Key Issues Dialogue: ITP - All About Bleeding · Biotherapies for Life™ Key Issues Dialogue: ITP...

Biotherapies for Life™

Key Issues Dialogue: ITPFeaturing Douglas B. Cines, M.D., Garrett Bergman, M.D.,

Joan Young and Dennis Jackman

From left to right, Dr. Douglas Cines, Joan Young, Dr. Garrett Bergman and Dennis Jackman

About ITP

D E N N I S J A C K M A N : What is ITP?

D R . D O U G L A S C I N E S : ITP is a bleeding disorder caused by a reduction in platelets

—the circulating cells that help prevent bleeding. ITP is an abbreviation for idiopathic

thrombocytopenic purpura, which technically means a condition of unknown origin

manifested by a low platelet count and red or purple discolorations of the skin. ITP is

also an abbreviation for immune thrombocytopenic purpura since most cases of ITP result

from an antibody response against platelets. Both terms are accurate. In ITP, antibodies

react with the patient’s own cells and lead to their removal from circulation—and in

some cases, a reduction in their production. When the platelet count in the bloodstream

falls to a certain level stressing the vascular system, internal bleeding can ensue.

D E N N I S : How common is ITP?

D O U G : It’s very difficult to know. We estimate that approximately five in 10,000

adults and children have the disorder; therefore, about 100,000 people in the United

States are afflicted. This statistic may, however, underestimate the prevalence of the

disorder because patients who have the disease, but have been successfully treated,

are no longer counted, and, alternatively, patients who have the disease but are not

afflicted have never been counted.

D E N N I S : Why is it so hard to come up with an accurate number?

D O U G : Partly it’s the criteria for making this diagnosis, and partly it’s the reporting of

the disease through coding procedures. There are inherent difficulties in coding, for

instance. How many low platelet counts does it take until the diagnostic code used in

the ICD-10 (International Classification of Diseases, 10th edition) becomes ITP? Not all

patients with low platelet counts have ITP. Also, it’s difficult to keep track of medical

disorders in many countries, especially the United States. We don’t have registries for

this condition.

D R . G A R R E T T B E R G M A N : And there are some patients who have ITP and

don’t have symptoms that would cause them to see a physician—cases that

may remain undiagnosed until the patient has a catastrophic hemorrhage.

Recently leaders from the ITP

community met to talk about

diagnosis, treatment,

developments and challenges

surrounding ITP.

This dialogue is part of a series

sponsored by CSL Behring for

patients, caregivers and

healthcare professionals in the

communities we serve.

Key Issues Dialogue: ITPFeaturing Douglas B. Cines, M.D., Garrett Bergman, M.D.,

Joan Young and Dennis Jackman

K E Y I S S U E S D I A L O G U E : I T P

A Diagnosis of Exclusion

D E N N I S : To what extent is ITP under-diagnosed?

J O A N Y O U N G : We think it’s under-diagnosed because we are aware of patients

who thought they were fine, but went to their physicians, had a blood test and

learned they had a problem. We also know that there are a lot of people diagnosed

with ITP who may actually have other diseases because the diagnosis is based on the

principle of exclusion. Then there are the mystery cases where a patient has a platelet

count of 100,000 one year, next year it’s 90,000 and the following year it’s 80,000.

A physician may not diagnosis this condition as ITP.

D E N N I S : What can be done to improve physicians’ ability to diagnose ITP?

D O U G : Right now ITP is, as Joan said, a diagnosis of exclusion. A physician has to

exclude other causes of thrombocytopenia before diagnosing ITP. This list of diseases to

exclude is very long. And a physician may not be able to acquire all the information he

needs to exclude every other possibility. For example, exposure to certain medications

or drugs may induce a low platelet count. Also, a patient could be exposed to toxins.

Or in some cases, there are familial causes but the physician doesn’t have all the family

members present. Then, too, there are concurrent medical problems, which may result

in thrombocytopenia, on which a diagnosis of ITP may be superimposed.

In fact, the best diagnostic test is how a patient responds to ITP directed therapy. One of

the best indicators of the disease is to give a patient a treatment regime unique for

ITP—one that does not impact other conditions—and see if the patient responds.

G A R R E T T: In children, however, it’s a little different. When children present symptoms,

usually physicians go through the same process of excluding other causes as they do

for adults. For example, when a child has bruises and petechiae, parents wonder, “Does

my child have leukemia?” Excluding leukemia as the cause of thrombocytopenia is,

therefore, a critical step in ITP diagnosis.

