Quality of life in children with sickle cell hemoglobinopathy
http://www.100md.com
《美国医学杂志》
Indira Gandhi Medical College, Nagpur, India
Abstract
OBJECTIVE: To identify specific domains and traits that are most affected in patients with sickle cell anemia and traits with respect to normal children. METHODS: Children attending the regional hemoglobinopathy center at IGMC, Nagpur in age group of 8-14 years were assessed. Of 52 children studied, 25 had sickle cell anemia (SCA), 12 had sickle cell trait (SCT) and 15 wre normal control. The (quality of life (QOL) was assessed using multidimensional interview based questionnaire. RESULTS: All domains, physical, psychosocial, cognitive and morbidity were affected. In SCA playing and mobility were most affected. There was feeling of sadness or disinterest and lack of support from teachers. The school attendance, vocational achievement perception, entertainment and participation in cultural activities were also affected. The intensity of weakness and pain was greater in SCA children who left that they were affected by a major illness. The unusual finding was that the SCT children also showed affection of all domains as compared to normal children, which was perhaps due to the stigma of the disease. CONCLUSION: QOL is affected in children with sickle cell disease (SCD) and to a lesser extent in SCT. Interventions to improve QOL should target the affected items. Improving awareness of the disease and its manifestation will help to alleviate the psychosocial affliction of children with SCT.
Keywords: Quality of life; Sickle cell anemia; Scale
WHO's definition of health is a state of complete physical, mental and social well-being and not merely absence of disease or infirmity.[1] Quality of life(QOL) is an essential aspect of mental and social well being that is often ignored. It is the individual's perceptions of their life in the context of their culture and value systems to which they belong and in relation to their goals, expectations, standards and concerns.[1] Sickle Cell Anemia is a disease, which is interspersed with morbidities. Estimation of QOL in these children is important to understand how and to what extent their quality of life is affected. Physicians, family members and the community can then intervene to improve the lives of these children by bettering those areas that are affected the most. The present study was conducted with the aim of assessing QOL in children with sickle cell disease by ( scd0 ) developing an interview based multidimensional disease specific scale. The objective was also to identify the specific domains and traits that are most affected in these children and the differences in QOL of children with SCA as compared to SCT.
Abbreviations used
FP - Domain: Function; Sub-domain: Physical.
FPsy - Domain: Function; Subdomain: Psychological.
FC - Domain: Function; Sub-domain: Cognitive.
FS - Domain: Function; Sub-domain: Social.
HP - Domain: Health perception.
O - Domain: Opportunity M-Domain: Morbidity
Materials and Method
This study was carried out at the Regional Haemoglobinopathy Centre, Indira Gandhi Medical College, Nagpur from April 2001 to October 2002. A focus group discussion with parents and children with this disease was conducted to identify domains for the QOL scale. Children aged 8 years and above were best able to express the impact of disease on their quality and the areas of affection or domains. They would be representative of younger ones most of whom had difficulties in expressing themselves. In addition was used the multidimensional conceptualization of quality of life by Patrick and Erickson[2], EUROQOL[3] and WHO QOL BREF[4] to develop a QOL scale for these children. This enhanced the criterion validity of the scale. Four domains i.e. Functional, Health Perception, Opportunity and Morbidities were identified. They were further divided into sub-domains. The Functional domain had the following sub-domains: Physical, psychological, cognitive and social. Each sub-domain had items which measured responses graded into three categories instead of five as described in the EUROQOL and WHO QOL table1, e.g., no difficulty, some difficulty and unable to carry on the concerned activity. In addition to the level of difficulty, a qualitative estimate of days (i.e. frequency, such as sick on most days, some days or none of the days) of sub-optimal activity was also assessed for all the traits in the respective domains. Three levels of responses were designed as children are not able to make the differentiation between "not at all" and "not much" i.e extremes of responses. Nor are they able to differentiate "a great deal" from "completely". So the responses were collapsed to a three point scale to make it easy to administer without losing the ability to describe the extent to which domains are affected. The scale was prepared in English and then translated into Marathi and Hindi, then re-translated into English. It was pre-tested in six children in both languages before forming the final scale. It was administered by a pediatric resident and a nurse who were involved in developing the scale. The number of items enhanced the internal consistency as well as the inter-rater reliability which was assessed using the Wilcoxon rank sum test which showed significant association between the scores of the two raters. Also the respondents were a homogenous group, which also improves reliability. The scale was empirically validated by two disease experts for content and construct. They agreed that the scale had the ability to measure its objectives of describing the domains of life that were affected. They also agreed that the traits had the construct to measure the different domains and the ability to differentiate normal persons on pre-test. Each scale had 37 questions which took an average of 30-45 minutes for each child. Some items appear to overlap in the responses they invoke. These items were retained for internal reliability. The weightage a child gives to one domain as compared to another would differ. If the scores were aggregatedm there would be an assumption of equal weightage. An overall aggregate score without individual weightage is not always reflective of an overall perception of illness. Therefore we did not aggregate the scores. The objective of the study was to describe the proportion of children who feel restricted in different domains and not to measure the extent within each domain or to have an aggregate measure of quality of life. This can be achieved by a well validated scale.
