Health-related quality of life in Thai thalassemic children treated with iron chelation

Thalassemia is a chronic hereditary disease in which patients with severe disease present with anemia during their first year of life. In Thailand, stem cell transplantation is not an option for most patients. Supportive treatments, such as blood transfusions and iron chelation are used. Little data exists regarding the Health Related Quality of Life (HRQoL) of these patients. We conducted a study of the four dimensions of quality of life: physical, emotional, social, and role (school) functioning, using the PedsQL 4.0 Generic Core Scale to measure the HRQoL among thalassemic patients at the Hematology Unit, Department of Pediatrics, Phramongkutklao Hospital, during December 1, 2006 - November 30, 2007 to evaluate the quality of life in thalassemic patients treated with three iron-chelating agents. Forty-nine thalassemic patients were enrolled and treated with iron-chelating agents. The mean (SD) age of the patients was 10.61 years (4.33). Fifteen thalassemic patients were treated with desferrioxamine, 18 with deferiprone and 16 with deferasirox. The quality of life (QOL) results show the mean (SD) total summary score was 74.35 (12.42). For the psychosocial health summary, the social and school functioning scores were 85.40 (16.67) and 62.14 (15.84), respectively. The QOL scores of the patients who received desferrioxamine, deferiprone and deferasirox were 75.29 (9.09), 73.91 (15.25) and 73.98 (12.32), respectively (p = 0.94). The QOL had no significant differences by age, gender, type of thalassemia or serum ferritin level. Multivariate regression analysis showed no significant differences in clinical severity, age of onset or pre-transfusion hematocrit levels.     

Health-related quality of life in perinatally HIV-infected children in the Netherlands

Combination antiretroviral therapy (cART) can alter HIV infection in children into a chronic condition. Studies investigating health-related quality of life (HRQoL) in HIV-infected children are scarce, and lacking from Western Europe. This study aimed to compare the HRQoL of clinically stable perinatally HIV-infected children to healthy, socioeconomically (SES)-matched controls as well as the Dutch norm population, and to explore associations between HIV and cART-related factors with HRQoL. HIV-infected and healthy children aged 8–18 years completed the Pediatric Quality of Life Inventory? 4.0 (PedsQL?). We determined differences between groups on PedsQL? mean scores, and the proportion of children with an impaired HRQoL per group (≥1 SD lower than the Dutch norm population). Logistic regression models were used to explore associations between disease-related factors and HRQoL impairment. In total, 33 HIV-infected and 37 healthy children were included. There were no differences in the mean PedsQL? subscales between HIV-infected children and both control groups. The proportion of children with an impaired HRQoL was higher in the HIV-infected group (27%) as compared to the healthy control group (22%) and the Dutch norm (14%) on the school functioning subscale (HIV vs. Dutch norm: P?=?.045). Mean scores of HRQoL of perinatally HIV-infected children in the Netherlands were not different from a SES-matched control group, or from the Dutch norm population. However, the HIV-infected group did contain more children with HRQoL impairment, suggesting that HIV-infected children in the Netherlands are still more vulnerable to a compromised HRQoL.     

The PedsQL in pediatric rheumatology: reliability, validity, and responsiveness of the Pediatric Quality of Life Inventory Generic Core Scales and Rheumatology Module

