Student saved by wonder drug NHS won’t pay for
A STUDENT has told how a revolutionary drug that treats cystic fibrosis saved her from a lung transplant. But last night thousands of sufferers of the killer condition were dealt a blow when NHS England announced it would not provide extra funds for the drug Orkambi, which tackles the genetic causes of the disease rather than treating its symptoms.
ConclusionsOlder age, lower FEV1 % of predicted, and lower MEP were independently linked to unfavorable outcomes. FACED and BSI were not accurate in predicting mortality in our cohort.
James Beeching, 34, has had the incurable genetic condition since he was two years old. Three lung transplants fell through before his surgery. He can finally walk his twins to school in Hastings.
Studies of large CF populations using registry data are important to identify people at high risk for death. Nkam et al. published a prognostic score developed on French CF registry data to predict death or lung transplantation (LT) over a 3-year period in the adult CF population. The goal of our study was to validate the proposed tool using the Canadian CF registry. Using data between 2011 and 2014, a total of 2043 adult CF patients were included. We found that the French prognostic score was a good predictor of death or LT in the Canadian CF population (OR for each unit increase: 3.12, 95% CI: 2.74 –3.55; p value
Mortality risk stratification is essential in lung transplantation (LTx) to allow listing, prioritization and mitigating strategies. In cystic fibrosis (CF) patients, predictors of post-LTx mortality are not established.
CONCLUSION: Readmissions are frequent among adults with CF and contribute to significant healthcare burden and cost among this population. PMID: 29709046 [PubMed - indexed for MEDLINE]
oup Abstract RATIONALE: The prognostic value of cardiopulmonary exercise testing (CPET) for survival in cystic fibrosis (CF) in the context of current clinical management, when controlling for other known prognostic factors is unclear. OBJECTIVES: To determine the prognostic value of CPET-derived measures beyond peak oxygen uptake (VO2peak) following rigorous adjustment for other predictors. MEASUREMENTS AND MAIN RESULTS: Data from 10 CF-centers in Australia, Europe and North America were collected retrospectively. 510 patients completed a cycle CPET between January 2000 and December 2007, of which 433 f...
We describe the use of a decontamination protocol that allowed for successful lung transplantation in a cystic fibrosis patient with necrotizing pneumonia from highly antibiotic-resistant pathogens (Burkholderia and psuedomonas species). This strategy may allow for successful lung transplantation in cystic fibrosis patients with multi-drug resistant infections previously considered non-transplantable.
Abstract Cystic fibrosis (CF) is the most common life-limiting genetic disease in Caucasian patients. Continued advances have led to improved survival, and adults with CF now outnumber children. As our understanding of the disease improves, new therapies have emerged that improve the basic defect, enabling patient-specific treatment and improved outcomes. However, recurrent exacerbations continue to lead to morbidity and mortality, and new pathogens have been identified that may lead to worse outcomes. In addition, new complications, such as CF-related diabetes and increased risk of gastrointestinal cancers, are c...
We describe the use of a decontamination protocol that allowed for successful lung transplantation in a cystic fibrosis patient with necrotizing pneumonia from highly antibiotic-resistant pathogens (Burkholderia and psuedomonas species). This strategy may allow for successful lung transplantation in cystic fibrosis patients with multi-drug resistant infections previously considered non-transplantable. PMID: 30316849 [PubMed - as supplied by publisher]
Background The aim of this study was to compare the pharmacokinetic profile, tolerability, and safety of a novel once-daily extended-release formulation of tacrolimus (LCPT) with that of once-daily prolonged-release tacrolimus (ODT) in stable adult lung transplant (LT) recipients. Methods Phase II, open-label, single-arm, single-center, prospective pilot pharmacokinetic study. Study population comprised 20 stable LT recipients receiving ODT, mean age 55.9 years (range, 38-67 years), 13 (65%) men. Patients were switched to LCPT in a 1:0.7 (mg/mg) conversion dose. Follow-up was 6 months, and cystic fibrosis patients wer...