Tuesday, February 7, 2012

Hepatitis News Ticker;HCV-Liver Cancer-Diabetes

Jacques Fabien Gautier d'Agoty 
In 1746 he published the "Anatomical Study" which contained color illustration/ diagrams of the body with the flesh pealed back showing the various organs and their placement within the body.

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Healthy You

In The News

Smoking Causes Rapid Mental Decline in Men; 3 Cancer Drugs Found to Raise Risk of Death; Positive Behavior Reinforcement May Reduce Bullying, Peer Rejection 

Treatment with three relatively new "targeted" cancer drugs has been linked to a slightly elevated chance of fatal side effects, according to a new analysis led by scientists at Dana-Farber Cancer Institute. They added that the risk remains low, but should be taken into account by physicians and patients.

The incidence of fatal complications was 1.5 percent in patients who received any of the three drugs, which block the vascular endothelial growth factor (VEGF) tyrosine kinase receptors in cancer cells, according to the study published February 6 in the Journal of Clinical Oncology. This is compared to a 0.7 percent incidence in patients given standard treatments or placebos.

The study looked at three drugs: sorafenib (Nexavar), sunitinib (Sutent), and pazopanib (Votrient). Sorafenib is approved to treat kidney and liver cancer, sunitinib to treat kidney cancer and gastrointestinal stromal tumor (GIST), and pazopanib to treat kidney cancer.

The authors of the study, led by Dana-Farber's Toni Choueiri, MD, suggest that physicians give full consideration of the potential risk before using the targeted drugs with patients at slightly high risk for bleeding or heart attacks - the most common fatal adverse events seen in clinical trials. They also recommended that physicians and patients be aware of the risks and to consider if those patients need to be closely monitored.

"There is no doubt for the average patient, these drugs have benefits and are FDA-approved for these indications," said Choueiri. "While the absolute incidence of these fatal side effects is very small, the relative risks are higher and patients and practitioners need to be aware of it."

For example, he said, it might be necessary to temporarily stop treating a patient with the drug or to cancel an elective surgery while a patient is taking one of these drugs. Choueiri added that these drugs should be used cautiously in patients who have had heart attacks. "The patient should be given all the information, and then he or she can balance the pros and cons in deciding whether to take the next step into treatment."

Choueiri said he believed the study is the first meta-analysis of published controlled trials to show a significantly increased risk of death from treatment with these VEGF-tyrosine kinase inhibitors. The majority of patients who died suffered fatal bleeding; the second most common cause was heart attack or heart failure; liver failure was also seen.

The 10 clinical trials subjected to the meta-analysis included 4,679 patients treated with the drugs.

Vascular endothelial growth factor receptor is a tyrosine kinase molecule that responds to chemical signals secreted by tumors to encourage the formation of new blood vessels for the purpose of providing nutrients to support tumor growth. However, humans need vascular endothelial growth factor (VEGF) at low levels to maintain critical to several physiologic processes in the body, including wound-healing, cardiac homeostasis, and formation of new blood vessels in normal tissues. As a result, blocking VEGF to treat cancer can interfere with these normal functions, increasing the odds of adverse effects.
View drug information on Nexavar; Sutent; Votrient.
Liver Cancer
Proton therapy has several advantages in treating liver cancer over traditional radiation therapy. Since the proton beam is so exact, larger dosages of radiation can be applied to the affected area, while sparing surrounding healthy tissue. Proton therapy also successfully addresses technical challenges, including liver movement during treatment when the patient breathes. Christopher Crane, M.D., Professor in the Department of Radiation Oncology at The University of Texas MD Anderson Cancer Center, discusses proton therapy as well as other options available to liver cancer patients.

Cancer Newsline - 01/16/2012
Cancer Newsline is a weekly audio podcast series from MD Anderson Cancer Center. The aim of Cancer Newsline is to provide the audience with current information on cancer research, diagnosis, treatment and prevention.

Click To Listen To Podcast

Jonathan Manning, Consultant Gastroenterologist, Scottish Borders. Reviewed by Luke Koupparis, general practitioner, Bristol.
Tuesday, 7 February 2012
Key learning points

* Hepatocellular carcinoma is a well recognised complication of chronic liver disease.
* Viral hepatitis remains the most common cause with alcohol also a significant contributor.
* Screening and surveillance is often performed to detect early lesions where possible.
* Surgery can be considered in fit patients with early stage disease.
* Palliative therapies are now available with lower toxicities and improved patient survival .
* Although key, endoscopy is not the only element in managing such patients.