OBJECTIVE: The Pediatric Quality of Life Inventory (PedsQL) is a modular instrument designed to measure health-related quality of life (HRQOL) in children and adolescents ages 2-18 years. The 23-item PedsQL 4.0 Generic Core Scales are multidimensional child self-report and parent proxy-report scales developed as the generic core measure to be integrated with the PedsQL disease-specific modules. The 22-item PedsQL 3.0 Rheumatology Module was designed to measure pediatric rheumatology-specific HRQOL. This study was undertaken to demonstrate the reliability, validity, and responsiveness of the PedsQL 4.0 Generic Core Scales and the PedsQL 3.0 Rheumatology Module in pediatric rheumatology. METHODS: The 4 PedsQL 4.0 Generic Core Scales (physical, emotional, social, and school functioning) and the 5 PedsQL 3.0 Rheumatology Module scales (pain and hurt, daily activities, treatment, worry, and communication) were administered to 231 children and 244 parents (271 subjects accrued overall) recruited from a pediatric rheumatology clinic. RESULTS: Internal consistency reliability for the PedsQL Generic Core total scale score (alpha = 0.91 for child self report, alpha = 0.93 for parent proxy report), physical health summary score (alpha = 0.87 for child self report, alpha = 0.89 for parent proxy report), and psychosocial health summary score (alpha = 0.86 for child self report, alpha = 0.90 for parent proxy report) were acceptable for group comparisons. The Rheumatology Module scales also demonstrated acceptable reliability for group comparisons (alpha = 0.75-0.86 for child self report, alpha = 0.82-0.91 for parent proxy report). Validity was demonstrated using the known-groups method. The PedsQL distinguished between healthy children and children with rheumatic diseases as a group. The responsiveness of the PedsQL was demonstrated through patient change over time as a result of clinical intervention. CONCLUSION: The results demonstrate the reliability, validity, and responsiveness of the PedsQL 4.0 Generic Core Scales and the PedsQL 3.0 Rheumatology Module in pediatric rheumatology.     

Factors affecting health-related quality of life in Thai children with thalassemia

Background

Knowledge of the factors associated with health-related quality of life (HRQOL) among patients with thalassemia is essential in developing more suitable clinical, counseling, and social support programs to improve treatment outcomes of these patients. In light of the limited research in this area, this study aims to examine factors associated with HRQOL among children and adolescents with thalassemia in Thailand.

Methods

A cross-sectional survey was conducted in three selected hospitals in Thailand during June to November 2006. PedsQL? 4.0 Generic Core Scale (Thai version) was used to assess HRQOL in 315 thalassemia patients between 5 and 18 years of age. Other related clinical characteristics of the patients were collected via medical record review.

Results

The mean (SD) of the total summary score was 76.67 (11.40), while the means (SD) for the Physical Health Summary score and Psychosocial Health Summary score were 78.24 (14.77) and 75.54 (12.76), respectively. The school functioning subscale scored the lowest, with a mean of 67.89 (SD = 15.92). The following factors significantly affected the HRQOL of the patients: age; age at onset of anemia and age at first transfusion; pre-transfusion hemoglobin (Hb) level; receiving a blood transfusion during the previous three months; and disease severity. In addition, iron chelation therapy had a significant negative effect on HRQOL in the school functioning subscale. In contrast, serum ferritin level, frequency of blood transfusions per year, and gender were not significantly related to HRQOL among these patients. The results from multivariate analysis also confirmed these findings.

Conclusions

To improve HRQOL of thalassemia patients, suitable programs aimed at providing psychosocial support and a link between the patient, school officials, the family and the physician are important, especially in terms of improving the school functioning score. The findings also confirmed the importance of maintaining a pre-transfusion Hb level of at least 9-10.5 g/dL. In addition, special care and attention should be given to patients with a severe condition, and those who are receiving subcutaneous iron chelation therapy.

     

Short stature and truncal shortening in transfusion dependent thalassemia patients: results from a thalassemia center in Malaysia

