Sunday, January 31, 2010
Catching Hospital Workers Dirty-Handed
by Gigi Douban
"Wash your hands" is a message Lindsey Ann Stone, a nurse at Baptist Princeton Hospital in Birmingham, Ala., and her colleagues have heard over and over again.
Stone ticks off the ways hand washing is promoted: "Through orientation, re-education; we have yearly updates that they put up, and bulletins and hand-hygiene pamphlets and posters. Everything that you can imagine, it's all over."
The standard protocol in hospitals is for doctors and nurses to wash their hands on the way in to see a patient, and on the way out. But that doesn't always happen — they get busy; they forget.
In fact, studies show that only about 40 percent of health care workers in the United States wash their hands as often as they should. So some hospitals are trying to monitor just how regularly their employees are washing up — by testing out new surveillance technologies.
Stone says she washes her hands a lot; she guesses at least 100 times a day. But her hospital's administrators aren't guessing — they have installed new devices to keep track.
With information transmitted wirelessly through a special badge Stone wears, the hospital can tell when she entered a patient's room, whether she washed her hands and whether she washed again on the way out. The information is sent to hospital officials, including the CEO.
When she first heard about the new system, Stone says she had mixed feelings.
"I was excited, but to be honest, I was a little nervous wondering how it was going to directly affect me as an RN," she says.
For one thing, she and her colleagues wondered how this information — a sort of nice and naughty list on hand washing — would ultimately be used.
"If they're habitually not complying, we can send them an e-mail or send them a text message, something that goes to them personally," says Harvey Nix, CEO of Proventix, the company that developed the monitoring system at Baptist Princeton.
Nix says the communication is meant to be more a gentle prod than punitive action. Still, most people aren't thrilled about being monitored.
The Centers for Disease Control and Prevention is trying to gauge how the surveillance affects health care workers. Focus groups are helping to tweak the technology, CDC epidemiologist Kate Ellingson says.
Researchers don't just want to know whether workers like it, Ellington says, but also the answers to questions like: "Do you see this as something that you would pay attention to? That would change your behavior?"
Improving hand hygiene among health care workers has been a real challenge, Ellingson says, but letting the problem slide comes at a price, too. According to the CDC, health care-associated infections kill about 100,000 Americans a year, at a cost of billions of dollars.
Saturday, January 30, 2010
An evolutionary approach to the high frequency of the Delta F508 CFTR mutation in European populations
Med Hypotheses. 2010 Jan 26.
An evolutionary approach to the high frequency of the Delta F508 CFTR mutation in European populations.
Departamento de Genética y Antropología Física, Universidad del País Vasco (UPV/EHU), Bilbao 48080, Spain.
The diffusion of the cattle pastoralism across Europe during the Neolithic period was probably accompanied by the emergence and spread of diverse contagious diseases that were unknown in the Paleolithic and that would have affected the frequency of genes directly or indirectly associated with differential susceptibility and/or resistance to infectious pathogens.
We therefore propose that the high frequency of the CFTR gene, and in particular, the common Delta F508 allele mutation in current European and European-derived populations might be a consequence of the impact of selective pressures generated by the transmission of pathogenic agents from domesticated animals, mainly bovine cattle, to the man.
Intestinal infectious diseases were probably a major health problem for Neolithic peoples. In such a context, a gene mutation that conferred an increased resistance to the diseases caused by pathogens transmitted by dairy cattle would have constituted a definite selective advantage, particularly in those human groups where cow's milk became an essential component of the diet. This selective advantage would be determined by an increased resistance to Cl(-)-secreting diarrheas of those individuals carrying a single copy of the Delta F508 CFTR mutation (heterozygote resistance).
This hypothesis is supported by the strong association between the geography of the diffusion of cattle pastoralism (assessed indirectly by the lactase persistence distribution), the geographic distribution of a sizeable number of HLA alleles (as indicative of potential selective pressures generated by epidemic mortality) and the geographic distribution of the most common mutation causing cystic fibrosis (Delta F508). The systematic interaction of humans with infectious pathogens would have begun in northern Europe, among the carriers of the Funnel Beaker Culture, the first farmers of the North European plain, moving progressively to the south with the dissemination of the cattle pastoralism. T
his gradual exposure to epidemic mortality among populations located further and further south in Europe as cattle pastoralism expanded could have generated differences in CFTR gene frequencies, thereby shaping the latitudinal frequency gradients observed in present-day European populations.
Wednesday, January 27, 2010
Wow I feel so fortunate to have had HFCC available to me for a very low cost since 1997.....
High Frequence Chest Wall Oscillation and Cystic Fibrosis
This study is not yet open for participant recruitment.
Verified by Imperial College London, January 2010
Sponsor: Imperial College London
Collaborators: Royal Brompton & Harefield NHS Foundation TrustHill-Rom
Purpose : High frequency chest wall oscillation (HFCWO) has been shown to increase tracheal mucus clearance compared with a control group. These observations led to the development of The Vest® which is a non-stretchable jacket connected to an air-pulse generator and worn by the patient over the chest wall. The generator rapidly inflates and deflates The Vest®, which gently compresses and releases the chest wall between 5 and 20 times per second. This generates mini-coughs that are said to dislodge mucus from the bronchial walls and to facilitate its movement up the airways. The Vest® has been shown to reduce the viscosity of mucus and this should further enhance mucus clearance.
