Table 4.

Patients with chronic ASMD grouped among disease severity (CETLv classification).

Group	Definition
Group outside the early access spectrum application	ASMD types B and A/B (NPD B or A/B) with a short-term vital prognosis (e.g., cancer)
Group 1: Patients with severe organ involvement	DLco a <50% and/ or dyspnoea Platelets <50 G/L ± recurrent bleeding or bruising Abdominal pain Urgent need to start treatment Break in growth curve (≥2 standard deviation)
Group 2: Patients in therapeutic trials	Patient continues to receive treatment as part of clinical trial or extension study
Group 3: Patients with moderate organ involvement	50% < DLco < 70% 50 G/L < Platelets < 100 G/L without bleeding or bruising Failure to thrive/break in growth curve (>1 standard deviation)
Group 4: Patients with mild organ involvement	DLco > 70% Platelets > 100 G/L Abnormal thoracic imagery and/or hepatosplenomegaly
Group 5: Patients with no symptoms	DLCO > 70% Platelets > 100 G/L No symptom Normal thoracic imagery No hepato-splenomegaly

ASMD, acid sphingomyelinase deficiency; DLco, diffusing capacity of the lungs for carbon monoxide; NPD, Niemann–Pick disease. ^a DLco not available for patients aged <6 years old.