One of the major complications in patients with transfusion dependent thalassemia is growth impairment secondary to iron overload. We studied the growth status in 66 patients with beta-thalassemia major and HbE-beta thalassemia who were transfusion dependent, aged from 2 to 24 years, and 66 controls matched for sex and age. The prevalence of short stature in transfusion-dependent thalassemics was 54.5% compared to 4.5% in control group (p<0.001). Short stature was more prevalent in those above the age of 10 years in this study group (83.3% vs 16.7%). Transfusion dependent thalassemics with short stature were found to have significantly lower mean standing height standard deviation scores (SDS), sitting height SDS and subischial leg length SDS values (p<0.001). There was also a significant difference between the mean sitting height SDS and the mean subischial leg length SDS in our thalassemics with short stature, suggesting that the short stature was due to disproportionate truncal shortening. Serum ferritin levels were significantly higher in transfusion dependent thalassemics who were short compared to those who were of normal height (p = 0.002). However, the mean pre-transfusion hemoglobin levels did not differ significantly between patients with short stature and those with normal height (p = 0.216). The prevalence of short stature also did not differ significantly between those with beta-thalassemia major and those with HbE-beta thalassemia (p = 0.32). This study highlighted the importance of providing optimal treatment in these patients, including monitoring of growth parameters and optimizing iron chelation therapy.     

The PedsQL Multidimensional Fatigue Scale in pediatric rheumatology: reliability and validity

OBJECTIVE:. The PedsQL (Pediatric Quality of Life Inventory) is a modular instrument designed to measure health related quality of life (HRQOL) in children and adolescents ages 2-18 years. The recently developed 18-item PedsQL Multidimensional Fatigue Scale was designed to measure fatigue in pediatric patients and comprises the General Fatigue Scale (6 items), Sleep/Rest Fatigue Scale (6 items), and Cognitive Fatigue Scale (6 items). The PedsQL 4.0 Generic Core Scales were developed as the generic core measure to be integrated with the PedsQL Disease-Specific Modules. The PedsQL 3.0 Rheumatology Module was designed to measure pediatric rheumatology-specific HRQOL. Methods. The PedsQL Multidimensional Fatigue Scale, Generic Core Scales, and Rheumatology Module were administered to 163 children and 154 parents (183 families accrued overall) recruited from a pediatric rheumatology clinic. Results. Internal consistency reliability for the PedsQL Multidimensional Fatigue Scale Total Score (a = 0.95 child, 0.95 parent report), General Fatigue Scale (a = 0.93 child, 0.92 parent), Sleep/Rest Fatigue Scale (a = 0.88 child, 0.90 parent), and Cognitive Fatigue Scale (a = 0.93 child, 0.96 parent) were excellent for group and individual comparisons. The validity of the PedsQL Multidimensional Fatigue Scale was confirmed through hypothesized intercorrelations with dimensions of generic and rheumatology-specific HRQOL. The PedsQL Multidimensional Fatigue Scale distinguished between healthy children and children with rheumatic diseases as a group, and was associated with greater disease severity. Children with fibromyalgia manifested greater fatigue than children with other rheumatic diseases. CONCLUSION: The results confirm the initial reliability and validity of the PedsQL Multidimensional Fatigue Scale in pediatric rheumatology.     

Comparison of twice-daily vs once-daily deferasirox dosing in a gerbil model of iron cardiomyopathy

OBJECTIVE: Despite the availability of deferoxamine chelation therapy for more than 20 years, iron cardiomyopathy remains the leading cause of death in thalassemia major patients. Effective chelation of cardiac iron is difficult; cardiac iron stores respond more slowly to chelation therapy and require a constant gradient of labile iron species between serum and myocytes. We have previously demonstrated the efficacy of once-daily deferasirox in removing previously stored cardiac iron in the gerbil, but changes in cardiac iron were relatively modest compared with hepatic iron. We postulated that daily divided dosing, by sustaining a longer labile iron gradient from myocytes to serum, would produce better cardiac iron chelation than a comparable daily dose. METHODS: Twenty-four 8- to 10-week-old female gerbils underwent iron dextran-loading for 10 weeks, followed by a 1-week iron equilibration period. Animals were divided into three treatment groups of eight animals each and were treated with deferasirox 100 mg/kg/day as a single dose, deferasirox 100 mg/kg/day daily divided dose, or sham chelation for a total of 12 weeks. Following euthanasia, organs were harvested for quantitative iron and tissue histology. RESULTS: Hepatic and cardiac iron contents were not statistically different between the daily single-dose and daily divided-dose groups. However, the ratio of cardiac to hepatic iron content was lower in the divided-dose group (0.78% vs 1.11%, p = 0.0007). CONCLUSION: Daily divided dosing of deferasirox changes the relative cardiac and liver iron chelation profile compared with daily single dosing, trading improvements in cardiac iron elimination for less-effective hepatic chelation.     

