|Year : 2014 | Volume
| Issue : 4 | Page : 222-226
Quality of life of Egyptian b-thalassemia major children and adolescents
Mohsen S Elalfy1, Maisa N Farid2, Jonair H Labib MD, PhD 1, Heba K RezkAllah3
1 Department of Pediatrics, Thalassemia Center, Ain Shams University, Cairo, Egypt
2 Department of Pediatrics, Institute of Postgraduate Childhood Studies, Ain Shams University, Cairo, Egypt
3 Department of Pediatrics, Sohag General Hospital, Sohag, Egypt
|Date of Submission||04-Dec-2014|
|Date of Acceptance||06-Dec-2014|
|Date of Web Publication||25-Mar-2015|
Jonair H Labib
Department of Pediatrics, Thalassemia Center, Ain Shams University, Cairo 11566
Source of Support: None, Conflict of Interest: None
Introduction b-Thalassemia major (B-TM) is a serious health problem in which children are in need of regular blood transfusions from a very young age to survive. They also need to receive iron chelation therapy to remove excess iron from their bodies, which imposes serious risk on their health and quality of life (QOL). Hence, this study was designed to assess the QOL of Egyptian B-TM children and adolescents in comparison with their healthy peers.
Materials and methods A total of 127 B-TM patients and 65 healthy volunteers were enrolled and interviewed at the Ain Shams University Thalassemia Center. QOL assessment was made using the Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scale.
Results The controls had higher QOL scores in all domains at the start of the study (P<0.0001). Compliant patients had higher total QOL scores (P = 0.004). High pretransfusion hemoglobin levels and low serum ferritin levels were independent predictors of better QOL scores.
Conclusion B-TM patients had a poor QOL; high hemoglobin level and low iron overload were associated with improved QOL scores. Egyptian J Haematol 39:-0 © 2014 The Egyptian Society of Haematology.
Keywords: β-thalassemia, iron chelation, quality of life
|How to cite this article:|
Elalfy MS, Farid MN, Labib JH, RezkAllah HK. Quality of life of Egyptian b-thalassemia major children and adolescents. Egypt J Haematol 2014;39:222-6
|How to cite this URL:|
Elalfy MS, Farid MN, Labib JH, RezkAllah HK. Quality of life of Egyptian b-thalassemia major children and adolescents. Egypt J Haematol [serial online] 2014 [cited 2019 Apr 18];39:222-6. Available from: http://www.ehj.eg.net/text.asp?2014/39/4/222/153963
| Introduction|| |
Children with b-thalassemia major (B-TM) are in need of regular blood transfusion from a very young age . They also have to receive iron chelation therapy (ICT) to remove excess iron from their bodies, which imposes serious consequences on their hearts ,. Advances in the treatment options for B-TM patients have led to longer life expectancies for them, increasing their chances for continuing education, advancing their career, and having a family ,. However, there is limited research on their (QOL), especially during childhood . Hence, this study aimed to assess QOL in Egyptian B-TM children and adolescents in comparison with their healthy peers.
| Materials and methods|| |
This case-control study was conducted in the Hematology Clinic, Children's Hospital, Ain Shams University. The inclusion criteria for enrollment of patients were diagnosis of B-TM, age older than 5 and less than 18 years, their willingness and their guardians' willingness to participate in the study, and regular follow-up at the center. The exclusion criteria were marked cognitive impairment, uncompensated organ failure, and severe psychological disorders. There were 127 B-TM patients (72 boys and 55 girls) in addition to 65 age-matched and sex-matched healthy volunteers (37 boys and 28 girls), who were of the same social class as the enrolled patients. The study complied with the World Medical Association Declaration of Helsinki of 1975, as revised in 2000, regarding ethical conduct of research involving human participants. It was accepted in August 2011 by the Pediatric Ethical Committee of Ain Shams University. Before participation in the study, consent was obtained from patients, controls, and/or their guardians after being informed of the study objectives and ensuring data confidentiality.
