Quality of Life in Short Stature Youth

1 Introduction1.1 Purpose of the Report1.2 Outline of Chapters1.3 The QoLISSY Project2 Short Stature2.1 Epidemiology and Definition2.2 Clinical Aspects of Short Stature2.2.1 Aetiology2.2.2 Diagnosis2.2.2.1 Growth Hormone Treatment2.2.2.2 Idiopathic Short Stature2.2.2.3 Constitutional Delay in Growth and Puberty2.2.2.4 Small for Gestational Age2.3 Underlying Mechanisms2.3.1 Growth Hormone2.3.2 Normal Human Growth Pattern2.3.3 Psychological Aspects of Short Stature2.4 Treatment2.4.1 Options2.4.2 Indications for Recombinant Human GH (RHGH)2.4.3 Psycho-social Interventions3 Health-Related Quality of Life3.1 Health-Related Quality of Life Research in Children and Adolescents3.2 Health Related Quality of Life in Short Stature3.2.1 Assessment Instruments for Adults3.2.2 Assessment Instruments for Children3.3 Guidelines for the Development of Patient Reported Outcomes (PRO)4 Development of the QoLISSY Instruments4.1 Rationale for the Development4.2 Challenges in Developing a HrQoL Instrument4.3 Review of the Literature4.4 Description of the QoLISSY Methodology5 Focus Group with Conceptual Model and Item Development5.1 Objectives5.2 Methods5.2.1 Planning of the Focus Groups5.2.2 Focus Group Procedure5.2.3 Statement Transcription5.2.4 Focus Group Conduct and Transcripts5.2.5 Item Writing5.2.6 Translation Process5.3 Focus Group Results5.3.1 Patient Sample5.3.2 Card Sorting Results5.3.3 Domains and Sub-Domains of Quality of Life5.3.4 Preliminary Conceptual Model5.3.5 Item Development6 Pilot Test with Cognitive Debriefing and Item Selection6.1 Objectives6.2 Methods6.2.1 Patient Recruitment6.2.2 Pilot Test Questionnaire6.2.3 Cognitive Debriefing6.2.4 Data Management and Data Analysis6.2.5 Statistical Procedure6.2.5.1 Psychometric Analysis6.2.6 Item Selection6.3 Results6.3.1 Patient Sample6.3.2 Qualitative Item Analysis6.3.3 Quantitative Analysis of Pilot Test Material6.3.4 Summary of Item Reduction from Quantitative and Qualitative Analysis7 Field Test and Retest with Psychometric Testing7.1 Objectives of the Field Test7.2 Methods7.2.1 Study Design7.2.2 Recruitment Procedures7.2.3 Sample Size and Sample Characteristics7.2.4 Study Instruments and Variables7.2.5 Data Management and Statistical Analysis7.2.5.1 Item and Scale Characteristics7.2.5.2 Reliability7.2.5.3 Construct Validity7.2.5.4 Convergent Validity7.2.5.5 Known Groups/Discriminant Validity7.2.5.6 Agreement between Parent and Child Scores7.3 Result of the Field Test7.3.1 Description of the Patient Sample7.3.1.1 Parent Sample Characteristics7.3.2 Quantitative Analysis of Field Test7.3.3 Psychometric Analysis Child Data7.3.3.1 Scale Characteristics7.3.3.2 Robustness of the Scales7.3.3.3 Factor Analysis7.3.3.4 Age and Gender Effects7.3.3.5 Scale Intercorrelations7.3.3.6 Convergent Validity7.3.3.7 Discriminant Validity7.3.3.8 Regression with Total Quality of Life7.3.3.9 Differences in Satisfaction with Height7.3.4 Psychometric Analysis Parents Data7.3.4.1 Scale Characteristics7.3.4.2 Robustness of the Scales7.3.4.3 Factor Structure7.3.4.4 Age and Gender Effects7.3.4.5 Scale Intercorrelation7.3.4.6 Convergent Validity7.3.4.7 Discriminant Validity7.3.4.8 Regression with Total Quality of Life Score7.3.5 Concordance between Parent and Child Versions7.4 Country Specific Data Analysis7.4.1 Cross Cultural Samples7.4.2 Sociodemografic and Clinical Data7.4.3 Scale Characteristics and Reliabilities7.4.3.1 Scale Characteristics and Reliabilities: Germany7.4.3.2 Scale Characteristics and Reliabilities: UK7.4.3.3 Scale Characteristics and Reliabilities: France7.4.3.4 Scale Characteristics and Reliabilities: Sweden7.4.3.5 Scale Characteristics and Reliabilities: Spain7.4.4 Test-Retest Reliability per Country7.4.5 Comparisons Across Countries8 The QoLISSY Manual - Summary of the Questionnaire Development Process and Users Guide8.1 Summary of the Questionnaire8.1.1 Purpose and Development8.1.2 Pilot Test8.1.3 Field Test8.1.3.1 Self Report Data from Children and Adolescents8.1.3.2 Parents Data8.1.3.3 Country Specific Data Analysis8.1.4 Further Use and Cross Cultural Performance of the QoLISSY Instrument8.1.5 Guidelines for Users8.1.5.1 Instrument Description8.1.5.2 Transformation and Reference Values8.1.5.3 Administration - Suggested Uses8.1.5.4 Uses of the QoLISSY Measures in Individual Patients8.2 Conditions for Use8.3 Summary Sheet9 LiteratureAppendixQoLISSY CQoLISSY P