Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

About the study

Background:

Sickle cell disease can often be treated with blood stem cell transplants. But for some
people the disease returns. This study will give a second transplant to people whose disease
has returned but still have some donor cells in their body.

Objective:

To cure people s sickle cell disease by giving a second treatment that makes more room in
their bone marrow for donor cells.

Eligibility:

People ages 4 and older with sickle cell disease who had a transplant but the disease
returned, and their donor relatives

Design:

Participants will be screened with medical history, physical exam, and blood tests.

Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow
sample, and teeth and eye exams. They must have a caregiver.

Donors will have 7-8 visits. They will take a drug for 5-6 days to prepare them for the
donation. For the donation, blood is taken from a vein in the arm or groin. The stem cells
are collected. The rest of the blood is returned. This may be repeated.

Recipients will get a long IV line in their arm or chest for about 1-2 months. They will take
drugs to help their body accept the donor cells. They will get the donor cells and red blood
cell transfusions through the line. They will stay in the hospital about 30 days after the
transfusion of donor cells.

In first 3 months after the infusion, recipients will have many visits. Then they will have
visits every 6 months to 1 year for 5 years. During those visits they will repeat some of the
screening tests….

Study point of contact

Julia M Varga
(301) 402-3595
julia.varga@nih.gov

Locations

1 United States site

Age

4 to 80 Years

Phase

Phase 1/Phase 2

Study type

Interventional

Gender

All

Interventions

Device

Compensation

Unknown

participation requirements

Inclusion criteria- recipient

1. Patient with history of SCD who underwent allogeneic hematopoietic stem cell
transplantation (HSCT)

2. Patient with recurrent SCD defined as HbS greater than or equal to 50% for donors with
sickle cell trait and greater than or equal to 10% for donors with HbAA with recurrent
clinical manifestations (for example but not limited to recurrent painful crises,
acute chest syndrome, priapism, or severe anemia)

3. Persistent donor chimerism levels

4. Age greater than or equal to 4 years

5. Negative beta-HCG

6. Ejection fraction greater than or equal to 35%

7. DLCO greater than or equal to 35%

Inclusion- donor

1. Original donor from the patient s prior transplant

2. Age greater than or equal to 18 years

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

4. Ability to comprehend and willing to sign an informed consent

participation restrictions

Exclusion criteria- recipient

1. ECOG performance status of 3 or more (see Appendix B)

2. Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking
medication and progression of clinical symptoms) within one month prior to signing the
consent

3. Patients with fever or suspected minor infection should await resolution of symptoms
before signing the consent

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

5. Pregnant or breastfeeding

6. Baseline alanine aminotransferase or direct bilirubin >3x upper limit of normal

7. History of liver cirrhosis

8. History of secondary malignancies (other than localized skin cancer)

Exclusion- donor

1. Pregnant or breastfeeding

2. HIV positive

3. Hemoglobin S >50%

4. History of congestive heart failure, unstable angina, or stroke

Locations

  • Bethesda, Maryland, United States, National Institutes of Health Clinical Center, 20892 [Recruiting]
Last updated 2021-05-12