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C H A N G E   I N   T O T A L   B O D Y  W A T E R   A S   A   P R E D I C T I V E   T O O L   F O R   G R O W T H   H O R M O N E   T R E A T M E N T   R E S P O N S E     Study  design   The  study  design  was  a  prospective  cohort  study  with  duration  of  follow-­‐up  of  12   months.   End   points:   Primary   end   points   were   growth   and   body   composition.   Growth   was   assessed   by   the   evaluation   of   height   during   one   year.   Body   composi-­‐ tion  was  assessed  by  measuring  the  total  body  water  (TBW)  by  the  Deuterium  dilu-­‐ tion  method  (doubly  labeled  water  (DLW)  method).     Measurements   Height   Height  and  weight  were  measured  at  the  start  of  therapy  and  at  3  months  intervals   during   at   least   one   year.   Height   was   measured   using   a   Harpenden   stadiometer.   Subject’s  shoes  and  socks  were  removed.  A  0.5  kg  weight  was  placed  on  the  head-­‐ board   to   flatten   hair.   The   patient   was   kept   in   the   correct   upright   position,   the   ‘Frankfurt  plane’,  while  a  gentle  upward  pressure  was  exerted  on  the  mastoid  pro-­‐ cesses   so   that   the   patient   was   fully   extended.   Height   was   expressed   in   standard   deviation  score  (SDS).  The  difference  in  SDS  over  one  year  (SDSt1-­‐SDSt0)  was  used   to  divide  the  patient  group  into  adequate  and  inadequate  responders.  Weight  was   measured  with  an  electronic  scale  with  digital  readings,  accurate  to  5  gram.  Empty   body  weight  was  measured  without  clothing,  directly  after  waking  up  and  voiding.   Deuterium  dilution  method  (DLW  method)   Body  composition  was  determined  by  measuring  the  TBW  with  the  stable  isotope   of  hydrogen  (Deuterium),  according  to  the  Maastricht  protocol16  at  t  =  0  and  t  =  6   weeks.   A   baseline   urine   sample   was   collected   before   the   labeled   water   was   con-­‐ sumed   to   determine   the   natural   abundance   of   Deuterium   in   the   urine.   The   child   drank  a  known  amount  of  the  doubly  labeled  water  in  the  evening,  before  it  went  to   bed.  Instructions  were  given  to  drink  all  the  water  and  to  rinse  the  bottle  to  be  sure   all  the  water  is  consumed.  A  second  urine  sample  was  collected  after  an  overnight   equilibration  after  the  first  voiding.  TBW  was  calculated  by  the  formula:  C1  x  V1  =  C2   x   V2   (C1   =   concentration   of   label   in   ingested   fluid,   V1   =   volume   ingested   fluid,   C2   =   concentration  tracer  in  second  urine  sample,  V2   =  distribution  volume).  The  distri-­‐ bution   volume   was   divided   by   1.04,   because   of   the   exchange   of   tracer   with   nonaqueous  substances  in  the  body,  to  calculate  the  TBW.  Because  patients  with  a   different  height  were  compared,  the  changes  in  TBW  after  6  weeks  were  corrected   for  height  in  accordance  with  the  Body  Mass  Index  (TBW/height2).  The  difference   of  this  value  between  t0  and  t6  is  plotted  against  the  change  in  height  SDS  after  one   year  of  therapy.     Based   on   the   mean   response   found   by   Ranke   et   al.17 a   cut   off   value   for   good   re-­‐ sponders  was  determined  as  a  change  of  more  than  0.7  SDS  over  one  year.  In  this   study  it  was  observed  that  during  the  first  year  on  rhGH  therapy  the  magnitude  of     47  


Proefschrift binnenwerk Manon Ernst_DEF.indd
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