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Protocol Details

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

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Participants currently recruited/enrolled
Gender: Male & Female
Min Age: 2
Max Age: 80

Referral Letter Required


Population Exclusion(s)

Pregnant Women and Fetuses

Special Instructions

Currently Not Provided


Peripheral Blood Stem Cells;
Host-Donor Chimerism;
Graft Versus Host Disease;
Donor Apheresis

Recruitment Keyword(s)



Sickle Cell Disease

Investigational Drug(s)


Investigational Device(s)



Procedure/Surgery: haploidentical stem cell transplant
Drug: sirolimus
Drug: campath
Drug: pentostatin
Drug: cyclophosphamide

Supporting Site

National Heart, Lung, and Blood Institute


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 days.

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


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.

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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 a maximal tolerated dose of hydroxyurea

--Complication-Acute chest syndrome; Eligible for hydroxyurea*-2 prior ACS; Eligible for HSCT-any ACS while on hydroxyurea.

*hydroxyurea at maximum tolerated dose for at least 6 months

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%


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 transplant

6. Pregnant or lactating

7. Anti-donor HLA antibodies

8. Patients seronegative for EBV who have EBV seropositive donors


1. Haploidentical relative donor

2. Weight > 20 kg (insofar that the weight difference between recipient and donor does not exceed a reasonable likelihood of being able to obtain an adequate cell dose from the donor within two aphereses)

3. Fit to receive filgrastim (G-CSF) and to give peripheral blood stem cells (blood counts and blood pressure within DTM standards)

4. No history of congestive heart failure or unstable angina, and no history of stroke

5. Ability to comprehend and willing to sign an informed consent; assent obtained from minors


Any of the following would exclude the donor from participating

1. Pregnant or breastfeedingl

2. HIV positive

3. Hemoglobin S > 50%

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Not Provided

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Principal Investigator

Referral Contact

For more information:

Courtney D. Fitzhugh, M.D.
National Heart, Lung and Blood Institute (NHLBI)

Courtney D. Fitzhugh, M.D.
National Heart, Lung and Blood Institute (NHLBI)
BG 10 RM 6N240A
(301) 402-6496

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: 1-866-411-1010

Clinical Trials Number:


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