ABSTRACT
Evidence for the extrapulmonary benefits of (CFTR) modulators is rapidly expanding. The use of CFTR modulators in CF patients who have undergone lung transplantation is not clear without guidance published in the medical literature to assist clinicians in the care of these patients. We discuss the potential benefits of CFTR modulators and provide insight into their use based on our experience in a small cohort of CF LTx recipients. We present pros and cons of CFTR modulator therapy for LTx recipients with CF. CFTR modulators should be considered in CF patients after lung transplantation for the time being until further research defines how to best use these therapies in transplant recipients.
Subject(s)
Aminophenols/therapeutic use , Benzodioxoles/therapeutic use , Cystic Fibrosis Transmembrane Conductance Regulator/metabolism , Cystic Fibrosis/drug therapy , Cystic Fibrosis/surgery , Indoles/therapeutic use , Lung Transplantation , Postoperative Care/methods , Pyrazoles/therapeutic use , Pyridines/therapeutic use , Quinolines/therapeutic use , Quinolones/therapeutic use , Adolescent , Biomarkers/metabolism , Combined Modality Therapy , Cystic Fibrosis/genetics , Cystic Fibrosis/metabolism , Cystic Fibrosis Transmembrane Conductance Regulator/genetics , Drug Combinations , Humans , Treatment Outcome , Young AdultABSTRACT
OBJECTIVE: To compare the occurrence of hypotension following administration of intermittent intravenous (IV) and enteral sildenafil for treatment of pulmonary hypertension (PH) in infants. We hypothesized there may be more adverse effects associated with intermittent IV sildenafil compared with enteral sildenafil. METHODS: This was a retrospective matched-cohort study conducted in a tertiary care children's hospital. Patients were included if they were less than 1 year of age and received intermittent sildenafil for PH. Exclusion criteria consisted of concurrent extracorporeal membrane oxygenation during the initiation of sildenafil, the utilization of sildenafil as a one-time dose, continuation of home-dosing regimen, or inclusion in the other cohort. A total of 40 patients were matched 1:1 based on postmenstrual age and primary diagnosis. RESULTS: There was no statistically significant difference in the primary outcome, as 30% (6/20) of patients receiving IV sildenafil required a hypotension intervention compared with 10% (2/20) in the enteral cohort (P = 0.24). The majority of interventions occurred within 24 hr of the initiation of sildenafil with 4/6 patients (67%) in the IV group and 2/2 patients (100%) in the enteral group, respectively. Baseline mean arterial pressure was significantly lower in the IV patients that required an intervention compared with those that did not (44 ± 6.3 vs. 65 ± 13.4 mmHg, P = 0.0024). CONCLUSIONS: There were no statistically significant differences in safety outcomes between intermittent IV and enteral sildenafil in infants with PH. Hemodynamic parameters should be monitored closely upon sildenafil initiation. Limitations include the retrospective nature and small sample size. Pediatr Pulmonol. 2017;52:232-237. © 2016 Wiley Periodicals, Inc.
