Von Willebrand disease type 1: a diagnosis in search of a disease

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Quite a few people will relate withe these findings on the below article.  vWD is not as easy to diagnos as Hemophilia.  Continue reading for more information…..

ABSTRACT:
Article written by: J. Evan Sadler

Von Willebrand disease (VWD) type 1 is reported to be common but frequently is difficult to diagnose. Many people have nonspecific mild bleeding symptoms, von Willebrand factor (VWF) levels display low heritability, and low VWF levels (15% to 50% of normal) are weak risk factors for bleeding. Therefore, bleeding and low VWF levels often associate by chance. Even with stringent diagnostic criteria based on a triad of bleeding symptoms, a low VWF level, and a positive family history, the prevalence of “false-positive” VWD type 1 is comparable to the published prevalence of the disease. Consequently, many patients diagnosed with VWD type 1 do not have a specific hemorrhagic disease at all, which limits the utility of the diagnosis. This unfortunate reality is a consequence of trying to force patients into binary categories of “diseased” or “healthy” that are incompatible with the continuous biologic context in which VWF functions. The problem may be avoided by substituting an empirical epidemiologic approach like that applied to other modest risk factors for disease such as elevated cholesterol and high blood pressure. Such a risk management strategy could be generalized to include other hemorrhagic and thrombotic risk factors.

Read the rest of the article here.

Mobile App to Improve Bleeding Disorder Diagnosis

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Novo Nordisk announced in February 2011 the creation of the first mobile application, available on both the iPhone and online, to aid in the diagnosis of bleeding disorders. Coags Uncomplicated features a Lab Value Analyzer allowing physicians to input test results and receive a list of possible diagnoses to consider, including a description of each bleeding disorder and the possible meanings of test results. The application also provides step-by-step suggestions for suitable lab tests to narrow potential diagnoses.

Coags Uncomplicated was developed in partnership with Craig Kessler, MD, Professor of Medicine and Pathology and Director of the Coagulation Laboratory, Georgetown University Hospital, Lombardi Comprehensive Cancer Center.

Coags Uncomplicated was created to meet a need identified by U.S. market research that some specialists do not diagnose the presence of a bleeding disorder in patients with abnormal clotting activity. The failure to recognize a possible bleeding disorder often leads to an overemphasis on the location of the bleed and too little focus on the cause. These findings were presented at the American Society of Hematology Annual Meeting in Orlando, FL, on December 6, 2010.

“While physicians are increasingly using hand-held devices to access medical information, no such tool has existed to help with the diagnosis of bleeding disorders, especially when getting to the correct diagnosis quickly may be critical,” said Eddie Williams, Vice President, Biopharmaceuticals at Novo Nordisk, Inc. “Novo Nordisk was pleased to collaborate with Dr. Kessler on this innovative approach to addressing an unmet need, further demonstrating our commitment to the bleeding disorder community.”

The Coags Uncomplicated app is available for free download at www.coagsuncomplicated.com. The application tools and additional information can also be accessed online.

Source: PRNewswire, February 4, 2011

CDC Guide Available for Hemophilia Healthcare Providers

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The National Hemophilia Foundation is making available a self-learning guide for hemophilia healthcare providers. Foundations: A Comprehensive Approach to Hemophilia Care was developed and published in 2009 by the Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities and the Centers for Disease Control and Prevention (CDC). The booklet is a 2nd edition; it was first published in 1996.

Foundations was created to enhance the knowledge base and skills of new healthcare providers to patients with hemophilia. When combined with continuing education and clinical practice, it is expected to improve hemophilia patient care. The booklet includes four education modules: “Introduction to Hemophilia”; “Bleeding Episodes”; “Complications of Hemophilia”; and “Psychosocial Implications of Hemophilia.”

The guide was developed in partnership with the CDC Foundation, through a grant from Baxter Healthcare Corporation.

To obtain a single copy of Foundations: A Comprehensive Approach to Hemophilia Care healthcare providers should contact HANDI, NHF’s information resource center: 800.424.2634 or handi@hemophilia.org.

Where did the “Specialty” in specialty Pharmacy go?

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The decline of the specialty pharmacy

By Mina Kimes, writerOctober 25, 2010: 12:57 PM ET

FORTUNE — When Elizabeth Purvis’ son Tater was born last year with hemophilia, she had a wide range of pharmacies available to her through her pharmaceutical benefits manager (PBM), Express Scripts. Because the clotting medicine Tater takes to prevent excessive bleeding is rare and difficult to administer, she can’t get it at her local pharmacy; it is only sold at so-called specialty pharmacies, which also offer nursing services. Two months later, Express Scripts dropped the pharmacy she chose, Coram, from its network, and sent her a letter with three choices. The first, “Hemophilia of the Sunshine State,” was an out-of-state pharmacy she had never heard of. She later found out that it was owned by Express Scripts.