D E N N I S : What are some solutions to improve diagnosis?

D O U G : The most rational long-term goal would be a test identifying what

physicians believe to be the central pathophysiology of this disorder, namely, the

anti-platelet antibody. If this disorder were always caused by these antibodies

and these antibodies are either not present or rare in other conditions, then we

would have an objective means of clinical diagnosis. As of yet, physicians do

not have that tool.

Education for Patients and Physicians

D E N N I S : What’s the role of both patient and physician education?

J O A N : To the extent we can educate patients on the different factors that can

potentially lower platelet counts—all those things that could be confused with ITP—

K E Y I S S U E S D I A L O G U E : I T P [ 2 ]

Right now ITP is a diagnosis

of exclusion. A physician has

to exclude other causes of

thrombocytopenia before

diagnosing ITP. This list of

diseases to exclude is very

long. And a physician may

not be able to acquire all

the information he needs

to exclude every other

possibility.

there’s the chance of a much stronger dialogue between the patients and physicians

and a much better chance of an accurate diagnosis.

D O U G : In straightforward cases, there are many pediatricians, internists, hematologists

and oncologists who not only make the initial diagnosis, but also provide primary

therapeutic interventions. However, oncologists care for a very high percentage of

patients with hematological disorders. In fact, it’s not uncommon for an oncologist to

have only one, two, or three ITP patients a year. For some oncologists, however, isolated

thrombocytopenia is not a differential diagnosis. For example, they may not think

to ask if anybody in a patient’s family had a familiar problem with bleeding after an

operation or had recurrent spontaneous abortions—both of which are conditions that

can be linked to ITP. ITP is a small subset of most physicians’ day-to-day activities. Due

to lack of exposure, physicians are less comfortable making diagnoses and providing

intervention, especially in complex cases.

D E N N I S : What steps can be taken to increase the awareness of physicians?

G A R R E T T: One of the things CSL Behring does to increase awareness is to set up

seminars and continuing education courses and bring in speakers like Dr. Cines to tell

us about the latest research for diagnostic or therapeutic interventions.

J O A N : At the Platelet Disorder Support Association, one of our primary goals is to

educate patients—we just started a program to educate hematologists as well—by

providing educational materials at our conferences and meetings, on our Web site

and in pamphlets.

What Role for Government?

D E N N I S : Does government have role in awareness, diagnosis and treatment?

D O U G : There’s a serious need for better understanding of autoimmune disorders,

of which ITP is one of the most common. Think of what we would learn from under-

standing how these auto-antibodies arise. ITP has a lot of treatments, but there are

other disorders that have no treatments. If we could understand the pathogenesis of

how these antibodies arise, we would be able to create directed therapeutics for this

and many other disorders.

J O A N : I agree. NIH could play a major role in pure research. Other government entities

like the CDC could also play a role. There are opportunities to develop educational

materials and to broaden the spectrum of treatment. EPA could look at environmental

triggers of disease since there’s a possibility that ITP could be triggered by an environ-

mental factor.

G A R R E T T: A national registry could be established as a database for research.

Registries have been beneficial in understanding the pathology and natural history of

diseases in other therapeutic areas.


When the child has

bruises and petechiae,

parents wonder, “Does my

child have leukemia?”

Excluding leukemia as the

cause of thrombocytopenia

is, therefore, a critical step

in ITP diagnosis.

D O U G : Yes, a registry is very important. So far, there is just one registry. It’s a pediatric

registry and physicians can assess the actual incidence of cerebral hemorrhage, which is

the most feared complication. This registry provides answers to fundamental questions,

such as how long after the diagnosis does cerebral hemorrhage generally occur? What

co-morbid factors are present? What is the platelet count? What interventions have

been used? Not one doctor, not even an expert, is going to see enough patients to be

able to make definitive statements about which kids are at risk and most in need of

initial therapy and which kids can be left alone because their chances of recovering

spontaneously are great.

About PDSA

D E N N I S : Joan, tell us what your organization, the Platelet Disorder Support

Association, does.

