Summary

Eligibility
for people ages up to 25 years (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

Summary

The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) for pediatric acquired severe aplastic anemia.

Details

A major challenge in treating pediatric Severe Aplastic Anemia (SAA) is the determination of best primary therapy for patients who lack a fully matched related donor for HSCT. Good survival outcomes have been seen with IST, but initial and late failures, CSA dependence, persistent cytopenias and secondary Myelodysplastic Syndrome (MDS) / Acute Myeloid Leukemia (AML) in a portion of patients leave considerable room for improvement. MUD HSCT survival in SAA has markedly improved, but a direct comparison of this approach with IST is necessary to determine whether this approach is feasible and will lead to better Event Free Survival. This trial will address the feasibility of randomization, test whether patients can be evaluated in a timely fashion and safely begin therapy with MUD HSCT or IST, and give a preliminary assessment of the safety of up-front MUD HSCT. If successful, this trial will lead to a future prospective trial comparing directly IST to MUD HSCT in this disease.

Keywords

Severe Aplastic Anemia, Anemia, Aplastic Anemia, Cyclosporine, Cyclophosphamide, Methotrexate, Fludarabine, Cyclosporins, Thymoglobulin, Antilymphocyte Serum, Immunosuppressive Agents, Matched Unrelated Donor Hematopoietic Stem Cell Transplant, horse anti-thymocyte globulin (ATG), rabbit anti-thymocyte globulin (ATG), low-dose total body irradiation (TBI), Immunosuppressive Therapy (IST), Immunosuppressive Therapy, Matched Unrelated Stem Cell Transplant

Eligibility

Locations

  • UCSF
    San Francisco California 94123 United States
  • Stanford Lucile Packard Children's Hospital
    Palo Alto California 94304 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Michael Pulsipher, MD
ID
NCT02845596
Study Type
Interventional
Participants
Expecting 40 study participants
Last Updated