Impact of SCOPE Program on Health-Related Quality of Life and Health Status of Children With Thalassemia: A Quasi-Experimental Study.

Background: Iron chelation, blood transfusions, and complication management are typical hospital requirements for children with beta-thalassemia major. This affects their health-related quality of life (HRQoL). The purpose of this study was to evaluate how the Supportive and Coping strategies, Ongoing Assessment, Prevention of Complications, and Empowerment (SCOPE) Program impacted the HRQoL and overall health of children with thalassemia. Method: The study employed a quasi-experimental pretest-posttest control group with a sequential follow-up design. A nonprobability purposive sampling technique was used to include 80 children with beta-thalassemia major in the sample, ranging in age from 6 to 18. Data were gathered using a Demographic Profile, PedsQLTM Version 4.0, and a Clinical Profile. The children in the intervention group received the SCOPE Program over the course of 6 months. The data collection included a pretest and a posttest with four follow-up evaluations. Results: During the pretest, children with thalassemia had a very low HRQoL. However, the final assessment after the intervention showed a significant difference in the mean scores between the two groups in the areas of physical functioning (p = .001), emotional functioning (p = .0001), social functioning (p = .001), and school functioning (p = .001). Growth indicators also demonstrated a notable improvement in the intervention group of children. Discussion: The SCOPE Program may be a thorough and efficient intervention for enhancing the general health of children with thalassemia. It can be used as a cooperative, well-organized, family-focused care strategy. Further study with a larger sample size is suggested.

Psychological challenges and quality of life in Pakistani parents of children living with thalassemia.

PURPOSE: Children living with thalassemia experience psychological challenges, but despite significant psychosocial burdens, caregivers' psychological wellbeing and quality of life remain understudied, particularly in lower-and-middle-income countries. DESIGN AND METHOD: The current study evaluated these relationships in 100 male and female Pakistani caregivers (23-45 years; 61% female) using Ryff's Psychological Well-Being Scale and the Singapore Caregiver Quality of Life Scale. Caregivers completed questionnaires during regularly scheduled clinic visits for their child. RESULTS: We found that Pakistani caregivers in our sample generally had significantly lower (30-40 points) quality of life than a referent sample of caregivers of older adults (ps < 0.001). Self-acceptance and personal growth were consistently significant predictors across quality of life domains. Further, significant interactions were observed. Female caregivers with less self-acceptance had worse mental health and wellbeing and impact on daily life (p < .05). Male caregivers with less personal growth had worse physical health wellbeing (p < .05). CONCLUSIONS: Our results demonstrate the importance of considering how distinct aspects of psychological wellbeing, rather than just the overall score, relate to the specific quality of life domains among male and female caregivers. PRACTICE IMPLICATIONS: Pediatric nurses are at the frontline of service delivery for children and are in a prime position to observe caregivers who could be at high risk for psychological challenges. Given our findings, future clinical interventions should prioritize support services promoting personal growth and self-acceptance for Pakistani caregivers of children living with thalassemia.

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.

Addressing the psychosocial needs of young people with thalassaemia undergoing bone marrow transplantation.

Beta thalassaemia major is an inherited condition that causes severe anaemia. Patients with the condition require regular blood transfusions. One curative treatment option available is bone marrow transplantation, but a bone marrow transplant is a high-risk, painful procedure requiring prolonged hospitalisation. Undergoing such a disruptive treatment can be a source of great anxiety for young people and their families, who will need honest, sensitive and empathetic communication, person-centred care, support to socialise and access education, involvement in decision-making and signposting to financial support. This article discusses the role of children's nurses in addressing the psychosocial needs of young people with thalassaemia who undergo bone marrow transplantation and in supporting young people's families.

Resilience in adolescents with thalassemia.