The Proceedings of the 17th International Conference on Chelation: Application of Effective Chelation Therapies in Iron Loading and Non Iron Loading Conditions,and the Gap in the Prevention and Treatment Policies on Thalassemia Between Developed and Developing Countries

Substantial progress in the use of chelating drugs for the treatment of iron overload and of non iron loading conditions has been presented during the 17th International Conference on Chelation (ICOC) held in November 2007 at Shenzhen, China. Major challenges lie ahead for the prevention and treatment of thalassemia in China, India, Thailand, Indonesia and many other developing countries where millions of heterozygote thalassemia carriers live and thousands of homozygote thalassemia patients are born annually. The progressive improvement of the economic climate in developing countries will increase the demand and resources for more prenatal and antenatal diagnoses, transfusions and chelation therapy in forthcoming years. Despite the major advances in diagnosis and treatment in developed countries, the vast majority of thalassemia patients in developing countries die untreated because they cannot afford the cost of transfusions and chelation therapy. New approaches and infrastructures and more efforts are needed to overcome the difficulties of supplying new techniques and treatments to patients in developing countries. International and local organizations need to be persuaded to act collectively and effectively to improve chelation and related treatments for thalassemia and other conditions, especially at this time that universally effective and inexpensive chelation therapies can be applied.     

Development of a parent-proxy quality-of-life chronic cough-specific questionnaire: clinical impact vs psychometric evaluations

BACKGROUND: Chronic cough affects at least 7% of children, and the impact of this on families is significant. Although adult cough-specific quality-of-life (QOL) instruments have been shown to be a useful cough outcome measure, no suitable cough-specific QOL for parents of children with chronic cough exists. This article compares two methods of item reduction (clinical impact and psychometric) and reports on the statistical properties of both QOL instruments. METHOD: One hundred seventy children (97 boys and 73 girls; median age, 4 years; interquartile range, 3 to 7.25 years) and one of their parents participated. A preliminary 50-item parent cough-specific QOL (PC-QOL) questionnaire was developed from conversations with parents of children with chronic cough (ie, cough for > 3 weeks). Parents also completed generic QOL questionnaires (eg, Pediatric Quality of Life Inventory, version 4.0 [PedsQL4.0] and the 12-item Short Form Health Survey, version 2 [SF-12v2]). RESULTS: The clinical impact and psychometric method of item reduction resulted in 27-item and 26-item PC-QOL questionnaires, respectively, with approximately 50% of items overlapping. Internal consistency among the final items from both methods was excellent. Some evidence for concurrent and criterion validity of both methods was established as significant correlations were found between subscales of the PC-QOL questionnaire and the scales of the SF-12v2 and PedsQL4.0 scores. The PC-QOL questionnaire derived from both methods was sensitive to change following an intervention. CONCLUSION: Chronic cough significantly impacts on the QOL of both parents and children. Although the PC-QOL questionnaires derived from a clinical impact method and from a psychometric method contained different items, both versions were shown to be internally consistent and valid. Further testing is required to compare both final versions to objective and subjective cough measures.     

