Cord Blood Transplant for Blood Diseases
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial uses stem cells from a baby's umbilical cord to treat patients who need new healthy stem cells. Patients first get strong medicine to clear out unhealthy cells, then receive the new stem cells, and take medications to prevent complications. Umbilical cord blood has been used in the treatment of various diseases, including leukemias, lymphomas, and immune system disorders.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is cord blood transplant generally safe for treating blood diseases?
Research shows that using busulfan, fludarabine, and melphalan in cord blood transplants is generally well-tolerated, with acceptable safety profiles in both children and adults. Some studies report manageable side effects like gastrointestinal issues and rare serious complications, but overall, these regimens have been used safely in various conditions.12345
How does the cord blood transplant treatment differ from other treatments for blood diseases?
This treatment is unique because it combines multiple chemotherapy drugs and total body irradiation to prepare the body for a cord blood transplant, which can be an alternative for patients without a matched donor. The combination of drugs like busulfan, fludarabine, and melphalan, along with total body irradiation, aims to reduce the risk of graft failure and relapse, although it may have higher toxicity compared to other regimens.25678
What data supports the effectiveness of the treatment Cord Blood Transplant for Blood Diseases?
The study on high-dose busulfan/melphalan for autologous transplantation in children with solid tumors showed that this combination is feasible and well-tolerated, with an acceptable mortality rate and proven antitumor activity, suggesting potential effectiveness in similar transplant settings.245910
Who Is on the Research Team?
Omar Aljitawi, MD
Principal Investigator
Professor - Department of Medicine, Hematology/Oncology (SMD)
Are You a Good Fit for This Trial?
This trial is for patients with various blood diseases, immune disorders, and cancers like leukemia, lymphoma, and solid tumors. Participants need to have a certain level of physical fitness (Karnofsky or Lansky score ≥ 70%) and good heart, lung, kidney, and liver function. They must not be pregnant or breastfeeding, HIV positive, or have had a recent autologous HSCT. A suitable HLA-matched donor should not be available.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Pre-transplant Conditioning
Participants receive chemotherapy +/- total body radiation as a pre-transplant conditioning regimen
Transplantation
Participants receive cord blood stem cells followed by GvHD prophylaxis
Follow-up
Participants are monitored for safety and effectiveness after transplantation
What Are the Treatments Tested in This Trial?
Interventions
- Busulfan
- Cord Blood Infusion
- Cyclophosphamide
- Fludarabine
- Melphalan
- Mesna
- Total Body Irradiation 1200 cGy
- Total Body Irradiation 200 cGy
Busulfan is already approved in United States, European Union, Canada, Japan for the following indications:
- Chronic myeloid leukemia
- Acute myeloid leukemia
- Malignant lymphoma
- Bone marrow transplantation conditioning
- Chronic myeloid leukemia
- Acute myeloid leukemia
- Bone marrow transplantation conditioning
- Chronic myeloid leukemia
- Acute myeloid leukemia
- Bone marrow transplantation conditioning
- Chronic myeloid leukemia
- Acute myeloid leukemia
- Bone marrow transplantation conditioning
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Rochester
Lead Sponsor