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Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease

Biegstraaten, M; Cox, TM; Belmatoug, N; Berger, MG; Collin-Histed, T; Vom Dahl, S; Di Rocco, M; ... Hollak, CE; + view all (2016) Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease. Blood Cells, Molecules, and Diseases 10.1016/j.bcmd.2016.10.008. (In press). Green open access

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Abstract

Gaucher Disease type 1 (GD1) is a lysosomal disorder that affects many systems. Therapy improves the principal manifestations of the condition and, as a consequence, many patients show a modified phenotype which reflects manifestations of their disease that are refractory to treatment. More generally, it is increasingly recognised that information as to how a patient feels and functions [obtained by patient- reported outcome measurements (PROMs)] is critical to any comprehensive evaluation of treatment. A new set of management goals for GD1 in which both trends are reflected is needed. To this end, a modified Delphi procedure among 25 experts was performed. Based on a literature review and with input from patients, 65 potential goals were formulated as statements. Consensus was considered to be reached when ≥75% of the participants agreed to include that specific statement in the management goals. There was agreement on 42 statements. In addition to the traditional goals concerning haematological, visceral and bone manifestations, improvement in quality of life, fatigue and social participation, as well as early detection of long-term complications or associated diseases were included. When applying this set of goals in medical practice, the clinical status of the individual patient should be taken into account.

Type: Article
Title: Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.bcmd.2016.10.008
Publisher version: http://doi.org/10.1016/j.bcmd.2016.10.008
Language: English
Additional information: Copyright © 2016 The Authors. Published by Elsevier B.V. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).
Keywords: Delphi study, Gaucher disease, Management goals, PROMs, Therapy
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: http://discovery.ucl.ac.uk/id/eprint/1545085
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