Stem Cell Transplant for Severe Aplastic Anemia
Trial Summary
What is the purpose of this trial?
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants. Objectives: To see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy. Eligibility: Recipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75 Design: Recipients will have: * Blood, urine, heart, and lung tests * Scans * Bone marrow sample Recipients will need a caregiver for several months. They may make fertility plans and a power of attorney. Donors will have blood and tissue tests, then injections to boost stem cells for 5-7 days. Donors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg. In the hospital for about 1 month, recipients will have: * Central line inserted in the neck or chest * Medicines for side effects * Chemotherapy over 8 days and radiation 1 time * Stem cell transplant over 4 hours Up to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests. At day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.
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.
What data supports the effectiveness of the treatment for severe aplastic anemia?
Is stem cell transplant using cyclophosphamide safe for humans?
Cyclophosphamide, used in stem cell transplants, has been studied for safety in humans and animals. It can cause serious side effects like bone marrow and heart toxicity, but with proper care, many patients recover well. Long-term studies show high survival rates in patients with severe aplastic anemia treated with high-dose cyclophosphamide.36789
How is the stem cell transplant treatment with cyclophosphamide unique for severe aplastic anemia?
This treatment is unique because it combines high-dose cyclophosphamide, a powerful drug that suppresses the immune system, with peripheral blood stem cells to help restore bone marrow function. Unlike traditional bone marrow transplants, this approach can be used even if a perfect donor match is not available, and it may offer a higher chance of complete remission without the need for additional immunosuppressive therapy.13456
Research Team
Richard W Childs, M.D.
Principal Investigator
National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria
This trial is for people aged 4-55 with severe aplastic anemia, myelodysplastic syndrome (MDS), or paroxysmal nocturnal hemoglobinuria (PNH) who haven't responded to standard treatments. They need a family member donor aged 4-75. Participants must understand the study and consent; minors will need guardian consent. Exclusions include certain heart, liver, kidney issues, active infections not responding to treatment, HIV positive individuals, pregnant women or those not using birth control.Inclusion Criteria
Exclusion Criteria
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
Pre-transplant Preparation
Recipients undergo blood, urine, heart, and lung tests, scans, and bone marrow sampling. Donors receive injections to boost stem cells for 5-7 days.
Transplantation
Recipients receive chemotherapy over 8 days and radiation 1 time, followed by a stem cell transplant over 4 hours.
Post-transplant Monitoring
Recipients stay near NIH for weekly physical exams and blood tests for up to 6 months after transplant.
Follow-up
Participants are monitored for safety and effectiveness after treatment, with tests at their doctor's office and NIH several times over 5 years.
Treatment Details
Interventions
- Cyclophosphamide (Alkylating agents)
- Peripheral Blood Stem Cells (Procedure)
Cyclophosphamide is already approved in Canada, Japan for the following indications:
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Heart, Lung, and Blood Institute (NHLBI)
Lead Sponsor
Dr. Gary H. Gibbons
National Heart, Lung, and Blood Institute (NHLBI)
Chief Executive Officer since 2012
MD from Harvard Medical School
Dr. James P. Kiley
National Heart, Lung, and Blood Institute (NHLBI)
Chief Medical Officer since 2011
MD from University of California, San Francisco