EQ-5D-3L health state utility values in transfusion-dependent thalassemia patients in Malaysia: a cross-sectional assessment.

PURPOSE: There is a gap of information describing the health state utility values (HSUVs) of transfusion-dependent thalassemia (TDT) patients in Malaysia. These values are useful in the assessment of health-related quality of life (HRQoL), economic evaluations and provide guidance to disease management decisions. The objective of this study was to estimate and derive HSUVs associated with the treatment and complications of TDT patients in Malaysia using the EQ-5D-3L instrument. METHODS: A cross-sectional survey using the EQ-5D-3L instrument was conducted between May to September 2018 across various public hospitals in Malaysia. Using a multi-stage sampling, patients diagnosed with TDT and receiving iron chelating therapy were sampled. The findings on the EQ-5D-3L survey were converted into utility values using local tariff values. A two-part model was used to examine and derive the HSUVs associated with the treatment and complications of iron overload in TDT. RESULTS: A total of 585 patients were surveyed. The unadjusted mean (SD) EQ-5D-3L utility value for TDT patients were 0.893 (0.167) while mean (SD) EQ VAS score was 81.22 (16.92). Patients who had more than two iron overload complications had a significant decline in HRQoL. Patients who were on oral monotherapy had a higher utility value of 0.9180 compared to other regimen combinations. CONCLUSION: Lower EQ-5D-3L utility values were associated with patients who developed iron overload complications and were on multiple iron chelating agents. Emphasizing compliance to iron chelating therapy to prevent the development of complications is crucial in the effort to preserve the HRQoL of TDT patients.

Caregiver Burden, Spirituality, and Psychological Well-Being of Parents Having Children with Thalassemia.

The research determined the relationship of caregiving burden, spirituality and psychological well-being of parents of Pakistani thalassemic patients in a crosssectional research design. The sociodemographic form, Montgomery-Borgatta burden measure (Montgomery et al. in Who should care for the elderly? An east-west value divide. World Scientific, River Edge, pp 27-54, 2000), Multidimensional Measure of Islamic Spirituality (Dasti and Sitwat in J Muslim Ment Health 8(2):47-67, 2014. doi: 10.3998/jmmh.10381607.0008.204 ) and Ryff Scale of Psychological Well-being (Ryff in J Pers Soc Psychol 57(6):1069-1081, 1989. doi: 10.1037/0022-3514.57.6.1069 ) were administered on a sample of 80 parents (32 fathers and 48 mothers) recruited from different Thalassemic Centers of Lahore city, Pakistan. Data were analyzed through correlation and mediational analyses. Results indicated that the caregiver burden was negatively correlated with the psychological well-being and the domains of spirituality, while the psychological well-being and spirituality were positively correlated. We identified that the caregiver burden has direct effect on the psychological well-being of the parents and it influences the psychological well-being through the pathway of the two domains of spirituality, i.e., self-discipline and meanness-generosity. These results highlighted the role of spirituality upon the psychological well-being of caregivers, which could be utilized to prevent pathological influences (such as hard feelings, hopelessness, depressed mood, anxiety, and relationship problems) of caregiver burden and enhance psychological well-being through spiritual counseling. Caregivers can work on their well-being and burden by disciplining their lives and forgoing hard feelings toward others.

Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review.

