Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission

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Brief Title

Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission

Official Title

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

Brief 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 Filgrastim 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.
    

Detailed Description

      Immune mechanisms are responsible for hematopoietic failure in most cases of acquired
      aplastic anemia (AA), a disease characterized by hypocellular bone marrow and pancytopenia.
      In aplastic anemia, much experimental data points toward an immune-mediated pathophysiology
      of destruction of hematopoietic progenitor and stem cells. Clinically, immunosuppressive
      therapies, usually anti-thymocyte globulin (ATG) and cyclosporine (CsA), have been shown to
      be effective in a large proportion of patients with severe AA. However, at 2 years after
      initial treatment as many as 35% of patients with initially good response and normal blood
      counts relapse and require repeated cycles of intense immunosuppression and/or chronic
      immunosuppressive regimens. Although relapse in previously immunosuppression-responsive
      patients has a generally good prognosis, there is an increased risk of complications and
      treatment-related toxicities. The outlook of patients who fail to respond to repeated
      intensive immunosuppression is poor. While it is likely that some of the treatment failures
      occurring with conventional immunosuppressive regimens (both at presentation and in relapsed
      patients) may be due to inability to suppress the autoimmune process leading to the bone
      marrow failure, more intense therapies such as cyclophosphamide have a high complication rate
      due to prolonged and dose-related myelosuppression. In this protocol, we propose that
      patients with severe AA who show a good response to the initial round of immunosuppression
      undergo stem cell mobilization, collection, and cryopreservation.

      This pilot study of 20 patients is designed to evaluate: 1) the CD34+ cell mobilization
      response to administration of standard doses of granulocyte-colony stimulating factor (G-CSF)
      and 2) the potential for collecting stem cells from patients with a history of severe AA who
      have been given G-CSF. Outcome parameters to be monitored are the mobilization response to
      G-CSF, and the safety profile and tolerance of G-CSF and leukapheresis. Effectiveness will be
      gauged by historical comparison of these parameters to normal healthy age-matched volunteers.

      It is important to point out that there is no therapeutic intent to the majority of this
      protocol or direct benefit for enrolled patients. We do plan, however, to cryopreserve the
      remainder of the mobilized cells collected by apheresis for possible autologous
      transplantation in the event of the patient's progression to leukemia or bone marrow failure
      in the future.
    

Study Phase

Phase 1

Study Type

Interventional




Condition

Aplastic Anemia

Intervention

G-CSF


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

20

Start Date

February 2001

Completion Date

February 2006


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 or growth
        factors or exhibit a spontaneous remission as defined by all peripheral counts as indicated
        below (must be at least 3 months following the initial course of immunosupressive or growth
        factor therapy and must be sustained for at least 3 week)

        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 the investigational nature of the protocol and be 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.

        Pregnant or lactating.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00011830

Organization ID

010083

Secondary IDs

01-H-0083


Study Sponsor

National Heart, Lung, and Blood Institute (NHLBI)


Study Sponsor

, , 


Verification Date

February 2006