Significant advances in the treatment of thalassemia in the past three decades have been accompanied by many new clinical research questions that can only be answered by multi-center trials with a sufficient number of eligible patients. To this end, the long- term objective of this proposal is to develop and participate in a Clinical Thalassemia Network that will design and implement collaborative research studies in thalassemia. The major specific aim related to this objective is to create the Mid- Atlantic Thalassemia Consortium that includes the Children's Hospital of Philadelphia, Thomas Jefferson Hospital/Alfred I. duPont Hospital for Children, St. Christopher's Hospital for Children, Johns Hopkins Hospital, and the Children's National Medical Center of Washington DC. This Consortium, which includes 82 patients with clinically significant thalassemia syndromes, takes advantage of the experience and expertise that exists in the five thalassemia programs. The Principal Investigator and the four co-investigators have a combined experience in clinical research in hemoglobinopathies of more than 100 years. The application proposes one short-term and one long-term clinical trial that are well suited for multi-center research within the Thalassemia Network. The goal of the short-term trial is to develop fetal hemoglobin enhancing agents as a safe and effective therapy of thalassemia intermedia.
The specific aim i s to conduct a one-year, randomized, placebo-controlled multi-center trial of a pulse-dose regimen of sodium phenylbutyrate to determine the effect on hemoglobin level and other hematologic parameters. The goal of the long-term trial is to determine the effect of storage time of donor red cells on the transfusion requirements and rate of iron loading in patients with thalassemia major, and to correlate these differences with findings using radiolabeled red cells.
The specific aim i s to conduct a three-year, randomized, crossover clinical trial of AS- 1 and AS-3 donor red cells stored for either 0-14 days or 21-35 days and to evaluate blood requirements and donor unit utilization. 51Cr studies of 24-hour red cell recovery, post-24- hour red cell survival and red cell availability in a subset of patients will complement the clinical trial. Both the short-term and long-term study take advantage of the specific expertise of investigators in the Mid-Atlantic Thalassemia Consortium. Moreover, both studies require the number of patients that can only be achieved through multi-center research as proposed for the Thalassemia Clinical Network. The successful development of the Clinical Thalassemia Network will provide a unique opportunity to address some of the most important problems affecting the length and quality of life of patients with thalassemia, and will serve as a model for the study of other diseases.
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