Adolescents with thalassemia often manifest with an increased risk of emotional and behavioral problems, as well as poor quality of life. However, some can be well-adapted and demonstrate evidence of resilience. This study aimed to explore resilience among those with thalassemia and determine the protective factors for their resilience. Sixty adolescents with thalassemia and 60 healthy adolescents as a comparison group participated in the study. Most adolescents with thalassemia demonstrated resilience. Eighty percent of them scored in the low-risk range of the SDQ total difficulties scale, and 91.7% scored in the low-risk range of the SDQ prosocial scale. The mean total difficulties scores of the thalassemia patients and the healthy controls were 11.38 and 11.27 respectively, whereas the mean prosocial scores were 7.28 and 6.65, without statistical significance. Despite the extensive demands of the illness, most adolescents with thalassemia appear to be adapted well, demonstrating evidence of resilience. Factors promoting resilience are lower BMI, less severe type of thalassemia, and younger age at the start of an iron chelator. Health care professionals who take care of adolescents with thalassemia should collaborate to improve their resilience.

Health-related quality of life among children with transfusion-dependent thalassemia: A cross-sectional study in Malaysia.

BACKGROUND: The treatment of children with transfusion-dependent thalassemia (TDT) in Malaysia has progressed since 2005. This study provides an updated health-related quality of life (HRQoL) assessment for children with the disorder and the factors affecting the HRQoL. METHODS: A cross-sectional HRQoL survey of Malaysian children with TDT was conducted using the PedsQL™ 4.0 Generic Core Scales. Patients with non-transfusion dependent thalassemia and other haemoglobinopathies were excluded. Parent-proxy and self-reported HRQoL scores were obtained using a multi-stage convenient sampling. The relationship between HRQoL scores and demographic factors were tested using association, correlation and regression analysis. RESULTS: A total of 368 patients were recruited. The mean (SD) Total Summary Score (TSS) was 80.12(13.87). Predictors for a lower TSS was an increasing age group and the use of dual chelating agents (R2 = 0.057, F (4, 359) = 5.40, p = < 0.001). The mean (SD) Physical Health Summary Score (PHSS) was 82.21 (16.82). Predictors of a higher PHSS score was being male, while predictors of a lower score was an increasing age group and parent-proxy reports(R2 = 0.075, F (5,358) = 5.80, p = < 0.001). The mean (SD) Psychosocial Health Summary Score (PCHS) was 79.39 (14.81). Predictors for a lower PCHS was the use of dual chelating agents(R2 = 0.041, F (1, 362) = 15.60, p = < 0.001). The school functioning score had the lowest mean (SD) score of 69.52(20.92) in the psychosocial dimension. CONCLUSION: The HRQoL of TDT children in Malaysia has improved over the last decade owing to the better access in treatment. However, further effort is needed to improve the school functioning dimension.

The transactional model of stress and coping as guidance for understanding adolescent patients' experience with thalassemia: Case report.

PURPOSE: This case report investigated the transactional model of stress and coping as guidance for nursing care of an adolescent patient with thalassemia. SOURCES USED: A case study of a 15-year-old female patient with ß-thalassemia major. Data were collected using patient medical records, an interview with the patient and physical examination. RESULTS: Four issues related to coping were isolated: Worsening physical symptoms; psychosocial consequences, coping process, and building supportive networks. These issues and the patient's adaption are explored via the transactional model. CONCLUSIONS: Having thalassemia was cognitively appraised by the patient as a stressful and taxing situation with detrimental consequences, such as changes in physical appearance, stigmatization, and depression. Nurses should evaluate each patient's physical and psychosocial needs, utilizing appropriate theoretical models for designing a suitable care plan. As the case study demonstrates, the transactional model was an effective guide for nurses in planning care of an adolescent patient with thalassemia.

Involvement of hemoglobins in the pathophysiology of Alzheimer's disease.

