Dr. Mehta will study the use of Quercetin, a naturally occurring flavonoid (antioxidant) in patients with Fanconi anemia Fanconi anemia: A rare inherited disorder that happens when the bone marrow does not make enough blood cells (red cells, white cells, and platelets). Fanconi anemia is diagnosed early in life. People with Fanconi anemia have a high likelihood of developing cancer. Genetic testing is used to diagnose Fanconi anemia. , one of the most common congenital bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… syndromes. She anticipates that the results of this pilot study will show that long term oral Quercetin therapy is feasible and well tolerated in patients with Fanconi anemia. These results will form the basis of the continuation phase of the study, which will demonstrate that Quercetin therapy in fact delays or prevents progressive marrow failure in children with Fanconi anemia.
Dr. Mehta is studying the use of Quercetin, a naturally occurring flavonoid (antioxidant) in patients with Fanconi anemia Fanconi anemia: A rare inherited disorder that happens when the bone marrow does not make enough blood cells (red cells, white cells, and platelets). Fanconi anemia is diagnosed early in life. People with Fanconi anemia have a high likelihood of developing cancer. Genetic testing is used to diagnose Fanconi anemia. , one of the most common congenital bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… syndromes. She anticipates that the results of this pilot study will show that long term oral Quercetin therapy is feasible and well tolerated in patients with Fanconi anemia. These results will form the basis of the continuation phase of the study, which will demonstrate that Quercetin therapy in fact delays or prevents progressive marrow failure in children with Fanconi anemia.
Progress in Dr. Mehta’s study was slower than expected due to some regulatory delays and the limited availability of Quercetin, but all approvals and quality assurance testing have been completed and she and her colleagues are in the process of contacting eligible study participants.