The enrollment started in September 2011 and continued for 6 months, during which time patients were interviewed sequentially on their scheduled appointments at the Hematology Clinic. Thereafter, the controls were interviewed in another outpatient facility. For every patient, full medical history was recorded, particularly age, sex, weight, height, BMI, date of attaining puberty, complications from iron chelation, and patient compliance. Compliance was categorized into four categories according to the percentage of the actual chelator dose taken: highly compliant category if the patient was taking more than 90% of the required dose, moderately compliant (51-90%), poorly compliant (1-51%), and noncompliant (0%) . In addition, there was documentation of the patients' and parents' education, and the laboratory results of the pretransfusion hemoglobin (Hb), serum ferritin (SF), C-reactive protein, and serum alanine transaminase levels. QOL assessment was made in the same setting through patient interviews.
QOL assessment tool
Assessment was made using the Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scale, provided by MAPI Research Institute in Lyon, France, after signing a user agreement with them. The PedsQ 4.0 Generic Core Scale contained four dimensions: three functional (physical, social, and school functioning) and one about emotional well-being. The Arabic forms used was provided by MAPI Research Institute. Forms included parallel child self-reports (age ranges 5-7, 8-12, and 13-18 years) during a standard recall period of the month before the study. Item responses were measured on a five-point rating scale ranging from 0 (almost always a problem) to 100 (never a problem).The mean domain score was computed as the sum of all item scores divided by the number of items answered in that domain. The mean total scale score was computed as the sum of all item scores divided by the number of items answered on all the scales, so that higher scores indicated better QOL.
Data were coded and processed using statistical program for social science (SPSS, Chicago, IL, USA), version 17, 2008. Data analysis was carried out using the c2 -test, analysis of variance test (F-test), Wilcoxon ranked-sum test (Z-test), Kruskal-Wallis test, and Pearson's correlation test, where appropriate. Logistic regression analysis was applied to identify independent predictors of high QOL scores among all studied factors. P-value was considered significant if P was 0.05.
| Results|| |
The demographic and clinical data of thalassemia patients and healthy controls are shown in [Table 1]. The healthy controls had statistically comparable level of education, but the mean weight, height, and BMI were significantly higher than that in the patient group. Among the B-TM patients, 62 (48.8%) were splenectomized and 22 (41.5%) were pubertal. The mean PedsQL scores of the case and control groups are shown in [Table 2]. The controls had higher QOL scores in all domains (P<0.0001). The order of the affected domains, according to severity, was as follows: physical functioning (the most severely affected), followed by school, social, and emotional functioning domains, respectively.
Among patients, there was no significant QOL difference between boys and girls as regards the total and individual domain scores. Upon splitting patients into a compliant group (n = 73, 57.5%) and a non/poorly compliant group (n = 54, 42.5%), the percentage of patients treated with oral deferiprone (DFP) and deferasirox (DFX) was found to be higher in the compliant group (66.66 and 21.74% compared with 44.83 and 7.25%, respectively, with P = 0.006). Compliant patients had higher total QOL scores compared with noncompliant patients (P = 0.004). Parental level of education was not statistically different between the two groups.
The comparison between patient groups, according to the type of their iron chelator, revealed that those receiving oral DFX had the lowest mean SF level followed by the DFP group. The former group had the highest scores in the physical, school, and total domains [Table 3].
|Table 3: Comparison of four groups of patients according to the iron chelator received|
Click here to view
Logistic regression analysis revealed that high pretransfusion Hb and low SF were significant independent predictors of better QOL scores (≥70) among all studied factors [Table 4].
| Discussion|| |
B-TM is a serious health problem that may lead to early death in the absence of blood transfusion. When children survive with the help of regular blood transfusion starting from infancy, they become susceptible to complications arising from iron overload and blood transfusions since a very young age. Therefore, the assessment of QOL in B-TM children is particularly important.
A comparison of Egyptian B-TM patients with their healthy peers revealed that the former reported lower growth parameters, as well as lower physical, social, emotional, school functioning, and total QOL scores. Lower growth parameters and QOL scores in B-TM patients are recognized observations in previous studies , that can be attributed to the chronic anemic state and/or the consequences of iron overload.