BACKGROUND: Patients with thalassemia major require iron chelation therapy (ICT) to prevent complications from transfusional iron overload. Deferoxamine is effective, but requires administration as a slow continuous subcutaneous or intravenous infusion five to seven times per week. Deferiprone is a three-times-daily oral iron chelator, but has limited availability in the United States. Deferasirox is a once-daily oral iron chelator that was approved in the United States in 2005 for patients older than 2 years of age with transfusional iron overload. STUDY DESIGN AND METHODS: Published evidence on rates of compliance with ICT and the association between compliance, and the incidence and costs of complications of iron overload, in patients with thalassemia major was reviewed. RESULTS: A total of 18 studies were identified reporting data on compliance with ICT, including 7 that examined deferoxamine only, 6 that examined deferiprone only, and 5 that compared deferoxamine and deferiprone; no studies reporting compliance with deferasirox were identified. In studies of deferoxamine only, estimated mean compliance ranged from 59 to 78 percent. Studies of deferiprone generally reported better compliance, ranging from 79 to 98 percent. Results of comparative studies of deferoxamine and deferiprone suggest that compliance may be better with oral therapy. Numerous studies demonstrate that that poor compliance with ICT results in increased risk of cardiac disease and endocrinopathies, as well as lower survival. Although data on the costs of noncompliance are limited, a recent model-based study estimated the lifetime costs of inadequate compliance with deferoxamine to be $33,142. CONCLUSIONS: Inadequate compliance with ICT in thalassemia major is common and results in substantial morbidity and mortality, as well as increased costs.

Caring for adults with thalassemia in a pediatric world.

Improved technology and medical advances have increased the life span for patients with thalassemia. Therefore, serious consideration must now be given to adult issues such as fertility, employment opportunities, medical insurance, and long-term coping with chronic illness. Since thalassemia is a childhood illness, most adults are seen in pediatric hospitals-often, in centers with a specialty for thalassemia. Compared to a decade ago, many more patients in thalassemia centers are adolescents or older. Unfortunately, pediatric hospitals are not fully equipped to meet the changing and complex needs of adults. Emergency room care, hospital admissions, decentralized care, comprehensive care, and psychosocial issues are current challenges that must be addressed. In this study, six adult patients were asked to keep track of their care for one month to further examine self-care for thalassemia, a high-maintenance disease. From a qualitative perspective, the issues and challenges that adults face are examined and solutions for improved care are discussed.

The impact of regular hospitalization of children living with thalassaemia on their parents in Sri Lanka: a phenomenological study.

This paper reports on a small phenomenological study that set out to describe what it means to have a child with thalassaemia regularly attending hospital for blood transfusion. Ten parents were invited to participate from the thalassaemia unit of a large hospital in a north-west province of Sri Lanka. Data were collected by in-depth interviews with parents. The participants' experiences are described as they reveal, in their own words, what it means to support children with this disease. With startling reality, this research portrays the problems associated with caring for these children and their regular admission to hospital. Three themes are used to present the participant's experience: worries, medical services, and helping and being helped. The findings of the study show that there have been improvements in the service and treatment of these children in Sri Lanka. However, the lives of the parents are still dire. This research should be useful locally in terms of increasing awareness of thalassaemia and raising the consciousness of Sri Lankan nurses and other health care workers, so that they are more aware of the parents' needs. Although phenomenological research is not generalizable, it is likely that the experience of the parents in this study has similarities throughout the developing world.

[Prevention of beta-thalassemia in the French speaking part of Belgium. I. Epidemiologic, clinical, psycho-social and economic justifications]. / La prévention des beta-thalassémies en Belgique francophone. I. Justifications épidemiologiques, cliniques, psycho-sociales et économiques.

A study was jointly conducted by three universities in order to determine whether the systematic prevention of beta-thalassaemia and related affections is justified in the French-speaking part of Belgium. In the entire geographical area considered, 19.1% of the population are foreign; 19.2% of births take place within foreign families. Around 59.7% of foreigners come from regions where beta-thalassaemia is endemic. The ethnic minorities are mostly concentrated in the cities and notably in Brussels. Around 4.1% of the minorities ethnically at risk are carriers of beta-thalassaemia. Births of new cases of thalassaemia major can be estimated at 2.9 per year, which would result, in the absence of prevention, in a population of 130 cases of thalassaemia major. In recent years, patient care has improved considerably, as demonstrated by an increasingly early diagnosis and institution of chelating therapy with an attendant gradual drop in ferritin levels. However, study of age histograms reveals that care of patients in non-endemic regions is not as satisfactory as in endemic countries where complete haemoglobinopathy control programmes have been developed. Psycho-social questioning of a sample of families confirmed that thalassaemia major is an affection which seriously impairs the quality of life. Finally, calculations projected over 20 years revealed that systematic prevention would cost from 2.3 to 2.8 times less than a purely curative approach. The authors conclude that a body of epidemiological, clinical, psycho-social, and economic arguments justify without any doubt the prevention of beta-thalassaemia and related affections in the French-speaking part of Belgium.