People with cystic fibrosis (CF), admitted to hospital with an acute infective pulmonary exacerbation, should increase the frequency and duration of their airway clearance sessions owing to the increase in quantity and viscosity of purulent bronchial secretions.In the United Kingdom, and in many other countries, the availability of physiotherapists to assist with the recommended number of daily treatments is insufficient to meet patient need. If the use of high frequency chest wall oscillation, in addition to 'usual' self airway clearance techniques, in the early morning and evening was to facilitate recovery from an exacerbation, this would indicate an important place for high frequency chest wall oscillation in the management of people with cystic fibrosis.
Hypothesis: The addition of high frequency chest wall oscillation to twice daily supervised physiotherapy is as effective as the addition of self treatment in facilitating recovery from an acute infective pulmonary exacerbation, as measured by improvement in lung function, specifically forced expiratory volume in one second (FEV1).
Device: High Frequency Chest Wall Oscillation (HFCWO)
Other: Usual airway clearance
Study Design: Treatment, Randomized, Open Label, Parallel Assignment, Efficacy Study
Official Title:The Use of High Frequency Chest Wall Oscillation During an Acute Infective Pulmonary Exacerbation of Cystic Fibrosis
Primary Outcome Measures:
Mean percentage change in forced expiratory volume in one second (FEV1) [ Time Frame: 7days ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
Wet weight of sputum expectorated [ Time Frame: 24 hours ] [ Designated as safety issue: Yes ]
Length of time to next course of intravenous antibiotics [ Time Frame: Within 6 monthsof completing study ] [ Designated as safety issue: Yes ]
Rate of change of C-reactive protein [ Time Frame: 7 days ] [ Designated as safety issue: Yes ]
Estimated Enrollment: 56
Study Start Date: February 2010
Estimated Study Completion Date: July 2011
Estimated Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Usual Airway Clearance Technique: Active Comparator
Two self administered treatment sessions a day and two treatments a day assisted by a Physiotherapist both using the patient's usual airway clearance method.
Other: Usual airway clearance
Airway clearance treatments using the active cycle of breathing techniques, autogenic drainage, positive expiratory pressure, manual techniques or oscillating positive expiratory pressure
High Frequency Chest Wall Oscillation (HFCWO): Experimental
Two self administered treatments a day using HFCWO and two treatment sessions a day assisted by a Physiotherapist using their 'usual' airway clearance method.
Contact: Elizabeth A Banks, MSc
0207 351 8935
Contact: Penny Agent
0207 352 8121 ext 8056
Royal Brompton & Harefield NHS Foundation Trust
London, United Kingdom
Sponsors and Collaborators
Imperial College London
Royal Brompton & Harefield NHS Foundation Trust
Imperial College London
Association of cystic fibrosis genetic modifiers with congenital bilateral absence of the vas deferens
Fertil Steril. 2010 Jan 23.
Association of cystic fibrosis genetic modifiers with congenital bilateral absence of the vas deferens.
Department of Medicine and Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama.
OBJECTIVE: To investigate whether genetic modifiers of cystic fibrosis (CF) lung disease also predispose to congenital bilateral absence of the vas deferens (CBAVD) in association with cystic fibrosis transmembrane conductance regulator (CFTR) mutations. We tested the hypothesis that polymorphisms of transforming growth factor (TGF)-beta1 (rs 1982073, rs 1800471) and endothelin receptor type A (EDNRA) (rs 5335, rs 1801708) are associated with the CBAVD phenotype.
DESIGN: Genotyping of subjects with clinical CBAVD.
SETTING: Outpatient and hospital-based clinical evaluation.
PATIENT(S): DNA samples from 80 subjects with CBAVD and 51 healthy male controls from various regions of Europe. This is one of the largest genetic studies of this disease to date.
MAIN OUTCOME MEASURE(S): Genotype analysis.
RESULT(S): For single nucleotide polymorphism (SNP) rs 5335, we found increased frequency of the CC genotype among subjects with CBAVD. The difference was significant among Turkish patients versus controls (45.2% vs. 19.4%), and between all cases versus controls (36% vs. 15.7%). No associations between CBAVD penetrance and polymorphisms rs 1982073, rs 1800471, or rs 1801708 were observed.
CONCLUSION(S): Our findings indicate that endothelin receptor type A polymorphism rs 5335 may be associated with CBAVD penetrance. To our knowledge, this is the first study to investigate genetic modifiers relevant to CBAVD.
Sunday, January 24, 2010
Have you had a genetic test prior to using warfarin?
We're all familiar with Vertex and 770 and 809 as well as PTC 124. Dare I say CF is at the forefront of this personalized medicine?
Wednesday January 13, 2010
Making Personalized Medicine Pay
Medco and other pharmacy benefit managers say future profits depend on matching drugs to patients based on their genes
By John Carey Business Week
The dirty little secret about drugs is that they only work in about half of the people who take them. So says an educational nonprofit called the Personalized Medicine Coalition, and many drug executives concede as much. Of the $292 billion spent in the U.S. on prescription drugs in 2008, as much as $145 billion went to medications that didn't help individual patients, said Jerel Davis, project manager at McKinsey, at a recent conference. And billions more are being spent to treat adverse drug reactions and other complications. "When you look at the data, it's shocking," says Dr. Robert S. Epstein, chief medical officer at Medco Health Solutions (MHS), a $51 billion company that manages drug prescriptions for 60 million Americans.
Researchers know how to solve this problem. First, figure out the differences between those patients who respond to a drug and those who don't, then treat only to those who will benefit. But this personalized medicine approach "has been slower to develop than we thought 10 years ago," says Richard K. Schatzberg, CEO of Generation Health, a startup that offers targeted medicine services. Lack of enthusiasm in the drug industry is a big reason; companies would lose billions of dollars if only those who actually benefit were to use such blockbuster drugs as antidepressants, arthritis medicines, and cholesterol pills.
