Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia
Status:
Terminated
Trial end date:
2012-07-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Baylor College of Medicine
Collaborators:
Center for Cell and Gene Therapy, Baylor College of Medicine Texas Children's Hospital The Methodist Hospital Research Institute The Methodist Hospital System