Protocol Number: 01-H-0083

Title:

A Pilot Study of G-CSF Induced Stem Cell Mobilization Potential in Patients with Relapsed Severe Aplastic Anemia

Number:

01-H-0083

Summary:

This study will examine 1) whether it is possible to collect enough stem cells (cells produced by the bone marrow that mature into white and red blood cells and platelets) from patients with aplastic anemia to use for future treatment, and 2) whether patients who have been treated successfully and relapse will benefit from autologous stem cell transfusion (transfusion of their own stem cells).

Patients 12 years of age or older with aplastic anemia who have been successfully treated with immunosuppressive drugs and are now in remission may be eligible for this study. Participants will undergo a complete history and physical examination, bone marrow biopsy (removal of a small sample of bone marrow from the hip bone) and blood tests, plus procedures to collect stem cells, as follows:

-G-CSF (Filgrastim) administration - G-CSF will be given by injection under the skin daily for up to 10 days. This drug causes stem cells to move from the marrow into the blood where they can be collected more easily.

- Apheresis - Stem cells will be collected through apheresis, usually starting the 5th to 6th day of Filgrastin injections. For this procedure, whole blood is collected through a needle in an arm vein. The blood circulates through a cell separator machine where the white cells and stem cells are removed. The red cells, platelets and plasma are returned to the body through a second needle in the other arm. The procedure takes about 5 hours. Up to five procedures, done on consecutive days, may be required to collect enough cells for transplantation. If enough cells are collected, they will be purified (treated to remove the white blood cells) using an experimental device. Removing the lymphocytes may reduce the chance of relapse of aplastic anemia following the stem cell transplant. The stem cells will be frozen for later use, if needed.

- Follow-up - Participants are followed at NIH at 6-month intervals.

Sponsoring Institute:

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Active Accrual Of New Subjects

Gender: Male & Female

Referral Letter Required: Yes

Population Exclusion(s): None

Eligibility Criteria:

INCLUSION CRITERIA

History of severe AA as defined by a hypocellular bone marrow and depression of two out of three peripheral counts as indicated below:

ANC less than 0.5 x 10 (9)/L;

platelet count less than 20 x10 (9)/L,

reticulocyte count less than 60 x 10 (9)/L.

Demonstrated hematologic response to first or second course of immunosuppression as defined by all of the following parameters:

ANC greater than 1.5 x 10 (9)/L

platelet count greater than 80 x10 (9)/L

hemoglobin greater than 10 g/dl (not transfused)

Weight > 18 kg

Age greater than or equal to 2 years

Able to comprehend and willing to sign an informed consent/assent.

EXCLUSION CRITERIA

Current diagnosis or past history of myelodysplastic syndrome, Fanconis anemia, dyskeratosis congenita or other congenital forms of aplastic anemia.

Evidence of uncontrolled infection

ECOG performance status of 2 or more

Inadequate organ function as defined:

bilirubin greater than 4.0 mg/dl and

transaminases greater than than 2 x ULN.

Current therapy for malignancy

HIV infection

Unfit to receive G-CSF and undergo apheresis (uncontrolled hypertension, currently active ischemic heart disease, unstable arrhythmia, history of chest pain, myocardial infarction, peripheral vascular disease, transient ischemic attack, or stroke).

Psychiatric, affective or any other disorder that would compromise ability to give informed consent

Moribund or patients with concurrent hepatic, renal, cardiac, metabolic disease of such severity that death within 1-4 weeks from the initiation of the therapy is likely.

An enlarged spleen by physical exam.

Special Instructions: Currently Not Provided

Keywords:

Stem Cell Cryopreservation

Leukapheresis

Recruitment Keywords:

None

Conditions:

Aplastic Anemia

Investigational Drug(s):

None

Investigational Device(s):

None

Contacts:

Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citations:

The treatment of severe aplastic anemia

Circulating activated suppressor T lymphocytes in aplastic anemia

The pathophysiology of acquired aplastic anemia

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department of Networks and Applications, CC.

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Bethesda, Maryland 20892. Last update: 10/16/2004