Hepatocellular carcinoma (HCC) refers to a liver tumour originating from within the liver tissue. Liver metastases are approximately 30 times more common than primary tumours and making this differentiation is imperative to further investigations and management. It ranks as the 5th most prevalent cancer in the world (7% male cancers and 3% female cancers – approximately ½ million cases per year).

Patients may in fact be asymptomatic and have their tumour picked up on routine scanning for other reasons. Others may present with one or a combination of fatigue, abdominal pain, ascites, weight loss, jaundice or abnormal liver blood tests. Any of these should lead to further investigations by the primary care physician, referral to a Gastroenterologist and/or other Specialist.

Cirrhosis is known to be the greatest risk factor for the development of HCC. Significant geographical differences are apparent in relation to this risk factor. HCC rates of 30% are reported in South Africa but lower rates of 20% in Great Britain and North America. This is likely to be related to the larger cohort of chronic Hepatitis B virus (HBV) infection seen in those countries compared to the developed world.
Figure 1: Severe liver cirrhosis with gross ascites.

In cirrhotic alcoholics there is a four-fold increase in HCC compared to the general non-cirrhotic population. Chronic viral carriage of HBV can increase the HCC risk even in the absence of cirrhosis. The same applies to chronic hepatitis C virus (HCV) infection as well, with a four-fold increase in HCC in HCV patients compared to HBV when cirrhosis is present.

The mechanism of HCC is thought to reflect disorganised DNA during cellular repair or nodule regeneration in the presence of cirrhosis or chronic inflammation. Addressing some of the other chronic liver conditions, iron overload, as seen in genetic haemochromatosis, is also high risk for HCC when cirrhosis is present.

Other high risk cirrhotic patients include alpha1-antitrypsin deficiency, type 1 glycogen storage disease and porphyria cutanea tarda. Primary biliary cirrhosis and chronic active auto-immune hepatitis with cirrhosis are both considered lower risk scenarios than those stated above. However, cirrhotic hepatitis B or C patients co-infected with HIV are possibly at the greatest risk.
Figure 2: Histology showing chronic hepatitis with ground glass hepatocytes.

Small lesions can be hard to see in a nodular cirrhotic liver and may simply represent a regenerative nodule. Once a liver lesion has been identified, often by ultrasound, several factors, including it’s likely aetiology, need to be considered. Multi-phase CT scanning, or MRI, may be required to define a lesion further and follow-up interval scanning performed as appropriate, if deemed low risk.

The current evidence suggests that the standard initial imaging interval should be 6-monthly. Higher risk patients do not warrant more intensive screening. In addition to appropriate imaging the serum α-fetoprotein level is often monitored as it can be elevated in HCC. However, it may not increase in 20-40% or so. It may also be elevated in the absence of HCC but in the presence of other cancers, such as cholangiocarcinoma. It is also raised in pregnancy.

Liver biopsy is avoided if at all possible due to the high incidence of tumour seeding, particularly if a potentially curative outcome is predicted.

This depends on the location, size and number of the tumours and also on the fitness of the patient. Small tumours (<2-3cm) will have a 1-year survival close to 85% but only a 50% 2-year survival. Surgical resection or liver transplantation both offer the only real chance of cure. Tumours that are invading the portal vein or breaching the liver capsule would be considered unresectable and perhaps only amenable to palliative therapies. A size of 5cm is the usual cut off for suitability of a single lesion resection.

Resection offers a greater than 50% 5-year survival with a 70% likelihood of recurrence. Patients with advanced cirrhosis are poor candidates for resection with an increased mortality and risk of liver failure post-operatively. Therefore, a liver transplant offers the best alternative. A shortage of donor organs has lead to live-donor right lobe transplants, with improving results.

Radiofrequency ablation (RFA) is a palliative therapy which can induce tumour necrosis. An electrode is passed via the peripheral vasculature into the liver. Heat can be delivered directly into the lesion and therapy repeated. It can be offered in patients with lesions >2cm, with a procedure-related mortality of 0.3%. Studies have shown upto a 70% 5-year survival with this treatment.

Transarterial embolisation (TAE) and transarterial chemoembolisation (TACE) are two other alternatives for palliation. Gelofoam or small metal coils can be inserted into the feeding vessels by interventional radiologists. TACE is the same as TAE but with prior injection of a chemotherapeutic agent into the lesion with improved results but higher associated toxicity.