Prokinetic agents in childen with poor appetite

BACKGROUND: Of the feeding disorders in children, poor appetite is probably one of the most common complaints notified by parents. Since gastric motility disorders may be the cause of this symptom, the aim of our study was to investigate the effect of prokinetics on children with poor appetite and delayed gastric emptying. METHODS: Poor appetite was graded by VAS at start and end of treatment. Delayed gastric emptying was assessed by 99mTechnetium tincolloid scintigraphy. Malnutrition was defined according to Waterlow criteria. After initial assessment behavioural feeding recommendations were provided and trimebutine given for 6 months. Scintigraphy was repeated during treatment. Anthropometrical measurements and daily energy intake calculations were performed monthly RESULTS: 21 children (M/F: 12/9) with a mean age of 7 +/- 3 years were studied. At the end of treatment, weight and height standard deviation scores improved significantly (-1.0 +/- 0.6 vs. -0.8 +/- 0.7, p = 0.008 and -1.1 +/- 1.0 vs. -0.8 +/- 0.9, p=0.003, respectively) and malnutrition rate decreased from 81% to 66.7%. Improvement of gastric emptying was shown in 11 out of 16 children but was not statiscally significant (P = 0.059). The visual analogue scale scores of appetite showed significant improvement with therapy (7.5 +/- 1.3 vs. 5.3 +/- 2.0, p = 0.012). CONCLUSION : Children with poor appetite who have delayed gastric emptying might benefit from prokinetic agent therapy combined with behavioural feeding recommendations.     

Quality of life in haemophilia A: Hemophilia Utilization Group Study Va (HUGS-Va)

Summary. This study describes health‐related quality of life (HRQoL) of persons with haemophilia A in the United States (US) and determines associations between self‐reported joint pain, motion limitation and clinically evaluated joint range of motion (ROM), and between HRQoL and ROM. As part of a 2‐year cohort study, we collected baseline HRQoL using the SF‐12 (adults) and PedsQL (children), along with self‐ratings of joint pain and motion limitation, in persons with factor VIII deficiency recruited from six Haemophilia Treatment Centres (HTCs) in geographically diverse regions of the US. Clinically measured joint ROM measurements were collected from medical charts of a subset of participants. Adults (N = 156, mean age: 33.5 ± 12.6 years) had mean physical and mental component scores of 43.4 ± 10.7 and 50.9 ± 10.1, respectively. Children (N = 164, mean age: 9.7 ± 4.5 years) had mean total PedsQL, physical functioning, and psychosocial health scores of 85.9 ± 13.8, 89.5 ± 15.2, and 84.1 ± 15.3, respectively. Persons with more severe haemophilia and higher self‐reported joint pain and motion limitation had poorer scores, particularly in the physical aspects of HRQoL. In adults, significant correlations (P < 0.01) were found between ROM measures and both self‐reported measures. Except among those with severe disease, children and adults with haemophilia have HRQoL scores comparable with those of the healthy US population. The physical aspects of HRQoL in both adults and children with haemophilia A in the US decrease with increasing severity of illness. However, scores for mental aspects of HRQoL do not differ between severity groups. These findings are comparable with those from studies in European and Canadian haemophilia populations.     

The relationship between health-related quality of life, pain and coping strategies in juvenile idiopathic arthritis

OBJECTIVES: To investigate the relationship between health-related quality of life (HRQL), experience of pain and pain coping strategies in children with juvenile idiopathic arthritis (JIA). To compare reports describing these variables obtained from children and their parents. METHODS: Participants were 59 children aged 8 to 18 yr with JIA and their parents. Parents and children completed the PedsQL generic core scales and arthritis module, the visual analogue scale of the Varni-Thompson Pediatric Pain Questionnaire, and the Waldron/Varni Pediatric Pain Coping Inventory. Parents rated children's functional disability using the Childhood Health Assessment Questionnaire. RESULTS: Parents reported significantly lower scores (indicating worse HRQL) than children on five of the eight PedsQL scales rating children's HRQL. Parents and children reported a significant negative relationship between pain levels and the PedsQL scores assessing children's physical, emotional and social functioning. They also reported a significant negative relationship between scores on several pain coping scales and scores on the PedsQL scales. However, the pattern of these relationships varied for reports from parents and children. CONCLUSIONS: Pain intensity and pain coping strategies have a significant and independent relationship with several domains that comprise the HRQL of children with JIA. However, parents and children have differing perceptions of the nature of these relationships. The differences emphasize the importance of clinicians obtaining information about children's HRQL, pain levels and pain coping strategies from both parents and children.     

