COSMIN database

A structured review of studies on health-related quality of life and economic evaluation in pediatric acute lymphoblastic leukemia

Pickard, A. S., Topfer, L. A., and Feeny, D. H.
BACKGROUND: A comprehensive review was made of the literature on the health-related quality of life (HRQL) and economic outcomes of children with acute lymphoblastic leukemia (ALL), the most common of all cancers in childhood.

OBJECTIVES: The primary objectives of the review were to locate and describe measures of HRQL used in pediatrics and in pediatric oncology that might be applicable to ALL, to summarize studies that have applied HRQL measures to ALL, to identify and summarize economic evaluations of the costs and consequences of care for pediatric ALL, and to identify areas requiring further research.

DATA SOURCES: To identify the HRQL literature in pediatric ALL, searches were run on the major biomedical and social science bibliographic databases. Search terms included a variety of MeSH and other thesaurus terms, text words, names of HRQL instruments, and the names of key authors in the field. The economic literature searches included most of the same databases, with the addition of the National Health Service Economic Evaluation Database and EconLit. Searches on specific authors and instruments and hand searches were also conducted.

STUDY SELECTION: Only English language studies published from 1975 through 2000 were included.

DATA EXTRACTION: Standardized data extraction forms were used to abstract information from HRQL and economic evaluation studies. Two reviewers independently screened the search results, and differences were resolved by consensus.

DATA SYNTHESIS: A number of generic HRQL measures, both adult and pediatric, have been applied in pediatric ALL. In addition, a number of pediatric oncology-specific instruments and pediatric oncology disease-specific instruments have been developed. Most of these instruments have been used to measure the health status of patients undergoing therapy. Despite the limited numbers of patients and resources available to assess HRQL measures in children with cancer, a fairly substantial body of literature has been published. Economic studies of pediatric ALL have only recently been undertaken. Most studies focus on a particular, narrow aspect of costs associated with the disease. There are relatively few cost-effectiveness studies that compare the costs and consequences of two or more treatment options. There are no published, comprehensive economic evaluations of pediatric ALL.

CONCLUSIONS: HRQL measures provide not only important information on the improvements offered by new therapies but also an outcome measure for economic evaluations. Recently developed HRQL measures and applications that include the direct assessments of children are important contributions. By the age of 7 or 8 years, children can generally provide reliable responses. Furthermore, children often provide information that is not available from parental reports (e.g., in the more subjective areas of pain and emotion). However, the use of multiple viewpoints, such as the patient, parent, and health professional perspectives, can provide valid and important complementary information. Expertise in HRQL measurement should be included in the design of most future trials. Funds for HRQL research should be made available to enhance the scope of HRQL activities by organizations such as the Children's Oncology Group. In the near future, further work to generate evidence of validity for available HRQL measures for use in children with ALL will be a high priority. Continuation of inquiries into the methods for HRQL assessment of younger children (i.e., preschoolers) is also a priority.
J Natl Cancer Inst Monogr
1052-6773 (Print)
Publication year:
General health perceptions / HRQoL:
Health-related quality of life
Children (0-18)
Diseases of and symptoms related to the blood and blood-forming organs and certain disorders involving the immune mechanism
Neoplasms and related symptoms
PRO / non-PRO:
Non-patient Reported Outcome
Patient Reported Outcome
Type of measurement instrument:
1 - Questionnaires
2 - Interviews
6 - Clinical rating scales
15D - 15 Dimensional Health-Related Quality Of Life Measure   
16D - Sixteen dimensional health-related measure
17D - Seventeen dimensional health-related measure
BASES - Behavioral, Affective, and Somatic Experiences Scale
BASES-C - Behavioral, Affective, and Somatic Experiences Scale - Child
BASES-P - Behavioral, Affective, and Somatic Experiences Scale-Parent
CHIP-AE - Child Health and Illness Profile-Adolescent Edition
CHQ - Child Health Questionnaire (versions: CHQ-SF; CHQ50)
CHRIs - Child Health Rating Inventories
DII-BMT - Disease Impairment Inventories—Bone Marrow Transplant
DUCATQOL - Dutch children’s quality of life questionnaire
FS II(R) - Functional Status II (R)
FSQ-S - Functional Status Questionnaire-Short
HAY - How Are You
HUI-2 - Health Utilities Index Mark 2
HUI-3 - Health Utilities Index-Mark 3
MPQOLQ - The Miami Pediatric Quality of Life Questionnaire
PCQL-32 - Pediatric Cancer Quality of Life Inventory
PCQL - Pediatric Cancer Quality of Life Inventory
PEDQOL® - quality of life in children and adolescents with cancer
PedsQL - Pediatric Quality of Life Inventory
PIE - Perceived Illness Experience
POQOLS - Pediatric Oncology Quality of Life Scale
PPSC - Play Performance Scale for Children
QWB - Quality of Well-Being Scale
RMH-PQLQ - Royal Marsden Hospital Paediatric Quality of Life Questionnaire [alias: RMH-PO-QOL]
Standard Gamble Utilities