Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission

Related Clinical Trial
Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia Long Term Follow-up Observational Study After Clinical Trials of AMG531 (Romiplostim) in Patients With Untreated Aplastic Anemia Efficacy and Safety of Lower-dose Decitabine in Refractory Aplastic Anemia Quantitative MRI of Bone Marrow Fat Fraction in Patients With Trepanobiopsy Avatrombopag Usage in NSAA Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7Abnormalities After Immunosuppressive Therapy REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) Investigation of the Cylex® ImmuKnow® Assay Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant Umbilical Cord Blood Transplantation From Unrelated Donors Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study Unrelated Umbilical Cord Blood (UBC)Transplantation New York Blood Center National Cord Blood Program Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor Nonmyeloablative Allo SCT for the Treatment of Hematologic Disorders Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis Effects of Aerobic Training and Inspiratory Muscle Training in Patients During Hematopoietic Stem Cell Transplantation A Phase II Study of Umbilical Cord Blood Transplantation Transplants With Unlicensed Preserved Cord Blood Safety Study of Cord Blood Units for Stem Cell Transplants Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells Trial of Two Central Venous Catheter (CVC) Flushing Schemes in Pediatric Hematology and Oncology Patients Blood Transplantation for Patients With Hematologic Malignancies or Bone Marrow Failure States Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies Extended Platelet Parameters as a Means to Differentiate Immune Thrombocytopenia From Hypo-proliferative Thrombocytopenias. Protection Against Benzene Toxicity Treatment of Menorrhagia in Women With Thrombocytopenia Using Platelets or Platelets and Hormones Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA Etiology of Blood Dyscrasias: Analysis of the International Agranulocytosis and Aplastic Anemia Study Data Multi-Center Trial of Anti-Thymocyte Globulin in Treatment of Aplastic Anemia and Other Hematologic Disorders King’s Invasive Aspergillosis Study II Identification of Mechanism in the Erythroid Response in Patients With Myelodysplasia Undergoing Chelation Therapy Aplastic Anemia Epidemiology: Incidence and Case-control Drug Etiology of Aplastic Anemia and Related Dyscrasias hATG+CsA vs hATG+CsA+Eltrombopag for SAA Non-Myeloablative Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Treatment of Hematologic Malignancies, Renal Cell Carcinoma, and Aplastic Anemia Conditioning Regimens for Patients With Severe Aplastic Anemia Transplanted With Marrow From an Unrelated Donor Study of Allogeneic Bone Marrow and T-Cell Depleted, CD34+ Peripheral Blood Stem Cell Transplantation in Patients With Aplastic Anemia Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia Study of MRI Monitoring in Patients With Aplastic Anemia and Low or Int-1 Risk of MDS Complicated With Iron Overload A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA) Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome Efficacy and Safety of Eltrombopag + CSA in Patients With Moderate Aplastic Anemia (EMAA) Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia Multi Center Case Control Study on Multiple Risk Factors of Aplastic Anemia NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA) Haploidentical Transplantation in Severe Aplastic Anemia A Description of Bacteria in the Mouths of Patients With Severe Aplastic Anemia Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia (SAA) Patient Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Aplastic Anemia Patients A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia Cyclophosphamide, Antithymocyte Globulin, and Total-Body Irradiation in Treating Patients With Severe Aplastic Anemia Undergoing Umbilical Cord Blood Transplant Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia Stem Cell Factor Medication for Aplastic Anemia Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome Extended Dosing With Eltrombopag for Severe Aplastic Anemia Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF) Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia Collection of Blood and Bone Marrow From Patients With Aplastic Anemia for Analysis of Adhesion Molecules, Chemokines and Their Receptors Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia Alefacept in Patients With Relapsed/Refractory Aplastic Anemia Oral Manifestations of Aplastic Anemia The Efficacy of Immunosuppressive Therapy Combined With Cord Blood Transfusion in Treatment of Severe Aplastic Anemia Clinical Study of Non Severe Aplastic Anemia Treated With Cyclosporine, Androgen and Levamisole Mesenchymal Stem Cells Transplantation to Patients With Relapsed/Refractory Aplastic Anemia. Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA) ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia A Novel TBI Free Conditioning Protocol for Haploidentical Transplant in Acquired Aplastic Anemia: Combination Therapy of Severe Aplastic Anemia Comparing Therapies for the Treatment of Severe Aplastic Anemia Mesenchymal Stem Cells Co-transplantation in Alternative Donor Transplantation of Severe Aplastic Anemia. Study of AMG531(Romiplostim) in Patients With Aplastic Anemia Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia Improving Immunosuppressive Treatment for Patients With Severe Aplastic Anemia Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia Alemtuzumab and Rituximab in Aplastic Anemia Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA) Safety and Efficacy of Patient’s Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

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



Aplastic Anemia




* 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


Estimated Enrollment


Start Date

February 2001

Completion Date

February 2006

Eligibility 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.


        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

        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.




N/A - N/A

Accepts Healthy Volunteers



, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Study Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Study Sponsor

, , 

Verification Date

February 2006