T he Ok lahoma ITP Reg is t ry N ew s let t er
July 2009
1
Hello!
1
The ITP Registry
1
Dr. G’s Perspective
3
Dee’s Dissertation
4
Patient Perspectives
about Steroids
7
Send Your Suggestions
7
Resources for ITP
Patients
7
Contact Information
Hello!
We would like to welcome you to our
fifth
Oklahoma
ITP
Registry
newsletter.
The ITP Registry
The Oklahoma ITP Registry began in
November 2001. Since that time we
have enrolled 92 people. With your
participation we hope to document the
clinical
course
and
long-term
outcomes of patients with ITP
Dr. G’s Perspective
Why are bone marrow
procedures
sometimes
done on ITP patients?
ITP is a disorder in which
platelets are destroyed too
quickly
although
platelet
production is normal, or nearly
normal. When I was training to be a
hematologist 40 years ago, it was
standard to examine the bone marrow
on all patients with suspected ITP to
be sure the low platelet count was not
associated with a bone marrow
disease
that
decreases
platelet
production (such as leukemia). The
bone marrow is where all blood cells
are made and examination of the bone
ITP Registry Newsletter
page 2
marrow can provide confidence that blood cell production is normal.
Although obtaining a marrow sample from the hip bone is painful, it is
a safe. In the past 15 years it has become clear that in most patients
with suspected ITP, careful examination of the blood can provide
confidence that platelet production is normal and the bone marrow
does not have to be examined. However in some patients
examination of the blood is not so clear. If some abnormalities of the
white blood cells and red blood cells are present; then direct
examination of the bone marrow is essential. Examination of the bone
marrow is also appropriate when there is no response to treatments
and in older patients, such as patients over 60 years old, when
disorders of platelet production are more common.
Are there any adverse side effects of the new ITP drug,
romiplostim, that was discussed in the last newsletter?
Romiplostim (brand name, Nplate) and eltrombopag (brand name,
Promacta) are very effective new agents that can increase platelet
production and platelet counts in most patients with ITP with very few
side effects. In our experience with 10 patients over the past 7
years, we have not observed any important side effects from
romiplostim.
However some patients have had problems that
required the treatment to be stopped. In a very few patients the
increase of platelet production in the bone marrow has been
associated with an increase of the tissue fibers that form the structure
of the marrow (called ‘reticulin’), which is evident from a bone
marrow biopsy. The problem has improved when romiplostim was
stopped. Increase of reticulin fibers has also occurred in a few
patients treated with eltrombopag. To document whether this may be
more common than we now suspect, there are current studies doing
repeated bone marrow tests on patients receiving romiplostim.
Because platelet counts can go very high as the dose of romiplostim
is being adjusted, there has been concern for risk of blood clots.
However this has not been observed. Finally, when patients stop
romiplostim, the platelet count may fall to even lower levels than
were present before treatment. However this is temporary and
platelet counts recover to their pre-treatment level within a few days.
ITP Registry Newsletter
page 3
Because these new drugs are so powerful and so effective, there has
been careful attention to the potential risks. But so far, they are
impressively safe treatments for ITP.
Is romiplostim safe for everyone?
The answer is “yes”, with a few exceptions. Romiplostim has not
been given to pregnant women and there is no information about
potential risks for the fetus; therefore romiplostim should not be used
in a woman who is pregnant or breastfeeding. Also the experience
with romiplostim is very limited in infants. However, even though
romiplostim is safe for almost everyone, it may not be appropriate
treatment for every patient with ITP. It is most important for patients
who have failed traditional treatments, such as a short course of
steroids, splenectomy, and rituximab. These treatments are important
to try first because they can stop the ITP, correct the platelet count,
and the patient may need no further treatment. The effect of
romiplostim is different. It does not change the ITP; it only increases
the platelet counts while the ITP persists. Therefore romiplostim and
eltrombopag must be taken forever. This is a big commitment and a
very big expense.
Dee’s Dissertation
Dee has recently finished her dissertation to receive her Ph.D. in
epidemiology (the study of disease). To graduate, she had to
complete an extensive research project, called a dissertation. Dee
chose ITP as the topic for her study.
Introduction: Idiopathic (also known as immune and autoimmune)
thrombocytopenic purpura (ITP) is an autoimmune blood disorder
defined by a low platelet count. That means a person with ITP is at
an increased risk for bleeding even with minimal trauma. Routine
things can no longer be taken for granted. Parents of toddlers with
ITP are told to watch their child carefully and make sure they avoid
head injuries. Children and teenagers diagnosed with ITP are often
told they can only play non-contact sports. An adult patient with ITP
ITP Registry Newsletter
page 4
might have to receive treatment to temporarily raise their platelet
count before a dental cleaning or a dermatologist visit.
A question patients and parents of patients with ITP often wonder is,
how many other people have ITP? The number of people living with a
disease is called the prevalence of the disease. There have only been
two previous studies completed in the US looking at the prevalence of
ITP. However results from these studies may or may not apply to the
general population. Both of these studies only included persons who
were privately insured.
The goal of the present study was to
determine the prevalence of ITP (regardless of age or insurance
status) in the state of Oklahoma.