J O A N : Let me begin by telling you how it all started. In 1992 I was diagnosed with

ITP. I had a really terrible case; zero platelet count at one time. I am one of the very

fortunate people who recovered and remained in remission. I have been in remission

since ‘96. I started PDSA because I wanted to help others avoid the difficulties with the

disease that I had endured. Since I was a computer professional I created a Web site to

inform patients about ITP. I was amazed by the number of people who visited the Web

site. It told me a great need existed for information and connection. After launching

the Web site, we founded the Platelet Disorder Support Association (PDSA).

PDSA started as a way for people to learn about ITP and to meet other people with the

same condition. This remains our core mission and the majority of our programs reflect

this mission. We have newsletters, conferences, monthly e-news, name exchange pro-

grams, local group meetings, regional meetings, booklets and a lot of other publications.

D E N N I S : How many people come to your organization?

J O A N : Thousands. That’s why I think that the numbers cited for the incidence and

prevalence may be a little low. When 3,000 people request information through our

Web site annually, it’s evident that more people are inquiring about the disease than

have been diagnosed with the disease. We currently have 24,000 people on our mailing

list. About 15 percent of these individuals live outside of the U.S. It’s definitely a global

organization.

D E N N I S : Are all your programs educational?

J O A N : Educational programs comprise about 80 percent of what we do, but we do

other things as well. In conjunction with other groups, we advocate on behalf of

patients with the disease. We have also conducted research, mostly surveys. In the

future, we intend to publish more information, offer more meetings and become

more active in advocacy.


PDSA started as a way for

people to learn about ITP and

to meet other people with

the same condition.

G A R R E T T: Joan, do people who register on your site and take advantage of what

your organization offers already have a diagnosis of ITP?

J O A N : Not necessarily. Some people have a low platelet count and are confused.

G A R R E T T: My guess would be that for the general public, ITP is not recognized as

an entity the way some other diseases are. I can’t remember ITP being profiled on TV

shows like ER and Grey’s Anatomy.

J O A N : Yes. Most people have never heard of the disease before their diagnosis.

The Face of ITP

D E N N I S : How would you describe the people affected by ITP?

D O U G : A wide variety of people are impacted by ITP. In rare cases, patients present

with serious, catastrophic internal bleeding. On the other hand, many affected individuals

have no symptoms or few symptoms. Such individuals only have trouble when

something else happens. For example, they take aspirin, develop an ulcer or need

surgery. Between these two extremes, are patients who have various degrees of

bruising, heavy menses, or epistaxis (bleeding from the nose).

J O A N : We see a range of patients similar to what Doug describes, but from a

different perspective. Some people with ITP go to work every day and the disease

remains a minor part of their life. Others really are at death’s door, and the disease

takes over their life. Every day, such patients watch for bleeding episodes. They worry

about hitting their heads or waking up with blood streaming from their noses. And

then, of course, between these two extremes, many patients accept the disease as

part of lives and they manage to move ahead. ITP affects individuals of all ages.

D E N N I S : If it is diagnosed and treated, what’s the prognosis?

D O U G : Quality of life depends on the efficacy and toxicities of each of the treatments.

The first step is making a proper diagnosis because appropriate therapies are relatively

specific for this disease. The second step is addressing the clinical presentation. Is the

patient asymptomatic, with a little bruising, or does the patient have a very serious

bleeding problem? At the next level, even if bleeding isn’t a major issue, does the

patient have a platelet count that is a useful surrogate marker for risk? It’s easy to

know if the patient has obvious clinical ITP. It’s much more difficult if the patient is

having a little bit of bleeding once in a while or maybe their menstrual period is a little

heavy. How much therapy should a physician provide for this patient?

Historically, I think patients have been over-treated. I think there has been a change in

practice over the past few decades. Physicians now recognize they don’t have to aim

for a near-normal platelet count in every individual every day.


Historically, I think patients

have been over-treated.

I think there has been a

change in practice over the

past few decades.

Physicians now recognize

they don’t have to aim for

a near-normal platelet

count in every individual

every day.

D E N N I S : Are there consensus guidelines on platelet count?

D O U G : In general, most physicians would be comfortable saying that a patient who

has no special risk because of co-morbid conditions or unusual life contingencies and

whose platelet count is above 50,000 probably doesn’t need any type of chronic ongo-

ing treatment. Many experts would agree that if the platelet count is below 20,000,

the patient probably is at some risk, but not every patient. Some patients do fine and

some don’t. So the question is, is there a magic number? How about 30,000? Does a

physician give patients a margin in case their count goes down during a viral illness or

if by accident they take a cold medication with aspirin? With a count between 30,000

and 50,000, most physicians would probably prefer not to treat. Other physicians

would tell you they treat for bleeding only. Unfortunately, we don’t have the data

that says unequivocally when treatment is and is not appropriate, in a way that can

be generalized to the entire population.

