Table 3.
Prior-ALIS cohort: safety profile among patients with up to 8 months of ALIS exposure at baseline (safety population)
| Parameter | Prior-ALIS Cohort (N = 73) |
|---|---|
| Any TEAE | 68 (93.2) |
| Grade 1: mild | 22 (30.1) |
| Grade 2: moderate | 30 (41.1) |
| Grade 3: severe | 13 (17.8) |
| Grade 4: life threatening | 1 (1.4) |
| Grade 5: death | 2 (2.7) |
| TEAE in ≥10% of patients | |
| Hemoptysis | 11 (15.1) |
| Nasopharyngitis | 10 (13.7) |
| Cough | 9 (12.3) |
| Dyspnea | 9 (12.3) |
| TEAE: pulmonary exacerbation* | 22 (30.1) |
| TEAE leading to discontinuation of ALIS | 6 (8.2) |
| TEAE leading to discontinuation of GBT | 4 (5.5) |
| TEAE leading to discontinuation of ALIS and GBT | 1 (1.4) |
| TEAE leading to death | 2 (2.7) |
| COPD exacerbation | 1 (1.4) |
| Lower respiratory tract infection | 1 (1.4) |
| Any serious TEAE † | 20 (27.4) |
| Serious TEAE occurring in ≥3% of patients | |
| Pneumonia | 3 (4.1) |
| Serious TEAE: pulmonary exacerbation | 7 (9.6) |
| Serious TEAE leading to discontinuation of ALIS | 3 (4.1) |
| Respiratory TEAEs of special interest | |
| Bronchospasm | |
| Dyspnea | 9 (12.3) |
| Wheezing | 1 (1.4) |
| Infective exacerbation of underlying disease | |
| Infective exacerbation of bronchiectasis | 7 (9.6) |
| COPD exacerbation | 4 (5.5) |
| Hemoptysis | |
| Hemoptysis | 11 (15.1) |
| Allergic alveolitis | |
| Alveolitis allergic | 2 (2.7) |
| TEAEs of special interest typically associated with parenteral amikacin | |
| Ototoxicity | |
| Hearing loss ‡ | 7 (9.6) |
| Dizziness | 2 (2.7) |
| Tinnitus | 1 (1.4) |
| Nephrotoxicity | |
| Hematuria | 4 (5.5) |
| GFR decreased | 2 (2.7) |
| Leukocyturia | 1 (1.4) |
| Proteinuria | 1 (1.4) |
| Azotemia | 1 (1.4) |
| Renal failure | 1 (1.4) |
| Neuromuscular disorders | |
| Peripheral neuropathy | 1 (1.4) |
Definition of abbreviations: ALIS = amikacin liposome inhalation suspension; COPD = chronic obstructive pulmonary disease; GBT = guideline-based therapy; GFR = glomerular filtration rate; TEAE = treatment-emergent adverse event.
Data are shown as n (%).
Adverse events are reported and classified based on Medical Dictionary of Regulatory Activities preferred terms.
Pulmonary exacerbation was defined based on the investigators’ best clinical judgment.
A serious adverse event is any untoward medical occurrence that at any dose resulted in death, was life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability or incapacity, or was a congenital anomaly or birth defect.
Hearing loss includes preferred terms hypoacusis, deafness neurosensory, deafness unilateral, and deafness.