Hemoglobins (Hbs) are heme-containing proteins binding oxygen, carbon monoxide, and nitric oxide. While erythrocytes are the most well-known location of Hbs, Hbs also exist in neurons, glia and oligodendroglia and they are primarily localized in the inner mitochondrial membrane of neurons with likely roles in cellular respiration and buffering protons. Recently, studies have suggested links between hypoxia and neurodegenerative disorders such as Alzheimer Disease (AD) and furthermore suggested involvement of Hbs in the pathogenesis of AD. While cellular immunohistochemical studies on AD brains have observed reduced levels of Hb in the cytoplasm of pre-tangle and tangle-bearing neurons, other studies on homogenates of AD brain samples observed increased Hb levels. This potential discrepancy may result from differential presence and function of intracellular versus extracellular Hbs. Intracellular Hbs may protect neurons against hypoxia and hyperoxia. On the other hand, extracellular free Hb and its degradation products may trigger inflammatory immune and oxidative reactions against neural macromolecules and/or damage the blood-brain barrier. Therefore, biological processes leading to reduction of Hb transcription (including clinically silent Hb mutations) may influence intra-erythrocytic and neural Hbs, and reduce the transport of oxygen, carbon monoxide and nitric oxide which may be involved in the (patho)physiology of neurodegenerative disorders such as AD. Agents such as erythropoietin, which stimulate both erythropoiesis, reduce eryptosis and induce intracellular neural Hbs may exert multiple beneficial effects on the onset and course of AD. Thus, evidence accumulates for a role of Hbs in the central nervous system while Hbs deserve more attention as possible candidate molecules involved in AD.

The Relationship Between Psychological Distress and Religious Practices and Coping in Malaysian Parents of Children with Thalassemia.

STUDY PURPOSE: This study was conducted to examine the relationship between religious practice, religious coping methods and psychological distress among parents caring for children with transfusion-dependent thalassemia. DESIGN AND METHODS: This is a cross-sectional survey. Data were collected on 162 parents of children diagnosed with thalassemia aged 12 years and younger in thalassemia day care centers of three public hospitals in Sabah, Malaysia. Data were collected using questionnaires, including General Health Questionaire-12 (GHQ-12), Duke University Religion Index (DUREL) and Brief RCOPE. RESULTS: Forty-two percent of parents had psychological distress with GHQ score ≥ 3 (mean score of 2.85 ±â€¯3.17). Ninety-five percent of parents used positive religious coping methods (mean P-COPE score 22.35 ±â€¯2.33) more than negative religious coping methods (mean N-COPE score was 12.19 ±â€¯5.23). They used Organized Religious Activities (mean ORA score of 4.20 ±â€¯1.27), and Non-Organized Religious Activities (NORA, the mean was 4.17 ±â€¯1.37). Positive and negative religious coping methods were significantly related to parents' psychological distress (P-COPE and GHQ-12 scores (rs (df) = 0.19, p < .05; N-COPE and GHQ-12 scores rs (df) = 0.38, p < .001)). CONCLUSION: The study findings showed the parents experienced psychological distress. They used positive religious coping methods more than negative religious coping methods. Psychological distress was significantly related to organized religious activities, non-organized religious activities and positive and negative religious coping methods. PRACTICE IMPLICATION: The study findings facilitate understanding of psychological distress and how parents use religious coping strategies to deal with the stress caring for their child.

Assessing the role of family well-being on the quality of life of Indian children with thalassemia.

BACKGROUND: The association between chronic diseases and psychological problems is well established. As thalassemia is chronic blood disorder with burdensome treatment procedures, patients are likely to have psychological health problems. Many studies reported evidences regarding the quality of life. But, factors influencing the health-related quality of life with focus on psychological well-being were minimally studied. We aimed to find the factors contributing to the health-related quality of life among thalassemia affected children and hypothesising whether the parent's psychological well-being, sociodemographic characteristics and transfusion interval have an impact on children's quality of life. METHOD: A cross-sectional analytical study conducted on 125 thalassemia patients and 125 parents (either father or mother) referred to the clinic of Thalassemia treatment center. KIDSCREEN-10 and Ryff Psychological well-being scale is used for measuring the health-related quality of life and well-being of children and parent respectively. RESULTS: We have found the three factors such as family income, children education, and, parent education significantly contributed to the children's health-related quality of life among thalassemia affected children. The average score of Health-related quality of life among children is 16.28 with a standard deviation of 3.432 and the mean psychological well-being score for the parent is 83.99 with a standard deviation of 11.41. A positive correlation exists between parent psychological well-being and children's health-related quality of life. CONCLUSION: Family well-being is the foundation for quality of life of the children. It was found that factors such as family income and parents' and children's education have a direct association with HRQoL of life of children with thalassemia. However, more studies need to be done in order to ascertain the factors contributing to HRQoL of children with thalassemia to improve the quality of life of thalassemia patients.

