Impact of educational programme regarding chelation therapy on the quality of life for B-thalassemia major children

Abstract

Objectives

Thalassemia is the most common genetic disorder in Egypt, with an estimated carrier rate of 9–10%. It is a genetic blood disorder which can be fatal if proper chelation is not received. The introduction of chelating agents capable of removing excessive iron from the body has dramatically increased life expectancy and improved the overall quality of life. The aim of this study was to assess the impact of educational programmes regarding chelation therapy on the quality of life of thalassemic children.

Methods

The study was carried out at the Mansoura University Children's Hospital in the period between March 2010 and May 2011. It included 173 B-thalassemia children (84 boys and 89 girls) with age ranging between 8–18 years. The researcher used a predesigned interviewing questionnaire to collect data regarding children's knowledge about thalassemia and its management, especially regarding chelation therapy. The paediatric quality-of-life inventory tool (Peds QL 4.0 generic core) was also used to assess the studied children's quality of life.

Results

There was a significant statistical difference of the studied children's knowledge regarding chelation therapy and their quality of life.

Conclusion

There was a positive effect of the educational programme in improving children's knowledge score and their quality of life. Application of educational programmes for thalassemic children and their nurses regarding chelation therapy and its importance in preventing thalassemia complications is established.

Keywords:

• Thalassemia
• Children
• Programme
• Chelation
• Quality of life
• Nurse

Introduction

Thalassemia is a group of inherited autosomal recessive blood disorders that originated in the Mediterranean region. The genetic defect, which could be either mutation or deletion, results in reduced rate of synthesis or no synthesis of one of the globin chains that make up haemoglobin. This can cause the formation of abnormal haemoglobin molecules, thus causing anaemia, the characteristic presenting symptom.¹ Thalassemias are classified according to the chain of the haemoglobin molecule that is affected. In α thalassemia, production of the α globin chain is affected, while in B thalassemia, production of the B globin chain is affected. The B globin chains are encoded by a single gene on chromosome 11; the α globin chains are encoded by two closely linked genes on chromosome 16.² The two major forms of the disease, alpha and beta, are prevalent in discrete geographical clusters around the world and they were associated with malarial endemicity in ancient times. Beta thalassemia is particularly prevalent among the Mediterranean people, and this geographical association is responsible for its naming. In Europe, the highest concentration of the disease is found in Greece and the coastal regions in Turkey.³

Β thalassemia major is a common health problem in Egypt; it has been estimated that 1000 children with B thalassemia are born annually for every 1.5 million live births. The carrier's rate of B thalassemia in Egypt was reported to be 9–10%.⁴ The intensive iron chelation therapy currently in use significantly increases the life span and improves the quality of life (QOL) of individuals with thalassemia.^5–7

A recent literature review revealed that few studies have focused on health-related QOL in patients with iron overload. Optimum chelation therapy reduces iron overload complications and provides a better QOL.^8,9

A nurse can fulfil the essential function of educating young patients about chelation therapy by stressing that with proper chelation therapy a child can grow, develop and live a normal life. The nurse has an opportunity to exchange information, and helps to decrease tensions and concerns about the treatment and develops an atmosphere of familiarity and routine in chelation therapy.¹⁰ The aim of this study is to assess the impact of educational programmes regarding chelation therapy on the QOL for children with B thalassemia.

Patients and methods

This is a quasi-experimental study conducted at the Mansoura University Children's Hospital (Hematology/Oncology Unit) in the period between March 2010 and May 2011.

By using epidemiology information programme 2000, statistical calculation division at power 80% and confidence interval 95%, the sample size was estimated to be 173 cases who fulfil the following criteria:

1.	Age: 8–18 years regardless of their gender, residence and educational level.
2.	Confirmed diagnosis of B-thalassemia major.
3.	Regular attendance for follow-up.

Exclusion criteria

Children having physical or mental chronic illness.

Ethical consideration

•	Ethical approval was obtained from the Research Ethics Committee of Faculty of Nursing, Mansoura University.
•	Written consent was obtained from the children studied and their caregivers.
•	The children studied were assured that the study is harmless, and that all the gathered data will be treated with confidentiality for research purpose and they are allowed to withdraw from the study whenever they want.