“They were pushing for all of us to use Express Scripts,” says Purvis, who lobbied for a local alternative and eventually found a nearby provider that could bill Tricare, the health insurer for military families (her husband is in the Army). “The little branches are able to provide so much more for us,” she explains, adding that she was apprehensive of depending on a large mail order operation based in another part of the country. “I didn’t just want to be another number in Express Scripts’ rolodex,” she says.

Specialty drugs, which treat rare diseases such as hemophilia, cancer, and multiple sclerosis, are one of the fastest-growing–and most lucrative–areas of healthcare. Due to their small patient volume (there are about 20,000 hemophiliacs in the U.S., compared to some 37 million people with high cholesterol) and complex manufacturing requirements, specialty treatments can cost tens of thousands of dollars. According to the 2010 Drug Trend Report put out by Medco, one of the country’s biggest PBMs, specialty drug prices climbed 14.7% last year, while regular brand-name drugs increased 9.2%.

Over the last decade, PBMs, which act as middlemen between retail pharmacies and employers or insurers, have started selling drugs, too; mostly through their massive mail order units, but also via their in-house specialty pharmacies. The big three companies–Express Scripts (ESRX, Fortune 500), CVS (CVS, Fortune 500) Caremark, and Medco (MHS, Fortune 500)–have all expanded their rare drug operations. When speaking about specialty drugs at a conference in June, Express Scripts CEO George Paz said: “This is going to be the growth driver.”

Are PBM’s steering customers away from speciality pharmacies?

Such pronouncements are worrisome to the owners of specialty pharmacies, who accuse the PBM industry of exploiting its position to capture more business. Russell Gay, the chairman of the Independent Specialty Pharmacy Coalition, says it is unfair that pharmaceutical benefits managers, who are supposed to evaluate drug transactions on behalf of payers, are also the ones executing those transactions. “How can you provide a check and balance against your own company?” he asks.

The shift has also affected clinics. Joe Pugliese, the head of the Hemophilia Alliance, a coalition of 83 federally funded, non-profit treatment centers, says the big PBMs have moved in recent months to shut out the centers from selling drugs, which he says they need to do to sustain their operations. “It is increasingly difficult for treatment centers to remain in network,” he says.

Dr. Steven Miller, the chief medical officer at Express Scripts, says that, while it is true that PBMs sometimes direct patients to their own specialty pharmacies, they do so because it’s in the best interests of customers and payers. “We’re looking for pharmacies that have the clinical expertise to support our patients, and also the buying power and systems to keep costs appropriate,” he says. “Many of the local specialty pharmacies can often match the safety factor…but often times they’re still not competitive from a cost standpoint.”

“The best confirmation is the marketplace,” says Mark Merritt, the CEO of the Pharmaceutical Care Management Association, which represents PBMs. Merritt points out that payers are sophisticated buyers who hire consultants to evaluate the plans.

But the savings generated by PBMs can be murky, even to experienced payers, says Terri Bernacchi, an analyst at healthcare research firm IMS who previously directed Wisconsin’s Blue Cross program. “Sometimes when mail order is promoted to a health plan, they don’t understand the implications from a cost perspective,” she says. “It’s like a big algebra equation.”

The new healthcare reform law calls for PBMs that participate in state insurance exchange to disclose detailed information on their pricing mechanisms and savings rates. The clause has become a battle ground issue for community pharmacists, who support it, and representatives of the PBM industry, who say it would hurt competition.

PBMs argue that they can cut costs because their networks are bigger and more efficient. Their specialty pharmacies also offer phone hotlines and contract with nurses who make home visits. But some specialty drug patients say that, given the nature of their illnesses, they need greater choice and personalized attention.

Purvis wasn’t the only Express Scripts member who saw her options whittled down after Tricare altered its agreement with the PBM last year. Hemophiliac patients and parents around the country received similar letters, and many were forced to switch providers. Though Express Scripts later widened the network, some customers, like Tricare member Colleen Pascua, still say their choices are insufficient.

Pascua, whose nine year old son has hemophilia and receives injections through a catheter embedded in his chest, lives in Redding, Calif., a rural town that is about two and a half hours away from Sacramento. When her son outgrows his catheter, she says, she will need the help of a nurse to inject medicine directly into his veins. But the only nurse in her area works for Accredo, which is owned by Express Scripts rival Medco and is not on her list of in-network providers.

