Eligibility

Eligible Ages

Between 16 Years and 60 Years

Eligible Sex

All

Accepts Healthy Volunteers

No

Criteria

Patient Eligibility:

1. Patients with sickle cell disease are eligible if they have any of the following
complications:

1.1 Stroke or central nervous system event lasting longer than 24 hours 1.2 Frequent
vaso-occlusive pain episodes, defined as ≥ 3 per year requiring emergency room,
acute care center, hospital admissions, or home bedrest leading to absence from work
or school. 1.3 Recurrent episodes of priapism, defined as ≥ 2 per year requiring
emergency room visits 1.4 Acute chest syndrome with recurrent hospitalizations,
defined as ≥ 2 lifetime events 1.5 Red-cell alloimmunization (≥ 2 antibodies) during
long-term transfusion therapy 1.6 Bilateral proliferative retinopathy with major
visual impairment in at least one eye 1.7 Osteonecrosis of 2 or more joints 1.8
Sickle cell nephropathy, defined by a GFR < 90mL/min/1.73m2 or the presence of
macroalbuminuria (urine albumin > 300 mg/g creatinine) 1.9 Pulmonary hypertension,
defined by a mean pulmonary arterypressure >25mmHg

2. Age 16-60 years

3. Karnofsky performance status of 60 or higher (Appendix A)

4. Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%

5. Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥
50% predicted (after adjustment for hemoglobin concentration)

6. Estimated GFR ≥ 50mL/min/1.73m2 as calculated by the modified MDRD equation

7. ALT ≤ 3x upper limit of normal

8. HIV-negative

9. Patient is not pregnant

10. Patient is able and willing to sign informed consent

11. Patient does not have a fully HLA-matched sibling donor

12. Patient has an HLA-haploidentical relative

Donor Eligibility Relatives (parents, offspring, siblings, aunts/uncles, cousins) will be
tested by molecular typing of HLA class I (A, B, and C) and class II (DRB1) at low
resolution. Only those that are an HLA-haploidentical match (≥ 4/8) will be considered as
a potential donor. NOTE: If during testing, a fully HLA-matched sibling donor is found
and is willing to donate his/her stem cells, the potential subject will not be eligible
for this protocol.

Donor consent will be obtained as per standard protocol of the bone marrow transplant
unit.