Akshay Sharma, Amanda Young, Yvonne Carroll, Himani Darji, Yimei Li, Belinda N Mandrell, Marquita N Nelson, Curtis L Owens, Mary Irvine, Mary Caples, Lauren P Jerkins, Yoram Unguru, Jane S Hankins, Liza-Marie Johnson
BACKGROUND: Sickle cell disease (SCD) is an inherited blood disorder that results in serious morbidity and early mortality. Novel therapies for SCD, most notably genetic therapies (GTs) and HLA-mismatched donor hematopoietic cell transplantation, are in clinical trials. While potentially curative, these interventions are some of the most intensive treatments for SCD and are associated with serious and life-altering side effects, which may manifest several years after treatment. Little is known about knowledge, beliefs, and attitudes of individuals with SCD, or their caregivers, toward existing and these emerging therapies...
March 28, 2023: Pediatric Blood & Cancer