Now, however, the promise of personalized drug treatments appears more realistic, thanks to new players on the sceneand a new business model. The recent entrants are pharmacy benefit managers (PBMs) such as Medco and CVS Caremark (CVS). Medco is testing patients for genetic variations that explain why they respond differently to drugs like warfarin, a widely used blood thinner, and tamoxifen for breast cancer. CVS Caremark has taken a majority stake in Generation Health and expects to launch a similar testing program in May. The move by the PBMs "is transformative," says Edward Abrahams, executive director of the Personalized Medicine Coalition, whose members include scientists, health-care providers, payers, and patients' groups. "We are talking about better care for millions of people and keeping costs down for employers, whose insurance costs are exploding. It could be the tipping point."
PBMs plan to make money by selling personalized medicine services to employers, which are willing to pay them higher fees for improved health outcomes and lower prescription costs. Medco and other PBMs also hope to win market share from their slower-moving competitors. "It is a differentiator for us," says Dr. Jane Barlow, vice-president for Medco's personalized medicine business. Plus, they expect genetic testing will increase the percentage of patients using certain cheaper generic drugs, thus increasing profits. Medco has signed up 200 employers to its program, representing 7 million people. "This has been the fastest adoption of a new program in Medco history," says Epstein. One early—and eager—adopter: IBM (IBM), which expects better health outcomes and cost savings, says Dr. Martin Sepulveda, an IBM vice-president for health matters.
The idea took a long time to bear fruit at Medco, even though it was an obsession for Epstein, an epidemiologist by training. "Back in 2000, Rob Epstein explained to me this would change the face of medicine—and make all the pharmaceutical companies nervous," recalls Schatzberg. "It took longer than he thought." The company's first foray into personalized treatments in 2002 foundered. Epstein wanted to do genetic testing on asthma patients to predict better which ones might end up in the hospital, "but I couldn't see the return on investment," he says.
The business case improved as scientists identified more genes linked to drug responses. For many medicines, enzymes produced in the liver are crucial. Some enzymes change drugs so that they are excreted from the body. Others convert drugs that have no effect when first administered into a medically active form. Because of variations in genes, these enzymes may work quickly or slowly or not at all. One example is tamoxifen, used to prevent breast cancer recurrence. In 20% of people, the enzyme that usually activates this drug is partially or completely ineffective, and the drug provides little or no benefit.
A turning point for Medco came in 2005, when a Food & Drug Administration advisory committee recommended that genetic information be considered in making treatment decisions with warfarin. The blood thinner is widely prescribed to prevent clots, but it's notoriously difficult to get the dose right. "We know that we kill people with warfarin all the time," says Dr. Issam Zineh, associate director for genomics at the FDA's Center for Drug Evaluation Research. Too much warfarin raises chances of bleeding and strokes caused by bleeding; too little allows deadly clots to form. The cost of using the wrong doses is estimated to be in the billions of dollars per year. With a genetic test, doctors can determine if people will need more or less warfarin than the standard 5-milligram dose.
When Epstein looked at Medco's medical records of its million patients on the drug, he discovered something alarming: As many as 25% of them ended up in the hospital within six months of starting on warfarin. "Avoiding one hospitalization could underwrite the cost of the test for 100 patients," Epstein reasoned. Medco worked with the Mayo Clinic to measure the clinical benefits and cost savings from genetic tests for warfarin. The final data won't be released for several months, but Medco found that employers were eager to sign on for the testing service anyway. Now, when Medco sees a prescription coming in for warfarin, it recommends genetic testing to the doctor and patient. In Medco's experience, 67% of doctors and 82% of patients agree to testing.
"THE INCENTIVES ALIGN"
The next drug Medco personalized was tamoxifen. Identifying women who can't metabolize the drug into its active form and putting them on a different drug reduces the cancer's chances of recurrence—and the costs of future treatment. Coming soon is a test for another blood thinner, the blockbuster Plavix. Pinpointing those who benefit will enable Medco to keep more patients on the drug when it goes generic, instead of switching to a more expensive alternative that doesn't require a test. A bonus is that the results from any given genetic test are usually applicable to many drugs. The same variation that determines the response to Plavix, for instance, can help determine how Valium, heartburn drugs like Nexium, and the antidepressant Celexa should be used.
The PBMs' foray into individualized treatments "is where the business rubber meets the road," says Michael Stocum, managing director of consultant Personalized Medicine Partners. "The incentives align. Patients want to get the right drug, and payers are willing to pay if they get a benefit."
Targeting drugs to those who benefit will obviously cut revenues for some drugmakers. But the pharmaceutical industry itself has started to back away from trying to sell the same medicines to everyone, says former Pfizer drug researcher Dr. Bruce H. Littman, now president of consultant Translational Medicine Associates. "The blockbuster mentality is still in place, but drugmakers are coming around," he says.
If they don't, the FDA may not be pleased. In the future, the agency may balk at approving drugs that can't be directed to the right patients—and payers may decline to reimburse. Amgen, for one, strongly backs the use of a test for a gene called KRAS for its $8,400-per-month colon cancer drug, Vectibix. About 40% of people have a variation of KRAS that prevents the drug from working. Drugmakers "see a future business model where they just want all of the smaller market of appropriate patients," says Generation Health's Schatzberg.
