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Although this section is open to the public, the information
within is intended for medical professionals and highlights
some of the latest clinical and research activities of
our staff. If you have any questions about your particular
situation, you should speak to a physician in your area.
For information geared towards patients, visit our section
on Understanding
Your Illness.
- “Long-term FEIBA prophylaxis does not prevent progression
of existing joint disease” M.W. Hilgartner, A. Kakipernaa
and D.M. DiMichele, Haemophilia (2003), 9, 261-268
- “Transjugular liver biopsy is safe and diagnostic
for patients with congenital bleeding disorders and hepatitis
C infection,” D.M. DiMichele, G. Mirani, P. Wilfredo
Canchis, D.W. Strosts and A.H. Talal, Haemophilia (2003),
9, 613-618.
- Immune Tolerance in Hemophilia A/High Titer Inhibitors:
(Donna DiMichele, M.D.) For the past three years as a co-principal
investigator for this project, Dr. DiMichele has been developing
the first international multicenter prospective randomized
study of immune tolerance therapy in hemophilia A patients
who develop high titer inhibitors (antibodies). This trial
began in the summer of 2002. Although this dosing trail
is clinical in scope, it will provide the opportunity to
study the role of genotype immunophenotype interaction
in inhibitor formation and disappearance through several
collaborative sub-studies.
- Laboratory Predictors of Thrombosis and End-Organ Failure
in Pediatric Patients Undergoing Stem Cell Transplant:
(Donna DiMichele, MD). In this American Cancer Society-sponsored
study, currently in the stage of final data collection
and analysis, clinical laboratory parameters of hypercoagulability
were measured longitudinally, pretransplant through engrafment.
Through the correlation of several prethrombotic laboratory
parameters and clinical even endpoints, the study will
attempt to identify one or more clinical laboratory predictors
of thrombosis and end-organ failure. Data from this study
will form the basis for a prophylactic intervention study
of anticoagulation.
- The Use of a Computational Model as a Predictor of Bleeding
in Severe Hemophilia A: (Leonardo Brandao, MD). The purpose
of this study is to investigate whether the use of this
new computerized method of blood clotting analysis can
predict/explain the bleeding tendencies in children with
severe hemophilia A. Additionally, this model will be tested
to see if it can also accurately predict bleeding tendencies
in children with severe hemophilia A who develop an inhibitor,
a substance or antibody made by the body against FVIII.
- The FEIBA (NovoSeven, Comparative Study, The FENOC Study):
(Donna DiMichele, MD). Our HTC is the coordinating US/Canadian
center for this study. The purpose of this study is to
compare the effectiveness of FEIBA and NovoSeven in the
treatment of joint bleeds in hemophilia A patients with
inhibitors.
- Hemostasis Fellowship Program: A fourth year pediatric
hematology/oncology fellowship with a clinical/clinical
research focus on hemophilia and general hemostasis and
thrombosis has been established at the Weill Cornell institution
and is overseen by Dr. DiMichele. Our first candidate’s
fellowship is under way for the 2002-2003 academic year.
This fellow will be offered an intensive combined clinical,
laboratory and research experience with the goal of developing
the following expertise: 1) diagnosis/management of hemophilia,
vWd, other congenital/acquired bleeding disorders as well
as congenital/acquired thrombophilia in the pediatric adult
patient: and 2) technical/interpretive aspects of the clinical
coagulation lab. His clinical research will be co-mentored
by the Children’s Clinical Research Center and accompanied
by coursework in epidemiology, statistics and research
design.
- Our Hemophilia Treatment Center is a designated International
Hemophilia Training Center through the World Federation
of Hemophilia. Annual fellowships are granted to physicians
in other countries who have an interest in learning more
about comprehensive care for patients with bleeding disorders.
During a course of training from 4-8 weeks candidates are
given the opportunity to participate in comprehensive clinics
at several of the HTC’s in our region, even traveling
as far as Puerto Rico, to get a better understanding of
the services available at a comprehensive care center and
the diverse population involved. Two trainees from Brazil
spent two months with us in 2002. In 2003, we had two trainees
from the Philippines for the same period of time.
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