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Fig. 3 | Orphanet Journal of Rare Diseases

Fig. 3

From: Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3)

Fig. 3

Changes in mean (± SD) DS3 score after initiation of GD1-specific treatment for patients with pre-treatment marked, moderate or mild disease as defined by baseline DS3 score a Baseline DS3 scores 6.00-14.87; b Baseline DS3 scores 3.00-5.93; c Baseline DS3 scores 0.40-2.93). Among 58 marked severity patients (baseline DS3 > 6), 38 patients (65.5 %) had a total of 82 new bone events (avascular necrosis, fracture, lytic lesions) while on treatment: 2.16 episodes per affected patient. Among 53 moderate severity patients (baseline DS3 3.00-5.99), 15 patients (28.3 %) had a total of 19 new bone events, 1.2 episodes per affected patient. Among 22 mild severity patients (baseline DS3 < 2.99), 4 patients (18.2 %) had a total of 4 events, 1.0 episode per affected patient

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172