Physical functioning was the most severely affected domain in relation to healthy controls. This was probably because many patients did not come regularly for blood transfusion and thus were suffering from anemic consequences that would adversely impact their physical activities and limit their exercise capacity. The presence of physical complications may also explain the worst scores in that domain ,. School functioning scores were better than the physical domain scores as patients attended the clinic for blood transfusion on Saturdays that were the schools' day off. This was in contrast with other the results of researchers who found that school functioning scored the lowest among QOL domains because of frequent absenteeism from school for hospital visits and lack of energy when performing academic activities ,, which negatively affected the total QOL score ,. School functioning as the second affected domain following physical functioning reflected the difficulty that young B-TM patients face in their education . Regarding the social functioning domain, the majority of Egyptian thalassemia children had no problems in getting along with other kids or being a member of a play team. This may be due to the absence of evident disease complications at a young age, resulting in decreased feeling of stigmatization. However, a significant difference remained between them and controls. The emotional QOL was probably affected because patients felt different from their peers and expressed negative thoughts about their lives and felt sadness, anger, and hurt toward their chronic illness. Children might develop psychological and emotional difficulties as early as the toddler stage. They seek the ability to satisfy some of their needs on their own and develop a sense of autonomy, but if caregivers refuse to let them perform these tasks because of their illness they may develop shame and doubt about their ability to handle problems . B-TM children, as they grew older, were becoming more aware of themselves as individuals, and more responsible toward themselves and their illness. They were becoming more reasonable to share and cooperate, but the disease prevented them from being more productive and a sense of inferiority might develop instead and lower their emotional domain scores . Moreover, the treatment was emotionally demanding, as transfusion and chelation therapy required repeated invasive procedures and hospital visits .
The sex difference had no significant impact on QOL scores in any of the domains, which is in agreement with other studies ,. In contrast, patient compliance had a great impact on QOL scores, as compliant patients had better total QOL scores compared with the rest of the patients who were noncompliant or poorly compliant. This could be due to the regular blood transfusion and regular ICT intake, which alleviated the anemia and the iron overload, leading to improved total QOL scores ,.
Regarding ICT, the study showed significant differences in all QOL domains among patients receiving different iron chelators; these differences were reported by other scholars . Patients receiving deferroxamine (DFO) had the least scores in physical, social, and school functioning, which might be due to patient noncompliance. Patients receiving DFO were often required to begin treatment at a very young age (2-3 years) and continue using the subcutaneous pump for 8 h a day, 5 days a week, throughout their life. The psychological impact on parents would be quite high as they have to endure the daily task of inserting a needle into their child and bear the constant battles with them in order to comply with this treatment regimen. These battles could start at a very young age and continue through adolescence and negatively affect the child-parent relationship. The DFO treatment regimen may also cause the children to become overdependent on their parents. The unsightly bumps and bruises caused by the subcutaneous infusion mostly affect adolescents and young adults, who reported inability to wear certain clothes and a sense of embarrassment on going outdoors. Local reactions from the DFO infusion were reported to contribute to inadequate compliance in many patients, as well as to higher morbidity and increased costs .
The DFX group had the lowest mean SF among all ICT groups, and had the highest total, physical, and school functioning scores. The difference between the DFP and DFX groups may be due to the more frequent daily doses and side effects of DFP, compared with DFX, which was taken once daily and was devoid of these side effects ,. QOL and patient satisfaction were reported to be better in those receiving oral iron-chelating agents compared with those taking subcutaneous infusions of DFO ,. Other authors stated that, in addition to DFO, DFP also appears to negatively impact QOL, probably because of its side effects .
Among the different factors that were expected to affect the QOL - namely, sex, parental education, growth parameters, laboratory results, combined treatment and patient compliance - only pretransfusion Hb and SF were found by logistic regression analysis to be independent predictors of high QOL scores. Both factors contributed to better physical condition and general health ; thus, each had a significant independent effect on QOL scores.
The main limitation of this study was the patient characteristics: for example, the low Hb level in most of them because of the national shortage of blood units, which may have an impact on QOL assessment. Furthermore, the low educational level and the religious profile of some patients and their guardians might have an unexpected reflection on the emotional and social parameters of QOL assessment. The lack of insight into the disease led to higher satisfaction and acceptance of the illness in some patients, despite the disease seriousness. This patient variability was not taken into account in this study, but further studies that do will be planned.
| Conclusion|| |
B-TM has a negative impact on patients' QOL, especially in the physical functioning domain. Better control of anemia as well as iron overload was associated with better QOL scores.
| Acknowledgements|| |
All authors contributed to concept, design, data acquisition, data analysis, statistical analysis, manuscript preparation, manuscript editing and manuscript review. The manuscript has been read and approved by all the authors, who believe that the manuscript represents honest work.