Given these trends, the once overhyped idea of personalized medicine "is really starting to get legs," says Dr. Eric Topol, chief academic officer at Scripps Health. "The old way of giving therapeutics will be obsolete."
A Revolution—or Not
The Language of Life, a new book from Dr. Francis Collins, director of National Institutes of Health, trumpets a revolution in personalized medicine. But Peter Aldhous, San Francisco bureau chief for New Scientist, finds the evidence thin. In a Jan. 7 entry on the New Scientist blog CultureLab, he calls Collins "a cheerleader for a revolution that hasn't arrived quite yet" and notes that many users of consumer gene testing services "have been underwhelmed by the insights gained."
I started work in June and I was doing pretty well until about the beginning of October (eating well, exercise, getting enough sleep and of course doing meds).
Fall and winter are the times that are the roughest on me - granted it's MUCH better than it was 10-15 years ago before I treated my allergies with shots, environmental controls, EXERCISE (oh yes, exercise reduces inflammation big time for me), anti-histamines and leukotriene agonists. But I still get a bit tighter in the fall/winter and it makes clearing mucus that much tougher.
Plus, things at my job were making me more tired around this time so I have to admit that my exercising went from about 3-4/week with Jillian to about 1x a week. Not sure about the rest of you all, but I don't fully grasp how crappy I feel until I start to feel better. I'm not sure if my self-awareness is just horrendous or if we CFer's are able to adapt so well to the ups and downs of our illnesses we just cope easily.
So as a result of less exercise, more stress, fatigue, and fall allergies, I had some pretty decent hemoptysis on Halloween. As you would expect, I was on 2 weeks of IV's where I didn't exercise a whole lot due to simple fear of coughing more blood / being wiped out from the IV's. I also threw another DVT so I was on blood thinners 'til mid december and of course that freaked me out because if I coughed up blood with Lovenox in my system, that would be BAD BAD news.
I got a 2 week break after the IV's before I caught a wicked cold where naturally I was preparing for some more hemoptysis. Thank God that didn't happen (lungs were probably in better shape because I had just finished IV's?) although the cold lasted for a good 2 weeks and I had to fight it with nebbed Colistin and PO Lequin. I felt better after 2 weeks of treatment, but never fully back to normal.
After about a week of the cold/ CF PA exacerbation being gone and I caught a stomach virus that was going around. Luckily no puking or the runs, but unbelievable stomach cramps, nausea, fevers and no appetite for about 4 days.
I also got my Mirena put in the 1st week of December and had a lovely period that lasted nearly 4 weeks. Cramps were pretty horrendous as well and I just decided to give myself the holidays off from exercising (not treatments) after a really tough 2 months.
So what's my point?
My point is, I started 2010 anew.
- My period finally ended and I feel so good with this Mirena. I mean really, really, really good. I can't wait to see if this continues.
- I am back on the exercise band wagon with Jillian Michaels' video that I wrote about last May. Yes, I took a good 3 months off from consistent exercising so it was pretty tough getting back in to shape. But MAN, do I feel good. I cough up 2-3 plugs/day about the size of my pinky finger nail and my problem area (right middle lobe) hasn't felt this good in probably well over a year. I have much more energy, and moods are amazing. Not goign to lie - when it's time to exercise I get pretty angry... I am not the type of person that likes to work out. At all. Buuuuuuuuuuuut I refer to my list that I made for 2010 to remind myself of the benefits of exercise and the consequences of not exercising:
-Lower chance of diabetes by creating muscle mass that contains more insulin receptors
-Lower chance of osteoporosis related fracture by creating muscle mass which leads to inreased bone mass
-Better moods which leads to better performance on the job, happier relationships and overall increased enjoyment of life
-Lower chance of hemoptysis because I'm clearing areas of my lungs that I couldn't otherwise clear doing CPT, IV's or coughing
-Lower chance of exacerbation because exercising decreases inflammation in my lungs, and inflammation can inhibit mucus clearance
-Increase FEV1 which is the best predictor of life expectancy. VX-809 may or may not come to fruition, but in case it does, I'm determined to have the best lungs possible to maximize the benefits of the drug
-Increase immune system, and in turn chances of catching a virus that could cause permanent lung damage
And the list could go on and on.... but for me, this is enough to get me motivated.
So my point? I feel off the exercise bandwagon for a host of reasons - the first time I've fallen off since I started consistent, regular exercise in June 2007. Seriously.
But, by undersatnding the factors that contributed to me falling off the bandwagon and setting myself up for success by understanding the long list of benefits of exercising just 45 minutes 3x/week, I am more likely to stay on the bandwagon the next time things get tough.
Have no respect for the status quo. FIGHT ON
Wednesday, January 20, 2010
This study is currently recruiting participants.
Verified by Stanford University, January 2010
First Received: January 14, 2010 Last Updated: January 15, 2010 History of Changes
Sponsor: Stanford University
Information provided by: Stanford University
Study Drug, SB-656933, is a selective CXCR2 antagonist in development as a novel, once-daily oral anti-inflammatory agent for the maintenance treatment of Cystic Fibrosis (CF) and Chronic Obstructive Pulmonary Disease (COPD). We want to find out if this experimental drug will help decrease inflammation and slow the progression of lung disease. This study compares how well different doses of the experimental study drug (SB-656933) control inflammation in patients with CF. Two doses of the study drug will be assessed against placebo to see which dose works best.