Technical design

Tools and technique of data collection

The data were collected by:

Interview questionnaire (pre-educational and post-educational programme)

This questionnaire was developed by the researcher after reviewing related literature in Arabic language. The validity and reliability were analysed. Each thalassemic child was interviewed individually to collect data related to their knowledge about thalassemia and its management, especially regarding chelation therapy. This tool comprised the following features:

1.	Socio-demographic characteristics of the children and their parents such as age, level of education, monthly income and family history of the disease.
2.	Children's knowledge and their reported practices about B-thalassemia and chelation therapy.

Scoring system for the studied children's knowledge about B-thalassemia and chelation therapy:

A total score of 50 grades was allocated for items of the interviewing questionnaire. The answers of the thalassemic children were evaluated using the model key answer sheet which was prepared by the researcher and graded as follows:

•	Good knowledge: >75 score
•	Average knowledge: 50–75 score
•	Poor knowledge: <50 score

Paediatric quality-of-life inventory tool (Peds QL 4.0 Generic Core)

This tool was developed by Varni (1998)¹¹ and the permission for using this tool was obtained from the author by signing the user agreement from MAPI Research Institute, Lyon, France. An English and an Arabic copy of the tool was obtained from the author. The validity and reliability of this tool were analysed by the author as it is an international tool.

The QOL tool integrates generic core scales which were designed to measure the core dimensions of physical functioning, emotional functioning, social functioning and school functioning. Each dimension consists of two reports: child self-report and parent-proxy report for the specific age group of children. Both reports will include questions about health-related problems in each dimension. The score for the questions in the four major dimensions was graded on the 5-point Likert scale from 0 (Never) to 4.

Scoring of dimensions:

Item scaling:

Scoring procedure:

Step 1: Transform score

Items are reverse scored and linearly transformed to a 0–100 scale as follows:

0 = 100, 1 = 75, 2 = 50, 3 = 25, and 4 = 0.

Step 2: Calculate scores

Score by dimensions:

•	If more than 50% of the items in the scale are missing, the scale scores should not be computed.
•	Mean score = Sum of the items over the number of items answered.

Total score: Sum of all the items over the number of items answered on all the scales. According to the total score of each child, the QOL was classified as follows:

• Low QOL: <50% of total score

• Moderate QOL: 50–75% of total score

• High QOL: >75% of total score

To assess the impact of health teaching programmes regarding chelation therapy on the QOL for thalassemic children.

Specific objectives:

1.	Define the meaning of thalassemia.
2.	Explain the prevalence of thalassemia.
3.	Explain the pathophysiology of thalassemia.
4.	Enumerate the types of thalassemia.
5.	List the clinical manifestations of thalassemia.
6.	Determine the complications of thalassemia.
7.	Identify methods of treatment of thalassemia.
8.	Identify the importance of blood transfusion.
9.	Recognise the possible signs and symptoms for blood reaction.
10.	Realise the benefit of splenectomy.
11.	Determine the importance of bone marrow transplantation.
12.	Identify the importance of chelation therapy.
13.	List the indications of chelation therapy.
14.	Determine the possible side effects of chelation therapy.
15.	List the types of chelation therapy.
16.	List the types of investigations that should be performed before starting chelation therapy.
17.	Recognise the benefits and functions of desferal.
18.	Enumerate the proper care for possible desferal reaction.
19.	Identify the process of desferal injection.

The contents of the programme consist of knowledge regarding:

•	Thalassemia: Definition, causes, prevalence, pathophysiology, types, clinical manifestations and complications.
•	Management of thalassemia: Blood transfusion, splenectomy and bone marrow transplantation.
•	Chelation therapy: Importance, function, indications, possible side effects, types, proper use, and injection process.

The programme was submitted in the form of five sessions; each session was 20–30 minutes long according to the physical and mental readiness of the children studied and used different strategies (lectures, demonstrations and group discussions). These children were also divided into groups, 10 children in each group, and they were motivated and rewarded for their active participation during the health teaching period.

Post-test questionnaire will be used immediately after the health teaching programme to determine the children's knowledge regarding chelation therapy.

A QOL inventory tool will be used after the health teaching programme twice at 3 and 6 months to evaluate the effectiveness of the programme on the children's QOL.

