Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease

Study point of contact

Courtney D Fitzhugh, M.D.
(301) 402-6496
[email protected]


1 United States site


2 to 80 Years


Phase 1/Phase 2

Study type









About the study


Peripheral blood stem cell transplantation procedures are used for people with sickle cell
disease. Researchers want to improve the success and reduce the complications for these
procedures. This might allow more people to have a transplant.


To see if a new transplant regime is effective, safe and well tolerated in people with sickle
cell disease.


Adults at least 18 years old with sickle cell disease and certain complications.

A relative who is a half tissue match.


Participants will be screened with medical history, physical exam, and blood tests.
Recipients will also have:

– Heart, lung, and mental health tests

– Chest x-rays

– Bone marrow taken from the pelvic bone

– Eyes and teeth checked

Recipients will have a large central line inserted into a vein for up to 6 months.

Donors will have their veins tested and have an IV inserted for 1 day or on rare occasions 2

Donors will get a drug to activate bone marrow. It will be injected for about 6 days.

Donors will have at least 1 five-hour procedure where bone marrow stem cells will be
collected. Blood will be taken from a vein in one arm or in rare cases from a groin vein and
put through a machine. Some blood will be saved and the rest will be returned. Stem cells
will be taken from the saved blood in a lab and frozen until ready to give to the recipient.

Recipients will have:

– Stems cells collected and frozen

– Hygiene lessons

– Bone density scans

– Low-dose radiation

– Drugs for their immune system

– Donor cells infused through their central line

– Transfusions

After about 30 days, recipients will leave the hospital. They must stay near NIH for 3 months
after the transplant and have frequent visits. After returning home, they will have 8 visits
over 5 years, then be contacted yearly.

participation requirements


Patients with any type of sickle cell disease who are at high risk for disease-related
morbidity or mortality, defined by having severe end-organ damage (A, B, or C) or
potentially modifiable complication(s) not ameliorated by hydroxyurea (D):

A. Stroke defined as a clinically significant neurologic event that is accompanied by an
infarct or hemorrhage on cerebral MRI or cerebral arteriopathy requiring chronic
transfusion therapy; OR

B. Tricuspid regurgitant jet velocity (TRV) of greater than or equal to 2.5 m/s at baseline
(without vaso-occlusive crisis) and/or pulmonary hypertension; OR

C. Sickle hepatopathy defined as either ferritin >1000 mcg/L and platelet count < 250,000/uL (without vaso-occlusive crisis) OR direct bilirubin > 0.4 mg/dL and platelet
count <250,000/uL (without vaso-occlusive crisis) D. Any one of the below complications: - Complication-Vaso-occlusive crises; Eligible for hydroxyurea*-At least 3 hospital admissions in the last year; Eligible for HSCT-Recurrent vaso-occlusive pain crises (at least 2 per year for the last 2 years) despite hydroxyurea - Complication-Acute chest syndrome; Eligible for hydroxyurea*-2 prior ACS; Eligible for HSCT-any ACS while on hydroxyurea. Non-disease specific: A. Age greater than or equal to 18 years B. Haploidentical relative donor available C. Ability to comprehend and willing to sign an informed consent D. Negative serum beta-HCG E. Ejection fraction greater than or equal to 35% F. Glomerular filtration rate >60 mL/min/1.73m^2 by cystatin C-based or othalamate-based or
other equivalent GFR testing

G. Adjusted DLCO greater than or equal to 35%

participation restrictions


Any of the following would exclude the subject from participating

1. Available 6/6 HLA-matched sibling donor

2. ECOG performance status of 3 or more

3. Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking
medication and progression of clinical symptoms) within one month prior to starting
the conditioning regimen.

4. Patients with fever or suspected minor infection should await resolution of symptoms
before starting the conditioning regimen.

5. Major anticipated illness or organ failure incompatible with survival from PBSC

6. Pregnant or breast-feeding

7. Donor specific anti-HLA antibodies (DSAs) greater than or equal to 2000 Mean
Fluorescence Intensity (MFI)

8. Patients seronegative for EBV who have EBV seropositive donors


  • Bethesda, Maryland, United States, National Institutes of Health Clinical Center, 20892 [Recruiting]