Condition Cystic Fibrosis
Intervention Drug: SB-656933
Phase Phase II
Study Design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Parallel Assignment, Safety/Efficacy Study
Official Title:A Randomized, Double Blind, Parallel Group, Placebo Controlled 28 Day Study to Investigate the Safety, Tolerability and Pharmacodynamics of SB-656933 in Patients With Cystic Fibrosis
Primary Outcome Measures: effect of study drug on safety and tolerability [ Time Frame: 28 days of dosing ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures: levels of neutrophil elastase in induced sputum and other sputum markers [ Time Frame: 28 days of dosing ] [ Designated as safety issue: No ]
Estimated Enrollment: 140
Study Start Date: January 2010
Estimated Study Completion Date: February 2011
Ages Eligible for Study: 18 Years and older
Genders Eligible for Study: Both
Accepts Healthy Volunteers: No
Inclusion Criteria: Diagnosis of CF based on the following: sweat chloride > 60 mEq/L and/or genotype with 2 identifiable mutations consistent with CF; (delta-F508 homozygote, or delta-F508 heterozygote with a second allele known to cause the disease, or two alleles known to cause a class I, II, or III mutation) and one or more clinical features consistent with CF.
Male and female subjects aged older or =18 years of age
A female subject is eligible to participate if she is of: Non-childbearing potential defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or postmenopausal defined as 12 months of spontaneous amenorrhea [in questionable cases a blood sample with simultaneous follicle stimulating hormone (FSH) > 40 MlU/ml and estradiol < 40 pg/ml (<140 pmol/L) is confirmatory]. Child-bearing potential and agrees to use one of the contraception methods listed in the Protocol for an appropriate period of time (as determined by the product label or investigator) prior to the start of dosing to sufficiently minimize the risk of pregnancy at that point. Female subjects must agree to use contraception until one week after the last dose.
Patients are non-smokers or former smokers by history. Former smokers will be defined as those who have not smoked for longer than or =6 months. Subjects who only use chewing tobacco products may be enrolled at the discretion of the Investigator and after consultation with the GSK medical monitor.
In the judgement of the investigator the patient is clinically stable with no change in symptoms or medication, no admissions to hospital, and no intravenous antibiotic therapy for at least 1 month prior to dosing.
Able to perform lung function tests reliably.
FEV1 >40% and <110% predicted.
Excluding periods of exacerbation, FEV1 has not decreased by >15% over the past 12 months
Clinically colonized by a bacterial organism commonly seen in cystic fibrosis other than Burkholderia cepacia (i.e. Pseudomonas spp., Staphylococcus aureus, Stenotrophomonas, B. Gladioli) as evidenced by identification in sputum culture within the past year. To be eligible a CF patient must have colonization of at least one typical CF organism.
To be eligible, female patients must have a negative pregnancy test (urine or serum) and not be nursing at screening or prior to dosing.
Subjects must have a QTcB or QTcF < 450 msec at screening as determined by the investigators review.
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) within twice (2x) the upper limit of normal at screening and bilirubin within 1.25x ULN at screening. AST, ALT, alkaline phosphatase and bilirubin smaller or =2.0 xULN (isolated bilirubin >2.0xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
Male subjects must agree to use one of the contraception methods listed in the Study Protocol. This criterion must be followed from the time of the first dose of study medication until one week after the last dose.
Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form.
The subject is able to understand and comply with protocol requirements, instructions and protocol-stated restrictions.
Any clinically relevant abnormality identified on the screening medical assessment, laboratory examination, or ECG, that is not associated with cystic fibrosis:
Neutrophil count < 1.5x109 /L
In the judgment of the PI, the patient:
suffers from clinically unstable pancreatic function
has clinically significant weight loss(bigger than or =5% after a previously stable period).
has evidence of uncontrolled hyperglycemia or recent hypoglycemia
has recent change in pancreatic enzyme requirements in the past 2 months.
Recent viral infection (within 4 weeks of dosing), with or without steroid or antibiotic treatment.
Presumed viral infection will be determined according to the judgment of the Investigator and no specific testing for virus will be required.
Subjects unable to produce a technically acceptable sputum sample.
Clinically significant hepatic impairment:
Evidence of cirrhosis
Patients with elevated INR that is due to suspected vitamin K deficiency may be enrolled at the discretion of the Investigator and after consultation with the GSK medical monitor
Blood pressure persistently >155/95 mmHg at screening.
Positive HIV, Hepatitis B surface antigen or Hepatitis C antibody at screening.
History of regular alcohol consumption averaging >7 drinks/week for women or >14 drinks/week for men. One drink is equivalent to (12 g alcohol) = 5 ounces (150 ml) of wine or 12 ounces (360 ml) of beer or 1.5 ounces (45 ml) of 80 proof distilled spirits) within 6 months of screening.
Urinary cotinine levels indicative of smoking.
Use of oral or parenteral corticosteroids within 4 weeks of screening; regular use (>3 x/wk) of high dose NSAIDS (e.g. >1.6g ibuprofen/day on a regular basis), within 4 weeks of screening.
Colonization with Burkholderia cepacia
Subjects currently being treated for mycobacterial infection
Subjects with presumed active Allergic Bronchopulmonary Aspergillosis (ABPA)
Subjects who have newly started therapy with azithromycin within the past 3 months.
In the judgment of the investigator, clinically significant hemoptysis (> 30 cc per episode) within the last 6 months
Donation of blood in excess of 500 mL within a 56-day period prior to dosing
Participation in a trial with any drug within 30 days or 5 half-lives (whichever is longer), or participation in a trial with a new chemical entity within 2 months prior to first dose of current study medication, unless in the opinion of the Investigator and sponsor the medication will not interfere with the study procedures or compromise subject safety.
