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Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease [BMT CTN 1507]
Protocol Number:
Phase II
Robert Brodsky
Johns Hopkins Kimmel Cancer Center in Baltimore
The purpose of this study is to find out how well and how safe haploidentical bone marrow transplantation (BMT) is in patients with sickle cell disease (SCD). There are two strata: (1) children with SCD with strokes and (2) adults with severe SCD. We will be looking at how well BMT works over time.
Age 5.00 – 14.99: Must have SCD (Hb SS or Sß° Thalassemia) who have stroke ischemia based on neuroimaging, clinical evidence of neurological injury lasting for 24 hours, or both. Age 15.00 – 45.99: Must have SCD (Hb SS or Sß° Thalassemia) with one of the following:a)Clinically significant stroke or neurologic deficit lasting over 24 hoursb)History of 2+ episodes of acute chest syndrome in the past 2 years despite supportive care measuresc)History of 3+ severe vaso-occlusive pain crisis per year in the past 2 years despite supportive care measures d)Administration of regular RBC transfusion therapy e)Significant echocardiogram finding of tricuspid valve regurgitant jet velocity All patients should be in good physical health, take contraceptive measures, and have a first-degree HLA-haploidentical donor who is willing and able to donate bone marrow. Patients should not have had a previous hematopoietic cell transplant.
After screening, patients start a 28-day pain diary leading up to a 70-day BMT preparation regimen. On day 0, patients undergo bone marrow transplant. Post-transplant, patients receive immunosuppressants and GVHD prophylaxis.
Last Update
08/07/2020 05:02 AM