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EHA 2018 | Developing a patient-reported outcome instrument for Gaucher disease

There are several genetic instruments available to measure the impact of Gaucher Disease (GD) on a patient’s quality of life, but no specific measure by which patients can report this information themselves. Speaking from the 23rd Congress of the European Hematology Association (EHA) 2018, held in Stockholm, Sweden, Esther Oliva, MD, Esther Oliva, MD, Chair of the European Haematology Association Scientific Working Group (EHA-SWG), from the Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, Italy, discusses her project to design such an instrument in order to help clinicians gain a more in-depth understanding of the impact of GD on patients and inform their treatment plan.