A written approval to conduct the study was obtained from the administrators of the study setting in addition to a written consent from the children studied and their parents. A review of literature that is relevant to the research problem including magazines, periodicalsand books to study the questionnaire tool and get acquainted with the previous aspects of the research problem is essential. A pilot study was conducted including 10% of the sample size, which was excluded later from the study sample. The purpose of this pilot study was to test the clarity of the questions and statements, and the feasibility, objectivity and consistency of the study tool. Based on the findings of the pilot study, the necessary modifications were made. The researcher was available 2 days per week from 9 a.m. to 3 p.m.; each child was individually assessed using the previously mentioned study tools. The interview questionnaire was used to assess the children's knowledge about chelation therapy before implementing the educational programme. The QOL inventory tool was also used to determine the children's QOL before implementing the programme.

Based on the actual educational-need assessment of the children studied, the educational programme was developed after reviewing-related literatures. The programme was submitted in the form of five sessions, each session was 20–30 minutes long according to the physical and mental readiness of the children studied. Different strategies were used such as lectures, demonstrations and group discussions. These children were also divided into sub-groups, each group had 10 children, and they were motivated and rewarded for their active participation during the study period. Post-test questionnaire was used immediately after the educational programme to determine the children's knowledge regarding chelation therapy compared with their preassessment knowledge. The QOL inventory tool was used after the educational programme twice after 3 and 6 months to evaluate the effectiveness of the educational programme on the studied children's QOL.

Statistical analysis

The collected data were coded and entered in the database using the excel programme for Windows. Frequency analysis and manual revision were used to detect any errors. Data were analysed using a computer program with a statistical package for social sciences (SPSS) version 13.

Descriptive measures included frequencies, percentage and statistical tests including Mann–Whitney U test and Qui square test to compare the difference between two medians of abnormally distributed quantitative variables. The level of significance selected for this study was P less than 0.05 and 0.001.

Results

Table 1 illustrates distribution of B-thalassemia children according to their age, gender, educational level, their age at the onset of the disease and duration of illness. About two-thirds of the children studied (68.2%) were aged 8–12 years and more than half of them (51.4%) were girls. With regard to the educational level, 59.5% of them were in primary school. In relation to their birth order, more than one-third of them (38.7%) were ranked as second order.

Table 1. Distribution of B-thalassemia children (n = 173) according to their age, gender, educational level, their age at the onset of the disease, and duration of illness

In relation to the age of thalassemic children at the onset of the disease, 79.8% were diagnosed as thalassemic children in their first year of age. As regards to the number of blood transfusions/month, 68.8% were receiving blood once/month while about one-third of them (31.2%) were receiving blood twice/month. Regarding the duration of illness, it was found that 32.9% of them had the disease for about 12–15 years.

Table 2 shows distribution of B-thalassemia children's parents according to their history of consanguinity, family history of the disease and number of the thalassemic siblings. As regards to the history of consanguinity, 32.4% had consanguineous parents, while 34.7% had a positive family history of the disease and about 21.4% of them had thalassemic siblings. In relation to the number of thalassemic siblings about 13.9% of them had one sibling, while 7.5% of them had two siblings.

Table 2. Distribution of B-thalassemia children's parents according to consanguinity, family history of the disease, and number of the thalassemic siblings

The distribution of B-thalassemia children according to their knowledge about function, side effects of chelation therapy and investigations performed before starting chelation therapy is presented in Table 3. It was observed from this table that there was an extremely significant statistical difference in relation to the studied children's knowledge pre-educational/post-educational programme implementation about the functions, side effects of chelation therapy and investigations carried out before starting chelation therapy. In relation to their knowledge about the functions and side effects of chelation therapy, it was found that 50.3 and 45.1% of them did not know either the function or the side effects of chelation therapy compared with 4.6 and 0% of them pre-educational/post-educational programme, respectively. Regarding their knowledge about the investigations they should carry out before starting chelation therapy, it was observed that only 14.5% of them know the investigations to be carried out pre-educational programme compared with 83.8% of them post-educational programme implementation.

Table 3. Distribution of thalassemic children according to their knowledge about function, method of administration, side effects of chelation therapy, and investigations done before starting chelation therapy

The distribution of B-thalassemia children according to their knowledge score pre- educational/post-educational programme implementation is recorded in Table 4. This table shows that there was an extremely significant statistical difference in relation to the studied children's knowledge score pre-educational and post-educational programme implementation (P = 0.001). It was noticed that 97.7 and 2.3% of them had poor and average score, respectively, pre-programme. On the other hand, it was observed that 61.8 and 38.2% of them had average and good score, respectively, post health teaching programme.

