Case Study 2:
Patient considering discontinuing Desferol therapy
Other studies
1.
2.
3.
4.
5.
6.
Sickle cell patient complains about shortness of breath
Patient considering discontinuing Desferol therapy
Pain Manangement
Sß+ Thalassemia patient with acute infarct
Patient with proteinuria
Pregnancy and sickle cell care
Results of Adult Sickle Cell Case Study No.2
19 year old man with sickle cell anemia, a history of an overt stroke at age 10, an abnormal MRI/MRA with areas of
silent stroke, who on transfusion therapy is transitioning into your practice. He otherwise is doing well, with no
hospitalizations since age 15, and has decided to discontinue his desferol chelation therapy now that he is an adult
and making his own decisions. His ferritin is 3572 ng/ml and he confesses he hasn't really chelated for 9 months
anyway. He adamantly refuses to resume chelation.
With regard to his chronic stroke prophylaxis would you (select more than one if it applies):
Response
Percent
Continue transfusion therapy despite lack of chelation
0%
therapy?
Change to a chronic exchange transfusion program?
56%
Discontinue transfusions unless he agrees to use
13%
chelation therapy?
Discontinue transfusions since they aren't necessary
0%
anymore?
Discontinue transfusions and switch to hydroxyurea therapy?
If so, would you perform phlebotomies to remove iron?
Yes
75%
Try to obtain an oral chelation agent (compassionate
13%
release or on a study)?
Other Comments:







Add HU then D/C transfusions w/ phlebotomies as tolerated
Use Vitamin C and green tea to help w/ iron overload
Check bone marrow for iron store
Check for other signs of iron overload i.e.; liver abnormalities
Currently it is unclear the risk of thrombosis after a certain period of transfusion. While transitioning pt. over
to HU continue w/ transfusions.
Start HU first + D/C transfusions after a couple of months on HU
Once FE overload occurs, FE removal is necessary to halt the oxidant mediated end-organ damage. FE
removal remains key whether transfusions are continued or not.
ADULT CARE PROVIDERS: Case # 2 Response
1. Discussion of recent case
I presented this case because over the years I have heard some providers comment that they would rather have a
patient risk the possibility of recurrent stroke than to develop iron overload, with the certainty of morbidity and
mortality that goes along with it. The risk of recurrent stroke in adults with a history of childhood stroke has never
been clearly defined. Also, it is not an unusual situation, as older teens and young adults sometimes express their
new freedom to make their own health care decisions to electing to stop chelation, transfusion, or both.
Interestingly, all who responded to this case would stop regular simple transfusion. A little more than half would
switch to exchange transfusion, which has been demonstrated to avoid iron accumulation (1), though it was
pointed out that the iron already accumulated needs to be removed to avoid end-organ damage. A majority of
people would also consider switching to hydroxyurea therapy, presumptively in an effort to preclude recurrent
stroke (or at least affect the course of the disease). It is unknown if hydroxyurea is as effective as transfusion in
preventing recurrent stroke. There have been small case series published in which hyroxyurea was used in children
with a history of stroke (2,3). Recurrent stroke rates varied from 0-19%, though most recurrence seemed to occur
during the first 3-4 months of therapy, when perhaps maximum benefit from hydroxyrea was not yet achieved.
More recent data were presented at the national sickle cell meeting. An abstract presented there (4) reported a 6.7
per 100 pt-yr recurrent stroke rate in 32 patients using hydroxyurea therapy in place of transfusion therapy over
approximately 40 months. Phlebotomy was successfully used to remove excess iron. This approach seems
promising, but needs further study. These data, as is almost always the case, are in children. A randomized trial
will be needed to decide if this represents a good alternative to transfusion therapy. In adult care, it has never been
established how long prophylactic transfusions and/or other therapies need to be continued for a person with a
history of a childhood stroke. Hopefully, future trials would take this question into account.




Hilliard LM, Williams BF, Lounsbury AE, Howard TH. Erythrocytapheresis limits iron accumulation in chronically
transfused sickle cell patients. Am J Hematol 1998 Sep;59(1):28-35.
Sumoza A, de Bisotti R, Sumoza D, Fairbanks V. Hydroxyurea (HU) for prevention of recurrent stroke in sickle
cell anemia (SCA). Am J Jematol 2002;71:161-165.
Ware RE, Zimmerman SA, Schultz WH. Hydroxyurea as an alternative to blood transfusions for the prevention
of recurrent stroke in children with sickle cell disease. Blood 1999 Nov;94(9):3022-3026.
Ware RE, Zimmerman SA, Sylvestre PB, Davis JS, MacKeigan SF, Treem WR, Schultz WH. Prevention of
secondary stroke and resolution of transfusional iron overload using hydroxyurea and phlebotomy in children
with sickle cell disease. Medicine, Science and Community: 30th Anniversary of the National Sickle Cell
Disease Program. September,2002, Washington DC.
2. In follow-up of case 1, we also asked for information about how many patients you follow with pulmonary
hypertension, and what therapies are their receiving. Only 5 providers responded, but this represents a modest
number of patients:
Responses
Number
Total # of pts. in overall population
700
Total # of pts. with recognized pulmonary hypertension
74
Total # of pts. receiving therapy
25
Transfusion
18
HU (for purposes of treating pulmonary hypertension)
5
HU (for purposes of treating underlying disease)
10
Oral calcium channel blockers
1
Iloprost
2
Bosentan
0
Other
0
I would be interested in hearing from anyone who has ever tried Bosenten in a sickle cell disease patient. We are
trying to get a research protocol going (for which we may need patients), and I'd like to hear if anyone has tried it
yet.
3. One of the care providers wanted to know what sorts of flow sheets are being used for sickle cell care. If you
have a good system in your practice, perhaps you'd be willing to share it. The fax number for that provider is: 614
293-7525
[This is the answer to the question.]
Back to Top
For additional information please contact: Colorado Sickle Cell Center
If you are experiencing problems using this website contact Webmaster