Since children are seldom interviewed regarding their performances or their feelings, they did experience some difficulty in articulating their feelings. The questions would have to be explained with examples to initiate a response. Most children would respond with confidence. It was administered to 25 children with sickle cell anemia, 12 with sickle cell trait and 11 normal children in their regional language. Children with sickle cell trait are expected to be asymptomatic. However our clinical experience indicates that once they are diagnosed and attend the sickle cell clinic it has an impact on their quality of life. This needed to be measured.
Results
Discussion
Children with chronic disease are 2.5 times more likely to have psychosocial problems in addition to their physical disabilities due to their illness as compared to normal children.[1] Unfortunately, there are few published studies on quality of life related to sickle cell disorders. Most of the studies have reported the impact of the illness on certain domains. Kumar et al measured the psychological effect of sickle cell anaemia on the self-concept, anxiety level and personal and social adjustments of school aged children.[5] A multidimensional disease specific scale to improve the coverage of different health aspects was developed. The impact of SCD on different health domains and its effect on daily lives of these children was assessed with this scale. The choice of dimensions in this scale was driven by the needs of children with SCD assessed using focus group discussion and with the help of multidimensional conceptualization of quality of life by Patrick and Erickson[2], EUROQOL[3] and WHO QOL BREF.[4] This interviewer-based scale, of three-level responses enabled the authors to explain the questions with examples, to add reliability, validity and enhance responsiveness to the scale. The greater the number of categories or levels, lessed is the agreement between interviewers and restricts reliability. This scale helped not only to determine the different areas of involvement in most children of SCD but also the differences seen in SCA as compared to SCT children. Translating the various domains and components of health into a quantitative value that indicate quality of life is a complex task, Drawing from the field of clinico-metrics, psychometrics and clinical discussion theory.[6] The chose to describe the domains that were affected in majority of the children rather than an aggregate score for each child.
The SCA children had greater restriction of physical activities such as performing household chore and play activities as compared to SCT children. Self care was seldom affected in these patients. In India, the Asian/Indian haplotype is associated with higher Hb F levels and milder course than the three African haplotypes.[7] This reflects in fewer episodes of crisis but does not change the physical limitations these children experience when sick. The feeling of sadness or disinterest, lack of support from teachers, school attendance, entertainment and participation in cultural activities, intensity of weakness and pain and a realization of being affected by a major illness was experienced to a larger extent by the SCA children as compared to the SCT children. Due to the stigma of the disease SCT children worried equally about their illness, felt jealous about their normal peers and felt that they looked different from normal peers. They also felt that their illness was an economic burden for their family and that their siblings got less attention than them. They both felt that their cognitive functions were affected but their exam performance was better than their perception about their abilities. Fowler et al found subtle neuropsychological and learning deficits when they compared SCA children who had experienced stroke with normal controls. None of the children in the present study had experienced stroke and were similar to the normal children in their cognitive abilities as measured by the questionnaire.[8]
Most children of SCT are expected to be asymptomatic, and not significantly different from normal children with respect to body pain or sick days. However in the present study, SCT children were also more affected in most domains as compared to normal children. This is attributed to the unusual manifestation of traits children to their awareness of having the gene. Unless educated otherwise, they seem to feel that any pain or sick days they experience are due to their underlying genetic disorder. This feeling gets reinforced by most general practitioners they see for their symptoms. This study showed its impact on their quality of life and emphasizes the role of health education in sickle cell trait.
