Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
University of Chicago
Age
2–25
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Hemoglobin SS, SC, S-β0 Thalassemia, or SO-Arab Sickle Cell Disease
Between the ages of 2 and 25 years (Stage 1: 10-25 years; Stage II: 2-25 years)
Lack a fully matched family donor or fully matched unrelated donor register in the National Marrow Donor Program
Partially-matched family member with hemoglobin AA (normal) or hemoglobin AS (sickle trait) phenotype
SCD with Severe Phenotype, defined by the following criteria: Neurologic manifestations of sickle disease including cerebral vascular accident (CVA), transient ischemic event (TIA) or abnormal MRI findings suggestive of silent infarct; Two or more episodes of acute chest syndrome (ACS) requiring admission for transfusional or respiratory support including supplemental oxygen within \[two years\] of enrollment in study despite hydroxyurea therapy. Patients who cannot tolerate hydroxyurea and who experience multiple episodes of ACS will also be eligible; History of severe vaso-occlusive (VOC) disease requiring hospitalization and intravenous narcotics on 3 or more occasions per year over the two years prior to enrollment despite hydroxyurea therapy. Patients who cannot tolerate hydroxyurea and who experience multiple episodes of VOC will also be eligible; Other severe phenotype as evidenced by end organ dysfunction related to sickle cell disease.
You may not be if
Karnofsky or Lansky score \< 60%
Acute hepatitis or evidence of moderate or severe portal fibrosis on biopsy. (Biopsy will be obtained if patient has been on chronic transfusion therapy \> 6 months or has a ferritin \> 1000 ng/ml) or AST or ALT \>5 times the upper limit of normal
Severe renal impairment (as evidenced by creatinine clearance of \<50ml/minute glomerular filtration rate (GFR) \< 50% predicted normal)
Cardiac function that demonstrates shortening fraction less than 26% by cardiac echocardiogram or pulmonary hypertension.
Pregnant Female.
Lactating female.
Pulmonary function with baseline O2 saturation \<85% or Diffusing Capacity for Carbon Monoxide (DLCO) on pulmonary function testing (PFT) with a DLCO \<40%.
Clareo Health | Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease
