The comprehensive assessment of health status in survivors of childhood cancer: application to high-risk acute lymphoblastic leukaemia

Feeny, David; Leiper, Alison; Barr, RD; Furlong, William; Torrance, G. W.; Rosenbaum, Peter; Weitzman, Sheila

doi:10.1038/bjc.1993.192

Cited by 88 publications

(56 citation statements)

References 27 publications

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“…Most limitations mentioned are lessened or overcome by the large number of patients for a rare disease, enhancing the power of the study and compensating for missing data. Other strengths are the longitudinal character of the study, with duration of 12½ years, and the use of standardized instruments that have been extensively validated in diverse populations 18, 27, 30, 44, 45. The latter also enables the comparison of the burden of VWM to other patient groups.…”

Section: Discussionmentioning

confidence: 99%

“…It has been used extensively as an outcome measure, also for neurological disorders, in patients from age 1 year 18, 19, 20, 21. It comprises distinct attributes of health status that enable valuation of function‐specific scores (single‐attribute utility scores) and generic multiattribute preference‐based measures of quality of life (HUI Health Index) at a point in time.…”

Section: Methodsmentioning

confidence: 99%

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Natural History of Vanishing White Matter

Hamilton

Lei

Vermeulen

et al. 2018

Annals of Neurology

173

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ObjectiveTo comprehensively describe the natural history of vanishing white matter (VWM), aiming at improving counseling of patients/families and providing natural history data for future therapeutic trials.MethodsWe performed a longitudinal multicenter study among 296 genetically confirmed VWM patients. Clinical information was obtained via disease‐specific clinical questionnaire, Health Utilities Index and Guy's Neurological Disability Scale assessments, and chart review.ResultsFirst disease signs occurred at a median age of 3 years (mode = 2 years, range = before birth to 54 years); 60% of patients were symptomatic before the age of 4 years. The nature of the first signs varied for different ages of onset. Overall, motor problems were the most common presenting sign, especially in children. Adolescent and adult onset patients were more likely to exhibit cognitive problems early after disease onset. One hundred two patients were deceased. Multivariate Cox regression analysis revealed a positive relation between age at onset and both preservation of ambulation and survival. Absence of stress‐provoked episodes and absence of seizures predicted more favorable outcome. In patients with onset before 4 years, earlier onset was associated with more severe disability and higher mortality. For onset from 4 years on, disease course was generally milder, with a wide variation in severity. There were no significant differences for sex or for the 5 eIF2B gene groups. The results confirm the presence of a genotype–phenotype correlation.InterpretationThe VWM disease spectrum consists of a continuum with extremely wide variability. Age at onset is a strong predictor for disease course. Ann Neurol 2018;84:274–288

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Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Natural History of Vanishing White Matter

Hamilton

Lei

Vermeulen

et al. 2018

Annals of Neurology

173

View full text Add to dashboard Cite

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“…When comparing these two groups in terms of their PIE scores and other measures, differences were found on one subscale only. Those who had completed treatment reported that the impact of the illness on integration in school was greater than for those still on maintenance treatment (mean (SD) [2][3][4][5] (1-6), p<005).…”

Section: Treatment Statusmentioning

confidence: 99%

Development of a measure to assess the perceived illness experience after treatment for cancer.

Eiser

Havermans

Craft

et al. 1995

Archives of Disease in Childhood

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The development of a scale to measure perceived illness experience in young people with cancer is described. Areas of concern were first identified through semistructured interviews conducted with children and adolescents. As a result, 78 items were generated to cover the main areas identified (physical appearance, interference with activity, peer rejection, integration in school, manipulation, parental behaviour, disclosure, preoccupation with illness, and impact of treatment). These items were rated (on five point scales) by 41 patients (mean age 14-6 years) and 35 oftheir parents. Measures of physical functioning (symptoms, functional disability, and restrictions) and psychological functioning (symptoms) were included for validation purposes. Test-retest reliability was calculated on the basis of ratings made by a subsample of parents on two separate occasions. A 34 item scale was constructed with four items in each of the areas identified above, except for physical appearance (n= 2). The scale has adequate internal reliability and validity. There were significant correlations between parents and their children on all subscales except for illness disclosure and impact of treatment, suggesting that parents may be less reliable informants for their children in these contexts. The scale has potential use in clinical contexts, in evaluating the psychosocial impact of different treatment regimens, and as an outcome measure in intervention work. (Arch Dis Child 1995; 72: 302-307)

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“…The HUI2 was originally developed to assess survivors of childhood cancer, 19 but has since been used to evaluate functional outcome in other pediatric populations. 17,18,[20][21][22] The HUI2 can describe 24,000 unique health states.…”

Section: Quality Of Life After Pediatric Liver Transplantmentioning

confidence: 99%

Health-related quality of life in long-term survivors of pediatric liver transplantation

et al. 2000

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The purpose of this study is to measure the health-related quality of life (HRQOL) in children who are long-term survivors of liver transplantation and to pilot the Liver Transplant Disability Scale (LTDS), a newly developed 12-point scale that quantifies chronic medical disability related to liver transplantation. This study is a crosssectional survey of 51 children surviving liver transplantation by at least 2 years, with a median age of 4.94 years. Functional capacity and utility scores were measured by the Health Utilities Index Mark II (HUI2), and chronic disease-specific medical disability was measured by the LTDS. HUI2 results were compared with a reference population. LTDS scores were compared with utility scores and patient survival 3 years later. Ninety percent of the study patients had functional deficits compared with 50% of controls. Functional impairment was typically mild. The resulting mean utility score, 0.86 ؎ 0.13 (0 ‫؍‬ dead, 1 ‫؍‬ perfect health), was significantly less than that of the reference population, 0.95 ؎ 0.07 (P F .001). LTDS scores ranged from 0 (no disability) to 6 (moderate disability). Seventy-one percent of the children had mild disability (scores 0 to 3), and 29% had moderate disability (scores 4 to 6). LTDS scores did not correlate with utility scores but were predictive of survival. The majority of pediatric liver transplant recipients have mild functional deficits. Their utility scores reflected a high level of HRQOL but were significantly less than those of a reference population. The majority also had mild medical disability, predominantly delayed growth. Medical disability did not correlate with HRQOL but predicted survival 3 years later. Liver Transpl 2000;6:333-339.)

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The comprehensive assessment of health status in survivors of childhood cancer: application to high-risk acute lymphoblastic leukaemia

Cited by 88 publications

References 27 publications

Natural History of Vanishing White Matter

Natural History of Vanishing White Matter

Development of a measure to assess the perceived illness experience after treatment for cancer.

Health-related quality of life in long-term survivors of pediatric liver transplantation

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