The influence of hemochromatosis mutations on iron overload of thalassemia major.

BACKGROUND AND OBJECTIVE: Hemochromatosis is a genetic form of iron overload due to a defective HFE gene. Secondary iron overload is the main complication in transfusion-dependent thalassemia patients. In this work we have examined the prevalence of HFE mutations in thalassemia major and evaluated the degree of iron overload of patients with and without HFE mutations. DESIGN AND METHODS: HFE mutations were studied in 71 Italian thalassemic patients and in 189 normal controls, using PCR and restriction enzyme analysis. The degree of iron overload, assessed by serum ferritin and liver iron concentration (LIC), was compared in 17 patients with mutations in the HFE gene, and in 17 subjects with wild type HFE genotype. The two groups of patients had comparable globin gene mutations, were matched for age and were homogeneous for transfusion and chelation history. In all cases the iron balance calculated on the basis of transfusion regimen and iron excreted by chelation was available. RESULTS: The allele frequencies of C282Y and H63D were respectively 1.4% and 12.7% in patients and 1.1% and 11.4% in controls. No case of C282Y homozygosity was recorded among patients. No significant difference was found in terms of serum ferritin, LIC, or the age at chelation start between patients with and without HFE mutations. The single patient with H63D homozygosity was severely iron-loaded. INTERPRETATION AND CONCLUSIONS: Our data suggest that the presence of a single mutation in the HFE gene does not influence the severity of iron loading in thalassemia patients following a regular transfusion and chelation program.     

Quality of Life Outcomes in a Pediatric Thalassemia Population in Egypt

Thalassemia is a disorder of hemoglobin (Hb) synthesis characterized by chronic hemolysis. In β-thalassemias major (β-TM), patients require regular transfusion at an early age due to severe anemia. Subsequently, intensive chelation therapy is initiated to mitigate the effects of the resultant iron overload. Clinical disease burden and the demanding treatment can affect health-related quality of life (HRQoL) outcomes in this population. The aim of this study was to assess HRQoL outcomes in Egyptian pediatric thalassemia patients. Patients were enrolled simultaneously from the hematology clinic at the National Research Institute in Cairo, Egypt. The Arabic version of SF36 tool was used to assess HRQoL outcomes. Socioeconomic data were collected by patient and parent interviews. Clinical data were collected by review of medical records. One hundred and thirty patients and 60 controls were enrolled, with a mean age of 5.4?±?3.2?years and 6.3?±?3.0, respectively. The HRQoL outcome scores were lower in all domains in the thalassemia group compared to the control group (p?=?0.0001). Transfusion-dependent (TD) patients had lower HRQoL scores compared to nontransfusion-dependent (NTD) patients (p?=?0.0001). Patient education and maternal education were independently associated with better HRQoL scores (p?=?0.007, p?=?0.028, respectively). Residents of rural areas reported lower scores compared to urban residents (p?=?0.026). Thalassemia was associated with lower HRQoL scores, in all domains, compared to HRQoL in unaffected controls. Chronic transfusion independence, patient education, and maternal education were all associated with higher HRQoL scores. Psychological, social, and economic support for families with thalassemia are all essential tools to improve HRQoL outcomes.     

The PedsQL™ in pediatric rheumatology: Reliability,validity, and responsiveness of the Pediatric Quality of Life Inventory™ generic core scales and rheumatology module

Objective

The Pediatric Quality of Life Inventory (PedsQL) is a modular instrument designed to measure health‐related quality of life (HRQOL) in children and adolescents ages 2–18 years. The 23‐item PedsQL 4.0 Generic Core Scales are multidimensional child self‐report and parent proxy‐report scales developed as the generic core measure to be integrated with the PedsQL disease‐specific modules. The 22‐item PedsQL 3.0 Rheumatology Module was designed to measure pediatric rheumatology–specific HRQOL. This study was undertaken to demonstrate the reliability, validity, and responsiveness of the PedsQL 4.0 Generic Core Scales and the PedsQL 3.0 Rheumatology Module in pediatric rheumatology.

