Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
Status:
Completed
Trial end date:
2010-12-01
Target enrollment:
Participant gender:
Summary
The purpose of this research study is to determine whether an experimental drug called
AMD3100 used in combination with another medication called G-CSF is safe and can help to
increase the amount of blood stem cells (called CD34+ stem cells) found in the peripheral
blood of patients with Fanconi anemia. While AMD3100 has been used successfully in adult
volunteers and cancer patients, it has not been used in children or patients with Fanconi
anemia and in only a few children with cancer.
Fanconi anemia is a rare genetic disease. Most Fanconi anemia patients eventually develop
bone marrow failure, a condition in which the bone marrow no longer produces red blood cells
(to carry oxygen), white blood cells (to fight infection), and platelets (to help blood
clot). The only successful treatment for patients with Fanconi anemia with bone marrow
failure is bone marrow transplantation. However, this treatment has many risks and is not
available to all patients with Fanconi anemia.
CD34+ cells include stem cells found in the bone marrow or peripheral blood which are capable
of making the red blood cells, white blood cells, and platelets. CD34+ stem cells can be
collected from bone marrow or peripheral blood and purified using an experimental device
called the CliniMACS. However, most Fanconi anemia patients do not have enough CD34+ stem
cells in their bone marrow or peripheral blood to be collected using standard methods that
work well in children and adults who don't have Fanconi anemia.