Table 4. Distribution of B-thalassemia children according to their knowledge score pre-/post-educational program implementation

Table 5 shows distribution of B-thalassemia children according to their QOL pre/post 3–6 months of programme implementation. This table shows that there was an extremely statistically significant difference between the studied children's QOL, pre-QOL, QOL post 3 months, and QOL post 6 months of health teaching programme implementation (P = 0.001 and 0.001, respectively).

Table 5. Distribution of B-thalassemia children according to their quality of life pre/post 3–6 months of program implementation

As regards to the studied children's QOL pre-educational programme implementation about 88.4 and 11.6% of them showed low and moderate degree of QOL, respectively. While their QOL post 3 months of the educational programme showed that 64.7 and 35.3% of them had low and moderate degree QOL, respectively, their QOL post 6 months of the educational programme showed that 38.7 and 61.3% had low and moderate degree QOL, respectively.

Discussion

Chelation therapy in children with B-thalassemia major has dramatically altered the prognosis of this previously fatal disease. There are several reasons for inadequate chelation such as lack of children's knowledge about chelation therapy, which can markedly reduce the life expectancy of thalassemic children.¹² So the only way to reduce the morbidity and mortality is by educating these children about the importance of chelation therapy. Optimum chelation therapy reduces iron overload complications and provides a better QOL.⁹

The QOL of children with B-thalassemia major has greatly improved during the past two decades, particularly with regular application of chelating agents and the new transfusion regimens.¹³

A nurse can fulfil the essential function of educating young patients about chelation therapy while stressing that with proper chelation therapy the child can grow, develop and live a normal life. The nurse has an opportunity to exchange information, and helps to decrease tensions and concerns about the treatment and develops an atmosphere of familiarity and routine in chelation therapy.¹⁰

The aim of this study was to assess the impact of health education programmes regarding chelation therapy on the QOL of B-thalassemia children. In relation to the characteristics of the studied thalassemic children, it was observed that boys constituted 48.6%, while girls were 51.4% of those who were affected. This result does not come in agreement with Rund and Rachmilewitz,¹⁴ who stated that thalassemia affects men and women equally.

This study revealed that more than three-quarters of the studied B-thalassemia children (79.8%) were diagnosed in the first year of their life. This result agrees with Giardina and Forget, who mentioned that children born with B-thalassemia major are normal at birth, but develop severe anaemia during the first year of life.³ It was indicated also by Datt and Parul¹⁵ that features of thalassemia usually develop after 6 months of age when haemoglobin synthesis switches from haemoglobin F to haemoglobin A. Moderate and severe thalassemias usually are diagnosed within the first 2 years of life; this is because signs and symptoms, including severe anaemia, often occur.¹⁶

In relation to the number of blood transfusion per month, it was also clear from Table 2 that more than two-thirds of the studied B-thalassemia children received blood transfusion only once per month. This result comes in agreement with Cazzola et al.,¹⁷ who mentioned that the recommended treatment for thalassaemia major involves lifelong regular blood transfusions, usually administered every two to five weeks, to maintain the pretransfusion haemoglobin level above 9–10.5 g/dl. This transfusion regimen promotes normal growth, allows normal physical activities, adequately suppresses bone marrow activity in most patients and minimises transfusional iron accumulation. This result also could be due to the difficulties that children face to get blood, that is why they come to the outpatient clinic every month when they are severely anaemic.

In relation to the history of consanguinity, it was observed that a minority of the studied B-thalassemia children's parents (32.4%) only were consanguineous. In contrast, Arif et al.¹² found that consanguinity was positive in 82.5% of the parents.

More than one-third of the studied B-thalassemia children showed a positive family history. This result almost comes in agreement with Arif et al.,¹² who found that extended family history of thalassemia positive was found in 40.8% of the cases.

Consanguineous marriages increase the birth prevalence of autosomal recessive disorders like thalassemia.¹⁸ This high rate of consanguineous marriage is basically due to our local cultural pattern and traditions where people prefer to marry within families without knowing its grave consequences. It was also reported by Pooya and Doroudchi¹⁹ that there is a significant association between B-thalassemia major and first cousin marriages. Most of thalassemia patients had a positive family history and they consider thalassemia to be an inherited disease.²⁰

This study revealed that 21.4 and 4% of the studied B-thalassemia children had a positive family history in siblings and cousins. This result is almost correspondent with Hashim's²¹ result who found that a positive family history is found in 40% of siblings and 6.6% of cousins.