TACE is considered the first line therapy for non-surgical patients with HCC that has no vascular invasion. However, it is much higher risk in those with Child-Pugh B or C due to the possibility of post-treatment liver failure.

Sorafenib, is a multikinase inhibitor, which has shown a survival benefit in palliative patients. Median survival is increased from 7.9 to 10.7 months. It is offered to those patients with preserved liver function who are not suitable for other treatment modalities.

1. Hashem B. El-Serag. N Engl J Med 2011; 365:1118-1127. Hepatocellular Carcinoma.
2. Nishant Merchant, Calvin S. David, and Steven C. Cunningham. Intl J Hepatol 2011; 2011:142085. Review Article: Early Hepatocellular Carcinoma: Transplantation versus Resection: The Case for Liver Resection.
3. Bruix J, Sherman M. Hepatol 2011 Mar;53(3):1020-2. AASLD - Management of hepatocellular carcinoma: an update.
4. Riccardo Lencioni. Oncology Haematology. In Press Jan 2012. Management of hepatocellular carcinoma with transarterial chemoembolization in the era of systemic targeted therapy.


New BHIVA guidelines recommend discussion with all patients on potential of HIV treatment to protect sexual partners
The British HIV Association today recommends that doctors should discuss the evidence for the effectiveness of antiretroviral treatment as prevention with all patients with HIV, and that it should be offered those who want to protect their partners from the risk of HIV infection - even if they have no immediate clinical need for treatment ... (more)


Coffee Lowers Liver Fibrosis Risk in Certain Patients
Laura Newman, MA
February 6, 2012 — Drinking caffeinated coffee protects against liver fibrosis in patients with nonalcoholic fatty liver disease, according to research published in the February issue of Hepatology.
In introducing the study, Jeffrey C. Molloy, MD, from the Division of Gastroenterology and Hepatology, Wilford Hall Medical Center, Lackland Air Force Base, Texas, and coauthors refer to previous research linking coffee with decreased progression of liver fibrosis in patients with alcoholic cirrhosis, cirrhosis in general, and elevated liver enzymes. "This news is significant," they write, "because any modality that decreases the progression of fibrosis in chronic liver disease, especially if it confers few adverse effects, has the potential to improve morbidity and mortality."

In summing up the import of their findings, they add: "Moderate coffee consumption may be a benign adjunct to the comprehensive management of patients with [nonalcoholic steatohepatitis (NASH)]."
However, Arthur L. Klatsky, MD, from the Kaiser Permanente Northern California Division of Research in Oakland, pins more benefit on "avoiding risk factors, such as obesity, high alcoholic intake, and viral hepatitis," he told Medscape Medical News. "In my opinion, these are much more important than suggesting that people drink moderate amounts of coffee."
He continued: "I don't think that the public should be advised to drink coffee." Dr. Klatsky was not involved in the present study, but he was the lead author of the first paper linking coffee intake to cirrhosis, published in 1992.

The new finding comes from a validated caffeine questionnaire administered to 4 patient groups at the Brooke Army Medical Center Hepatology Clinic in Fort Sam Houston, Texas: negative controls, bland steatosis/not-NASH, NASH stage 0 to 1, and NASH stage 2 to 4. Between March 2010 and March 2011, 306 patients completed the caffeine questionnaires, which also tabulated tea drinking. Overall, coffee drinking accounted for 71.5% of all caffeine consumed. Patients were also questioned about their intake of other caffeinated beverages, such as various teas, cocoa, caffeine-fortified drinks, chocolate, and caffeine pills. No other caffeinated beverages showed a correlation with any dimension of liver protection (eg, NASH, insulin resistance, diabetes, liver enzymes).

Patients with no to early fibrosis received 57.5% of their caffeine from regular coffee, in comparison with those with advanced fibrosis, who consumed only 35.9% of their caffeine from coffee (P = .041). Average caffeine intake varied significantly across the 4 groups on analysis of variance (P = .024). Coffee intake specifically varied between the 4 groups (P = .011).
The authors flagged the finding that patients with bland steatosis/not-NASH and control patients drank less coffee than those patients with NASH/steatosis. They speculate: "It may be that coffee is only beneficial to those [patients with nonalcoholic fatty liver disease] with a propensity for fibrosis (i.e., NASH patients)."

