PMID- 23777770
OWN - NLM
STAT- MEDLINE
DCOM- 20131107
LR  - 20220318
IS  - 1528-0020 (Electronic)
IS  - 0006-4971 (Print)
IS  - 0006-4971 (Linking)
VI  - 122
IP  - 7
DP  - 2013 Aug 15
TI  - Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing 
      outcome and costs since the 1970s.
PG  - 1129-36
LID - 10.1182/blood-2012-12-470898 [doi]
AB  - Prophylactic treatment in severe hemophilia is very effective but is limited by 
      cost issues. The implementation of 2 different prophylactic regimens in The 
      Netherlands and Sweden since the 1970s may be considered a natural experiment. We 
      compared the costs and outcomes of Dutch intermediate- and Swedish high-dose 
      prophylactic regimens for patients with severe hemophilia (factor VIII/IX < 1 
      IU/dL) born between 1970 and 1994, using prospective standardized outcome 
      assessment and retrospective collection of cost data. Seventy-eight Dutch and 50 
      Swedish patients, median age 24 years (range, 14-37 years), were included. 
      Intermediate-dose prophylaxis used less factor concentrate (median: Netherlands, 
      2100 IU/kg per year [interquartile range (IQR), 1400-2900 IU/kg per year] vs 
      Sweden, 4000 IU/kg per year [IQR, 3000-4900 IU/kg per year]); (P < .01). Clinical 
      outcome was slightly inferior for the intermediate-dose regimen (P < .01) for 
      5-year bleeding (median, 1.3 [IQR, 0.8-2.7] vs 0 [IQR, 0.0-2.0] joint bleeds/y) 
      and joint health (Haemophilia Joint Health Score >10 of 144 points in 46% vs 11% 
      of participants), although social participation and quality of life were similar. 
      Annual total costs were 66% higher for high-dose prophylaxis (mean, 180 [95% 
      confidence interval, 163 - 196] x US$1000 for Dutch vs 298 [95% confidence 
      interval, 271-325]) x US$1000 for Swedish patients; (P < .01). At group level, 
      the incremental benefits of high-dose prophylaxis appear limited. At the patient 
      level, prophylaxis should be tailored individually, and many patients may do well 
      receiving lower doses of concentrate without compromising safety.
FAU - Fischer, Kathelijn
AU  - Fischer K
AD  - Van Creveldkliniek, Department of Hematology, University Medical Center Utrecht, 
      Utrecht, The Netherlands. k.fischer@umcutrecht.nl
FAU - Steen Carlsson, Katarina
AU  - Steen Carlsson K
FAU - Petrini, Pia
AU  - Petrini P
FAU - Holmstrom, Margareta
AU  - Holmstrom M
FAU - Ljung, Rolf
AU  - Ljung R
FAU - van den Berg, H Marijke
AU  - van den Berg HM
FAU - Berntorp, Erik
AU  - Berntorp E
LA  - eng
PT  - Comparative Study
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20130618
PL  - United States
TA  - Blood
JT  - Blood
JID - 7603509
RN  - 0 (Coagulants)
RN  - 9001-27-8 (Factor VIII)
RN  - 9001-28-9 (Factor IX)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Child
MH  - Child, Preschool
MH  - Coagulants/*administration & dosage
MH  - Dose-Response Relationship, Drug
MH  - Factor IX/*administration & dosage
MH  - Factor VIII/*administration & dosage
MH  - Female
MH  - Hemophilia A/*drug therapy/*economics
MH  - Humans
MH  - Infant
MH  - Male
MH  - Netherlands
MH  - Prospective Studies
MH  - Quality of Life
MH  - Retrospective Studies
MH  - Sweden
MH  - Time Factors
MH  - Treatment Outcome
MH  - Young Adult
PMC - PMC3744988
EDAT- 2013/06/20 06:00
MHDA- 2013/11/08 06:00
PMCR- 2013/08/15
CRDT- 2013/06/20 06:00
PHST- 2013/06/20 06:00 [entrez]
PHST- 2013/06/20 06:00 [pubmed]
PHST- 2013/11/08 06:00 [medline]
PHST- 2013/08/15 00:00 [pmc-release]
AID - S0006-4971(20)54267-5 [pii]
AID - 2012/470898 [pii]
AID - 10.1182/blood-2012-12-470898 [doi]
PST - ppublish
SO  - Blood. 2013 Aug 15;122(7):1129-36. doi: 10.1182/blood-2012-12-470898. Epub 2013 
      Jun 18.