RT Book, Section
A1 Minniti, Caterina P.
A1 Hankins, Jane S.
A2 Gladwin, Mark T.
A2 Kato, Gregory J.
A2 Novelli, Enrico M.
SR Print(0)
ID 1179345519
T1 Therapeutic Options and Combination Therapy
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=1179345519
RD 2024/04/18
AB The  therapeutic  landscape  for  patients  with  sickle  cell  disease  (SCD)  has  recently  expanded,  and  health  care  practitioners  now  have  several  options  of  disease-modifying  drugs  that  are  sickle  cell  specific.  As  of  2020,  in  the  United  States,  there  are  4  Food  and  Drug  Administration  (FDA)-approved  drugs  for  patients  living  with  SCD:  hydroxyurea,  L-glutamine,1  voxelotor,2  and  crizanlizumab.3  Many  more  agents  are  currently  being  tested  in  phase  II  or  III  trials;  therefore,  we  foresee  that  several  new  targeted  therapies  will  be  available  by  the  end  of  the  current  decade.  The  treatment  of  individuals  with  SCD  will  become  increasingly  complex  and,  at  the  same  time,  mechanistically  based,  as  a  large  number  of  these  drugs  are  specifically  being  developed  to  affect  and/or  change  the  multifaceted  pathophysiology  of  SCD.