Quality of life outcomes in thalassaemia patients in Saudi Arabia: a cross-sectional study.

BACKGROUND: Research focusing on health-related quality of life (HRQoL) in thalassaemia patients remains limited in Saudi Arabia. AIMS: To report on HRQoL outcomes in thalassaemia patients, and study associations with psychosocial and clinical factors. METHODS: Thalassaemia patients attending King Abdulaziz University Hospital were sequentially approached for enrolment. HRQoL outcomes were assessed using a validated Arabic version of the Medical Outcomes Study Short form (SF-36). RESULTS: This study included 105 individuals with ß-thalassaemia major, with a mean age of 22.9 (± 11.7) (range 5-35) years, and 52.4% were male. Participants aged ≥ 14 years had a mean physical HRQoL score of 43.7 (± 10.1) and mental HRQoL score of 46.6 (± 10.5). Participants aged < 14 years, had corresponding scores of 48.4 (± 10.7) and 52.9 (± 8.0), respectively. Physical functioning scores were significantly worse for older patients, and those reporting chronic pain. Lower scores of vitality were associated with older age, chronic pain and history of splenectomy. Older age and history of splenectomy were associated with lower scores of mental health. Controlling for other variables, younger age and higher pretransfusion haemoglobin were associated with better mental HRQoL. Professional workers had better physical HRQoL scores compared to nonprofessional workers.. CONCLUSIONS: Controlling for other variables, higher pretransfusion haemoglobin and younger age were associated with better mental HRQoL outcomes in thalassaemia patients. Professionals reported better physical HRQoL outcomes, compared to non-professionals. Preventive and comprehensive care models are needed in Saudi Arabia to improve HRQoL outcomes in thalassaemia patients.

Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias.

BACKGROUND: Adherence to long-term chelation therapy in transfusion-dependent patients is critical to prevent iron overload-related complications. Once-daily deferasirox dispersible tablets (DT) have proven long-term efficacy and safety in patients ≥2 years old with chronic transfusional iron overload. However, barriers to optimal adherence remain, including palatability, preparation time, and requirements for fasting state. A new film-coated tablet (FCT) formulation was developed, swallowed once daily (whole/crushed) with/without a light meal. METHODS: The open-label, Phase II ECLIPSE study evaluated patient-reported outcomes (PROs) in transfusion-dependent thalassemia or lower-risk myelodysplastic syndromes patients randomized 1:1 to receive deferasirox DT or FCT over 24 weeks as a secondary outcome of the study. Three PRO questionnaires were developed to evaluate both deferasirox formulations: 1) Modified Satisfaction with Iron Chelation Therapy Questionnaire; 2) Palatability Questionnaire; 3) Gastrointestinal (GI) Symptom Diary. RESULTS: One hundred seventy three patients were enrolled; 87 received the FCT and 86 the DT formulation. FCT recipients consistently reported better adherence (easier to take medication, less bothered by time to prepare medication and waiting time before eating), greater satisfaction/preference (general satisfaction and with administration of medicine), and fewer concerns (less worry about not swallowing enough medication, fewer limitations in daily activities, less concern about side effects). FCT recipients reported no taste or aftertaste and could swallow all their medicine with an acceptable amount of liquid. GI summary scores were low for both formulations. CONCLUSIONS: These findings suggest a preference in favor of the deferasirox FCT formulation regardless of underlying disease or age group. Better patient satisfaction and adherence to chelation therapy may reduce iron overload-related complications. TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT02125877; registered April 26, 2014.