The authors extend their gratitude to the MAPI Research Institute in Lyon, France, for providing the QOL assessment tool 'Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scale', free of charge for our governmental university.
Conflicts of interest
There are no conflicts of interest.
| References|| |
Vullo R, Modell B, Georganda B. What is thalassemia?
ed. Nicosia, Cyprus: the Thalassemia International Federation; 1995.
Anionwu E, Atkin K. The politics of sickle cell and thalassemia
. Buckingham, UK: Open University Press; 2001.
Levine L, Levine M. Health care transition in thalassemia: pediatric to adult-oriented care. Ann N Y Acad Sci 2010; 1202
Pakbaz Z, Treadwell M, Yamashita R, Quirolo K, Foote D, Quill L, et al.
Quality of life in patients with thalassemia intermedia compared to thalassemia major. Ann N Y Acad Sci 2005; 1054
Baghianimoghadam MH, Sharifirad G, Rahaei Z, Baghianimoghadam, B, Heshmati H. Health related quality of life in children with thalassemia assessed on the basis of SF-20 questionnaire in Yazd, Iran: a case-control study. Cent Eur J Public Health 2011; 19
Lee WS, Toh TH, Chai PF, Soo TL. Self-reported level of and factors influencing the compliance to desferrioxamine therapy in multitransfused thalassemias. J Paediatr Child Health 2011; 47
Ismail A, Campbell MJ, Ibrahim HM, Jones GL. Health related quality of life in Malaysian children with thalassemia. Health Qual Life Outcomes 2006; 2
Cheuk DKL, Mok ASP, Lee ACW, Chiang AKS, Ha SY, Lau YL, et al
. Quality of life in patients with transfusion-dependent thalassemia after hematopoietic SCT. Bone Marrow Transplant 2008; 42
Musallam KM, Khoury B, Abi-Habib R, Bazzi L, Succar J, Halawi R, et al.
Health-related quality of life in adults with transfusion-independent thalassemia intermedia compared to regularly transfused thalassemia major: new insights. Eur J Haematol 2011; 87
Thavorncharoensap M, Torcharus K, Nuchprayoon I, Riewpaiboon A, Indaratna K, Ubol BO. Factors affecting health-related quality of life in Thai children with thalassemia. BMC Blood Disord 2010; 10
Saeed N. My life. United Kingdom Thalassemia Society Matters
. 2004; 99:10.
Cantaan D, Ratip S, Kaptan S, Cosan R. Psychological burden of beta thalassemia major in Antalya, South Turkey. Soc Sci Med 2003; 56
Gharaibeh H, Amarneh BH, Zamzam SZ. The psychological burden of patients with beta thalassemia major in Syria. Pediatr Int
El Dakhakhny AM, Hesham MA, Mohamed SE, Mohammad FN. Quality of life of school age thalassemic children at Zagazig City. J Am Sci
Torcharus K, Pankaew T. Health-related quality of life in thalassemia treated with iron chelation. Royal Thai Army Medl J
Tefler P, Constantinidou G, Andreou P, Christou S, Modell B, Angastiniotis M. Quality of life in thalassemia. Ann N Y Acad Sci
Cianciulli, P. Treatment of iron overload in thalassemia. Pediatr Endocrinol Rev
Delea TE, Edelsberg J, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, et al.
Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion 2007; 47
McLeod C, Fleeman N, Kirkham J, Bagust A, Boland A, Chu P, et al.
Deferasirox for the treatment of iron overload associated with regular blood transfusions (transfusional haemosiderosis) in patients suffering with chronic anaemia: a systematic review and economic evaluation. Health Technol Assess (Rockv) 2009; 13
Elalfy MS, Massoud W, Elsherif NH, Labib JH, Elalfy OM, Elaasar S, et al.
A new tool for the assessment of satisfaction with iron chelation therapy (ICT-Sat) for patients with b-thalassemia major. Pediatr Blood Cancer
Ho PJ, Tay L, Lindeman R, Catley L, Bowden DK. Australian guidelines for the assessment of iron overload and iron chelation in transfusion-dependent thalassemia major, sickle cell disease and other congenital anaemias. Intern Med J 2011; 41
[Table 1], [Table 2], [Table 3], [Table 4]