TY  - CHAP
M1  - Book, Section
TI  - Clinical Trials: State of the Art and Lessons Learned
A1  - De Castro, Laura M.
A1  - Kalpatthi, Ramasubramanian
A1  - Telen, Marilyn J.
A2  - Gladwin, Mark T.
A2  - Kato, Gregory J.
A2  - Novelli, Enrico M.
PY  - 2021
T2  - Sickle Cell Disease
AB  - In  1973,  patients  with  sickle  cell  disease  (SCD)  due  to  homozygosity  for  hemoglobin  S  (HbSS)  were  estimated  to  have  a  median  life  expectancy  of  slightly  more  than  14  years,1  and  thus,  the  disease  was  essentially  a  disease  of  childhood.  However,  in  the  observational  Cooperative  Study  of  Sickle  Cell  Disease  (CSSCD),  data  collected  between  1977  and  1988  showed  that  approximately  85%  of  children  with  HbSS  lived  to  adulthood.2  Furthermore,  in  the  CSSCD,  child  mortality  peaked  early,  occurring  among  children  aged  1  to  3  years,  and  was  due  predominantly  to  infections  such  as  Streptococcus  pneumoniae  sepsis.  Since  the  CSSCD  natural  history  study,  hallmark  interventional  clinical  trials  have  significantly  impacted  the  disease’s  natural  history  by  establishing  standards  of  care,  helping  prevent  serious  complications,  and  improving  quality  of  life  and  survival  in  SCD.  In  this  chapter,  we  first  review  those  clinical  trials  that  have  had  major  impact  on  clinical  practice  and  patient  survival  (Figure  24-1  and  Table  24-1).  We  also  summarize  the  major  observational  studies  that  have  substantially  affected  clinical  practice  in  the  care  of  patients  with  SCD  in  Table  24-2.  Subsequent  sections  in  this  chapter  address  the  key  achievements  and  challenges  of  clinical  trials  in  SCD.  
SN  - 
PB  - McGraw-Hill Education
CY  - New York, NY
Y2  - 2024/04/18
UR  - hemonc.mhmedical.com/content.aspx?aid=1179345141
ER  -