Several studies have reported different aspects of QOL in affected SCA children as compared to normal children. They have been more domain specific and few if any; have addressed the differences observed in SCA as compared to SCT children. Palermo et al reported a parental assessment of children with sickle cell disease. They compared health related quality of life scale of 58 children with sickle cell disease to demographically similar sample of 120 healthy children applying Child Health Questionnaire. Their results also demonstrated that these children had more limited physical, psychological and social well-being than healthy children. The findings indicated that sickle cell disease significantly affects the HRQL of these youths.[9] Kater et al determined differences in quality of life between children with sickle cell disease and healthy children in Amsterdam. These children scored less in physical, motor, daily functioning independently and had more negative emotions.[10] Fuggle et al examined the frequency and severity of sickle related pain and its impact on quality of life and concluded that careful assessment of pain at home effectively supports children resilience and improves quality of life.[11] Using such a multidimensional scale would enable a more objective assessment of the impact of different interventions for pain management on different domains. Midence K et al also described the family and social issues in sickle cell disease and concluded that like all chronic illnesses SCA children have major psychosocial and practical consequences.[12]
In conclusion, the present study findings reflect earlier ones in that majority of the children of SCA having limitations in physical activities psychosocial aspects perception about their own cognitive abilities, health and future opportunities. This assessment would enable a humanistic element into health care of these patients in not only alleviating their symptoms but improving their abilities. This study also highlighted the importance of appropriate health education in improving the QOL in children with SCT. Palliative treatment or interventions can thus target improvement in the most affected domains and also the change they bring about in the QOL over the course of the disease can be measured. The estimated advantage of one treatment over another in sickle cell anemia, using the cost-utility method is possible if a quality of life scale is available for that population.[13] More studies in this area will help to improve the reliability of this scale.
References
1. World Health Organization, World Health Organization Constitution. Basic documents, Geneva 1948.
2. Patrick D, Erickson P. Quality of life in health care evaluation and research allocation. New York Oxford Univ. Health Status and Health Policy Press 1992.
3. The EuroQol Group. EuroQol-a new facility for the measurement of health-related quality of life. Health Policy 1990; 16: 199-208.
4. WHO QOL-BREF Introduction, administration, scoring and generic version of assessment; Field Trail, December 1996; Version 5.2.
5. Kumar S, Powars D, Allen J, Haywood LJ. Anxiety, self-concept, personal and social adjustment in children with sickle cell anemia. J Pediat r 1976; 88: 859-63.
6. Feinstein AR. Clinico metrics. New Haven. Connecticut. Yale University Press 1987.
7. Kulozik AE, Kar BC, Satapathy RK et al. Fetal hemoglobin levels and bs - globin haplotypes in Indian population with sickle cell disease. Blood 1987; 69: 1724-26.
8. Fowler MG, Whitt JK et al. Neuropsychologic and academic functioning of children with sickle cell anaemia. J Dev Behave Pediatr 1988; 9: 213-220.
9. Palermo TM, Schwartz L et al. Parental report of health related quality of life in children with sickle cell disease. J Behav Med 2002; 25: 269-283.
10. Kater AP, Heijboer H et al. Quality of life in children with sickle cell disease in Amsterdam area. Ned Tijdschr Geneesked 1999; 143: 2049-2053.
11. Fuggle P, Shand PA et al. Pain, quality of life, and coping in sickle cell disease. Arch Dis Child 1996; 75: 199-203.
