Version May 2024
| Category | Management goals |
| Anemia-related symptoms | Eliminate blood transfusion dependency |
| Increase hemoglobin levels within 12 to 24 months to >11.0 g/dL for females and children and >12.0 g/dL for males | |
| Bleeding tendency | Increase platelet counts during the first year of treatment sufficiently to prevent surgical, obstetrical, and spontaneous bleeding |
| In patients with splenectomy – Normalization of platelet count by one year of treatment | |
| In patients with an intact spleen – Achieve platelet count of ≥100,000/mm3 by 3 years of treatment | |
| Mobility | Lessen bone pain that is not related to irreversible bone disease within 1 to 2 years |
| Decrease bone marrow involvement, as measured by a locally used scoring system (eg, BMB score or DGS) in patients without severe irreversible bone disease at baseline | |
| Increase BMD by 2 years in adults for patients with a T-score below –2.5 at baseline | |
| Attain normal or ideal peak skeletal mass in children | |
| Normalize growth such that the height of the patient is in line with target height, based upon population standards and parental height, within 2 years of treatment | |
| Visceral complications | Avoid splenectomy (may be necessary during life-threatening hemorrhagic events) |
| Alleviate symptoms due to splenomegaly: abdominal distension, early satiety, new splenic infarction | |
| Eliminate hypersplenism | |
| Reduce spleen volume to <2 to 8 times normal (or in absence of volume measurement tools reduce spleen size) by year 1 to 2, depending on baseline spleen volume | |
| Reduce the liver volume to 1 to 1.5 times normal (or in absence of volume measurement tools aim for normal liver size) by year 1 to 2, depending on baseline liver volume | |
| General well-being | Improve scores from baseline of a validated quality-of-life instrument within 2 to 3 years or less depending on disease burden |
| Reduce fatigue (not anemia related) as measured by a validated fatigue scoring system | |
| Improve or restore physical function for carrying out normal daily activities and fulfilling functional roles |
From: Biegstraaten M, Cox TM, Belmatoug N, et al. Management goals for type 1 Gaucher disease: An expert consensus document for the European working group on Gaucher disease. Blood Cells Mol Dis 2018; 68:203. Copyright © 2018 Elsevier, Inc. Available at: https://www.sciencedirect.com/science/article/pii/S1079979616301917?via%3Dihub (Accessed November 20, 2017). Reproduced under the terms of the Creative Commons Attribution License.
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