HLA Haploidentical Stem Cell Transplant with Pretransplant Immunosuppression for Patients with Sickle Cell Disease

Sickle cell disease (SCD) is 1 of the most common genetic diseases that affects millions of individuals worldwide. The severity of symptoms cannot be predicted based solely on the genotype. However, in most patients it is associated with early mortality and unfavorable impact on quality of life [1], with recurrent episodes of pain accompanied by progressive damage to vital organs, such as lung, brain, spleen, and kidney [2,3]. Advances in supportive care, including usage of hydroxyurea, have led to improvements in early survival, creating a growing proportion of young adults with chronic health issues related to SCD [4,5].

Source: Biology of Blood and Marrow Transplantation - September 19, 2017 Category: Hematology Authors: Anna B. Pawlowska, Jerry C. Cheng, Nicole A. Karras, Weili Sun, Leo D. Wang, Alison D. Bell, Lisa Gutierrez, Joseph Rosenthal Source Type: research