|Timothy Olson, MD, PhD|
|Barb McGlynn, RN, BSN|
1 United States site
2 to 25 Years
Hemoglobin SS, Hemoglobin SC
Severe Sickle Cell Disease
– Genotype: Hemoglobin SS, Hemoglobin SC, Hemoglobin SD, SOArab, or Hemoglobin SBeta
– Must have at least one of the following disease manifestations
– Clinically symptomatic neurologic event (stroke) or any neurologic deficit lasting
greater than 24 hours at any time prior to enrollment
– History of two or more episodes of vaso-occlusive events (VOE) per year in the 2 years
preceding enrolment. Patients must be refractory to hydroxyurea, defined as developing
VOE despite receiving hydroxyurea for at least 6 months. Patients who are intolerant
of hydroxyurea may also be enrolled.
Vaso-occlusive events include:
– Acute chest syndrome
– Pain episodes requiring intravenous pain management and/or hospitalization
– Splenic sequestration (defined as a 2 g/dL drop in hemoglobin in the setting of an
acutely enlarging spleen. This will be determined as part of clinical care and prior
to the research)
– Administration of regular red blood cell (RBC) transfusion therapy, defined as
receiving ≥ 8 RBC transfusions in the year preceding enrollment to prevent sickle
cell-related complications of any kind per treating hematologist’s judgment.
Beta Thalassemia Major
– Genotype: Confirmed Beta Thalassemia genotype by molecular genetic testing (May
include E/Beta0 and Beta0/Beta+ genotypes)
– Must meet clinical diagnosis of transfusion-dependent thalassemia, defined as need for
≥ 8 RBC transfusions per year in the two years preceding study enrollment.
– Patients who do not meet disease, organ or infectious criteria.
– Previous Hematopoietic stem cell transplant (HSCT)
– Patients with no suitable unrelated donor available. Patients with suitable fully
matched related donor are also not eligible.
– Pregnant females. All females of childbearing potential must have negative pregnancy
– Participation in a clinical trial in which the patient receives an investigational
drug must be discontinued prior to the time of initiation of transplant therapy.
Specifically transplant chemotherapy should not begin until at least 3 half-lives
after last use of the investigational drug.
– Severe RBC alloimmunization, defined as inability to receive packed RBC transfusion
therapy due to anti-RBC antibodies. Patients with high titer anti-donor human
leukocyte antigen (HLA) antibodies detected on screening may be enrolled if they are
willing to undergo HLA antibody desensitization therapy.