The subject has a positive pre-study drug/alcohol screen. A minimum list of drugs that will be screened for include amphetamines, barbiturates, cocaine, opiates, and cannabinoids. Subjects who use benzodiazepines or other anxiolytic on a regular basis can be included at the discretion of the investigator and in consultation with the GSK medical monitor
Patients may not be on an inhaled antibiotic during the study (i.e. must be an "off- TOBI" month; cessation of TOBI or other inhaled antibiotics commences from one week prior to dosing until final PK draw). Patients on maintenance therapy with hypertonic saline solution or inhaled DNase may continue these therapies.
Unwillingness or inability to follow the procedures outlined in the protocol.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01051453
Contact: Colleen Dunn
United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Contact: Colleen Dunn 650-736-0388 firstname.lastname@example.org
Principal Investigator: Richard B Moss
Sub-Investigator: Carlos Milla
Sub-Investigator: Carol K. Conrad
Sub-Investigator: Paul K Mohabir
Sub-Investigator: David Weill
Sub-Investigator: Zoe Davies RN
Sub-Investigator: Colleen Dunn RCP
Sub-Investigator: Martha Hamilton
Sub-Investigator: Andrea Core
Sub-Investigator: Yan Ki Angela Leung
Sponsors and Collaborators
Richard B Moss
Tuesday, January 19, 2010
On January 10, Vertex said that the VX-770 Phase 3 registration program is advancing rapidly, and it expects to obtain Phase 3 data for VX-770 early in 2011.
Additionally, Vertex also expects to obtain clinical data from a Phase 2 trial of VX-809 in the coming weeks that could potentially support the evaluation of VX-770 and VX-809 as part of a combination regimen in patients with the most common mutation of this disease.
Thursday, January 7, 2010
December 29, 2009 — The American Diabetes Association (ADA) revised clinical practice recommendations for diabetes diagnosis promote hemoglobin A1c (A1c) as a faster, easier diagnostic test that could help reduce the number of undiagnosed patients and better identify patients with prediabetes. The new recommendations are published December 29 in the January supplement of Diabetes Care.
"We believe that use of the A1c, because it doesn't require fasting, will encourage more people to get tested for type 2 diabetes and help further reduce the number of people who are undiagnosed but living with this chronic and potentially life-threatening disease," Richard M. Bergenstal, MD, ADA president-elect of medicine & science, said in a news release. "Additionally, early detection can make an enormous difference in a person's quality of life. Unlike many chronic diseases, type 2 diabetes actually can be prevented, as long as lifestyle changes are made while blood glucose levels are still in the pre-diabetes range."
The A1c test, which measures average blood glucose levels for a period of up to 3 months, was previously used only to evaluate diabetic control with time. An A1c level of approximately 5% indicates the absence of diabetes, and according to the revised evidence-based guidelines, an A1c score of 5.7% to 6.4% indicates prediabetes, and an A1c level of 6.5% or higher indicates the presence of diabetes.
For optimal diabetic control, the recommended ADA target for most people with diabetes is an A1c level no greater than 7%. It is hoped that achieving this target would help prevent serious diabetes-related complications including nephropathy, neuropathy, retinopathy, and gum disease.
Unlike fasting plasma glucose testing and the oral glucose tolerance test, A1c testing does not require overnight fasting. Compliance with screening may therefore be improved through use of the A1c test, which can be determined from a single nonfasting blood sample.
Recommendation Changes for 2010
Specific changes in the 2010 Clinical Practice Recommendations are as follows:
A section on diabetes related to cystic fibrosis has been added to "Standards of Medical Care in Diabetes." New evidence has shown that early diagnosis of cystic fibrosis-related diabetes and aggressive treatment with insulin have narrowed the gap in mortality between patients with cystic fibrosis with and without diabetes and have eliminated the sex difference in mortality rates. New recommendations for the clinical management of cystic fibrosis-related diabetes, based on a 2009 consensus conference, will be published in 2010 in a consensus report.
Revision of the section "Diagnosis of Diabetes" now includes the use of the A1c level for diabetes diagnosis, with a cutoff point of 6.5%.
The section formerly named "Diagnosis of Pre-diabetes" is now named "Categories of Increased Risk for Diabetes." Categories suggesting an increased risk for future diabetes now include an A1c range of 5.7% to 6.4%, as well as impaired fasting glucose and impaired glucose tolerance levels.
Revisions to the section "Detection and Diagnosis of GDM [Gestational Diabetes Mellitus]" now include a discussion of possible future changes in this diagnosis, according to international consensus. Screening recommendations for gestational diabetes are to use risk factor analysis and an oral glucose tolerance test, if appropriate. Women diagnosed with gestational diabetes should be screened for diabetes 6 to 12 weeks postpartum and should have subsequent screening for the development of diabetes or prediabetes.
Extensive revisions to the section "Diabetes Self-Management Education" are based on new evidence. Goals of diabetes self-management education are to improve adherence to standard of care, to educate patients regarding appropriate glycemic targets, and to increase the percentage of patients achieving target A1c levels.
Extensive revisions to the section "Antiplatelet Agents" now reflect evidence from recent trials suggesting that in moderate- or low-risk patients, aspirin is of questionable benefit for primary prevention of cardiovascular disease. The revised recommendation is to consider aspirin treatment as a primary prevention strategy in patients with diabetes who are at increased cardiovascular risk, defined as a 10-year risk greater than 10%. Patients at increased cardiovascular risk include men older than 50 years or women older than 60 years with at least 1 additional major risk factor.