Factors affecting quality of life in children and adolescents with thalassemia in Iraqi Kurdistan.

OBJECTIVES: To assess the health related quality of life (HRQoL) in Iraqi Kurd children and adolescents with thalassemia, and identify the factors that affect it. METHODS:  In the period between May and June 2018, 100 thalassemic patients and 100 healthy subjects between the ages of 6-18 years were enrolled. The patients included 73 with thalassemia major (TM) and 27 with intermedia (TI). Patients were clinically re-evaluated, and the pediatric quality of life inventory (PedsQL) 4.0 was administered by both child and parent reports. Results: The mean HRQoL score of thalassemic patients was significantly lower than that of healthy subjects, with lowest scores in physical functioning. Furthermore, the mean HRQoL of TM was significantly lower than that of TI subgroup. Significantly lower mean HRQoL scores were seen in those taking ≥6 transfusions/year, with hepatitis C infection, with illiterate parents, and those on oral iron chelation. Pearson correlation revealed that HRQoL was negatively associated with age, frequency of transfusions, and serum ferritin, but positively correlated with age at starting transfusion and age at diagnosis. Only age and serum ferritin remained significant by multivariate analysis. CONCLUSION:  This study shows that among Iraqi Kurds with thalassemia, the disease has a significant negative impact on quality of life, with age and serum ferritin being identified as independent predictors.  Psychosocial, educational, and patient-centered management programs may be needed to improve HRQoL in this disease.

Quality of life in Sardinian patients with transfusion-dependent Thalassemia: a cross-sectional study.

PURPOSE: The aim of this study has been to evaluate the physical, psychological, and social well-being in a large group of Sardinian adult patients with transfusion-dependent beta-Thalassemia when compared with a group of healthy subjects of the same age and geographical extraction. METHODS: Male or female patients ≥ 18 years of age with Thalassemia major on regular transfusion at Thalassemia Center in Cagliari (Italy) were requested to complete the World Health Organization Quality of life-BREF (WHOQOL-BREF) questionnaire. The WHOQOL-BREF was also made available online to age- and sex-matched non-thalassemic adult subjects living in Sardinia. RESULTS: Two hundred and seven subjects with Thalassemia were invited to participate in the study. The questionnaire was also completed by 211 age- and sex-matched non-thalassemic subjects living in Sardinia. Scores suggestive of a good quality of life were obtained in all the areas investigated. Thalassemia patients had scores at least as good as those of non-thalassemic subjects in all items and the percentage of those with a score ≥ 60 was higher among patients. The analysis of demographic actually highlights that the disease has a little effect on their personal and social lives. There was a positive association between subjective well-being and effective clinical conditions. Moreover, the association between health perception and adherence to treatment suggests that compliance with treatment contributes to the well-being of the patient, both physically and psychologically. CONCLUSIONS: Adult subjects with Thalassemia who live in Western countries have a good quality of life in accordance with the advances in the management of the disease.

Quality of Life (QoL) and the Factors Affecting it in Transfusion-dependent Thalassemic Children.

OBJECTIVES: To evaluate the Quality of life (QoL) and the factors affecting it in transfusion-dependent thalassemic children. METHODS: Current study was conducted at Department of Pediatrics, Dr. SNMC, Jodhpur. Ninety-three transfusion-dependent thalassemic children aged between 8 and 18 y were enrolled. Quality of life was assessed using Pediatric Quality of Life (PedsQL) measurement model in thalassemic children. RESULTS: Mean age of children was 11.8 ± 3.2 y. Lowest mean score was observed in School functioning (57.4) while in Physical functioning, Emotional functioning and Social functioning it was 73.9, 82.2 and 83.49 respectively. Mean PedsQL score was higher in 8-12 y age group as compared to 13-18 y (p = 0.024). PedsQL score was higher in children who required transfusion once in a month (p = 0.028) and children on combined chelation therapy as compared to children who were on injectable chelator (p = 0.039). Health Related QoL was not affected with gender, education status, family history of thalassemia, ferritin level, splenectomy or consanguinity. CONCLUSIONS: Factors related with low QoL in current study were higher age, increased transfusion frequency and injectable mode of chelation. In order to improve the QoL in thalassemic children appropriate programmes and interventions should be started targeting above domains.

