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Recommendations for measuring health‐related quality of life in children on anticoagulation

2012; Elsevier BV; Volume: 10; Issue: 12 Linguagem: Inglês

10.1111/jth.12018

1538-7933

A.K. Bruce, Mary Bauman, Sophie Jones, M. Patricia Massicotte, Paul Monagle,

Infant Development and Preterm Care

10:Bruce AK, Bauman ME, Jones S, Massicotte MP, Monagle P. Recommendations for measuring health‐related quality of life in children on anticoagulation. J Thromb Haemost 2012; 2596–8. Bruce AK, Bauman ME, Jones S, Massicotte MP, Monagle P. Recommendations for measuring health‐related quality of life in children on anticoagulation. J Thromb Haemost 2012; 2596–8. Quality of life (QOL) is an abstract concept that has been defined by the World Health Organization (WHO) as ‘an individual’s perception of their position in life in the context of the culture and value systems … in relation to their goals, expectations, standards, and concerns’ [1Sabaté E. Adherence to Long‐Term Therapies: Evidence for Action. WHO, 2003Google Scholar]. The concept of health‐related QOL (HRQOL) has emerged, focusing on dimensions of QOL specifically related to health and therapeutic management strategies [2Jenney M.E. Campbell S. Measuring quality of life.Arch Dis Child. 1997; 77: 347-50Crossref PubMed Scopus (122) Google Scholar]. HRQOL in patients must be measured in order to allow the development of effective therapeutic choices, the creation of research strategies, and changes in policy for improvement of healthcare adherence and outcomes. What is considered desirable or undesirable by patients impacts on their personal QOLs, although this is not necessarily associated with morbidity as measured through medical definitions and methods. Therefore, HRQOL is now considered to be the ‘gold standard’ measurement for patient‐relevant outcomes [3Newcombe P.A. Sheffield J.K. Juniper E.F. Marchant J.M. Halsted R.A. Masters I.B. Chang A.B. Development of a parent‐proxy quality‐of‐life chronic cough‐specific questionnaire: clinical impact vs psychometric evaluations.Chest. 2008; 133: 386-95Abstract Full Text Full Text PDF PubMed Scopus (63) Google Scholar]. In clinical trials, HRQOL inventories can help to distinguish between drugs with apparently equal efficacy and safety, and also to provide patient‐reported adverse outcomes that might not be captured otherwise. QOL measurement in clinical trials has been endorsed by many organizations, such as the WHO, the Food and Drug Administration, the European Agency for the Evaluation of Medical Products, and the National Institute of Health [4Apolone G. De Carli G. Brunetti M. Garattini S. Health‐related quality of life (HR‐QOL) and regulatory issues: an assessment of the European Agency for the Evaluation of Medicinal Products (EMEA) recommendations on the use of HR‐QOL measures in drug approval.Pharmacoeconomics. 2001; 19: 187-95Crossref PubMed Scopus (81) Google Scholar, 5World Health OrganizationMeasurement of Quality of Life in Children. WHO, 1994Google Scholar, 6World Health OrganizationWHOQOL‐BREF: Introduction, Administration, Scoring and Generic Version of the Assessement. WHO, 1996Google Scholar]. Growing numbers of children are requiring thromboprophylaxis. At present, children on long‐term anticoagulation therapy (>3 months) are prescribed vitamin K antagonists or low molecular weight heparins, and this therapy alters their QOL [7Bruce A.A.K. Bauman M.E. Black K. Newton A. Legge L. Massicotte M.P. Development and preliminary evaluation of the KIDCLOT PAC QL©: a new health‐related quality of life measure for pediatric long‐term anticoagulation therapy.Thromb Res. 2011; 126: 116-212Abstract Full Text Full Text PDF Scopus (26) Google Scholar]. Although new agents are currently under investigation for evaluation of their pharmacodynamic, pharmacokinetic, safety and efficacy profiles in children, the impact of therapy on QOL, and hence patient preferences, must also be considered. Although pediatric patient numbers are small as compared with adults, children represent several treatment years, as many start long‐term anticoagulation in their early years or infancy [8Christensen T.D. Self‐management of oral anticoagulant therapy: a review.J Thromb Thrombolysis. 