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Full-Text Articles in Translational Medical Research

Exploring Attitudes Towards Newly Approved Therapeutics In Prenatal Genetic Counseling Practice, Charlotte Close May 2021

Exploring Attitudes Towards Newly Approved Therapeutics In Prenatal Genetic Counseling Practice, Charlotte Close

Human Genetics Theses

Cystic fibrosis (CF) care has rapidly evolved over the past decade due to the introduction of CFTR modulators and continued quality improvement efforts, yet research from 2013 suggests that prenatal genetic counselors often did not feel knowledgeable discussing newer CF treatments with patients and expressed the need to refer to a CF specialist (Elsas, 2017). Given that the intervening years have allowed for collection of long-term data about CFTR modulators and that the recently approved modulator Trikafta (tez/elx/iva) is available to 90% of the CF population, it is appropriate to re-evaluate the status of prenatal genetic counselors’ awareness of and …


Investigation Of Medication Adherence And Rejection In Liver Transplant For Cystic Fibrosis (Cf), Lucia Sanchez, Elizabeth Rand, Md, Phoebe Wood Feb 2021

Investigation Of Medication Adherence And Rejection In Liver Transplant For Cystic Fibrosis (Cf), Lucia Sanchez, Elizabeth Rand, Md, Phoebe Wood

Phase 1

Introduction: 40% of transplant recipients are non-adherent to medications, causing increased risk of rejection and diminished survival. The relationship between adherence measured as Medication Level Variability Index (MLVI) and graft rejection rates in pediatric liver transplant recipients with CF compared to those without CF will be evaluated. We hypothesize that MLVI is higher and rejection rates are lower in subjects with CF.

Methods: Retrospective chart review of pediatric liver transplants performed at the Children’s Hospital of Philadelphia between 1995-2018 identified 9 CF liver transplant recipients and 9 age matched controls. Medication adherence was assessed using MLVI. An unpaired t-test was …