Express Scripts says it accepts out-of-network requests, but doing so would require Pascua to pay a steep co-payment on a drug that she says costs some $20,000 a month. A spokesman for Express Scripts wrote in an email: “In the tough economic climate, some of our clients are having to make difficult decisions, including restricting their networks.” Tricare, Pascua’s insurance plan, did not immediately respond to requests for comment.

Pascua is skeptical of the PBM’s motives for changing her options. “They’re funneling all of these specialty patients because they want their business,” she says. “It’s a fox in the henhouse. They want to wipe out their competition.”

From CNNMoney.com

Hemophilia Care – Past, Present, and Future

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Haemophilia care then, now and in the future

This is a link to an article from “Haemophilia: the Official Journal of the World Federation of Hemophilia” about the past, current, and future pediatric care of Hemophilia patients, as well as current and future geriatric care for Hemophilia patients.  Volume 15, Issue s1, 2009.Pages: 2–7

The article recently became viewable online without a fee.  I hope you find the information relevant.  Life Expectancy data, long-acting FVIII products, and demographic data was included in the report.

ADVOCACY

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Advocacy: (noun) : The act of pleading or arguing in favor of something, such as a cause, idea, or policy; active support.

Not a easy word to influence someone to action.  We all need to advocate for Hemophilia.  We are a minority and we need to be a vocal minority.

Where do we start?  What should my first step be?  The following it taken from Hemaware Magazine

Can You Be an Advocate?

The idea of becoming an advocate can be overwhelming for a first-timer. Questions arise, such as:

  • How do I get started?
  • How much of a time commitment do I have to make?
  • Do I have the right skills?

Advocacy is not a unique skill that only some people have, Gray says. “Everyone in the bleeding disorders community can do it and be really effective.”

Parents are great advocates if they understand that they have always advocated for something or other, points out Kim Bernstein, JD, former director of the Hemophilia Health Services’ A.C.C.E.S.S. program (Advocating for Chronic Conditions, Entitlements and Social Services) in Tampa, Florida. Bernstein is now president of Grassroots Advocacy Strategy & Solutions, which advocates for individuals and communities who need access to healthcare.

“You are an advocate when you go to the ER and the staff asks you how long you have had hemophilia or something else crazy like that, or when you say, ‘I need you to call my treatment center,’” says Bernstein. People may view this as different from going to Congress, but it is not, she says. It’s about educating people on the details of bleeding disorders and how the disease affects lives.

Start by working with your local hemophilia chapter and take advantage of the information it can provide. The bleeding disorders community is most effective when it speaks with one voice. Local state chapters are likely to have a summary of the issues the community is facing, along with helpful fact sheets.

The Advocacy Center on NHF’s Web site is also an important resource in learning more about federal advocacy issues that may affect you and your state. Kerry ­Fatula, executive director of NHF’s Western Pennsylvania Chapter in Pittsburgh, recommends finding a mentor when starting out in advocacy. Any chapter is a good resource, she notes, even if it doesn’t have an advocacy committee, because it is familiar with most government agencies.

The important think is to start!  Don’t be pushy and rude.  Be a resource to your Government Representatives, Hospitals,  School, etc.  Get Involved and EDUCATE!

Bleedings Disorders Bill

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Bleeding Disorders Bill Introduced in Congress
On March 15, 2010, Congresswoman Carolyn McCarthy introduced H.R. 4846 entitled The Bleeding Disorders Screening, Awareness, and Further Education Act of 2010.” 
 
If passed the bill will:
  • Increase research funding for all bleeding disorders
  • Provide greater physician and public education on bleeding disorders
  • Screen college students for von Willebrands (vWD) at institutions of higher education
  • Enhance diagnosis rates and treatment of bleeding disorders
  • Decrease the number of unnecessary hysterectomies performed each year due to undiagnosed cases of vWD.
Each aspect of this measure is important to the bleeding disorders community.  Education and awareness of bleeding disorders has the potential for earlier diagnosis, which can lead to quality care and better health outcomes.  Furthermore, additional screening mechanisms, increased funding for research and a research plan can positively impact the lives of those who remain undiagnosed.   You can read the full bill by clicking here.
 

Experts Reach Consensus on Diagnosis, Treatment of Bleeding Disorders in Women

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Taken from: DocGuide.com

NEW YORK — June 2, 2009 — Because bleeding from the reproductive tract is a naturally occurring event during menstruation and childbirth, women who exhibit menorrhagia, or excessive bleeding after their menstrual cycle, may have underlying diseases that are underdiagnosed.

In order to address important issues related to the diagnosis and management of reproductive tract bleeding in women with bleeding disorders, a consensus conference was convened. Results are published in the July 2009 issue of the American Journal of Obstetrics & Gynecology.