2004; 18: 127-43Crossref PubMed Scopus (31) Google Scholar]. This position paper recommends that HRQOL be evaluated in pediatric clinical trials for emerging anticoagulation agents. Pediatric patient numbers, combined with the heterogeneity of the population, limit the ability to capture significant results with traditional outcome measurements such as safety and efficacy. Evaluation of HRQOL in clinical trials should include the following: 1Both specific and generic QOL inventories must be utilized when novel anticoagulants for children are evaluated. The use of condition‐specific (HRQOL) in addition to generic QOL inventories has merit in providing a comprehensive picture of the child’s and parents’ perception of their therapy. Generic QOL inventories assess global QOL, whereas condition‐specific HRQOL inventories assess the influence of a particular disease or condition on QOL. Condition‐specific HRQOL inventories assess aspects of a specific health condition that are left unexplored in generic measures, and must be developed in relation to the condition. They are more sensitive to change over time, and are better discriminators of differences between subgroups within a disease category [9Marino B.S. Shera D. Wernovsky G. Tomlinson R.S. Aguirre A. Gallagher M. Lee A. Cho C.J. Stern W. Davis L. Tong E. Teitel D. Mussatto K. Ghanayem N. Gleason M. Gaynor J.W. Wray J. Helfaer M.A. Shea J.A. The development of the pediatric cardiac quality of life inventory: a quality of life measure for children and adolescents with heart disease.Qual Life Res. 2008; 17: 613-26Crossref PubMed Scopus (94) Google Scholar]. Generic pediatric QOL inventories are widely available, validated, and reliable [10Varni J.W. Burwinkle T.M. Lane M.M. Health‐related quality of life measurement in pediatric clinical practice: an appraisal and precept for future research and application.Health Qual Life Outcomes. 2005; 3: 34-43Crossref PubMed Scopus (343) Google Scholar, 11Varni J.W. Burwinkle T.M. Seid M. Skarr D. The PedsQL 4.0 as a pediatric population health measure: feasibility, reliability, and validity.Ambul Pediatr. 2003; 3: 329-41Abstract Full Text Full Text PDF PubMed Scopus (1576) Google Scholar, 12Varni J.W. Limbers C.A. The pediatric quality of life inventory: measuring pediatric health‐related quality of life from the perspective of children and their parents.Pediatr Clin North Am. 2009; 56: 843-63Abstract Full Text Full Text PDF PubMed Scopus (223) Google Scholar].2The QOL inventories used must have undergone acceptable psychometric testing. The following should be considered when the inventory is designed: purpose, focus, origins of items, opportunity for self‐reporting, the threat of negative wording damaging self‐esteem, number of items, time needed to complete the inventory, proxy reporting, adequate psychometric properties, and culture and language of population to be tested. Adequate psychometric properties of the QOL inventory include reliability, validity, and responsiveness testing. Further testing that is recommended by the National Institutes of Health [13National Institutes of HealthQuality of Life Methodology. National Institutes of Health, 2011Google Scholar] is either item response theory testing (IRT) or the Rasch method of testing; however, although these are laudable goals, the numbers of patients required for the performance of IRT or Rasch constitute a major limiting factor for most condition‐specific QOL inventories in pediatrics. In order to maintain rigor and continue developing condition‐specific QOL inventories, many authors are using new methods to compensate for the small patient volumes [14Klaassen R.J. Blanchette V.S. Barnard D. Wakefield C.D. Curtis C. Bradley C.S. Neufeld E.J. Buchanan G.R. Silva M.P. Chan A.K.C. Young N.L. Validity, reliability, and responsiveness of a new measure of health‐related quality of life in children with immune thrombocytopenic purpura: the kids’ ITP tools.J Pediatr. 