Interview: Dr. Patricia Shi, Mt. Sinai Medical Center
June 8, 2011 – CAF speaks with Dr. Patricia A. Shi, who runs the adult hemoglobinopathy program at Mt. Sinai Medical Center in New York City.
CAF: How many thalassemia patients are seen at the Mt Sinai thalassemia program?
PS: We have always been known as one of the comprehensive sickle cell programs in New York City. We have not been traditionally known as a thalassemia program, however, so we currently have only a few patients, transferred from the pediatric program led by Dr. Anne Hurlet. The pediatric program sees about 8 patients currently.
|Dr. Patricia A. Shi|
CAF: How long have you been involved with the thalassemia program at Mt. Sinai?
PS: I took over the sickle cell program in January of 2009 from one of my mentors, Dr. George Atweh, when he moved from Mt. Sinai to become chairman of the Hematology-Oncology division at University of Cincinnati. Previous to that, being dual-certified in Blood Banking and Hematology, I was working primarily in Transfusion Medicine as the director of cellular therapy collections for the bone marrow transplantation program. I have been at Mount Sinai since completing my post-doctoral training at the National Institutes of Health in 2002.
CAF: How did you get interested in/involved in thalassemia?
PS: Dr. Atweh was interested in thalassemia as well as sickle cell, and he sparked my interest. The two diseases are related in that they are both disorders that affect genes involved in hemoglobin production and cause anemia. Although the pathophysiology in these disorders is obviously very different, they share an increased risk for complications such as gallstones, thrombosis, osteoporosis, leg ulcers, gout, pulmonary hypertension, iron overload, and aplastic crises from parvovirus B19 infection. Currently available treatment options also overlap those being transfusion, iron chelation, hydroxyurea, and bone marrow transplantation.
CAF: Tell me more about the thalassemia program at Mt. Sinai.
PS: Dr. Anne Hurlet and I direct the pediatric and adult patient programs, respectively, so patients seamlessly transition from one program to the other. To be cared for by hematologists specifically committed and interested in taking care of patients with thalassemia, which at the minimum means keeping up to date with the current literature in thalassemia, is important. That Mount Sinai is a tertiary care center is also important, because of the need to access advanced technologies and the expertise of other subspecialties in the care of patients with thalassemia. Mount Sinai has highly nationally ranked programs in endocrinology, cardiology, obstetrics/gynecology, pulmonology, gastroenterology, and nephrology that allow us to provide optimal care and coordination of care to patients. Lastly, our clinical dedication also extends to our basic research endeavors, where Dr. James Bieker, Dr. Ron Hoffman, and Dr .Anna Rita Migliaccio are carrying out laboratory investigations related to thalassemia.
CAF: Who else comprises the thalassemia program staff at Mt. Sinai?
PS: We were fortunate to recruit Jena Srivastava as the nurse practitioner for our program, specifically and solely to take care of our adult sickle cell and thalassemia patients. Our clinical program also has an excellent social worker, Meredith Ruden. On the basic research side, we have several investigators interested in thalassemia research. One is Dr. James Bieker, who is studying a class of molecules called peptide nucleic acids for their ability to increase hemoglobin F levels. Dr. Ron Hoffman is also exploring chromatin modifying agents such as DNA methyltransferase inhibitors and histone deacetylase inhibitors for their ability to increase Hb F. Both he and Dr. Anna Rita Migliaccio are studying the capacity of red cells grown in vitro to improve transfusion therapy for patients with thalassemia. We eventually hope to translate these laboratory-based research endeavors into clinical trials.
CAF: Is the program able to offer access to R2 and/or T2* technology?
PS: The program has on-site access to liver and cardiac MRIs using T2/T2* technology to accurately assess iron burden under the leadership of Dr. Bachir Taouli and Dr. Javier Sanz in the Division of Radiology and Cardiology, respectively.
CAF: Can you tell me about the Mt. Sinai’s experience with bone marrow transplantation (BMT) and thalassemia?
PS: We have an active bone marrow transplant program in thalassemia under the leadership of Dr. Gustavo Del Toro for pediatric patients. Since arriving to Mount Sinai in 2000, Dr. Del Toro has performed four unrelated cord blood transplants, in two patients with β-thalassemia major and two with HbE/β-thalassemia. The patients ranged in age from 6 months to 5 years of age, and all have been cured of their disease and are doing well. We also have an active adult program in bone marrow transplantation under the leadership of Dr. Luis Isola, but we have not transplanted any adults with thalassemia due to the high morbidity and mortality risk. We hope to participate in gene therapy trials in the near future.
CAF: Are there any thalassemia-related clinical trials in which Mt. Sinai is currently involved?
PS: Because of our currently small patient numbers, we do not have any treatment trials active. We do have an ongoing laboratory-based trial with Dr. Barry Coller, looking at levels of platelet activation in thalassemia patients, in order to better understand the increased thrombosis risk in thalassemia. This simply requires an extra tube of blood to be drawn at the time of the routine blood draw with their clinic visit. As our clinic numbers expand, however, we will certainly open up new trials. Some of the trials that we plan to pursue, as I mentioned, involve reducing red cell alloimmunization and transfusion frequency and increasing HbF levels. We are also interested in gene therapy trials in collaboration with Dr. Michel Sadelain.
CAF: What else would you like to share?
PS: We hope to expand the hours of clinic to include evening hours once a week, in order to better accommodate the lifestyles of our patients. This reflects our commitment to providing the best standard of clinical care for patients with thalassemia and also raising these standards by developing improved and novel therapies.