Methods: De-identified data (no names or information that could
link back to an individual person) was obtained from hematologists in
the state of Oklahoma. In November 2007, there were 93
hematologists in Oklahoma who cared for ITP patients. Our
preliminary data documented that hematologists saw > 92% of all
patients with ITP and that primary care physicians in Oklahoma
reported they were ‘likely’ to refer 85% of patients with platelet
counts <30,000/μL and bleeding symptoms to a hematologist. All
hematologists were asked to provide information on all (new and
existing) ITP patients seen from 1/03-12/04. Ninety-five percent
(88/93) of the hematologists in the state were able to provide these
data. Prevalence estimates were calculated separately for children
(age <16 years) and adults (age > 16 years).
Results: The average annual prevalence estimate for children was
8.1 per 100,000 children-so for every 100,000 children in Oklahoma
there are 8.1 children who have ITP; the estimate for adults was 12.1
per 100,000 adults-so for every 100,000 adults in Oklahoma there
are 12.1 with ITP. Children were 51% female and the average age
was 6 years; adults were 57% female and the average age was 55
years.
ITP Registry Newsletter
page 5
Conclusion: This is the first population based study to determine the
prevalence of ITP for an entire defined geographic region, regardless
of insurance status and age. These are the most generalizable
estimates of prevalence currently available on which to determine the
burden of ITP. This information is important not only for patients with
ITP, but also pharmaceutical companies, regulatory agencies, and
heath care authorities.
Comparison of Patient and Physician Perspectives Results!
The results are in from the study that our student Jacqueline
designed. Thank you to everyone who completed the survey!
As you may know, steroids are standard initial treatment for patients
with idiopathic thrombocytopenic purpura (ITP), but side effects may
become severe. We hypothesized that patients with ITP may be more
concerned about the side effects of steroid treatment and less
concerned about the risk for serious bleeding than their
hematologists. We surveyed 80 patients in the Oklahoma ITP Registry
and all the hematologists in the State of Oklahoma with questions
about the occurrence and severity of 18 steroid side effects and risks
for serious bleeding.
Response rates were 80% (patient) and 71% (hematologists). The
responses of patients and hematologists were significantly different
(indicated by stars on the graph on the next page) regarding the
frequency of severe steroid side effects The survey specifically asked
patients and hematologists about 38 different side effects related to
steroid treatment, grouped into the 18 categories The numbers in the
graph correspond to the numbers of each corticosteroid side effect
listed next to the graph.
The difference between the patients’ responses and the physicians’
responses was statistically significant for 14 out of 18 categories. Of
those 14 significant differences, 13 reported the patients experienced
a lot more bother from that category of side effects than their
hematologist recognized.
ITP Registry Newsletter
page 6
80
70
*
Patient
Hematologist
Percent
60
50
*
40
30
20
**
10
0
1 2 3 4 5
* *
*
* * *
*
*
*
*
* *
*
*
6 7 *8 9 10 11 12 13 14 15 16 17 18
*
Side Effects
1. Moon face, bloating, swelling
2. Weight gain/increased appetite
3. Hair loss
4. Acne
5. Stretch marks
6. Insomnia, restlessness, and/or
trouble sleeping
7. Anxiety and/or nervousness
8. Depression and/or stress
9. Anger and/or irritability
10. Generalized weakness, fatigue
11. Muscle weakness 12. Body pain
(joint stiffness, muscle cramps,
osteoporosis)
13. Hot flushes and/or sweating
14. Visual problems (light sensitivity/
decreased visual acuity)
15. Nausea, upset stomach, vomiting,
diarrhea
16. Dizziness, headaches
17. Trouble with blood glucose levels,
diabetes
18. High blood pressure
In addition, significantly fewer patients (31%) with platelet counts
<10,000/µL reported they had been very worried about serious
bleeding, compared to 69% and 93% of hematologists, responding to
two comparable case scenarios.
Physicians need patients to communicate with them so that they
know how much a side effect is bothering their patients. Just as
physicians must educate patients about their illness, patients have a
responsibility to educate their physicians about the problems they are
experiencing. We hope this study will be influential in the ways that
ITP patients and physicians communicate and the way that steroids
are prescribed for ITP patients in the future.
For the full results of this study, use the citation below or contact our
office.
Corticosteroid side-effects and risk for bleeding in immune thrombocytopenic
purpura: patient and hematologist perspectives.
Guidry JA, George JN, Vesely SK, Kennison SM, Terrell DR.
Eur J Haematol. 2009.
ITP Registry Newsletter
page 7
Send Your Suggestions
Is there anything you’d like to see in the next newsletter? We’d like to hear
from you! Please contact us if you have any suggestions as to what you would
like to see in this newsletter in the future either by emailing Dee Terrell at deeterrell@ouhsc.edu or calling at (405)271-8001 extension 48386.
Resources for ITP Patients
Visit our website, Platelets on the Internet, at http://www.ouhsc.edu/platelets.
There is also an informative website from the United Kingdom you can visit at
www.itpsupport.org.uk. This site includes a support group with newsletters,
publications, and information on ITP. Dr. George contributes “An American
Perspective” found on this page, where you can find additional topics about ITP.
www.itpsupport.org.uk/american.htm
Contact Information
Phone:
(405) 271-4222
Mailing address:
James George, MD
Attention: ITP Registry
OU Health Sciences Center
Hematology-Oncology Section
P.O. Box 26901 CHB #333
Oklahoma City, OK 73126
Website:
http://www.ouhsc.edu/platelets
The Oklahoma ITP Registry
James George, MD
OU Health Sciences Center
Hematology-Oncology Section
P.O. Box 26901
Oklahoma City, OK 73126