A Cross-Sectional Retrospective Study of Non-Splenectomized and Never-Treated Patients with Type 1 Gaucher Disease

• Published in: Journal of clinical medicine Vol. 9; no. 8; p. 2343
• Main Authors: Serratrice, Christine, Stirnemann, Jérôme, Berrahal, Amina, Belmatoug, Nadia, Camou, Fabrice, Caillaud, Catherine, Billette de Villemeur, Thierry, Dalbies, Florence, Cador, Bérengère, Froissart, Roseline, Masseau, Agathe, Brassier, Anaïs, Hivert, Bénédicte, Swiader, Laure, Bertchansky, Ivan, de Moreuil, Claire, Chabrol, Brigitte, Durieu, Isabelle, Leguy Seguin, Vanessa, Astudillo, Leonardo, Humbert, Sébastien, Pichard, Samia, Marcel, Catherine, Hau Rainsard, Isabelle, Bengherbia, Monia, Yousfi, Karima, Berger, Marc G.
• Format: Journal Article
• Language: English
• Published: Basel MDPI AG 22.07.2020; MDPI
• Subjects: Clinical medicine; Enzymes; Hematology; Hemoglobin; Human health and pathology; Laboratories; Life Sciences; Parkinson's disease; Patients; Surveillance; Thrombocytopenia; France; United States > US; risk factors; Gaucher disease; enzyme replacement therapy; disease course; lysosomal storage disorder; prognosis
• ISSN: 2077-0383; 2077-0383
• DOI: 10.3390/jcm9082343

Patients with type 1 Gaucher disease (GD1) present thrombocytopenia, anemia, organomegaly, and bone complications. Most experts consider that the less aggressive forms do not require specific treatment. However, little is known about the disease course of these forms. The objective of this cross-sec...