Psychometric Evaluation of the Persian Translation of the Thalassemia-Specific Quality of Life Questionnaire.

BACKGROUND AND PURPOSE: There is no Persian scale for assessing the quality of life of patients with thalassemia major. Thus, this study was conducted to translate and validate the 36-item thalassemia-specific Transfusion-Dependent Quality of Life (TranQoL) questionnaire. METHODS: The questionnaire was first translated into Persian and then was backward-translated into English. Afterward, panels of experts commented on the Persian translation, and then its face validity was confirmed and content validity index (CVI) and content validity ratio (CVR) were calculated. The factorial structure and the reliability of the translated questionnaire were assessed using exploratory factor analysis (EFA) and internal consistency method, respectively. RESULTS: The CVI and CVR of the questionnaire were .95 and .84, respectively. Five items were deleted during EFA, and 9 factors were extracted from the 31 remaining items. The reliability of the questionnaire was confirmed through a Cronbach's alpha coefficient of .9. CONCLUSION: The Persian translation of the TranQoL questionnaire is highly reliable and valid, and thus, it can be used as a specific measure for assessing the quality of life among patients with thalassemia.

Health-related Quality of Life and its Predictors Among Transfusion-dependent Thalassemia Patients.

PURPOSE: With regular blood transfusions and modern medical management strategies, transfusion-dependent thalassemia (TDT) patients are currently living into adulthood, but they still suffer from the chronicity of the disease and its complications. This study was aimed to assess the health-related quality of life (HRQoL) scores in pediatric TDT patients compared with healthy controls. PATIENTS AND METHODS: The PedsQL 4.0 Generic Core Scales were administered to 80 pediatric TDT patients (aged 5 to 18 y, child self-report) and their parents (parent-proxy report), as well as age-matched and sex-matched healthy controls (80 participants and their parents). RESULTS: Patients with TDT and their parents rated lower HRQoL scores in all domains (physical, emotional, social, and school functioning) compared with the healthy population (P<0.01). The univariate analysis indicated that the total HRQoL score for children's self-reports was negatively predicted by high ferritin levels and the presence of any complication. The only negative predictor of the total HRQoL score in multivariate analyses was a high ferritin level (>1800 ng/dL). CONCLUSIONS: This study showed that thalassemia has a negative impact on HRQoL when compared with a healthy population. For a better quality of life, intensive treatment should be applied for complications and high ferritin levels.

Quality of Life in Children with Thalassemia and their Caregivers in India.

OBJECTIVES: To assess and compare the Quality of Life (QOL) of children with beta-thalassemia major on regular transfusion therapy with normal children, and of the caregivers of children with beta-thalassemia major to that of caregivers of normal children. METHODS: A cross-sectional comparison of QOL in 75 thalassemic and 80 non-thalassemic children was conducted using the PedsQL™ 4.0 generic core scale. Also self-rated health was assessed in their caregivers using Short Form-36 Health Survey. RESULTS: The total QOL score according to child-self report [83.7 (10.8) vs. 97.6 (3.3); p < 0.001] and parent-proxy report [84.2 (11.9) vs. 96.7 (3.5); p < 0.001] was significantly lower in cases as compared with controls. It was found that a significantly higher proportion of caregivers of cases reported poor health compared with caregivers of controls (29.2% vs. 2.5%, p < 0.001). Even after adjusting for age, sex, socio-economic status, and total QOL score by the parent, it was found that caregivers of thalassemic children were significantly more likely to report poor health compared with those of controls (odds ratio: 15.8, 95% confidence intervals: 2.8-89.9). CONCLUSIONS: Health Related QOL is significantly affected in children with beta-thalassemia major on regular transfusion across all age groups, gender and socio-economic classes and also in their caregivers.