Cystic Fibrosis

Feb 142018
 
FDA Approves New Cystic Fibrosis Drug Combo

Vertex Pharmaceuticals won FDA approval for its third drug to treat cystic fibrosis, the company announced. Ivacaftor (Kalydeco) combined with tezacaftor, a new agent, will be sold as Symdeko. Symdeko was approved for patients age 12 or older who are homozygous for the F508 deletion of the CFTR gene, or who have another mutation that [… read more]

Dec 222017
 
FDA Approves Angiotensin-II for Septic Shock

The FDA approved angiotensin-II (Giapreza) as a new intravenous vasopressor for septic shock and other forms of distributive shock. The first new FDA-approved vasopressor in decades, angiotensin-II could significantly change the management of severe septic shock. FDA based its expedited approval (under priority review) on the ATHOS-3 trial enrolling 321 patients with shock refractory to [… read more]

Aug 242013
 
More children to be considered for adult lung transplants

More Children Under 12 to Be Considered for Adult Lung Transplants The Organ Procurement and Transplantation Network (OPTN), which is responsible for the life-and-death policies for allocation of lung transplants and other organs, announced it will allow children needing lung transplants to file expedited appeals to be listed for adult lungs. Children under 12 with [… read more]

Dec 192012
 
FDA approves ivacaftor (Kalydeco), new cystic fibrosis drug (Guess what it costs?)

Kalydeco: New Drug Helps Some with Cystic Fibrosis On January 31, the FDA approved ivacaftor (Kalydeco), the first-ever drug that treats an underlying cause of cystic fibrosis. In about 4% of cystic fibrosis patients, defective chloride ion channel transporters (CFTRs) are trafficked to the cell membrane, but don’t work properly. Kalydeco “unlocks” and improves function [… read more]

Jun 072012
 
Inhaled hypertonic saline for young kids with cystic fibrosis: no benefit? (RCT)

(image: Bennett Gamel blog) Inhaled hypertonic saline (7% NaCl) increases clearance of mucus by airway cilia, making it an attractive treatment for cystic fibrosis. Hypertonic saline has been a cornerstone of daily therapy for CF ever since a 2006 NEJM randomized trial showed that ~80 adults and kids older than 6 using hypertonic saline for [… read more]

Nov 172011
 

Most people with cystic fibrosis have mutations in the CFTR gene that prevent sufficient quantities of the assembled channels from making it to the cell membrane surface. That’s a hard problem to fix. However, a small minority (4-5%) have mutations on the G551D allele that impair the function of  the CFTR ion channel once it [… read more]

Nov 122011
 

Intracranial hemorrhage is a subject neuro-intensivists spend years learning about and refining their knowledge and skills on. A few key points: Myocardial “stunning” with depressed ejection fraction and pulmonary edema should be expected, due to a form of tako-tsubo cardiomyopathy, most commonly in subarachnoid hemorrhage. It’s not due to ischemia and gets better over weeks. [… read more]

Aug 302011
 

It’s long been known in the pediatric cystic fibrosis population that exacerbations accelerate decline of lung function. de Boer et al show that exacerbations are just as bad or worse in adults. Following 446 adults with CF in Ontario for 3 years, those with >2 exacerbations per year had a hazard ratio of 4.05 for [… read more]

Jun 022011
 

Although universally recommended, there was no evidence that nebulized hypertonic saline actually reduced inflammation in the airways of cystic fibrosis patients. Reeves et al showed that it can (specifically, by disrupting IL-8 from glycosaminoglycan matrices before they can recruit neutrophils by chemotaxis to wreak havoc). AJRCCM 2011;183:1517-1523.

May 052011
 

CF patients are instructed to inhale dornase alpha 30 minutes before airway clearance techniques. This has always been based on lore and plausibility. Dentice & Elkins publish their meta-analysis of 5 studies, including a total of 122 patients who were randomized and crossed-over to test the effect of the timing of dornase inhalation on outcomes. [… read more]

Mar 092011
 

Accurso et al report phase 3 results from industry-funded TIGER-1, testing t.i.d. inhaled denufusol (an ion channel regulator that alters sodium/chloride transport to improve mucociliary clearance) vs. placebo in early cystic fibrosis (FEV1>74% predicted). After 24 weeks, FEV1 was 45 mL better in the treated group, most of whom were younger than age 19. There [… read more]