Allogeneic Stem Cell Transplantation for Patients with Severe Aplastic Anemia


  • Study type

  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • Site IRB
    • Rochester, Minnesota: 06-006216
    NCT ID: NCT00578266
    Sponsor Protocol Number: 06-006216

About this study

For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of allogenic stem cell transplantation from either a matched unrelated donor or a mismatched reacted donor using the conditioning regimen of Cytoxan, reduced total body irradiation (TBI) and Campath IH. The principle measures of safety and efficacy will be :

  1. Patient survival probability at 100 days, 1 year and 2 years.
  2. Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years.
  3. Engraftment at 6 months, 1 year and 2 years

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • Diagnosis of SAA based on bone marrow aspirate and biopsy results
  • Failure to respond to immunosuppressive therapy
  • Lack of an HLA identical family member
  • A 6/6 or 5/6 HLA matched unrelated donor or a 5/6 matched related donor available after high resolution typing

Exclusion Criteria:

  • Aplastic anemia and active infection must be treated to maximally resolve this problem before beginning the conditioning regimen
  • HIV seropositive
  • Clonal cytogenetic abnormalities or a myelodysplastic syndrome
  • Greater than 60 years of age
  • Pregnant or nursing
  • Active hepatitis
  • Severe cardiac dysfunction defined as shortening fraction <25%
  • Severe renal dysfunction defined as creatinine clearance < 40ml/mim/1.73m2
  • Severe pulmonary dysfunction with FEV1, FVC and DLCO 40% of predicted or 3 SD below normal

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Shakila Khan, M.D.

Closed for enrollment


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