12. Midence K, Shand P. Family and social issues in sickle cell disease. Health Visit 1992; 65: 441-443.
13. Testa MA, Lenderking WR. Interpreting Pharmocoeconomic and quality of life clinical trial data for use in therapeutics. Pharmocoeconomics 1992; 2: 107-117.(Patel Archana B, Pathan H)
Abstract
OBJECTIVE: To identify specific domains and traits that are most affected in patients with sickle cell anemia and traits with respect to normal children. METHODS: Children attending the regional hemoglobinopathy center at IGMC, Nagpur in age group of 8-14 years were assessed. Of 52 children studied, 25 had sickle cell anemia (SCA), 12 had sickle cell trait (SCT) and 15 wre normal control. The (quality of life (QOL) was assessed using multidimensional interview based questionnaire. RESULTS: All domains, physical, psychosocial, cognitive and morbidity were affected. In SCA playing and mobility were most affected. There was feeling of sadness or disinterest and lack of support from teachers. The school attendance, vocational achievement perception, entertainment and participation in cultural activities were also affected. The intensity of weakness and pain was greater in SCA children who left that they were affected by a major illness. The unusual finding was that the SCT children also showed affection of all domains as compared to normal children, which was perhaps due to the stigma of the disease. CONCLUSION: QOL is affected in children with sickle cell disease (SCD) and to a lesser extent in SCT. Interventions to improve QOL should target the affected items. Improving awareness of the disease and its manifestation will help to alleviate the psychosocial affliction of children with SCT.
Keywords: Quality of life; Sickle cell anemia; Scale
WHO's definition of health is a state of complete physical, mental and social well-being and not merely absence of disease or infirmity.[1] Quality of life(QOL) is an essential aspect of mental and social well being that is often ignored. It is the individual's perceptions of their life in the context of their culture and value systems to which they belong and in relation to their goals, expectations, standards and concerns.[1] Sickle Cell Anemia is a disease, which is interspersed with morbidities. Estimation of QOL in these children is important to understand how and to what extent their quality of life is affected. Physicians, family members and the community can then intervene to improve the lives of these children by bettering those areas that are affected the most. The present study was conducted with the aim of assessing QOL in children with sickle cell disease by ( scd0 ) developing an interview based multidimensional disease specific scale. The objective was also to identify the specific domains and traits that are most affected in these children and the differences in QOL of children with SCA as compared to SCT.
Abbreviations used
FP - Domain: Function; Sub-domain: Physical.
FPsy - Domain: Function; Subdomain: Psychological.
FC - Domain: Function; Sub-domain: Cognitive.
FS - Domain: Function; Sub-domain: Social.
HP - Domain: Health perception.
O - Domain: Opportunity M-Domain: Morbidity
Materials and Method
This study was carried out at the Regional Haemoglobinopathy Centre, Indira Gandhi Medical College, Nagpur from April 2001 to October 2002. A focus group discussion with parents and children with this disease was conducted to identify domains for the QOL scale. Children aged 8 years and above were best able to express the impact of disease on their quality and the areas of affection or domains. They would be representative of younger ones most of whom had difficulties in expressing themselves. In addition was used the multidimensional conceptualization of quality of life by Patrick and Erickson[2], EUROQOL[3] and WHO QOL BREF[4] to develop a QOL scale for these children. This enhanced the criterion validity of the scale. Four domains i.e. Functional, Health Perception, Opportunity and Morbidities were identified. They were further divided into sub-domains. The Functional domain had the following sub-domains: Physical, psychological, cognitive and social. Each sub-domain had items which measured responses graded into three categories instead of five as described in the EUROQOL and WHO QOL table1, e.g., no difficulty, some difficulty and unable to carry on the concerned activity. In addition to the level of difficulty, a qualitative estimate of days (i.e. frequency, such as sick on most days, some days or none of the days) of sub-optimal activity was also assessed for all the traits in the respective domains. Three levels of responses were designed as children are not able to make the differentiation between "not at all" and "not much" i.e extremes of responses. Nor are they able to differentiate "a great deal" from "completely". So the responses were collapsed to a three point scale to make it easy to administer without losing the ability to describe the extent to which domains are affected. The scale was prepared in English and then translated into Marathi and Hindi, then re-translated into English. It was pre-tested in six children in both languages before forming the final scale. It was administered by a pediatric resident and a nurse who were involved in developing the scale. The number of items enhanced the internal consistency as well as the inter-rater reliability which was assessed using the Wilcoxon rank sum test which showed significant association between the scores of the two raters. Also the respondents were a homogenous group, which also improves reliability. The scale was empirically validated by two disease experts for content and construct. They agreed that the scale had the ability to measure its objectives of describing the domains of life that were affected. They also agreed that the traits had the construct to measure the different domains and the ability to differentiate normal persons on pre-test. Each scale had 37 questions which took an average of 30-45 minutes for each child. Some items appear to overlap in the responses they invoke. These items were retained for internal reliability. The weightage a child gives to one domain as compared to another would differ. If the scores were aggregatedm there would be an assumption of equal weightage. An overall aggregate score without individual weightage is not always reflective of an overall perception of illness. Therefore we did not aggregate the scores. The objective of the study was to describe the proportion of children who feel restricted in different domains and not to measure the extent within each domain or to have an aggregate measure of quality of life. This can be achieved by a well validated scale.
