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.
Source: Daily Express - Health - Category: Consumer Health News Source Type: news

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In this study, the median age at LT for CFLD was 15.7 years. Notably, 10 of 13 (77%) CF explants had>5% steatosis and 8 of 13 (61.5%) demonstrated variable fibrosis. The median age, sex, type of transplant (liver vs liver-lung), pancreatic insufficiency status, body mass index (BMI) percentile, genotype, and prevalence of diabetes were comparable in those with and without explant steatosis. More than half of allograft biopsies showed significant steatosis (17/31, 54.8%) and lobular inflammation (16/31, 51.6%). Hepatocyte ballooning was less frequent (5/31, 16.1%). Overall, 6 patients (46.2%) had allograft steatosis that...
Source: Journal of Pediatric Gastroenterology and Nutrition - Category: Gastroenterology Tags: Original Articles: Hepatology Source Type: research
Ruby Walker, from Angus, Scotland, is at the top of the national 'super urgent' list. Her family worry 'the window of time for her transplant is closing' as she has waited for more than two years.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
We describe two cystic fibrosis patients infected with pandrug-resistantBurkholderia cepacia complex, with the exception of ceftazidime –avibactam, who received prophylaxis with this antibiotic during lung transplantation. Although both patients had a post-operative relapse of respiratory infection, one with positive blood cultures, ceftazidime–avibactam treatment yielded a favourable outcome. 12 months after transplantation, on e patient presented an excellent clinical outcome. However, the other patient died 10 months later due to severeB. cepacia sinusitis with intracranial invasion.
Source: Infection - Category: Infectious Diseases Source Type: research
Publication date: January 2019Source: The Annals of Thoracic Surgery, Volume 107, Issue 1Author(s): Sarah Cullivan, Karen Redmond, Carole Ridge, Oisin J. O’ConnellA 21-year-old patient presented with a short history of fatigue and dyspnea on a background of double-lung transplantation for cystic fibrosis and preexisting chronic superior vena cava obstruction. Computed tomography of the chest demonstrated a 3-cm mass occluding the right pulmonary veins, with associated right upper and lower lobe pulmonary parenchymal infiltrates. Two invasive procedures were performed, with similar complications in both procedures.
Source: The Annals of Thoracic Surgery - Category: Cardiovascular & Thoracic Surgery Source Type: research
Christina VanDerVelden, 17, of Jacksonville, Florida, slipped into a week-long coma after her healthy rapidly declined. The cystic fibrosis sufferer now needs a double lung transplant to survive.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
ABSTRACT The study of the human microbiome-and, more recently, that of the respiratory system-by means of sophisticated molecular biology techniques, has revealed the immense diversity of microbial colonization in humans, in human health, and in various diseases. Apparently, contrary to what has been believed, there can be nonpathogenic colonization of the lungs by microorganisms such as bacteria, fungi, and viruses. Although this physiological lung microbiome presents low colony density, it presents high diversity. However, some pathological conditions lead to a loss of that diversity, with increasing concentrations of so...
Source: Jornal Brasileiro de Pneumologia - Category: Respiratory Medicine Source Type: research
Authors: Regard L, Martin C, Chassagnon G, Burgel PR Abstract Introduction Cystic fibrosis (CF) is a genetic disease that primarily affects the respiratory system and often leads to respiratory failure and premature death. Although pulmonary complications contribute to 85% of deaths, non-pulmonary complications are responsible for significant morbidity and mortality in adults with CF. Areas covered This review summarizes acute and chronic non-pulmonary complications in CF patients, with emphasis on emerging complications and in the context of the current growth and ageing of the CF adult population. It also address...
Source: Expert Review of Respiratory Medicine - Category: Respiratory Medicine Tags: Expert Rev Respir Med Source Type: research
Conclusion: As for cardiac or hepatic transplantations, despite a strong immunosuppressive regimen, a high frequency of transient PF4/H Ab is observed in patients undergoing BLT. Their appareance is not related to thrombocytopenia and/or thrombotic events. However, they could be an early marker of a cellular reaction againts the graft.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - Category: Hematology Authors: Tags: 311. Disorders of Platelet Number or Function: Poster II Source Type: research
Conference abstracts
Source: The Aspergillus Website - updates - Category: Respiratory Medicine Authors: Source Type: news
Conclusion: There was 100% survival with all patients being discharged home within an average of 20.5 days post Tx. 100% were mobilised with physiotherapists within 12 hours of extubation, with an average increase in 6MWT on discharge of 360 mtrs. No patients required 02 therapy on discharge from (19.02.2108)Hirche et al., 2014 ‘Practice Guidelines: Lung Transplantatation in Patients with Cystic Fibrosis’ Pulm Med:621342
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Cystic fibrosis Source Type: research
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