As regards to the method of taking the chelation therapy, it was observed from this study that 19.6, 32.4, 0, 17.9, and 23.1% of the studied thalassemia children took the medicines via oral, intravenous, intramuscular, subcutaneous infusion via pump and combined more than one iron intake method. The route of administration of chelation therapy is critical in achieving the goal of therapy, which is to reach a negative iron balance (i.e. excreting more iron than acquired from intestinal absorption and transfusion). As the agent is not absorbed in the gut, it must be administered parenteraly, whether intramuscularly, intravenously or subcutaneously via an infusion pump and combinations of two iron chelators (parenteral DFO plus the oral chelator) have been demonstrated to produce additive and synergistic effects. Such an approach would enable a flexible schedule and improve compliance and overall QOL.²²

In relation to the studied B-thalassemia children's knowledge about the side effects of chelation therapy, this study showed that 11.6% of them pre-educational programme implementation only knew the answer compared with 76.9% of them post-programme who mentioned that the side effects of chelation therapy are local reactions, bone deformities, tachypnea, urticaria, vision, and hearing loss. Kwiatkowski listed the adverse effects of iron chelators as follows: local reaction, e.g. redness and induration, vision and hearing abnormalities, allergic reaction, pulmonary complications as tachypnea, hypoxia, neurologic complications and bone deformities.²³

Regarding the studied B-thalassemia children's knowledge about investigations they should carry out before starting chelation therapy, this study showed that a minority of them (14.5%) know the investigations preprogramme compared with the majority of them (83.8%) post-programme who mentioned hearing and vision assessment, cardiac function, hepatic function, renal function, growth and development assessment and endocrine function. It is known that prior to beginning chelation therapy, baseline assessment of hearing, vision, cardiac function, hepatic function, renal function and growth and development are recommended. Annual reassessment of the above parameters, calcium metabolism and endocrine function (including thyroid, parathyroid and sex hormones, glucose tolerance) are suggested.²⁴

There was an extremely significant statistical difference in relation to the studied children's knowledge score preprogramme and post-programme implementation. This study showed that the majority of them had a poor score, while very few of them had an average score preprogramme. But 61.8 and 38.2% of them had an average and good score post-programme, respectively. This result comes in accordance with Armeli et al.,²⁵ who found that B-thalassemia education programmes in Italy appear to have dramatically increased awareness of the disorder.

There was a statistically significant difference between the studied children's QOL degree, pre-QOL, QOL post 3 months, and QOL post 6 months. With regard to their QOL preimplementation of health teaching programmes regarding chelation therapy, about 88.4 and 11.6% of patients had low and moderate degree of QOL, respectively. Their QOL post 3 months showed that 64.7 and 35.3% of them had low and moderate degree of QOL, respectively. But their QOL post 6 months showed that 38.7 and 61.3% had low and moderate degree of QOL, respectively. The QOL of non-chelated and fully chelated thalassaemia patients differs greatly, while the fully chelated patients had a QOL almost similar to that of normal children, except with regard to body pain.²⁶

Transfusion-dependent thalassaemia patients on an optimum chelation therapy had higher QOL scores.⁹ Optimum chelation therapy could lower the levels of serum ferritin, which was associated with a lower rate of iron overload complications and better QOL. On the contrary, Ismail et al.²⁷ and Shaligram et al.²⁸ mentioned that due to the effects of the disease and its treatment, like chelation therapy the existing evidence indicates that thalassemia has a negative impact on health related QOL. Wonke stated that management of a patient with thalassemia is based on adequate safe blood transfusion and iron chelating agents used in innovative ways to improve the QOL and survival of patients with B thalassemia.²⁹

We can conclude that there was a positive effect of the educational programme in improving B-thalassemia children's knowledge regarding thalassemia and chelation therapy and their QOL. Educational programmes should be conducted for the nurses working with thalassemia patients about the importance of chelation therapy and its regular use. Periodic educational programmes based on the actual need assessment of thalassemia children to improve their QOL are important.

Disclaimer statements

Contributors All authors contributed equally to this manuscript.

Funding None.

Conflicts of interest None.

Ethics approval Ethical approval was obtained from the Research Ethics Committee of Faculty of Nursing, Mansoura University.