Table 2– Treatment-emergent adverse events and laboratory abnormalities
Ataluren dose level/adverse event CTCAE grade#
LowHigh+
12341234
Adverse events
 CF exacerbation3 (25.0)2 (16.7)
 Diarrhoea1 (8.3)
 DIOS1 (14.3)
 Fungal infection1 (8.3)
 Nasopharyngitis1 (8.3)1 (14.3)
 Pseudomonas infection1 (8.3)
 Respiratory tract infection1 (8.3)
 Rhinitis2 (16.7)2 (28.6)
 Urinary tract infection1 (14.3)
 Viral infection1 (8.3)
 Arthralgia1 (14.3)
 Transient ischaemic attack1 (8.3)
 Dysuria2 (16.7)1 (14.3)
 Cough7 (58.3)4 (57.1)
 Dyspnoea1 (8.3)
 Rash1 (14.3)
Laboratory abnormalities§
 Haemoglobin ↓1 (8.3)1 (14.3)
 Platelets ↓1 (8.3)
 Sodium ↓1 (8.3)2 (28.6)
 Potassium ↑2 (16.7)1 (14.3)
 Bicarbonate ↓1 (14.3)
 BUN ↑4 (33.3)
 Creatinine ↑2 (16.7)1 (14.3)
 Calcium ↓5 (41.7)3 (42.9)
 Uric acid ↑4 (33.3)
 Glucose ↑6 (50.0)2 (16.7)1 (14.3)1 (14.3)4 (57.1)
 Albumin ↓1 (8.3)
 Bilirubin ↑1 (8.3)
 AST ↑1 (8.3)4 (57.1)
 ALT ↑1 (8.3)1 (8.3)2 (28.6)
 GGT ↑1 (14.3)
 Creatine kinase ↑1 (8.3)1 (14.3)
 LDH ↑2 (16.7)1 (8.3)1 (14.3)
 Alkaline phosphatase ↑3 (25.0)
 Total cholesterol ↑1 (8.3)1 (14.3)
 Triglycerides ↑2 (16.7)1 (8.3)1 (14.3)

  • Data are presented as n (%) and blank spaces in table are intentionally blank. #: a patient with at least one occurrence of the same type of event is only listed once, by worst severity grade; : 4, 4 and 8 mg·kg−1, n = 12; +: 10, 10 and 20 mg·kg−1, n = 7; §: only patients with shifts to worse grades are listed. CTCAE: Common Terminology Criteria for Adverse Events; CF: cystic fibrosis; DIOS: distal ileal obstruction syndrome; ↑: increase; ↓: decrease; BUN: blood urea nitrogen; AST: aspartate aminotransferase; ALT: alanine aminotransferase; GGT: gamma glutamyl transferase; LDH: lactate dehydrogenase.