G A R R E T T : Doug, if you had a patient whom you decided you would prefer not to

treat, let’s say with a platelet count above 50,000 but not much above 50,000, what kind

of recommendations would you give about activity restrictions?

D O U G : This is the art of medicine. There are options that could theoretically cure. There

are options that could ameliorate. We would now have to ask, what are the patient’s life

expectations? Is the patient a college football player? If a patient is rendered ineligible for

a scholarship due to an abnormal platelet count, then a physician would be taking away

a lifestyle option. In these types of negotiations, the patient plays the dominant role. What

are the chances this patient’s platelet count will plummet to nothing? What’s the chance

this patient is going to need a surgical or invasive procedure because of their age? Does

the patient bleed or not bleed? How does the patient tolerate therapy? The less compelling

the medical case for intervention, the more important the patient’s preferences become.

Treatment Options

D E N N I S : Let's talk about the treatment options.

D O U G : The first option is “no treatment”. The second option would present in a mild

emergency, where the patient is having mild petechia—little red spots —with reduced

platelets or increased bruising. If a patient has a true emergency, that patient is probably

going to get a combination of medications. There is, again, no single standard of care.

But I do think it is wise for an adult with serious bleeding to be treated with multiple

therapies upfront because each therapy has a percent response rate and a percent

non-response rate. So if the patient is actively, acutely bleeding, the physician also

needs to consider giving them a platelet transfusion, even though they are destroying

their own platelets—and would destroy new ones as well.

Then there are interventions aimed at decreasing platelet destruction. These interventions

include intravenous gamma globulin, intravenous anti-D, such as Rhophylac®, and various

doses of cortical steroids. And, finally, there are other treatments that can be used in

an attempt to paralyze the destructive process.


There is, again, no single

standard of care. But I do

think it is wise for an adult

with serious bleeding to be

treated with multiple

therapies upfront because

each therapy has a percent

response rate and a percent

non-response rate.

D E N N I S : What are your concerns about chronic treatment?

D O U G : The success rate is very high for aggressive, multi-modality therapy upfront.

Of course, depending on the patient’s overall health status, the physician may want to

minimize the treatment burden.

D E N N I S : But you also don't want a recurrence.

D O U G : No, a physician does not want a patient having episodic, potentially catastrophic

bleeds. Therefore, in making a decision regarding the appropriate long-term treatment

regime, a physician should consider a patient’s natural history. A physician needs to ask

himself/herself, “What's going to happen if I don't do anything for this patient?” This

is an example where patient registries would be very helpful by providing a better

understanding of long-term ITP outcomes.

In determining appropriate treatment options, children and adults fall in different

categories. About 80 percent of children have a self-limited disorder, which will

improve within six to 12 months. Therefore, options appropriate for an adult may

be deferred in a young child.

Evolution in Treatment

D E N N I S : What evolution do you see in treatment?

D O U G : There’s understanding now that the prognosis for ITP is improving. With

treatments like anti-D and more recently with high-dose pulses of cortical steroids,

there may be a population of patients who will attain adequate, if not normal, platelet

counts. This may not be a majority of patients, but a substantial minority. After a period

of six to 12 months, such patients need little or no subsequent care and are unlikely to

suffer catastrophic relapses.

D E N N I S : Do you have a percentage estimate for such patients?

D O U G : I’m making an estimation because these are very small subsets... let’s say 30 or

40 percent. Before progressing to second-line treatments, most hematologists have a

designated window during which they are comfortable watching patients and monitoring

symptoms and platelet counts.

D E N N I S : For those patients who don’t resolve, what do you do?

D O U G : Traditional treatment would be a combination of therapies that act on different

aspects of the ITP process. But there are toxicities, inconvenience of administration and

costs that affect lifestyles as well. Physicians must gauge the rate with which to remove

the steroids and rely upon IVIg or anti-D as a maintenance therapy.

What Quality of Life?

D E N N I S : What is the quality of life for patients who are appropriately diagnosed

and treated?


