Comparing Therapies for the Treatment of Severe Aplastic Anemia
Severe Aplastic Anemia (SAA) is a rare and very serious blood disorder in which the bone marrow stops producing the cells which make up blood; red blood cells, white blood cells, and platelets.
Researchers believe this is caused by an autoimmune reaction, a condition in which the natural defense system of the body begins attacking itself. In SAA the immune system begins attacking the bone marrow. Red blood cells are responsible for carrying oxygen to all of the organ systems in the body, and low numbers (anemia) can cause difficulty breathing and fatigue. Platelets are responsible for normal blood clotting and low numbers can result in easy bruising and bleeding which can be deadly. White blood cells are responsible for fighting infections, and low numbers of these can lead to frequent infections, the most common cause of death in patients with aplastic anemia.
SAA can be treated by bone marrow transplant (BMT) or by drugs designed to slow down the immune system (immunosuppressants). BMT can be successful, but it requires a donor with matched bone marrow, making this therapy available only to a few patients. BMT with unmatched bone marrow can fail and cause dangerous side effects.
Presently, the two drugs used to treat SAA by slowing down the immune system (immunosuppression) are antithymocyte globulin (ATG) and cyclosporin A (CSA). When used in combination these two drugs can improve most patients condition. However, one third of the patients who respond to this therapy experience a relapse of SAA. In addition, some patients treated with ATG/CSA can later develop other disorders of the blood.
Recently, researchers have found that another immunosuppressive drug called cyclophosphamide, has been successful at treating patients with SAA. In addition, patients treated with cyclophosphamide do not experience relapses or develop other disorders of the blood.
In this study researchers would like to compare the combinations of antithymocyte globulin (ATG) and cyclosporin A (CSA) to cyclophosphamide and cyclosporin A (CSA) for the treatment of SAA.
|Aplastic Anemia Hematologic Disease||Drug: Antithymocyte globulin & Cyclosporin A Drug: Cyclophosphamide & Cyclosporin A||Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Randomized Trial of Antithymocyte Globulin and Cyclosporine Versus Cyclophosphamide and Cyclosporine in the Treatment of Severe Aplastic Anemia|
- Compare the sustained response proportions among patients with SAA treated with immunosuppressive therapy with either ATG/CSA or high dose cyclophosphamide and CSA. [ Time Frame: 12 weeks. ]
- Overall and event-free survival rate. [ Time Frame: 12 months ]
|Study Start Date:||May 2, 1997|
|Study Completion Date:||March 3, 2008|
|Primary Completion Date:||September 30, 2002 (Final data collection date for primary outcome measure)|
1 hour intravenous infusion of 50 mg/kg of cyclophosphamide daily for 4 days as an alternative immunosuppressive agent for treatment of severe aplastaic anemia.
Drug: Cyclophosphamide & Cyclosporin A
1 hour IV infusion of 50 mg/kg daily for 4 days
Active Comparator: 2
40 mg/kg of ATG daily for 4 days as immunosuppressive agent for treatment of severe aplastic anemia
Drug: Antithymocyte globulin & Cyclosporin A
40 mg/kg daily for 4 days
Please refer to this study by its ClinicalTrials.gov identifier: NCT00001626
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Neal S Young, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|