Table 2.

Key monitoring parameters and frequency of assessments for patients with ASMD type B forms.

Exam	Baseline Visit	Follow-Up Visit in the First 6 Months	Follow-Up Visit Every 3 Months for the First Year	Follow-Up Visit Every Year
Pulmonary assessment
Respiratory functional exploration (DLco—not before 6 years)	X	X		X
CT: thoracic	X			X a
Hepato-splenic assessment
Splenic and hepatic volume (ultrasound +/− abdominal MRI in adults; ultrasound in children)	X	X		X
Portal hypertension (abdominal ultrasound with Doppler)	X	X		X
Transaminases, alkaline phosphatase, bilirubin	X	X		X
Haematological assessment
CBC, platelets, haemostasis assessment (PT, factor V, fibrin, APTT, ferritin)	X		X	X
Lipid blood-test including total, LDL and HDL cholesterol and triglycerides	X		X	X

^a If normal, then every 5 years. APTT, activated partial thromboplastin time; CBC, complete blood count; CT, computed tomography; DLco, diffusing capacity of the lungs for carbon monoxide; HDL, high density lipoprotein, LDL, low density lipoprotein; MRI, magnetic resonance imaging; PT, prothrombin time.