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Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

From: Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study

Fig. 2

Survival from birth to 12 months of age. Kaplan-Meier analyses of (a) historical control group of untreated patients with early growth failure (n = 21) from natural history study of infants with LAL deficiency (LAL-1-NH01; data on file, Alexion Pharmaceuticals, Inc.) [7] and (b) the primary efficacy set of the present phase 2/3 study (N = 9). Patients in the historical control group were considered untreated if they had not received hematopoietic stem cell transplant, liver transplant, or enzyme replacement therapy. Growth failure was defined by 1) decreased body weight across ≥2 of the 11 major centiles on a standard WHO weight-for-age chart, or 2) body weight in kilograms below the 10th centile on a standard WHO weight-for-age chart and no weight gain for the previous 2 weeks, or 3) loss of ≥5% of birth weight in children who are >2 weeks of age

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