• Medicina clinica · Nov 2024

    Identification of patient-reported outcomes measures (PROMs) and patient-reported experiences measures (PREMs) in Gaucher disease in Spain.

    • Pilar Giraldo, María Camprodón, Paloma Cerro Alcolea, Elena Gras-Colomer, Daiana Ibarretxe, Sunil Lakhwani, Elvira Mora, María Purificación Velasco Calderón, and Montserrat Morales-Conejo.
    • Hematology, Hospital Quironsalud, Zaragoza, Spain; Fundación Española para el Estudio y Tratamiento de la Enfermedad de Gaucher (FEETEG), Zaragoza, Spain. Electronic address: giraldocastellano@gmail.com.
    • Med Clin (Barc). 2024 Nov 15; 163 (9): 449457449-457.

    BackgroundPatient-reported outcome measures (PROMs) and patient-reported experiences measures (PREMs) are crucial for understanding the impact of GD on quality of life and patient's perceptions on care, but also to guide decision-making processes. Nevertheless, no specific PREMs in GD have been published, neither PROMs for Spanish GD patients have been developed.MethodsTwo project coordinators selected key-points to be included in a PROMs/PREMs questionnaire, and the scientific committee and a group of expert patients contributed to the initial draft. Then, 9 meetings with experts were held to discuss controversial points. After, a questionnaire with 103 items regarding symptomatology, aspects of daily life and care experience was developed. Finally, it was conducted a Delphi survey among a multidisciplinary group of experts in GD.ResultsConsensus was reached on 85 out of the 103 items. Recommendations on PROMs and PREMs regarding symptomatology, aspects of daily life and care experience were obtained. Consensus was reached on the importance of considering fatigue, concentration problems, and communication issues in GD patients using 5-step analog scales. Panelists recommended asking GD patients about the impact on social functioning and work/school performance. Finally, consensus was reached on considering care experiences, such as treatment satisfaction, treatment interruptions or transitions and healthcare professionals involved in patient's management to perceive patient's perceptions.ConclusionThis expert consensus may help developing GD-specific PROMs/PREMs for improving GD management. Properly developed and validated PROMs/PREMs may help decision-making, establishing patient-tailored therapeutic and follow-up goals.Copyright © 2024 The Authors. Published by Elsevier España, S.L.U. All rights reserved.

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