Patients are actively

exploring alternatives to

treatment options. Patients

are also very interested in

side effect profiles.

Increasingly, I think the

cost of treatment is also

very important.


D O U G : With all existing therapies, probably five to 10 percent of patients today have

serious, life-altering, if not life-threatening, disease.

D E N N I S : Does that mean that 80 to 90 percent of patients actually have a

managed condition and lead relatively normal lives?

D O U G : Patients range from individuals doing extremely well, not worrying about

their 80,000-platelet count, to individuals on the edge with platelet counts of 28,000,

32,000 and everything in between. Patients must consider questions such as, how

frequently do I need to get a platelet count? If I travel, do I need to see a doctor? How

often do I need my medication changed? What am I not doing that I would like to be

doing with my life? So patients manage; yes, they manage.

J O A N : I’ll mention some patient perspectives on the treatments. In one of our

surveys, we asked patients what treatments worked well and which treatments didn’t.

Cortical steroids were the most hated treatment. The other thing that hasn’t been

mentioned here is splenectomy. Some years ago the standard treatment was

Prednisone followed by splenectomy. It was just kind of one-two; it was how the

disease was treated. And splenectomy is probably the second most disliked treatment

from a patient perspective. So patients are actively exploring alternatives to these two

treatment options. Patients are also very interested in side effect profiles. Increasingly, I

think the cost of treatment is also very important. More and more patients don’t have

insurance or don’t know how they’re going to pay for treatments. Even those who

have insurance sometimes can’t afford the co-payments.

Getting Reimbursed

D E N N I S : How much of an issue is reimbursement?

D O U G : I’m in the ivory tower to some extent, so for me reimbursement is usually

not a problem. When I write letters to the insurers, I usually see a response. Moreover,

universities often provide social services, along with payment plans and forgiveness.

In my practice, I can do what I think is right.

But in the real world, where doctors are ordering drugs or paying for the drugs, they

hope to be reimbursed and support their practice. For example, if a doctor is treating a

non-paying patient, he could just as well be treating a paying patient. And then doctors

have other costs including their own insurance payments. With significant expense

burdens, how much money does a doctor actually recoup for a given modality and

time invested in patient care? These are real issues for physicians and, therefore,

become real issues for patients.

G A R R E T T: Can you rank the different kinds of treatments in terms of cost?

D O U G : It’s very difficult because some treatments are administered repetitively. IVIg is

given every 14 days, 17 days or 20 days; therefore, it is the most expensive proposition.

Repetitive anti-D is less expensive, but costly. Splenectomy is a one-time cost. Recently,

With significant expense

burdens, how much money

does a doctor actually

recoup for a given modality

and time invested in patient

care? These are real issues

for physicians and,

therefore, become real

issues for patients.


various drugs that stimulate platelet production have entered the market and are intend-

ed for indefinite, chronic use. It has not yet been determined how these drugs will be

marketed. Namely, how will these drugs be priced and reimbursed? Potentially, these

drugs could cost a lot more than steroids.

J O A N : I’ve always wanted a good study done of cortical steroids. The pill is cheap,

but what about the ensuing path of destruction? I mean in terms of diabetes, hip

replacements and terrible things like divorce or even psychosis.

The true cost of corticosteroid treatment, in terms of its impact on patients, is not really

reflected in the treatment cost. Not even splenectomy. I don’t have a spleen, and it

concerns me. Without a spleen, I’m permanently immuno-compromised. Will I ever go

to a third-world country? Not in my lifetime, because I can’t fight off diseases to which

I might be exposed. Have I taken 10 years off my life because now I’m more susceptible

to communicable diseases? Perhaps.

Then there is the increased risk of sepsis, malaria and immunosuppression from spleen

removal. And, recently, concerns about pulmonary hypertension resulting from spleen

removal have arisen. We need to look at a higher order of pharmo-economics, where

the impact upon the entire medical condition, not just the cost of the drug and its

administration, is considered.

New Paradigm for Reimbursement: Quality of Care

D E N N I S : What is needed to change the reimbursement paradigm?

D O U G : If we could shift the paradigm of reimbursement to quality of care, then we

could have a significant impact. We could have groups composed of not just ITP

experts but also economists and patients, which would come together to define

guidelines for quality of care. The guidelines developed need not be prescriptive, just

suggestive. With cause, we could deviate in either direction at any time. A physician

has individual responsibilities to the patient, but quality care guidelines would provide

a baseline of care. This approach would also ensure that insurance companies did not

deviate from these guidelines.