Fundus photography may be used as a screening strategy for retinopathy, as described in the section "Retinopathy Screening and Treatment." However, although high-quality fundus photographs detect most clinically significant diabetic retinopathy, they should not act as a substitute for an initial and dilated comprehensive eye examination. Retinal examinations should be carried out annually or at least every 2 to 3 years among low-risk patients with normal eye examination results in the past.
Extensive revisions to the section "Diabetes Care in the Hospital" now question the benefit of very tight glycemic control goals in critically ill patients, based on new evidence.
Extensive revisions to the section "Strategies for Improving Diabetes Care" are based on newer evidence. Successful strategies to improve diabetes care, which have resulted in improved process measures such as measurement of A1c levels, lipid levels, and blood pressure, include the following:
Delivery of diabetes self-management education.
Adoption of practice guidelines developed with participation of healthcare professionals and having them readily accessible at the point of service.
Use of checklists mirroring guidelines, which help improve adherence to standards of care.
Systems changes, including providing automated reminders to healthcare professionals and patients and audit and feedback of process and outcome data to providers.
Quality improvement programs, in which continuous quality improvement or other cycles of analysis and intervention are combined with provider performance data.
Practice changes, which may include access to point-of-care A1c testing, scheduling planned diabetes visits, and clustering dedicated diabetes visits into specific times.
Tracking systems with either an electronic medical record or patient registry to improve adherence to standards of care.
Availability of case or (preferably) care management services using nurses, pharmacists, and other nonphysician healthcare professionals following detailed algorithms under physician supervision.
"The most successful practices have an institutional priority for quality of care, involve all of the staff in their initiatives, redesign their delivery system, activate and educate their patients, and use electronic health record tools," the guidelines authors conclude. "It is clear that optimal diabetes management requires an organized, systematic approach and involvement of a coordinated team of dedicated health care professionals working in an environment where quality care is a priority."
Diabetes Care. December 29, 2009; January 2010 Supplement.
Tuesday, January 5, 2010
Fatty Acid Supplements Improve Respiratory, Inflammatory and Nutritional Parameters in Adults with Cystic Fibrosis
In addition to be aggressive about abx, the Swedes are also into supplementing essential fatty acids.
Arch Bronconeumol. 2009 Dec 30.
Fatty Acid Supplements Improve Respiratory, Inflammatory and Nutritional Parameters in Adults with Cystic Fibrosis
Servicio de Endocrinología y Nutrición, Hospital Regional Universitario Carlos Haya, Málaga, España; CIBERDEM, CIBER de Diabetes y Enfermedades Metabólicas, Instituto de Salud Carlos III, Madrid, España.
INTRODUCTION AND AIMS: Chronic inflammation plays a major role in lung deterioration in cystic fibrosis (CF) patients and anti-inflammatory strategies have beneficial effects. To study the changes seen after a one-year course of low-dose dietary supplements with a mixture of fatty acids in adult patients with CF in chronic inflammation, pulmonary status (lung function, respiratory exacerbations and antibiotic consumption), quality of life and anthropometric parameters.
PATIENTS AND METHOD: Seventeen adult subjects with CF received 324mg of eicosapentaenoic, 216mg of docosahexaenoic, 480mg of linoleic and 258mg of gammalinolenic acid daily. We assessed inflammation markers, spirometry parameters, number and severity of respiratory exacerbations, antibiotic consumption, quality of life (St George's QoL), anthropometric parameters and serum phospholipid fatty acid composition.
RESULTS: At the end of the treatment period TNF alpha levels fell significantly and its soluble receptors (60 and 80) rose significantly. Levels of IgG and IgM anti-oxidized LDL antibodies fell significantly. Spirometry improved significantly. Annual respiratory exacerbations and days of antibiotic treatment fell significantly. The improvement in QoL was not significant. Serum levels of docosahexaenoic, total omega-3 and linoleic acid rose significantly and more favourable profiles were seen in monoenoic acids, arachidonic acid and the arachidonic/docosahexaenoic ratio. The fat-free mass and hand grip dynamometry improved significantly.
CONCLUSIONS: Low-dose supplements of n-3 and gammalinolenic fatty acids over a long period (one year) appears to improve pulmonary status (lung function, respiratory exacerbations and antibiotic consumption), inflammatory and anthropometric parameters in adults with CF.
Friday, January 1, 2010
Top Empowered Patient tips for 2010
By Sabriya Rice, CNN Medical Producer
To be an empowered patient, do more than the minimum -- take charge of your health care
(CNN) -- Being an empowered patient means doing more than the bare minimum. It means taking an active part in your own health care.
Over the past year, we've brought you the extraordinary stories of ordinary people who took health problems into their own hands -- from a young girl who diagnosed her own condition in science class, to a wife whose memory of a disco tune saved her husband's life.
Your actions, however, don't have to be extraordinary: It's often the little, everyday things that mean the difference between a good health care experience and a bad one. Several common themes run through the stories of the people we've profiled. Here are the top lessons that emerged that can help you to become a more empowered patient in 2010.
1. Don't believe everything you hear (get a second opinion)
"Getting a second or even third opinion is critical, says Dr. Angelo Cuzalina, president-elect of the American Academy of Cosmetic Surgery (AACS). "The extra effort and additional consultations will undoubtedly educate you further... which is always self-empowering."
2. Ask a ton of questions
You've heard the phrase "there's no such thing as a stupid question," and experts couldn't agree more.