The goals of the consensus conference were to highlight the problems these women experience and to provide clinical information and recommend strategies to guide practicing obstetricians and gynaecologists.

Although the majority of women who present with menorrhagia do not have a bleeding disorder, the conference participants identified more than a dozen symptoms that suggest further evaluation, including menorrhagia since puberty, a family history of a bleeding disorder, and personal history of 1, but usually several, of the following symptoms: nosebleeds (generally bilateral > 10 minutes), more than once in the past year; notable bruising without injury (and with bruises > 2 cm in diameter); minor wound bleeding from trivial cuts lasting > 5 minutes; or prolonged or excessive bleeding following dental extraction.

A haematologic evaluation of the patient’s platelet number and function and her coagulation factor profile should be assessed in collaboration with a haematologist. Meeting participants also agreed that haematologic evaluations should be repeated to confirm the diagnosis of a bleeding disorder.

“Obstetricians and gynaecologists should be aware of bleeding disorders such as [Von Willebrand Disease], rare bleeding disorders and platelet disorders, which remain underdiagnosed in women with menorrhagia and potentially in other cases of abnormal bleeding such as postpartum hemorrhage,” said co-author Andra H. James, MD, Women’s Hemostasis and Thrombosis Clinic, Duke University Medical Center, Durham, North Carolina.

“Clues, including a family or personal history of bleeding events, should provoke suspicion of an underlying bleeding disorder. Responding to these clues facilitates collaboration among obstetrician-gynaecologists and haematologists that could lead to a decrease in the diagnosis of idiopathic menorrhagia and allow more effective management of bleeding events.”

SOURCE: Elsevier Health Sciences

von Willebrand Disease and Sports

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The 5-foot-6 Brooke Cortes became interested in gymnastics while watching the Olympics when she was 2. Cortes patted her mother on the leg and said, “I want to go to ’lympics.”

Christine Cortes enrolled her daughter in a recreation class and Brooke began competing when she was 7.

Shortly after quitting gymnastics, Cortes was diagnosed with von Willebrand disease — an inherited blood disorder caused by a defect or deficiency of a blood clotting protein called von Willebrand factor.

Cortes’ mother and her two sisters also have the disorder, but Brooke has the most severe case.

“It is hard, but I try not to let it get in my way,” Brooke said. “I just don’t really think about it and I feel that even though I have this blood disease I should be treated normal. There are still things I want to do.”

Cortes takes an oral medication and a nasal spray to help control the disorder.

She plans to attend a camp this summer to learn how to self infuse Humate-P, which raises the level of von Willebrand factor in the blood.

A few months ago, Cortes had a tonsillectomy because of recurring bouts of strep throat. The procedure is usually done on an outpatient basis, but Cortes remained in the hospital for seven days to control the bleeding and missed four weeks of practice.

MDs refer profitable patients to their ASCs

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ASC = An Ambulatory Surgery Center can also known as outpatient surgery centers or same day surgery centers

This isn’t too surprising, but it’s not something policymakers will like, either. A new study of referral patterns in the Philadelphia and Pittsburgh metros suggests that physicians who are high-admitters to physician-owned ASCs send far more privately insured patients to those facilities, while sending Medicaid enrollees to hospital outpatient departments, in many cases. The study, published in Health Affairsand completed through the University of Chicago, looks at whether having a stake in an ASC affects the types of patients a physician would refer to a facility. Research found that for the top 50 percent of physician referrals to ASCs, 45 percent of Medicaid patients were sent to hospital outpatient departments, while 92 percent of privately insured patients were sent to physician-owned ASCs.

Folks, these numbers seem to pretty much clinch the argument that ASCs keep the best-paying patients for themselves, while blocking the poor from getting whatever benefits the specialized ASC services might offer. Looks like this could kick off some heated discussions on Capitol Hill.

To get more information on the study:
- read the Health Affairs report
- read this Modern Healthcare article (reg. req.)

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They (Doctors) are already screening us by our health insurance type.  Why not let them continue to screen us by our ablility to pay and our payment history.   Will the Hippocratic Oath change from “First do no harm” to “First , Check the credit score and insurance, THEN do no harm”.  Look here for the NIH version of the Hippocratic Oath.  Want to read the Original Greek Oath or translation, go here.

NOVA produced a great peice, “The Hippocratic Oath Today: Meaningless Relic or Invaluable Moral Guide?”  Most medical schools have changed the original oath to suit their ends.  For example, only 14 percent of modern oaths prohibit euthanasia, 11 percent hold convenant with a deity, 8 percent foreswear abortion, and a mere 3 percent forbid sexual contact with patients—all maxims held sacred in the classical version.  What oath did your doctor take? 

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