The authors acknowledge several limitations to their analysis; namely, that their study "was not prospective and thus did not follow the effects of differences in fibrosis on clinical outcomes over time," as well as a lack of blinding of the interviewers. They advise further study of the specific components of coffee and their effects on metabolic activities of the liver.

The study's senior author has disclosed that he consults for Amylin. Dr. Klatsky and the other authors have disclosed no relevant financial relationships.
Hepatology. 2012;55:429-436. Abstract

Authors and Disclosures
Laura Newman, MALaura Newman is a freelance writer for Medscape.

Disclosure: Laura Newman, MA, has disclosed no relevant financial relationships.
Idenix Pharmaceuticals to Present at Two Upcoming Investor Conferences

CAMBRIDGE, Mass., Feb. 7, 2012 /PRNewswire/ -- Idenix Pharmaceuticals, Inc., (NASDAQ: IDIX) announced today that Idenix management will present a corporate overview at the 14th Annual BIO CEO & Investor Conference on February 14, 2012 at 10:00 a.m. ET at the Waldorf Astoria, New York City and at the Leerink Swann 2012 Global Healthcare Conference on February 15, 2012 at 3:00 p.m. ET at the Waldorf Astoria, New York City. 

The live and archived webcasts of the company presentations can be accessed under "Calendar of Events" in the Idenix Investor Center at www.idenix.com. Please log in approximately 5-10 minutes before each event to ensure a timely connection. The archived replay will be available on the Idenix website for two weeks following the conferences.

About Idenix
Idenix Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts, is a biopharmaceutical company engaged in the discovery and development of drugs for the treatment of human viral diseases. Idenix's current focus is on the treatment of patients with chronic hepatitis C infection. For further information about Idenix, please refer to http://www.idenix.com.
Idenix Pharmaceuticals Contacts:
Kelly Barry (617) 995-9033 (media)
Teri Dahlman (617) 995-9807 (investors)
SOURCE Idenix Pharmaceuticals, Inc.

 New Websites to Track Adverse Events
February 6, 2012
Press Highlights Section Editor: Grace L. Su, MD, University of Michigan Medical School
Story By: Kristine Novak, PhD, Science Editor, AGA Journals

Two websites have been created to help physicians and patients find and better interpret information about adverse events from drugs and medical devices.

One of the sites, AdverseEvents.com/ has streamlined the complicated U.S. Food and Drug Administration (FDA) database, simplifying searches for adverse event reports (AERs) on more than 4500 drugs. Another site, Clarimed.com has done the same for reports filed with the FDA on 130,000 medical devices—covering everything from syringes to stents to tanning beds to diagnostic tests.

The websites were launched in September by entrepreneurs who felt that adverse event data posted by the FDA was too difficult to interpret. According to The Wall St. Journal, AdverseEvents Inc. President and co-founder Brian Overstreet came up with the idea for the website when a friend's wife suffered a severe drug reaction—he went looking for information and found the FDA's AER files to be indecipherable.

The FDA has collected millions of AERs, ranging from fatigue to fatal heart attacks, for thousands of prescription drugs since 1969—it now has 6.4 million reports. The reports can be filed and read on the FDA’s AERs website. However, it is difficult to get an overall idea of the number or type of AERs for any specific drug, or a summary of interactions with other drugs. People seeking adverse event information can download raw quarterly data, but it isn't cumulative and requires technical expertise to use.

The system for devices, the Manufacturer and User Facility Device Experience (MAUDE), was started in the 1990s. According to The Wall St. Journal, 759,000 reports were filed for drugs and 238,000 for devices in 2010 alone. But reporting is voluntary, and represents only a fraction of all the side effects and malfunctions.

There is also a large amount variation in the reports, which come from drug and device manufacturers, patients, physicians, family members—even lawyers—and often contain errors and inconsistencies. "There are at least 440 different ways to spell Ambien and each has a separate file at the FDA," Overstreet told The Wall St. Journal.

AdverseEvents Inc. therefore developed an algorithm that filters duplicates and combines spelling variations, reducing over 200,000 drug names to about 4500. It also made the data easily searchable and comparable for thousands of conditions and side effects, back to 2004.

Gastroenterology Press Highlights

The FDA acknowledges the limitations of the data provided on its AERs site. The homepage reads: “there is no certainty that the reported event was actually due to the product. FDA does not require that a causal relationship between a product and event be proven, and reports do not always contain enough detail to properly evaluate an event. Further, FDA does not receive all adverse event reports that occur with a product…AERs cannot be used to calculate the incidence of an adverse event in the US population”.