To make sure you get all of your questions answered:
• Take a list with you. Let's face it, doctors are often rushed these days, so it's up to the patient to be as prepared as possible. One good rule of thumb is to write down your questions beforehand, and take them with you. This can save both you and the doctor time.
• Schedule time for questions. When possible, try to schedule an appointment that gives you enough time to get all of your questions in. "Tell the receptionist you'd like an extended session," recommends Dr. Linda Reid Chassiakos of the David Geffen School of Medicine at the University of California, Los Angeles, adding that "many providers set up longer visits at day's end where you can have their uninterrupted attention." Once you're there, get the stuff that's really troubling you out of the way first. "If you save the questions about the burning pain in your groin for a 'by the way' at the end of the visit, there won't likely be time to address your real issue fully."
• Be prepared for answers. Asking the right questions is great, but it's equally important to be prepared for the response."When the visit concerns a potentially serious problem, anxiety, fear, disbelief and denial are omnipresent ... the chance of you hearing exactly what the doctor says and remembering what you are supposed to do about it are slim," bioethicist Art Caplan says. He suggests bringing along someone you trust to be a second set of ears; if no one is available, bring a tape recorder and ask the doctor for permission to record what you are being told.
3. If you are going to use the Web, search smart
According to research from the Pew Internet and American Life Project, eight out of 10 Internet users have searched the Web specifically for health information.
The Web is a wonderful resource for any empowered patient, but it can also be a pit of misinformation. "Remember that anyone can create a Web site about 'health' or 'medical treatments,' " says Dr. Rhonda Medows, commissioner of the Georgia Department of Community Health.
On the other hand, a proper search can render information that is useful, even cutting-edge. "Read medical literature to learn the documented facts about your procedure of interest," advises Cuzalina of the AACS. "It's worth it when you get facts that are supported by empirical evidence."
Here are some tips for surfing for health information on the Web:
• Check your URL. Dr. Jennifer Shu, a pediatrician and Living Well expert for CNNHealth.com, says it's best to narrow your search to specific URLs. "Look for sites that end in .gov, .org or .edu, which generally provide credible information." But don't just stop there. Shu says that if your Internet research turns up something interesting or useful, share it with your doctor. "When patients do this in my office, I can confirm the information they have is medically sound, or suggest other sites that may be either more accurate or easier to understand."
• Protect your privacy. "Health insurance is a very personal transaction, so don't give out personal information," says Sande Drew, a patient advocate and consultant for Ehealthinsurance. She says to look for sites that allow you to initially search anonymously, prompting you to enter only your ZIP code, date of birth and gender.
• Compare and contrast. In many instances, you can check prices, hospital ratings and what services are offered online before your visit. "When a serious medical condition or emergency occurs, customers typically rush to the closest hospital, which may not be the best hospital," says Dr. Archelle Georgiou, president of a health care strategy firm in Minneapolis, Minnesota. Web sites such as Hospital Compare, My Health Compare,Health Grades and Ucompare Health Care allow you to compare hospital quality and patient satisfaction. Knowing your preferences in advance can help you decide quickly how to proceed if you ever need to rush to the emergency room.
4. Free and discounted care is out there
"In today's tough economic times, everybody is bargain hunting, trying to save pennies here and there," says UCLA's Chassiakos. But beware of scams, she warns. "If a bargain is too good to pass up, be prepared that there may be a catch."
Here are some ways to find dependable help:
• Pay what you can. The Department of Health and Human Services provides assistance through federally funded health centers. You pay what you can afford based on your income level. The services include everything from preventive care to dental work. Click here for more information.
• Find an advocate. When you get laid off and lose your health insurance, you may need someone in your corner. Several groups specialize in helping people find affordable insurance and free care, including: Coverage for All,Ehealthinsurance,Healthcare Advocacy,Patient Advocate Foundation and Patient Services Incorporated.
• Get prescription drug help. If you can't afford health insurance, or if your insurance doesn't include good prescription drug benefits, look for $4 generic drugs at many major supermarkets and drug store chains. Also, your state may offer a discount drug program. You can also check these private groups that offer prescription assistance: Chronic Disease Fund,FamilyWize discount drug card,HealthWell Foundation, Needy Meds, Partnership for Prescription Assistance, Rx Assist and Rx Hope.
• COBRA. If you're voluntarily or involuntarily laid off from your job, or if you experience a large reduction in work hours, you may be eligible for COBRA, a program that allows you to keep your employer's insurance. But there's one big catch: You have to pay the premium in its entirety, which can sometimes be upwards of $1,000 per month. As part of the congressional stimulus package passed earlier this year, people who involuntarily lost their jobs can have the government pay 65 percent of their COBRA premiums. Several rules apply. For more information, go the Department of Labor's Web site.
5. Paying attention can save your life
You know your body better than anyone else. If your gut tells you something just isn't right, then listen to it.
Here are some other ways paying attention can make a difference:
• Know your medications. Keeping track of what medications you're taking is crucial. Medows, from the Georgia Department of Community Health, recommends writing a word or two on the bottle to remind you of what the medication is for. "Life can get too complicated to depend on memory alone to keep track of everything," Medows says.
• Track your progress. If your treatment involves a prescription, change of diet or physical therapy, make sure you understand what you have to do each step of the way before you leave the doctor's office. "Clear communication is always a challenge. It's even harder when the stakes are your health," says Caplan, the ethicist. Write down the steps and keep track of how long before you should see improvement and when you should schedule a follow-up appointment.
CNN's Elizabeth Cohen, John Bonifield and Jennifer Pifer-Bixler contributed to this report.