Since children are seldom interviewed regarding their performances or their feelings, they did experience some difficulty in articulating their feelings. The questions would have to be explained with examples to initiate a response. Most children would respond with confidence. It was administered to 25 children with sickle cell anemia, 12 with sickle cell trait and 11 normal children in their regional language. Children with sickle cell trait are expected to be asymptomatic. However our clinical experience indicates that once they are diagnosed and attend the sickle cell clinic it has an impact on their quality of life. This needed to be measured.
Results
Discussion
Children with chronic disease are 2.5 times more likely to have psychosocial problems in addition to their physical disabilities due to their illness as compared to normal children.[1] Unfortunately, there are few published studies on quality of life related to sickle cell disorders. Most of the studies have reported the impact of the illness on certain domains. Kumar et al measured the psychological effect of sickle cell anaemia on the self-concept, anxiety level and personal and social adjustments of school aged children.[5] A multidimensional disease specific scale to improve the coverage of different health aspects was developed. The impact of SCD on different health domains and its effect on daily lives of these children was assessed with this scale. The choice of dimensions in this scale was driven by the needs of children with SCD assessed using focus group discussion and with the help of multidimensional conceptualization of quality of life by Patrick and Erickson[2], EUROQOL[3] and WHO QOL BREF.[4] This interviewer-based scale, of three-level responses enabled the authors to explain the questions with examples, to add reliability, validity and enhance responsiveness to the scale. The greater the number of categories or levels, lessed is the agreement between interviewers and restricts reliability. This scale helped not only to determine the different areas of involvement in most children of SCD but also the differences seen in SCA as compared to SCT children. Translating the various domains and components of health into a quantitative value that indicate quality of life is a complex task, Drawing from the field of clinico-metrics, psychometrics and clinical discussion theory.[6] The chose to describe the domains that were affected in majority of the children rather than an aggregate score for each child.
The SCA children had greater restriction of physical activities such as performing household chore and play activities as compared to SCT children. Self care was seldom affected in these patients. In India, the Asian/Indian haplotype is associated with higher Hb F levels and milder course than the three African haplotypes.[7] This reflects in fewer episodes of crisis but does not change the physical limitations these children experience when sick. The feeling of sadness or disinterest, lack of support from teachers, school attendance, entertainment and participation in cultural activities, intensity of weakness and pain and a realization of being affected by a major illness was experienced to a larger extent by the SCA children as compared to the SCT children. Due to the stigma of the disease SCT children worried equally about their illness, felt jealous about their normal peers and felt that they looked different from normal peers. They also felt that their illness was an economic burden for their family and that their siblings got less attention than them. They both felt that their cognitive functions were affected but their exam performance was better than their perception about their abilities. Fowler et al found subtle neuropsychological and learning deficits when they compared SCA children who had experienced stroke with normal controls. None of the children in the present study had experienced stroke and were similar to the normal children in their cognitive abilities as measured by the questionnaire.[8]
Most children of SCT are expected to be asymptomatic, and not significantly different from normal children with respect to body pain or sick days. However in the present study, SCT children were also more affected in most domains as compared to normal children. This is attributed to the unusual manifestation of traits children to their awareness of having the gene. Unless educated otherwise, they seem to feel that any pain or sick days they experience are due to their underlying genetic disorder. This feeling gets reinforced by most general practitioners they see for their symptoms. This study showed its impact on their quality of life and emphasizes the role of health education in sickle cell trait.