D O U G : If Medicare were to prescribe to quality of care outcome measurements, then

everybody else is going to follow.

J O A N : From a patient perspective, I think there is too much variability in the disease

to say, okay, do this first, this second, this third, and everything is going to be okay.

I don’t think the disease works that way.

D O U G : In other words, if either 90 percent or 10 percent of a physician’s patients

are getting splenectomies, then physicians are probably not managing the disease

appropriately. On the one hand, physicians are probably using too much steroid

treatment. On the other hand, they are not trying other available modalities.

We need to look at a

higher order of pharmo-

economics, where the

impact upon the entire

medical condition, not

just the cost of the drug

and its administration,

is considered.

K E Y I S S U E S D I A L O G U E : I T P [ 1 0 ]

J O A N : What comes to mind is, who are we treating here—the insurance companies,

the doctors or the patients?

D O U G : What’s lost in all this is: “what’s the best outcome for the patient?”

D E N N I S : Joan, do you have an idea what percentage of your patients might be

on Medicare?

J O A N : One of the interesting findings recently is that there are more Medicare patients

with ITP than we had originally thought. At one point, it was regarded as more of a

young person’s disease and a disease of women of childbearing age. But now we’re

hearing from seniors, and among seniors it seems to affect both men and women equally.

Setting the Priorities

D E N N I S : What are the priorities for ITP from here?

D O U G : We certainly need clinical care and outcomes research. Equally important is

basic science inquiry into the etiology and pathophysiology of this particular, very common

selective autoimmune disorder. I think this research will bear fruit widely in other areas.

If we could develop technologies to identify the evolution of antibodies in this disorder,

that knowledge could be extended to many other autoimmune disorders.

J O A N : I’d like to see more research into the causes of the disease. One question I’m

asked a lot is, what caused this? How did I get this? Why did I get this? If a patient

understands the causes, there is a possibility of prevention. We’ve talked a lot about

treating the disease. But this disease can be very difficult and life threatening.

Prevention of this disease is a very important goal.

D O U G : As physicians, we hope that the normal immune response is more robust than

auto-antibody production, which we are trying to control. But we are not doing any-

thing specific to identify and eliminate the T-cell, the B-cell or the antigen that

precipitates this disorder. There is work being done in this area, but...

D E N N I S : Is work being done in this area?

D O U G : Yes. But most hematologists are not trained in basic immunology and most

immunologists don’t see hematological problems. So the immune oriented grants

given by the NIH in this field tend to be relatively limited compared to grants for other

immunologic disorders.

G A R R E T T: We may find ITP consists of multiple diseases with multiple causes.

D O U G : That may well be, but wouldn’t it be wonderful to know if that is the case?

Not only to identify the panel of antibodies responsible, but also the genes that are

likely to be active in this disease process.

J O A N : Or which treatments worked well and which didn’t—so a patient could try

another one.

We certainly need clinical

care and outcomes research.

Equally important is basic

science inquiry into

the etiology and

pathophysiology of this

particular, very common

selective autoimmune

disorder.


G A R R E T T: How well do we understand the mechanism/action of the treatments that

do work?

D O U G : It’s relatively primitive despite work in the field. Rituxan is often used early in

the disease process. We have no idea how it should be used not only in ITP, but also in

any other immune disorder. We just use the same regimen that is used for lymphoma.

However, ITP is not lymphoma and we don’t know how to monitor the efficacy of

rituxan in ITP. There is nothing to follow but the endpoint of the platelet count.

The Patient’s Attitude

D E N N I S : How does a patient’s attitude/situation impact their therapeutic outcome?

J O A N : Attitude and life situation can have a huge impact. Some patients question their

physician’s recommendations and engage in a dialogue. Other patients decide to do their

own thing. And then many patients look beyond hematology to alternative and inte-

grated medicine. Some try herbs and change their diet and lifestyle. Additionally, there

has also been a lot of press and credence given to prayer and positive thinking. So many

patients are engaged in alternate modalities that could potentially have an impact.

D E N N I S : As a physician, how do you see the role of patient attitude and patient

awareness impacting their outcomes?

D O U G : When patients understand what we’re talking about, we all have an advantage.

