Inherited Anemias: Slow, Measured Progress

Naveen Manchanda, MD

No meeting in the general field of hematology is complete without reports of progress in sickle cell anemia and thalassemia. While far from where we would like to be, progress in understanding the molecular dynamics of sickle cells as well as newer approaches to iron chelation therapy in thalassemia has given vigor for the benign hematologist.

During this morning’s education session on sickle cell disease (9:30 a.m. in Room 311 of the Orange County Convention Center), Dr. Nigel Key will provide a timely update on the clinical relevance of sickle cell trait. While the trait form of sickle cell hemoglobin has been thought to be mostly a benign condition, the recent news story about an NCAA athlete with this trait sustaining grievous injury reminds us of some of the dangers in this otherwise benign process. Dr. Wally Smith will provide a timely update on the topic of pain in sickle cell disease. He will outline progress in understanding the biological basis of pain. Painful crises in this illness can be recurrent, disabling, and can rob an individual of the best years of his or her life. While long known to be a lifelong accompaniment to this illness, pain has continued to be treated ineffectively. Further insights into the biological basis of this pain are urgently needed, and this talk will be a timely update. Dr. Z. Jim Wang will present a comprehensive review of the physical, biological, social, and clinical aspects of pain in these patients. This session was also presented yesterday at 4:00 p.m. 

Progress in the attack on thalassemia continues in the form of iron chelation therapy, gene therapy, and hematopoeitic stem cell transplantation (HSCT). The education session on thalassemia, scheduled for 7:30 a.m. in Room 224 of the Orange County Convention Center, will feature three speakers. Dr. Ellis Neufeld will provide a timely update on iron chelators, their effectiveness or lack thereof, and options for therapy; Dr. Emanuele Angelucci will discuss different approaches to HSCT in patients at different stages of the disease process; and Dr. Punam Malik will provide a tantalizing update on gene therapy outlining recent developments and possible transfer of technology from bench to bedside to offer a potential cure for this disease. This education session also took place yesterday at 9:30 a.m.

Dr. Manchanda indicated no relevant conflicts of interest.