Patient services: Development of the PedsQL Sickle Cell Disease Module

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The determination of health-related quality of life (HRQL) for children with sickle cell disease 1) provides a broader method to assess the impact of sickle cell disease and its treatments on children's health and 2) may improve the care provided to these children. Prior studies of the HRQL of adults and children with sickle cell disease have utilized generic measures of HRQL and shown the poor HRQL of those who suffer from this disease. However, use of such generic measures likely reduce the ability to detect disease-specific health issues as well as the ability to detect changes in clinical status over time. Because there are no pediatric disease-specific HRQL questionnaires for children with sickle cell disease, this prior work may limit our ability to evaluate additional aspects of well being that are specific to these children, such as chronic pain, effect of medications and hospitalizations. Ideally, both a generic and disease-specific instrument should be used to measure HRQL. The PedsQL¿ is the most widely used generic measure of HRQL in children that has companion disease-specific modules. This proposal seeks to develop and validate a disease-specific HRQL module for use in children with sickle cell disease. The underlying hypothesis of this work is that the PedsQL Sickle Cell Disease module will be an effective tool to measure the impact of sickle cell disease and treatment from the perspective of the child and parent. The project proposed here will lay the foundation for testing this hypothesis. The specific aims of this proposal encompass the four phases of isntrument development and are as follows: to identify disease-specific HRQL items representing the physical, social, emotional and cognitive well being of children with sickle cell disease, to construct a PedsQL Sickle Cell Disease Module that incorporates those HRQL areas found to be relevant to children with sickle cell disease and to determine pilot standard scores and psychometric properties for the PedsQL Sickle Cell Disease HRQL module. This project will result in a reliable and valid measure of HRQL of children with sickle cell disease that can be utilized to facilitate outcomes research as well as provide an alternative method to measure the effect of therapies in clinical trials for these children. The ultimate goal is to use this tool in clinical practice to improve the health and well being of children with sickle cell disease. This disease-specific questionnaire will be made available without fees for all academic researchers.