K. Body Composition in Adult Patients with Thalassemia Major. Int J Endocrinol. 2016;2016:6218437. doi: 10.1155/2016/6218437. Epub 2016 Nov 10. PMID 27956899

Идентификаторы

NCT: NCT07204015 · Body Composition,Thalassemia

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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