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scientific articles

Severe sickle cell disease- pathophysiology and therapy

key information

source: Biology of Blood and Marrow Transplantation 

year: 2009

authors: George Buchanan, Elliott Vichinsky, Lakshmanan Krishnamurti, Shalini Shenoy

summary/abstract:

Background

Over 70,000 people live with sickle cell disease in the United States and multitudes worldwide. About 2,000 afflicted babies are born in this country each year. In African countries such as Nigeria, over 100,000 babies are born with the disease each year. Great strides have been made in the conservative management of sickle cell disease. However, the medical and psychosocial cost of supporting patients with this chronic illness is enormous and spans a lifetime. Hematopoietic stem cell transplant (HSCT) can abrogate sickle cell disease manifestations and is the best option for cure today. Yet, this treatment modality is underutilized as less than 500 transplants are reported in the Center for International Blood and Marrow Transplant Research database due to its significant risk of morbidity and mortality. There is growing understanding of the pathophysiology of the disease, and this, coupled with advances in transplantation and new approaches to therapy continue to improve care of patients with sickle cell disease both in children and during adulthood. Continuing investigation seeks to predict the course of the disease and to determine timing and modality of therapy in order to optimize outcomes

organisation: University of Texas Southwestern at Dallas, TX; Children’s Hospital and Research Center at Oakland, CA; Children’s Hospital of Pittsburgh, University of Pittsburgh, PA; Washington University School of Medicine and St. Louis Children’s Hospital, MO

DOI: 10.1016/j.bbmt.2009.10.001

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