According to The Wall St. Journal, the FDA defends its own use of the data for safety surveillance and issues frequent MedWatch alerts on potential problems. FDA analysts mine the data for worrisome trends that prompt further investigation and sometimes stronger warning labels or even removal from the market. For example, the cholesterol-lowering drug Baycol was withdrawn in 2001 after rhabdomyolysis was found to be an adverse event.

For consumers who want more information on drug side effects, "the best source is to read the product label and talk to your doctor or pharmacist," said Gerald Dal Pan, director of surveillance and epidemiology in the FDA's Center for Drug Evaluation and Research. AdverseEvents.com "is useful and necessary, but it may not be sufficient, given the problems with the FDA data," said Joe Graedon, a pharmacologist who runs ThePeople'sPharmacy.com, a website where visitors often discuss their own experiences with drugs.

According to The Wall St. Journal, consumer advocates have complained that the FDA is too understaffed to investigate all the potential problems with the AERs website, that regulatory actions can take years, and that consumers and health-care professionals need better safety information in the meantime.

Experts told The Wall St. Journal that AdverseEvents.com and Clarimed.com can be helpful, as long as users understand the caveats. "If you just want an impression of the side effects of a drug, those impressions are pretty accurate," said Thomas J. Moore, a senior scientist at the Institute for Safe Medication Practices.

Overstreet said that consumers looking for information "can base some conclusions on the 3 million-plus reports we have in this database, or they can go with what a couple of people are saying in an online discussion board—that's what a lot of people do now." The bias in the information provided on these discussion boards is one reason that both websites were created. Although basic searches are free, AdverseEvents started to charge consumers $10 a month for access to full drug reports starting Feb 1. It will offer health-care professionals and businesses more detailed information for additional fees. Clarimed might do the same. Both websites also offer a way to file reports to the FDA.

Off The Cuff
Not to be missed:  last week’s BMJ published an analysis by a team at Dartmouth Medical School led by Steven Woloshin and Lisa Schwartz – “Influence of medical journal press releases on the quality of associated newspaper coverage.

 This is an important contribution to our understanding of the food chain of the dissemination of research news to the American public:  medical journals feed journalists who feed the American public what they get out of journals – sometimes driven largely by what’s in journal news releases.  If the information at the source is complete and high quality, the flow of information from journalists to the public is more likely to be complete and high quality as well.  But this analysis also suggests that “low quality press releases might make (associated newspaper stories) worse.”.. 
4.08PM 07 February 2012
Professor Edzard Ernst 
Several years ago, I was refused from donating blood because I had recently received acupuncture as a participant of a trial. Even today, the NHS is very clear on this point: the current leaflet handed out to all blood donors states, 'you must not give blood if you have had acupuncture, unless this was done in the NHS or by statutory registered health care professional'.

But is this wise?

A Californian team investigated 494 patients with hepatitis C in order to determine the risk factors for this infection.1 Specifically they wanted to find out whether the risk factors differ between various ethnic groups. 55% of all patients were Caucasian, 20% Hispanic and 25% Asian.
The laboratory profiles of these sub-groups were similar. 94% and 86% of Caucasians and Hispanics had commonly known risk factors for hepatitis C such as blood transfusion, drug injection or tattooing. For Asian patients, the picture was significantly different: in this population, acupuncture was a prominent risk factor. Some 50% of this subgroup had had acupuncture prior to the infection (Caucasians = 31%, Hispanics = 20%).

Many British GPs regularly see Asian patients; these findings from the US therefore beg the question whether UK Asians might be at similar risks. Patients can, of course, only be infected, if the acupuncture needle is not sterile. Thus the chances of acquiring an infection via adequately handled disposable needles should be zero. All regulated acupuncturists are told to use proper and safe techniques. It follows that, in the UK, the risk of hepatitis C infection through acupuncture should be zero.

But what about the unregulated acupuncturists who Asian patients might consult? What about the TCM-outlets in our high streets? What about amateur Asian acupuncturists who are on no register at all? To the best of my knowledge, there is no research to answer these questions.
To be on the safe side, therefore, acupuncture should be considered as a potential source of hepatitis C, particularly in Asian patients. To prevent such infections, GPs should urge their Asian patients not to frequent unregistered acupuncturists. And to prevent infections via blood donors, the categorical statement by the NHS mentioned above seems correct.

