Patient Monitoring

Treating physicians will determine the actual frequency of necessary assessments according to a patient’s individualized need for medical care and routine follow-up, as well as to published or local guidelines, as appropriate. As a minimum, however, a recommended schedule of assessments has been developed based on input of physicians from the international medical community with expertise in the care of patients with Gaucher disease. The recommended schedule represents the core assessments that are currently thought to monitor Gaucher-related clinical manifestations and to stage disease progression across the life-long course of the disease. The assessments include hematologic, visceral, skeletal assessments, and quality of life evaluations.

In addition to the outcomes included in the Minimum Recommendations for Monitoring Patients with Non-Neuronopathic (Type 1) Gaucher Disease, the following data should also be routinely collected and reported:

• Height
• Weight
• Incidence of Pregnancy
• Bone Pain
• Bone Crisis
• Mobility Status
• Enzyme Replacement Therapy (as applicable)
• Chronic Diseases
• Surgical Procedures
• Other Chronic Medications, specifically:

Adherence to these recommendations is encouraged since they facilitate the Registry’s intent for consistent and thorough clinical evaluations, which may reveal changes in a patient’s status and provide information for a database to fulfill the scientific objectives of the Registry.

Download Recommended Schedule of Assessments (PDF).