Several studies have reported different aspects of QOL in affected SCA children as compared to normal children. They have been more domain specific and few if any; have addressed the differences observed in SCA as compared to SCT children. Palermo et al reported a parental assessment of children with sickle cell disease. They compared health related quality of life scale of 58 children with sickle cell disease to demographically similar sample of 120 healthy children applying Child Health Questionnaire. Their results also demonstrated that these children had more limited physical, psychological and social well-being than healthy children. The findings indicated that sickle cell disease significantly affects the HRQL of these youths.[9] Kater et al determined differences in quality of life between children with sickle cell disease and healthy children in Amsterdam. These children scored less in physical, motor, daily functioning independently and had more negative emotions.[10] Fuggle et al examined the frequency and severity of sickle related pain and its impact on quality of life and concluded that careful assessment of pain at home effectively supports children resilience and improves quality of life.[11] Using such a multidimensional scale would enable a more objective assessment of the impact of different interventions for pain management on different domains. Midence K et al also described the family and social issues in sickle cell disease and concluded that like all chronic illnesses SCA children have major psychosocial and practical consequences.[12]
In conclusion, the present study findings reflect earlier ones in that majority of the children of SCA having limitations in physical activities psychosocial aspects perception about their own cognitive abilities, health and future opportunities. This assessment would enable a humanistic element into health care of these patients in not only alleviating their symptoms but improving their abilities. This study also highlighted the importance of appropriate health education in improving the QOL in children with SCT. Palliative treatment or interventions can thus target improvement in the most affected domains and also the change they bring about in the QOL over the course of the disease can be measured. The estimated advantage of one treatment over another in sickle cell anemia, using the cost-utility method is possible if a quality of life scale is available for that population.[13] More studies in this area will help to improve the reliability of this scale.
References
1. World Health Organization, World Health Organization Constitution. Basic documents, Geneva 1948.
2. Patrick D, Erickson P. Quality of life in health care evaluation and research allocation. New York Oxford Univ. Health Status and Health Policy Press 1992.
3. The EuroQol Group. EuroQol-a new facility for the measurement of health-related quality of life. Health Policy 1990; 16: 199-208.
4. WHO QOL-BREF Introduction, administration, scoring and generic version of assessment; Field Trail, December 1996; Version 5.2.
5. Kumar S, Powars D, Allen J, Haywood LJ. Anxiety, self-concept, personal and social adjustment in children with sickle cell anemia. J Pediat r 1976; 88: 859-63.
6. Feinstein AR. Clinico metrics. New Haven. Connecticut. Yale University Press 1987.
7. Kulozik AE, Kar BC, Satapathy RK et al. Fetal hemoglobin levels and bs - globin haplotypes in Indian population with sickle cell disease. Blood 1987; 69: 1724-26.
8. Fowler MG, Whitt JK et al. Neuropsychologic and academic functioning of children with sickle cell anaemia. J Dev Behave Pediatr 1988; 9: 213-220.
9. Palermo TM, Schwartz L et al. Parental report of health related quality of life in children with sickle cell disease. J Behav Med 2002; 25: 269-283.
10. Kater AP, Heijboer H et al. Quality of life in children with sickle cell disease in Amsterdam area. Ned Tijdschr Geneesked 1999; 143: 2049-2053.
11. Fuggle P, Shand PA et al. Pain, quality of life, and coping in sickle cell disease. Arch Dis Child 1996; 75: 199-203.
12. Midence K, Shand P. Family and social issues in sickle cell disease. Health Visit 1992; 65: 441-443.
13. Testa MA, Lenderking WR. Interpreting Pharmocoeconomic and quality of life clinical trial data for use in therapeutics. Pharmocoeconomics 1992; 2: 107-117.(Patel Archana B, Pathan H)