Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia

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Brief Title

Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia

Official Title

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors (SAA MUD)

Brief Summary

      Patients have been diagnosed with severe Aplastic Anemia that have not responded to treatment
      with immunosuppressive therapy (drugs that suppress the immune system, for example Steroids).
      The immune system is the system in the body that helps protect the body and fights bacterial,
      viral and fungal infections.

      Research studies have shown that patients with Aplastic Anemia have improved survival (may
      live longer) after receiving a HLA (Human Leukocyte Antigen) identical sibling (brother and
      sister) stem cell transplants. Patients who do not have matched siblings can undergo
      immunosuppressive therapy, which has also shown to improve outcome. Unfortunately patients
      who do not respond to immunosuppressive therapy usually die. The best chance of survival for
      these patients is an HLA matched unrelated or mismatched related stem cell transplant as
      described below.

      Stem cells are created in the bone marrow. They mature into different types of blood cells
      that people need including red blood cells which carry oxygen around the body, white blood
      cells which help fight infections, and platelets which help the blood to clot and prevent
      bleeding. For a matched unrelated stem cell transplant, stem cells are collected from a
      person (donor) who is not related to the patient but who has the same type of stem cells. For
      a mismatched related stem cell transplant, stem cells are collected from a donor who is
      related to the patient and whose stem cells are almost the same as those of the patient but
      not exactly. The patient then receives high dose chemotherapy. This chemotherapy kills the
      stem cells in the patient's bone marrow. Stem cells that have been collected from the donor
      are then given to the patient to replace the stem cells that have been killed.

      The major problems associated with these types of stem cell transplants are graft rejection
      (where the patient's immune system rejects the donor stem cells) and severe graft versus host
      disease (GVHD), where the donors stem cell reacts against the patient's tissues in the body.

Detailed Description

      Before patients receive any treatments, they will have the following:

        -  Complete history and physical exam

        -  Dental consultation

        -  Bone marrow aspirate

        -  Blood tests

        -  Urinanalysis

        -  Tests to look at your lungs (Chest X-ray and PFT's and DLCO)

        -  Tests to look at your heart (EKG, MUGA, or echocardiogram)

        -  Pregnancy test for females of childbearing age.

      Once the decision has been made by the patients to have a mismatched related or matched
      unrelated stem cell transplant, the patient will receive treatment called a "conditioning
      regimen" to prepare their body for the transplant. The purpose of this conditioning regimen
      is to kill the stem cells in the bone marrow, so that the patients immune system is
      suppressed (lowered or stopped from working), so their body will not reject the donors stem
      cells (transplant).

      The medicines which will be given as part of the conditioning regimen include
      Cyclophosphamide (Cytoxan) and Campath 1H. Both of these medications are approved by the Food
      and Drug Administration. Cyclophosphamide will be given for four days in a row.
      Cyclophosphamide is broken down into different chemicals and removed from the body in the
      urine. These different chemicals can cause bleeding in the bladder. Mesna is a drug that will
      be given with Cytoxan to prevent the build up of these chemicals that cause bleeding from the
      bladder. Campath 1H will also be given for four days. After the medicines, the patient will
      receive total body irradiation (TBI). Total body irradiation is strong doses of radiation
      (like x-rays) given to almost the whole body. One dose of TBI will be given to patients
      receiving a matched stem cell infusion while two doses of TBI will be given to patients
      receiving a mismatch stem cell infusion. After the conditioning regimen, the patient will
      then receive the stem cells (transplant). These stem cells will be given as an infusion
      (drip) into a vein.

      Patients will receive the drugs Tacrolimus (FK506) and Methotrexate to help prevent the
      complication of graft versus host disease. Methotrexate will be given on days 1, 3, 6 and 11
      after they receive the stem cells. During this time, the patient will be on the stem cell
      transplant unit in protective isolation to help protect from developing infections.

      The following evaluations will be done as part of the stem cell transplant.


        -  Physical examination daily until discharged, then weekly.

        -  Peripheral Blood for chimerism studies (looks at the presence of cells from the donor
           and from the patient existing together after the transplant)

        -  Complete blood count (CBC) and platelets daily until discharge, then at least weekly

        -  Blood chemistry (Electrolytes, BUN, creatinine, glucose, calcium, uric acid, total
           protein, albumin, ALT, AST, LDH, total bilirubin, LDH, magnesium) checked daily until
           discharge, then at least weekly

        -  FK506 blood levels twice a week until discharge, then at least weekly

        -  Blood tests for CMV (a type of virus) weekly

      DAY 100-365:

        -  Physical examination at least monthly through Day 365

        -  Peripheral blood for chimerism studies

        -  Follow-up for patients with chronic GVHD as needed

        -  Chest X-ray

        -  CBC and Blood chemistry tests as above checked monthly


        -  Biopsies as needed (taking tissue samples for testing)

        -  Chest x-ray

        -  CBC and Blood chemistry evaluations as listed above

        -  Peripheral blood for chimerism studies


      We will also be looking at the patients immune function. To do this, we will take 30 ml (2
      tablespoonfuls) of blood every two weeks for two months, then monthly for 6 months, and then
      every 3 months for 2 years. When possible, the blood that is taken will be taken through an
      existing IV line. However, at times drawing the blood will require another stick with a
      needle. These blood tests will be done for research purposes as part of this study. All other
      blood tests listed above are done as part of good clinical care for patients receiving a stem
      cell transplant.

Study Phase

Phase 2

Study Type


Primary Outcome

Number of Subjects Alive at 100 Days Post Transplant

Secondary Outcome

 Number of Patients With Engraftment Rate at 100 Days Post Transplant


Aplastic Anemia



Study Arms / Comparison Groups

Description:  Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Cytoxan, Campath, TBI-Total Body Irradiation, FK-506, Methotrexate, Stem Cell Infusion


* 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 2002

Completion Date

July 2012

Primary Completion Date

July 2012

Eligibility Criteria

        Inclusion Criteria:

          1. Diagnosis of Severe Aplastic Anemia (SAA) based on bone marrow aspirate and biopsy

          2. Failure to respond to immunosuppressive therapy.

          3. Lack of an Human Leukocyte Antigen (HLA) identical family member.

          4. A 6/6 or 5/6 HLA matched unrelated donor or a 5/6 matched related donor available
             after high resolution HLA typing.

          5. Age from birth to 60 years.

        Exclusion Criteria:

          1. Severe disease other than aplastic anemia that would limit the probability of survival
             during the graft procedure. Patients who present with active infection must be treated
             to maximally resolve this problem before beginning the conditioning regimen.

          2. Human immunodeficiency virus (HIV) seropositive patients

          3. Patients who have clonal cytogenetic abnormalities or a myelodysplastic syndrome.

          4. Patient greater than 60 years of age.

          5. Women who are pregnant or nursing.

          6. Patients with active hepatitis

          7. Patients with severe cardiac dysfunction defined as shortening fraction < 25%.

          8. Patients with severe renal dysfunction defined as creatinine clearance < 40

          9. Patient with severe pulmonary dysfunction with forced expiratory volume in the first
             second (FEV1), forced vital capacity (FVC) and diffusing capacity of the lung for
             carbon monoxide (DLCO) 40% of predicted or 3 standard deviations (SD) below normal.




N/A - 60 Years

Accepts Healthy Volunteers



Kathryn Leung, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID

10915-SAA MUD

Secondary IDs


Responsible Party

Principal Investigator

Study Sponsor

Baylor College of Medicine


 The Methodist Hospital Research Institute

Study Sponsor

Kathryn Leung, MD, Principal Investigator, Baylor College of Medicine

Verification Date

March 2016