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. 2022 Feb 10;11(4):920. doi: 10.3390/jcm11040920

Table 5.

Criteria in ASCEND (adults and peds) study for use of olipudase alfa.

Criteria ASCEND (Adults) ASCEND Peds
Lung Inclusion criteria:

  • DLco ≤ 70%

Exclusion criteria:

  • Invasive ventilation

  • Non-invasive ventilation > 12 h/day

 Exclusion criteria:

  • Invasive ventilation

  • Non-invasive ventilation > 12 h/day
Spleen Inclusion criteria:

  • Spleen volume ≥6 MN, measured by MRI; patients who have had a partial splenectomy may be included if the procedure was performed ≥1 year prior to screening and the residual spleen volume is ≥6 MN

  • SRS score ≥ 5 over one week

 Inclusion criteria:

  • Spleen volume ≥5 multiples of normal (MN), measured by MRI; patients who have had a partial splenectomy may be included if the procedure was performed ≥1 year prior to screening and the residual spleen volume is ≥5 MN
Liver Exclusion criteria:

  • Active hepatitis B or hepatitis C

  • ALT or AST > 250 IU/L or total bilirubin > 1.5 mg/dL (except for patients with Gilbert’s syndrome)

  • Major organ transplantation (liver, bone marrow)

  • The patient is unwilling or unable to refrain from taking any potentially hepatotoxic medication or herbal supplement 10 days before and 3 days after the liver biopsies

 Exclusion criteria:

  • Active hepatitis B or hepatitis C

  • Cirrhosis (determined by clinical assessment)

  • Major organ transplantation (liver, bone marrow)

  • ALT or AST > 250 IU/L or total bilirubin > 1.5 mg/dL
Blood Exclusion criteria:

  • Platelet count < 60 G/L

  • INR > 1.5

  • The patient is unwilling or unable to avoid taking any medication or herbal supplements that may cause or prolong bleeding, 10 days before and 3 days after the liver biopsies

 Exclusion criteria:

  • Platelet count < 60 G/L

  • INR > 1.5
Bone/growth retardationBone damageDelayed growth

  • No mention of bone criteria

Inclusion criteria:

  • Size of −1 Z-score or lower

  • No mention of bone criteria
Central nervous system

  • No mention of criteria

 Exclusion criteria:

  • Acute or rapidly progressive neurological abnormality

  • Homozygosity for the R496L, L302P, and fs330 mutations or any combination of these 3 mutations

  • Delayed motor skills
Cardiovascular Exclusion criteria:

  • Significant heart disease

  • Major organ transplantation (liver, bone marrow)

  • No mention of dyslipidaemia/coronary calcifications

 Exclusion criteria:

  • Significant heart disease

  • No mention of dyslipidaemia/coronary calcifications
Pain/fatigue Inclusion criteria:

  • SRS score ≥ 5

  • No mention of specific criteria

ALT, alanine aminotransferase; AST, aspartate transaminase; DLco, diffusing capacity of the lung; INR, international normalised ratio; MN, multiples of normal; MRI, magnetic resonance imaging. SRS (Splenomegaly Related Symptom) score: a score composed of 5 items (abdominal pain, abdominal discomfort, early satiety, self-image, ability to stoop). This score is derived from the Myeloproliferative Syndrome Assessment Score (MF-SAF) (JAKARTA, NCT# 01437787). The questions assess the effect over the last 24 h of the splenomegaly in patients with NPB disease.