Regenerative Medicine to Cure Sickle Cell Anemia. Robert A. Brodsky, MD Johns Hopkins Family Professor of Medicine and Oncology Director: Division of Adult Hematology. Glossary of terms. BMT B one m arrow t ransplantation B lood or m arrow t ransplantation Stem cell transplantation
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Regenerative Medicine to Cure Sickle Cell Anemia
Robert A. Brodsky, MD
Johns Hopkins Family Professor of Medicine and Oncology
Director: Division of Adult Hematology
Number of Transplants
SAFETY and Donor availability
Non-myeloablative or “mini” BMT
Matched sibs available <30% pts
The Holy Grail of BMT?
Szydloet al JCO 1997
Emadi, Jones and Brodsky. Nat Rev ClinOncol 2009
SCD kills an estimated half-million people worldwide annually.
"When one considers the additional contributions of sickle cell disease associated with
reduced quality of life, uncompensated care, lost productivity, and premature mortality,
the full burden of sickle cell disease is likely to be quite higher."
Kauf et al, Am J Hematol. 2009
Reduced intensity haploidentical BMT
with post-transplant Cyclophosphamide (CY)
ATG Day -9 to -7
Bolanos-Meade et al, Blood 2012
* Hgb reflects transfusion of RBCs within last 90 days