The purpose of this research study is to test the safety and efficacy of a drug called Plerixafor. Plerixafor is approved by the US FDA for use in increasing blood stem cell counts before collection in cancer patients. It is not yet approved for patients with sickle cell disease. The investigators want to find out if Plerixafor can be used to increase cell counts in patients with sickle cell disease.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Weill Cornell Medical College
New York, New York, United States
safety
Safety is assessed using a dose limiting toxicity (DLT) endpoint. Definition of a DLT is the occurrence of any of the below events that meets the following criteria: The occurrence of a vasoocclusive crisis requiring hospitalization, acute chest syndrome, CNS acute event, or any other disease related ischemic-based adverse event (AE) should be considered as a DLT, if occurring in the 48 hours DLT observation period.
Time frame: up to 30 days
efficacy
Efficacy is defined as 100% of evaluable patients reaching a PB CD34 concentration ≥ 30/uL.
Time frame: ≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor.
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