Clinical trial NCT03161899: Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital
Official title: Evaluation of Nutritional Status in Patients With Thalassemia Major in Assiut University Children Hospital
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Thalassemia Major
Keywords: thalassemia - nutrition -growth
Lead sponsor: Assiut University
Sponsor class: OTHER
First posted: 2017-05-22
Enrollment: 246
Sex: ALL
Minimum age (years): 6
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: all beta thalassemic patients attending the children Hematology Clinic, Children Hospital, Assiut University, during the period of 1st of June 2017 till 31 th of may 2018.

Brief summary: Thalassemia is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha \& Beta thalassemia. Alpha thalassemia: occurs when a gene or genes related to the alpha globin protein are missing or mutated.

Beta-thalassemia syndromes are a group of hereditary blood disorders characterized by reduced or absent beta globin chain synthesis. Beta-thalassemias can be classified into:

Silent carrier: completely asymptomatic with normal hematological parameters. Beta-thalassaemia minor (beta-thalassaemia trait): usually asymptomatic; diagnosis is made during a work-up for mild anemia.

Beta-thalassaemia intermedia: usually a similar presentation to beta-thalassaemia major; symptoms are usually less pronounced and the course is usually more insidious.

Beta-thalassaemia major : In which there is complete absence of hemoglobin A

Detailed description: In Egypt beta thalassemia-major is the most common type with carrier rate of 5.3 to ≥9%and 1000 new cases born with beta-thalassemia major per 1.5 million live births per year.

Children born with thalassemia major are normal at birth, but develop severe hemolytic anemia during the first year of life. Symptoms are those of anemia (lethargy, poor feeding, pallor...etc.) failure to thrive and organomegaly. Later on they develop signs of extra medullary hematopoiesis .

Optimal nutritional status is important for growth, immune function, bone health and pubertal development . Various reports suggest the incidence of poor growth ranges from 25% to 75% depending on thalassemia syndrome and severity of disease. This marked growth deficits raise a red ﬂag for any pediatrician to evaluate nutritional status of thalassemic patients and detect possible nutritional deficiencies and associated factors.

Etiology of delayed growth and malnutrition reported in thalassemic patient is multifactorial including :

1. Nutritional deprivation with or without feeding difficulties arising from fatigue and breathlessness
2. Increased energy expenditure secondary to hyper metabolism with or without heart failure
3. Gastrointestinal hypoxia which consequently produces anorexia and malabsorption
4. Reduction of biosynthetic activity of liver.
5. Disturbance of the endocrine function
6. Impaired synthetic hepatic function secondary to hemosiderosis and hepatitis.

The main aim of this study is to assess the nutritional status of thalassemic patient attending Assiut university Children hospital and to determine the factors involved.

Eligibility criteria: Inclusion Criteria:

* patients diagnosed to have thalassemia

Exclusion Criteria:

* patient with any underlaying systemic diseases other than B thalassemia major.

Primary outcomes:
measure: percentage of malnutrition
timeFrame: 12 month
Secondary outcomes:
measure: aetiology poor growth
timeFrame: 12 month
measure: decrease morbidity
timeFrame: 18 month
Other outcomes:
Locations:
city: Assiut
country: Egypt
facility: Asmaa Refaat Abdelmonem
Study officials:
affiliation: Assuit , faculty of medicine, egypt
name: asmaa refaat, MBBCH
role: PRINCIPAL_INVESTIGATOR
References:
citation: Soliman AT, El-Matary W, Fattah MM, Nasr IS, El Alaily RK, Thabet MA. The effect of high-calorie diet on nutritional parameters of children with beta-thalassaemia major. Clin Nutr. 2004 Oct;23(5):1153-8. doi: 10.1016/j.clnu.2004.03.001.
citation: Fung EB. Nutritional deficiencies in patients with thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:188-96. doi: 10.1111/j.1749-6632.2010.05578.x.
citation: Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010 May 21;5:11. doi: 10.1186/1750-1172-5-11.