2007; 150: 510-5Abstract Full Text Full Text PDF PubMed Scopus (75) Google Scholar].3In children, the parent–child dyad, the medical team–child relationship, language, social and developmental changes and the child’s life trajectory must be considered. Children’s perspectives, goals, choices and aims change as they develop. HRQOL inventories need to be developmentally appropriate, both in content and in level of comprehension, and need to be an appropriate length for the child’s developmental age [15Theunissen N.C.M. Vogels T.G.C. Koopman H.M. Verrips G.H.W. Zwinderman K.A.H. Verloove‐Vanhorick S.P. Wit J.M. The proxy problem: child report versus parent report in health‐related quality of life research.Qual Life Res. 1998; 7: 387-97Crossref PubMed Scopus (542) Google Scholar, 16Quittner A.L. Avani M. Ivette C. Systematic review of health‐related quality of life measures for children with respiratory conditions.Paediatr Respir Rev. 2008; 9: 220-32Crossref PubMed Scopus (44) Google Scholar, 17Eiser C. Morse R. Quality‐of‐life measures in chronic diseases of childhood.Health Technol Assess. 2001; 5: 1-147Crossref PubMed Scopus (606) Google Scholar, 18Eiser C. Morse R. A review of measures of quality of life for children with chronic illness.Arch Dis Child. 2001; 84: 205-11Crossref PubMed Scopus (501) Google Scholar].4To complement any data collected from child HRQOL inventories, parent‐proxy inventories should be included [15Theunissen N.C.M. Vogels T.G.C. Koopman H.M. Verrips G.H.W. Zwinderman K.A.H. Verloove‐Vanhorick S.P. Wit J.M. The proxy problem: child report versus parent report in health‐related quality of life research.Qual Life Res. 1998; 7: 387-97Crossref PubMed Scopus (542) Google Scholar, 16Quittner A.L. Avani M. Ivette C. Systematic review of health‐related quality of life measures for children with respiratory conditions.Paediatr Respir Rev. 2008; 9: 220-32Crossref PubMed Scopus (44) Google Scholar]. There is consensus among authors that parent‐proxy questionnaires alone do not provide adequate and reliable data about the QOL of children, and should therefore be used as an adjunct inventory to provide an additional perspective. [15Theunissen N.C.M. Vogels T.G.C. Koopman H.M. Verrips G.H.W. Zwinderman K.A.H. Verloove‐Vanhorick S.P. Wit J.M. The proxy problem: child report versus parent report in health‐related quality of life research.Qual Life Res. 1998; 7: 387-97Crossref PubMed Scopus (542) Google Scholar, 19Clarke M. daCruz E. Koehler J. Kaufman J. A multicenter survey of heparin prophylaxis practice in pediatric critical care.J Intensive Care Med. 2010; 26: 314-7Crossref Scopus (10) Google Scholar]. Parents commonly report their child’s health and QOL differently from how the child reports it [16Quittner A.L. Avani M. Ivette C. Systematic review of health‐related quality of life measures for children with respiratory conditions.Paediatr Respir Rev. 2008; 9: 220-32Crossref PubMed Scopus (44) Google Scholar, 20Berkes A. Varni J. Pataki I. Kardos L. Kemény C. Mogyorósy G. Measuring health‐related quality of life in Hungarian children attending a cardiology clinic with the Pediatric Quality of Life Inventory™.Eur J Pediatr. 2009; 169: 333-47Crossref PubMed Scopus (27) Google Scholar, 21Casey F.A. Craig B.G. Mulholland H.C. Quality of life in surgically palliated complex congenital heart disease.Arch Dis Child. 1994; 70: 382-6Crossref PubMed Scopus (64) Google Scholar, 22Ghez O. Liesner R. Karimova A. Ng C. Goldman A. van Doorn C. Subcutaneous low molecular weight heparin for management of anticoagulation in infants on Excor ventricular assist device.ASAIO J. 2006; 52: 705-7Crossref PubMed Scopus (10) Google Scholar], so a combination of self‐report and parent‐proxy reporting is ideal to give an accurate representation of QOL.5Cultural and language adaptations are required, owing to the international nature of many clinical trials, to produce versions that are conceptually equivalent [23Swaine‐Verdier A. Doward L.C. Hagell P. Thorsen H. McKenna S.P. Adapting quality of life instruments.Value Health. 