| Shelby Gruntorad, MS | |
| 7737026554 | |
| [email protected] |
1 United States site
2 to 25 Years
SS, SC
N/A
Interventional
All
Device
Unknown
The purpose of this study is to develop a safe and curative stem cell transplant approach to
treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem
cell transplantation using αβ+ T-cell depletion for children and adolescents with severe
sickle cell disease (SCD).
– 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.
– 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%.