Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Status:
Completed
Trial end date:
2016-06-01
Target enrollment:
Participant gender:
Summary
Transplantation with stem cells is a standard therapy in many centers around the world.
Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other
cancers, aplastic anemia and other non-malignant diseases, has led to prolonged disease-free
survival or cure for some patients. However, the high doses of pre-transplant radiation and
chemotherapy drugs used, and the type of drugs used, often cause many side effects that are
intolerable for some patients. Slow recovery of blood counts is a frequent complication of
high dose pre-transplant regimens, resulting in a longer period of risk for bleeding and
infection plus a longer time in the hospital.
Recent studies have shown that using lower doses of radiation and chemotherapy (ones that do
not completely kill all of the patient's bone marrow cells) before blood or bone marrow
transplant, may be a better treatment for high risk patients, such as those with Dyskeratosis
Congenita (DC) or Severe Aplastic Anemia(SAA). These low dose transplants may result in
shorter periods of low blood counts, and blood counts that do not go as low as with
traditional pre-transplant radiation and chemotherapy. Furthermore, in patients with
Dyskeratosis Congenita or SAA, the stem cell transplant will replace the blood forming cells
with healthy cells.
It has recently been shown that healthy marrow can take and grow after transplantation which
uses doses of chemotherapy and radiation that are much lower than that given to patients with
leukemia. While high doses of chemotherapy and radiation may be necessary to get rid of
leukemia, this may not be important to patients with Dyskeratosis Congenita or SAA. The
purpose of this research is to see if this lower dose chemotherapy and radiation regimen
followed by transplant is a safe and effective treatment for patients with Dyskeratosis
Congenita or SAA.