1. Ho EY, Ha NB, Ahmed A, Ayoub W, Daugherty T, Barcia G et al. Prospective study of risk factors for hepatitis C acquisition by Caucasian, Hispanic, and Asian American patients. J Viral Hepatitis 2012; 19:e105-e111.


By Kristina Fiore, Staff Writer, MedPage Today
Action Points
Note that in a new guideline, the American College of Physicians (ACP) recommends monotherapy with metformin as the initial pharmacologic therapy for most patients with type 2 diabetes.
Note that the ACP recommends the addition of a second agent when patients have persistent hyperglycemia despite metformin and lifestyle modification.

Metformin should be the first drug of choice in oral therapy for type 2 diabetics who don't respond to diet and lifestyle changes, according to a new guideline.

A second agent can be added if metformin monotherapy fails to control hyperglycemia, but there's insufficient evidence to recommend one secondary agent over another, Amir Qaseem, MD, PhD, director of clinical policy at the American College of Physicians, and colleagues wrote in the organization's new guideline for the management of type 2 diabetes, published in the Annals of Internal Medicine.

"We found that most diabetes medications reduced blood sugar levels to a similar degree," Qaseem said in a statement. "However, metformin is more effective compared to other type 2 diabetes drugs in reducing blood sugar levels when used alone and in combination with other drugs."
There are currently 11 classes of drugs approved for treating hyperglycemia in type 2 diabetes, the researchers wrote, and most patients receive more than one class of diabetes medication.
To determine the optimal treatment strategy with these agents, Qaseem and colleagues conducted a comparative safety and effectiveness analysis of studies published between 1966 and April 2010.
All three recommendations in the guideline are strong and based on high-quality evidence, they said. First among the guidelines: Put patients on oral therapy when diet, exercise, and weight loss have failed to control hyperglycemia.

 There are no data as to the best time start oral therapy; instead, clinicians should take into account other complicating factors including life expectancy, microvascular and macrovascular complications, risk for adverse events related to glucose control, and patient preference, they wrote.
The patient's HbA1c target should also be based on an individual risk assessment, though Qaseem and colleagues noted that below 7% is a reasonable goal for many patients.

They also recommend that the first oral agent should be metformin monotherapy. Even though the evidence shows that most drugs reduce HbA1c to a similar degree, metformin appeared to be more effective than the others, they wrote.

It also conferred the additional benefits of reduced weight and improved cholesterol profiles and was associated with less hypoglycemia, they said. It does, however, carry increased risks for gastrointestinal side effects.

When metformin alone fails to control blood glucose, Qaseem and colleagues recommended using it in combination with another oral agent, noting that there's no good evidence to support one second-line therapy over another.

Generic sulfonylureas are the cheapest second-line agents, but adverse effects, especially hypoglycemia, are generally worse with combination therapies that include a sulfonylurea, they cautioned.

They also warned about well-known risks of heart failure associated with the thiazolidinedione class, which includes the rosiglitazone (Avandia) and pioglitazone (Actos).

Combining more than two agents wasn't evaluated in this review, they added, and patients with persistent hyperglycemia despite oral therapy may eventually need insulin.

Qaseem and colleagues noted that low-quality or insufficient evidence made it difficult to draw conclusions about the comparative effectiveness of medications on all-cause and cardiovascular mortality, cardiovascular and cerebrovascular morbidity, and microvascular outcomes.
Primary source: Annals of Internal Medicine

Wilson's Disease

Geneva, Switzerland: The first European Clinical Practice Guidelines (CPGs) for the diagnosis and management of Wilson's disease are published today by the European Association for the Study of the Liver (EASL) on the EASL website -- www.easl.eu.(1) Developed to assist physicians and healthcare providers in the clinical decision making process, the guidelines describe best practice for the diagnosis and treatment of patients with Wilson's disease -- a rare genetic(2) disorder that, if left untreated, is fatal.

Approximately one in 30,000 people worldwide are affected by Wilson's disease -- a condition in which copper is not excreted by the body effectively, leading to excess copper build up, liver failure and damage to the brain. While Wilson's disease may manifest at any age, the majority of patients present between the ages of 5 and 35.