2004; 7: S27-30Abstract Full Text PDF PubMed Scopus (164) Google Scholar]. This includes forward and backward translation, and revalidation with the specific patient group within each culture. The requisite adaptations face additional challenges in children and novel strategies have been investigated to maintain sound methodology with small patient numbers [24Price V.E. Klaassen R.J. Bolton‐Maggs P.H.B. Grainger J.D. Curtis C. Wakefield C. Dufort G. Riedlinger A. Soltner C. Blanchette V.S. Young N.L. Measuring disease‐specific quality of life in rare populations: a practical approach to cross‐cultural translation.Health Qual Life Outcomes. 2009; 7: 92-100Crossref PubMed Scopus (42) Google Scholar]. There is currently one HRQOL anticoagulation inventory that meets the majority of the criteria for development as outlined in this article. The KIDCLOT PAC QL [7Bruce A.A.K. Bauman M.E. Black K. Newton A. Legge L. Massicotte M.P. Development and preliminary evaluation of the KIDCLOT PAC QL©: a new health‐related quality of life measure for pediatric long‐term anticoagulation therapy.Thromb Res. 2011; 126: 116-212Abstract Full Text Full Text PDF Scopus (26) Google Scholar] is designed for the measurement of HRQOL for children on vitamin K antagonists, but can be easily modified for implementation as novel anticoagulants are evaluated, with the goal for approval for use in children. Cross‐cultural and language validation is underway. The influence of long‐term anticoagulation on children’s and family’s HRQOL is that they experience difficulty with the ‘tasks’ of warfarin, such as changing the dose, capillary blood collection, venous blood collection, and remembering to give the medication [7Bruce A.A.K. Bauman M.E. Black K. Newton A. Legge L. Massicotte M.P. Development and preliminary evaluation of the KIDCLOT PAC QL©: a new health‐related quality of life measure for pediatric long‐term anticoagulation therapy.Thromb Res. 2011; 126: 116-212Abstract Full Text Full Text PDF Scopus (26) Google Scholar]. Anticoagulation does affect how children/adolescents participate in social/physical activities, and they report that their interactions with friends, diet, alcohol, rough housing and sports are negatively altered. Parents report concern regarding long‐term effects of anticoagulation: family stress, feeling trapped, feeling that anticoagulation is a burden in their lives, and having their children limit their activities [7Bruce A.A.K. Bauman M.E. Black K. Newton A. Legge L. Massicotte M.P. Development and preliminary evaluation of the KIDCLOT PAC QL©: a new health‐related quality of life measure for pediatric long‐term anticoagulation therapy.Thromb Res. 2011; 126: 116-212Abstract Full Text Full Text PDF Scopus (26) Google Scholar]. The impact that a single medication has on the lives of the patients and families cannot be understated. The emotional and physical burdens described in association with long‐term anticoagulation may lead to non‐adherence [25Casais P. Meschengieser S.S. Sanchez‐Luceros A. Lazzari M.A. Patients’ perceptions regarding oral anticoagulation therapy and its effect on quality of life.Curr Med Res Opin. 2005; 21: 1085-90Crossref PubMed Scopus (57) Google Scholar]. Non‐adherence results in decreased treatment safety and efficacy, clinical complications, and avoidable healthcare costs for both families and society [1Sabaté E. Adherence to Long‐Term Therapies: Evidence for Action. WHO, 2003Google Scholar]. Therefore, if practitioners can identify the barriers that patients and families face with long‐term anticoagulation, anticipatory interventions can be instituted. Consequently, as the licensing process is commenced for novel anticoagulants for use in pediatrics, HRQOL measurement will provide critical information for its implementation. HRQOL assessment enables more informed choices to be made about anticoagulant therapy, encouraging participation of the child and family in their health management with the goal of maximizing adherence. The ease of incorporating HRQOL measurement into clinical trial protocols and producing meaningful results consolidates the significant contribution of such an assessment. The authors state that they have no conflict of interest.