Methods

The 4 PedsQL 4.0 Generic Core Scales (physical, emotional, social, and school functioning) and the 5 PedsQL 3.0 Rheumatology Module scales (pain and hurt, daily activities, treatment, worry, and communication) were administered to 231 children and 244 parents (271 subjects accrued overall) recruited from a pediatric rheumatology clinic.

Results

Internal consistency reliability for the PedsQL Generic Core total scale score (α = 0.91 for child self report, α = 0.93 for parent proxy report), physical health summary score (α = 0.87 for child self report, α = 0.89 for parent proxy report), and psychosocial health summary score (α = 0.86 for child self report, α = 0.90 for parent proxy report) were acceptable for group comparisons. The Rheumatology Module scales also demonstrated acceptable reliability for group comparisons (α = 0.75–0.86 for child self report, α = 0.82–0.91 for parent proxy report). Validity was demonstrated using the known‐groups method. The PedsQL distinguished between healthy children and children with rheumatic diseases as a group. The responsiveness of the PedsQL was demonstrated through patient change over time as a result of clinical intervention.

Conclusion

The results demonstrate the reliability, validity, and responsiveness of the PedsQL 4.0 Generic Core Scales and the PedsQL 3.0 Rheumatology Module in pediatric rheumatology.

     

Pilot study on parental stress and behavioral adjustment to the thalassemia major disease process in children undergoing iron-chelation in western Taiwan

Thalassemia was first described by Cooley and Lee in 1952 in several Italian children as a severe anemia with spleen and liver enlargement, skin discoloration, and bony changes. Great strides in management and intervention have not been matched by progress in psychosocial rehabilitation. Because parental stress and adaptation are of concern, this study focuses on parental stress and adjustment in response to the disease process of their afflicted children in western Taiwan. The parents of 18 thalassemia major patients (under 12 years of age) were interviewed (in two sessions) to determine their feelings, sources of stress, and support during their childrens' disease process. The study found that: 1) many parents suffer from stress as a result of the disease process, 2) all parents had similar concerns about iron chelation treatment, and 3) some resilience factors were present in the support system.     

Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort

Morbidity and mortality in thalassemia are associated with iron burden. Recent advances in organ-specific iron imaging and the availability of oral deferasirox are expected to improve clinical care, but the extent of use of these resources and current chelation practices have not been well described. In the present study, we studied chelation use and the change in iron measurements in 327 subjects with transfusion-dependent thalassemia (mean entry age, 22.1 ± 2.5 years) from 2002-2011, with a mean follow-up of 8.0 years (range, 4.4-9.0 years). The predominant chelator currently used is deferasirox, followed by deferoxamine and then combination therapies. The use of both hepatic and cardiac magnetic resonance imaging increased more than 5-fold (P < .001) during the study period, leading to an 80% increase in the number of subjects undergoing liver iron concentration (LIC) measurements. Overall, LIC significantly improved (median, 10.7 to 5.1 mg/g dry weight, P < .001) with a nonsignificant improvement in cardiac T2* (median, 23.55 to 34.50 ms, P = .23). The percentage of patients with markers of inadequate chelation (ferritin > 2500 ng/mL, LIC > 15 mg/g dry weight, and/or cardiac T2* < 10 ms) also declined from 33% to 26%. In summary, increasing use of magnetic resonance imaging and oral chelation in thalassemia management has likely contributed to improved iron burden.     