Psychosocial and economic profile of a sample of families with thalassaemic children in Lebanon.

Thalassaemia major is a relatively common disease in Lebanon. This study of 41 families with 54 patients attending the American University of Beirut Medical Center was conducted to define some aspects of the disease in Lebanon and to assess the attitudes of affected families on relevant psychosocial and economic issues. We conclude that because of the high frequency of consanguineous marriage, thalassaemia major is more common in Lebanon than might be expected on the basis of the incidence of the trait. Most patients are diagnosed early in life, but their treatment is generally far from adequate; securing desferrioxamine and paying for follow up visits to the doctor seem to be the most important financial burdens. The general population of the country is not properly informed yet and about 70% of the families had not heard about the disease before having an affected child. The inherited nature of the disease is not clear in the minds of a high percentage of the families, and in about 30% of cases the family had not been told about the advisability of screening to detect heterozygotes. The great majority of families favour a preventive approach to thalassaemia, based on heterozygote screening and the possibility of prenatal diagnosis.

Screening and genetic counseling for beta-thalassemia trait in a population unselected for interest: comparison of three counseling methods.

We have assessed the effects of screening and genetic counseling for beta-thalassemia trait on knowledge, attitudes, and behavior in a prospective, controlled study of randomly selected adult members of a health maintenance organization. We report here that knowledge of manifestations and of inheritance of thalassemia, previously reported to be high immediately after counseling, were well maintained at 2 and 10 months following counseling. There was no detectable impairment of self-concept. Marital adjustment improved, and sexual activity increased significantly. Mood, assessed immediately before and after counseling, showed no undesirable changes. A patient-structured counseling method, designed to minimize negative psychological effects via discussion of feelings, was not superior to conventional and programmed methods, described in our previous reports, in terms of learning or attitude change.

Genetic counseling for beta-thalassemia trait following health screening in a health maintenance organization: comparison of programmed and conventional counseling.

Providing adequate counseling of patients identified in genetic screening programs is a major responsibility and expense. Adults in a health maintenance organization, unselected for interest, were screened for beta-thalassemia trait as part of preventive health care. Counseling was provided by either a trained physician (conventional counseling) or by a videotape containing the same information followed by an opportunity to question a trained physician (programmed counseling). Immediately before and after counseling, knowledge of thalassemia, knowledge of genetics, and mood change were assessed by questionnaire. Comparable mood changes and similar learning about thalassemia and genetics occurred with both counseling methods. Thus, as judged by immediate effects on knowledge and mood, videotaped instruction can greatly reduce professional time required for genetic counseling and facilitate the incorporation of genetic screening into primary health care.

Screening and genetic counseling for beta-thalassemia trait in a population unselected for interest: effects on knowledge and mood.

To evaluate the effects of genetic screening and counseling in a population unselected for interest, adults in a health maintenance organization (HMO) were screened for beta-thalassemia trait as part of health care or multiphasic screening. Counseling was provided by either a trained physician or a videotape containing the same information, followed by an opportunity to question a trained physician. Knowledge of thalassemia, knowledge of genetics, and mood were assessed by standardized questionnaires and by interview immediately before and after counseling. Compared to controls, trait subjects demonstrated significant learning about thalassemia (P less than .001) and about genetics (P less than .001) and recorded significant mood changes, namely, surprise (startle) (P less than .05), increased alertness (decreased deactivation) (P less than .05), and decreased skepticism (P less than .01). Screening and genetic counseling for beta-thalassemia trait conducted as part of multiphasic screening of the population of a HMO, essentially and unselected population, can result in significant overall learning with acceptable effects on mood.