But, understanding is not always easy to achieve. Patients differ intellectually and

emotionally. Also, psychological, familial and social circumstances can be either assets or

impediments. For example, a single mom taking care of several kids who loses her job

because she has to go to her doctor to be treated may differ greatly from somebody

who has a strong support system, is financially secure and can readily understand and

integrate treatment of this disorder.

The Power of Partnership

D E N N I S : Garrett, we’re hearing a lot about educating patients. What’s the role

of industry in that education?

G A R R E T T: There is a lot the industry can do for patients that have the disease

and are seeking help from their physician. At CSL Behring, we can help prepare patient

educational materials that reinforce the physician’s recommendations. We also partner

with Joan and her organization to find ways to reach out to the general public and

increase awareness of ITP. For example, if you or someone you know bruises easily and

has nosebleeds—seek an assessment. Maybe you don’t need help. Maybe you don't

have a disease. Maybe there is no intervention. But at least raise the question. That’s

one of the things that we can do as an industry.

J O A N : It’s a partnership. I think we’re all interested in making patients well, and to

that end, we all have a role to play.

At CSL Behring, we can

help prepare patient

educational materials that

reinforce the physician’s

recommendations. We

also partner with Joan and

her organization to find

ways to reach out to the

general public and

increase awareness of ITP.

About the Participants

and Development, at Octagen Corporation.

Dr. Bergman holds a doctorate in medicine

from Jefferson Medical College and a

master’s degree in business administration

from Temple University School of Business

and Management.

Joan Young

Joan Young is president and founder of

the Platelet Disorder Support Association

(PDSA). Founded in 1998, PDSA is a non-

profit organization providing information

and support services, while also encour-

aging research about ITP and other

platelet disorders.

The roots of PDSA’s founding trace back

to 1997 when Ms. Young began a Web

site that provided a place to share ITP

stories—including her own. PDSA was

created as a result. Today thousands of

people come to the organization. Besides

patient education, support services and

public education, PDSA encourages

research in the treatment of ITP and

other platelet disorders through surveys

and collaboration with the National

Institutes of Health, physicians and

researchers. The organization has a global

reach; 15 percent of the 24,000 people

on the mailing list live outside the U.S.

Douglas B. Cines, M.D.

Dr. Douglas B. Cines is a board-certified

hematologist, professor of pathology and

vice-chair for academics in the Department

of Pathology and Laboratory Medicine

at the Hospital of the University of

Pennsylvania (HUP). He is also director

of the Hematology Laboratory and the

Coagulation Laboratory at HUP. Dr. Cines

has published more than 139 peer-

reviewed articles, received numerous

grants and holds several patents. He is

also a member of several professional

and honorary societies. Dr. Cines holds

a doctorate in medicine from New York

University School of Medicine.

Garrett E. Bergman, M.D.

Dr. Garrett E. Bergman is senior director

of U.S. Medical Affairs at CSL Behring.

After a distinguished academic career in

medicine, Dr. Bergman has been working

in the biopharmaceutical industry, in

research and development and medical

affairs, since 1987. Dr. Bergman served

at Rhone Poulenc Rorer and at Armour,

which merged with Behringwerke AG to

become Centeon LLC—a predecessor

company of CSL Behring. Most recently,

Dr. Bergman was vice president, Research


Dennis Jackman

Dennis Jackman is senior vice president

of Public Affairs at CSL Behring. Mr.

Jackman is is responsible for optimizing

stakeholder impact on CSL Behring’s

ability to provide lifesaving therapies

worldwide. Previously he served as

Executive Director of the Plasma Protein

Therapeutics Association for North

America, the industry group for

advocacy, quality standards and

communications. He has held senior

public affairs positions in the pharma-

ceutical and biotechnology industries

since 1989. Previously, he was senior

legislative aide to U.S. Senator Arlen

Specter of Pennsylvania, focusing on

pharmaceutical, trade and tax policy.

About CSL Behring

CSL Behring is a global leader in plasma

protein biotherapeutics. Dedicated to

saving lives and improving the quality

of life for patients with rare diseases

worldwide, the company provides

safe and effective plasma-derived and

recombinant products and offers

patients a wide range of related services.

From left to right, Dr. Douglas Cines, Dr. Garrett Bergman, Joan Young and Dennis Jackman

Key Issues Dialogue: ITP - All About Bleeding · Biotherapies for Life™ Key Issues Dialogue: ITP...

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