Pilot Study on Parental Stress and Behavioral Adjustment to the Thalassemia Major Disease Process in Children Undergoing Iron-Chelation in Western Taiwan

Thalassemia was first described by Cooley and Lee in 1952 in several Italian children as a severe anemia with spleen and liver enlargement, skin discoloration, and bony changes. Great strides in management and intervention have not been matched by progress in psychosocial rehabilitation. Because parental stress and adaptation are of concern, this study focuses on parental stress and adjustment in response to the disease process of their afflicted children in western Taiwan. The parents of 18 thalassemia major patients (under 12 years of age) were interviewed (in two sessions) to determine their feelings, sources of stress, and support during their childrens' disease process. The study found that: 1) many parents suffer from stress as a result of the disease process, 2) all parents had similar concerns about iron chelation treatment, and 3) some resilience factors were present in the support system.     

Safety and efficacy of early start of iron chelation therapy with deferiprone in young children newly diagnosed with transfusion‐dependent thalassemia: A randomized controlled trial

Iron overload is inevitable in patients who are transfusion dependent. In young children with transfusion‐dependent thalassemia (TDT), current practice is to delay the start of iron chelation therapy due to concerns over toxicities, which have been observed when deferoxamine was started too early. However, doing so may increase the risk of iron accumulation that will be manifested as toxicities later in life. This study investigated whether deferiprone, a chelator with a lower affinity for iron than deferoxamine, could postpone transfusional iron overload while maintaining a good safety profile. Recently diagnosed TDT infants (N = 64 their age ranged from 10 to 18 (median 12) months, 54.7% males; receiving ≤6 transfusions; serum ferittin (SF) >400 to < 1000 ng/mL were randomized to “early start deferiprone” (.ES‐DFP) at a low dose (50 mg/kg/day) or to “delay chelation” (DC), and remained in the study until their serum ferritin (SF) level reached ≥1000 μg/L. 61 patients continued the study Levels of transferrin saturation (TSAT) and labile plasma iron (LPI) were measured as well. By approximately 6 months postrandomization, 100% of the subjects in DC group had achieved SF > 1000 µg/L and TSAT > 70% compared with none in the ES‐DFP group. LPI level > 0.6 µM was observed in 97% vs. 40% of the DS and ES groups, respectively, (P < 0.001). The time to reach SF > 1000 µg/L was delayed by 6 months in the ES‐DFP group (P < 0.001) without escalating DFP dose. No unexpected, serious, or severe adverse events were seen in the ES‐DFP group.     

Reliability,Validity, and Responsiveness of the Pediatric Quality of Life Inventory™ (PedsQL™) Generic Core Scales and Asthma Symptoms Scale in Vulnerable Children With Asthma

Background. Patient-reported outcomes such as health-related quality of life (HRQOL) are increasingly used as primary endpoints in clinical trials. The Pediatric Quality of Life Inventory? (PedsQL?) is widely used as a measure of HRQOL and may be a particularly suitable primary outcome in pediatric asthma clinical trials. Objectives. To examine the reliability, validity, and responsiveness to clinical change of the PedsQL? 4.0 Generic Core Scales and PedsQL? Asthma Module Asthma Symptoms Scale in a sample of vulnerable children with persistent asthma recruited from Federally Qualified Health Centers. Methods. Children (N = 252; ages 3 to 14 years) with persistent asthma (27% mild, 40.9% moderate, 32.1% severe) and their parents (93.7% mother, 83.3% Hispanic, 76.9% Spanish-speaking, 72.6% less than a high school diploma) enrolled in a clinical trial completed the PedsQL? 4.0 Generic Core Scales, the PedsQL? 3.0 Asthma Module Asthma Symptoms Scale, and a measure of asthma symptom frequency (used as an indicator of clinical change) at baseline and 3-month follow-up. Results. The PedsQL? demonstrated adequate internal consistency reliability and convergent and discriminative validity. Based on intra- and intersubject change, effect sizes, and standard errors of measurement, the PedsQL? demonstrated responsiveness to clinical change. Conclusions. For both child self-report and parent proxy-report, the PedsQL? Generic Core Scales Total Scale score and the PedsQL? Asthma Symptoms Scale are suitable for use as primary asthma clinical trial outcomes.