RT Book, Section
A1 Rees, David
A1 Lanzkron, Sophie M.
A2 Gladwin, Mark T.
A2 Kato, Gregory J.
A2 Novelli, Enrico M.
SR Print(0)
ID 1179346034
T1 Overview
T2 Sickle Cell Disease
YR 2021
FD 2021
PB McGraw-Hill Education
PP New York, NY
SN 9781260458596
LK hemonc.mhmedical.com/content.aspx?aid=1179346034
RD 2024/04/24
AB Although  sickle  cell  disease  (SCD)  is  probably  the  most  common  severe  genetic  disorder  in  the  world,  there  are  still  only  3  therapeutic  interventions  that  have  been  convincingly  shown  to  alter  the  natural  history  of  the  condition  in  clinical  trials:  penicillin  prophylaxis  (prevention  of  invasive  pneumococcal  disease),  hydroxyurea  (reduction  of  acute  complications,  primary  stroke  prevention),  and  regular  blood  transfusion  (primary  stroke  prevention).  Although  there  are  other  treatments  that  are  undoubtedly  beneficial,  including  vaccination,  analgesia,  intermittent  blood  transfusion  for  symptomatic  anemia,  and  hematopoietic  stem  cell  transplantation,  the  treatment  options  in  SCD  are  currently  very  limited.