Lead author Professor Peter Ferenci said: "The clinical presentation of Wilson's disease can vary widely, but it must be considered in any patient who presents with a combination of unexplained liver disease and neurological or neuropsychiatric disorders. In the absence of Kayser-Fleischer rings(3) -- which are typical, but not always present -- the guidelines recommend measurement of urinary copper excretion and hepatic parenchymal copper as diagnostic methods of choice. Notably, age alone should not be the basis for eliminating a diagnosis of Wilson's disease."
The CPGs, based on a systematic review of existing literature, provide best practice diagnosis and treatment protocols with an emphasis on:
  • Clinical presentation and prognosis
  • Diagnostic strategies (e.g. serum ceruloplasmin, basal 24-hour urinary copper excretion, genetic analysis)
  • Importance of family screening
  • Treatment options (e.g. chelating agents, zinc, liver transplantation)
With treatment, prolonged survival has become the norm for Wilson's disease patients. The guidelines recommend chelating agents -- drugs that bind to copper and remove it from the body (D-penicillamine or trientine) -- as the initial treatment for symptomatic patients and that, unless liver transplantation is performed, treatment is maintained for life.

Professor Roderick Houwen added: "Unfortunately, as there are no optimally designed randomized controlled trials conducted in Wilson's disease, there is a lack of high-quality evidence to estimate the relative treatment effects of the available drugs. Our evaluation is mostly based on large case series that have been reported in recent decades, which highlights a clear need to conduct more robust randomized controlled trials to better understand treatment for this rare condition."

Professor Mark Thurz, EASL Secretary General, added: "EASL is dedicated to promoting hepatology research and education to improve the worldwide treatment of liver disease. Its series of Clinical Practice Guidelines aims to promote best practice to drive better clinical outcomes and inform both the scientific community and the wider public of the latest developments in the field. We hope these new Wilson's disease guidelines provide clinicians with the most up-to-date, evidence based methods for the management of affected patients."

The Wilson's disease CPGs will be published in the March issue (Volume 56, No. 3) of the Journal of Hepatology -- EASL's official journal.

About EASL EASL is the leading European scientific society involved in promoting research and education in hepatology. EASL attracts the foremost hepatology experts and has an impressive track record in promoting research in liver disease, supporting wider education and promoting changes in European liver policy.
EASL's main focus on education and research is delivered through numerous events and initiatives, including:
  • The International Liver CongressTM which is the main scientific and professional event in hepatology worldwide
  • Meetings including Monothematic and Special conferences, Post Graduate courses and other endorsed meetings that take place throughout the year
  • Clinical and Basic Schools of Hepatology, a series of events covering different aspects in the field of hepatology
  • Journal of Hepatology published monthly
  • Participation in a number of policy initiatives at European level
1. Ferenci P, et al (2011) Wilson's disease: EASL Clinical Practice Guidelines. European Association for the Study of the Liver. Available at http://www.easl.eu/_clinical-practice-guideline
2. Due to mutations of the ATP7B gene on chromosome 13.
3. Kayser-Fleischer rings are a golden-brown discolouring of the eye's corneal rim. The cornea is the transparent front part of the eye that covers the iris, pupil, and anterior chamber.
For Your Reading Pleasure

Grand Rounds is the weekly summary of the best healthcare writing online, featuring stories, opinion and analysis from doctors, nurses, patients, researchers and administrators, as well as journalists. Each Tuesday, a different blogger takes the helm, publishing a new edition of Grand Rounds on their site. Each edition features the host’s picks for the ten best healthcare links of the week.

This weeks host EndoGoddess 

I am a total news junkie and always have been (which is probably why I started out college as a journalism major before deciding that I wanted to be a doctor). So, this week's edition of Grand Rounds features the news themes of the prior week and their relationship to health: politics, football fever, the power of facebook, red heart disease awareness, and the impact of pink.

The Impact of Pink:

Last week, the pink powers at Susan G. Komen Foundation first announced their intentions to defund future grant commitments to Planned Parenthood as an ‘ineligible’ organization, followed by a series of drifting explanations, and ultimately the rescission of their decision some 48+ hours post announcement. Healthcare executive, Gregg Masters, keyed into the social media response influencing these events in his blog post last week: http://xanatemedia.com/2012/02/how-to-kill-brands-goodwill-in-24-hours-or-less/ . The politics of women’s health care was summarized from the point of view of neuropsychologist Dominic Carone: http://t.co/hVl7XoI1 . The following post now circulating on pinterest proposed a new call to action and a good way to make the conflict eventually benefit the mission of ending breast cancer: http://bcaction.org/2012/02/03/a-victory-but-a-small-one-challenging-the-status-quo-of-breast-cancer/

Read More From The EndoGoddess 

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