In this protocol, the investigators propose to evaluate the biochemical effects of imatinib
on sickle red blood cells (RBCs). Patients will be administered imatinib mesylate orally
following the guidelines previously established for use of imatinib in other disorders. The
biochemical effects of imatinib on sickle RBCs will be examined, including changes in their
levels of band 3 tyrosine phosphorylation and the abundances of RBC-derived microparticles in
their blood. In addition, the patients will be monitored for symptoms of sickle cell disease
(SCD). The investigators expect band 3 tyrosine phosphorylation to decrease dramatically in
patients treated with imatinib. The investigators also anticipate a reduction in the numbers
of RBC-derived microparticles in circulation (quantitated by assaying the number of
glycophorin A positive microparticles in peripheral blood samples by flow cytometry. Most
importantly, the investigators expect to see a reduction in the frequency of vaso-occlusive
crises, and possibly acute chest syndrome and utilization of opioids. The study duration is
planned as 6 months in order to provide adequate time for potential change in the primary
endpoints (e.g. percent irreversibly sickled cells).
Phase:
Early Phase 1
Details
Lead Sponsor:
Indiana University
Collaborators:
Children's Hospital Medical Center, Cincinnati Purdue University