Outcomes in Patients with Alagille Syndrome and Complex Pulmonary Artery Disease.
The Journal of pediatrics
To assess outcomes in a large cohort of patients with Alagille Syndrome (ALGS) who underwent pulmonary artery reconstruction surgery for complex PA disease.Patients with ALGS who underwent PA reconstruction surgery at Lucile Packard Children's Hospital Stanford were reviewed. Patients were examined as an overall cohort and based on the primary cardiovascular diagnosis: severe isolated branch PA stenosis, tetralogy of Fallot (TOF) without major aortopulmonary collateral arteries (MAPCAs), or TOF with MAPCAs RESULTS: Fifty-one patients with ALGS underwent PA surgery at our center: 22 with severe branch PA stenosis, 9 with TOF without MAPCAs, and 20 with TOF and MAPCAs. Forty-one patients (80%) achieved a complete repair. Five of the patients with TOF with MAPCAs (25%) had a complete repair at the first surgery, compared with 8 (89%) and 19 (86%) with TOF without MAPCAs and isolated branch PA stenosis, respectively. At a median follow-up of 1.7 years after the first surgery, 39 patients (76%) were alive, 36 with a complete repair and a median PA:aortic systolic pressure ratio of 0.38. Nine patients (18%), 8 with isolated branch PA stenosis, underwent liver transplantation.Most patients with ALGS and complex PA disease can undergo complete repair with low postoperative right ventricular pressure. Patients with TOF/MAPCAs had the worst outcome, with higher mortality and more frequent PA interventions compared with patients with TOF without MAPCAs or isolated branch PA stenosis. Complex PA disease is not a contraindication to liver transplantation in patients with ALGS.
View details for DOI 10.1016/j.jpeds.2020.09.053
View details for PubMedID 32980376
Safety, Efficacy and Pharmacokinetics of Repeat Subcutaneous Dosing of Avexitide (Exendin 9-39) for Treatment of Post-Bariatric Hypoglycemia.
Diabetes, obesity & metabolism
AIMS: To evaluate the safety, efficacy, and pharmacokinetics of repeat dosing of two formulations of subcutaneous (SC) avexitide (exendin 9-39) in patients with post-bariatric hypoglycemia (PBH).METHODS: In this Phase 2, multiple-ascending-dose study conducted at Stanford University, 19 women with PBH underwent a baseline oral glucose tolerance test (OGTT) with metabolic and symptomatic assessments. Fourteen participants were then sequentially assigned to receive 1 of 4 ascending dose levels of twice daily (BID) lyophilized (Lyo) avexitide by SC injection for 3 days. On the basis of safety, efficacy and tolerability, 5 additional participants then received a novel liquid formulation (Liq) of avexitide by SC injection at a fixed dose of 30 mg BID for 3 days. All 19 subjects underwent a repeat OGTT on Day 3 of dosing to quantify metabolic, symptomatic, and pharmacokinetic responses.RESULTS: Treatment with Lyo avexitide reduced the magnitude of symptomatic hyperinsulinemic hypoglycemia at all dose levels, with dose-dependent improvements in glucose nadir, insulin peak and symptom score; doses ?20 mg BID did not require glycemic rescue (administered at glucose <50 mg/dL). Participants receiving Liq avexitide 30 mg BID did not require any glycemic rescue, and on average achieved a 47% increase in glucose nadir, 67% reduction in peak insulin, and 47% reduction in overall symptom score. Equivalent doses of Liq vs Lyo avexitide yielded higher and more sustained plasma concentrations. Both formulations were well tolerated.CONCLUSIONS: In patients with PBH, BID administration of SC avexitide effectively raised the glucose nadir and prevented severe hypoglycemia requiring rescue intervention. Avexitide may represent a viable therapy for PBH.
View details for DOI 10.1111/dom.14048
View details for PubMedID 32250530
When a Child Becomes Terminal: Discrepant Adolescent and Parent Perspectives on Discussing a Terminal Prognosis With Adolescents With Cardiomyopathy, Heart Failure, and Heart Transplant
LIPPINCOTT WILLIAMS & WILKINS. 2018
View details for Web of Science ID 000528619400075
- Repeat Subcutaneous Dosing of Exendin 9-39 Reduces Hyperinsulinemic Hypoglycemia and Neuroglycopenic Symptoms in Patients with Post-Bariatric Hypoglycemia American Diabetes Association 77th Scientific Sessions 2017