PMID- 16427383
OWN - NLM
STAT- MEDLINE
DCOM- 20060412
LR  - 20161124
IS  - 0037-1963 (Print)
IS  - 0037-1963 (Linking)
VI  - 43
IP  - 1 Suppl 1
DP  - 2006 Jan
TI  - Glanzmann's thrombasthenia treatment: a prospective observational registry on the 
      use of recombinant human activated factor VII and other hemostatic agents.
PG  - S33-6
AB  - Glanzmann's thrombasthenia (GT) is a rare congenital bleeding disorder caused by 
      deficiency or dysfunction of platelet surface glycoprotein (GP) IIb/IIIa 
      receptor. Platelet transfusion is the standard treatment for bleeding that 
      remains non-responsive to conservative measures, and for surgical coverage. 
      Platelet transfusions, however, may result in the development of antibodies to 
      GPIIb/IIIa and/or human leukocyte antigen (HLA), rendering further transfusions 
      ineffective. Recombinant human activated factor VII (rFVIIa; NovoSeven/Niastase 
      [Canada], Novo Nordisk, Bagsvaerd, Denmark) has documented efficacy in GT 
      patients, and is approved in the European Union for the treatment of GT patients 
      with platelet antibodies and platelet refractoriness. However, there are 
      insufficient data to determine the optimal rFVIIa regimen (eg, for major surgery) 
      or to allow thorough safety evaluation (eg, thrombotic risk). A post-marketing, 
      prospective, observational, multinational registry has been developed to collect 
      data on the efficacy and safety of rFVIIa in the treatment and prevention of 
      bleeding in GT patients with platelet antibodies or platelet refractoriness. 
      Patients treated with other hemostatic agents or rFVIIa to avoid the development 
      of antibodies against GPIIb/IIIa will also be reported. Standardized data will be 
      collected using a customized internet-based (www.glanzmann-reg.org) data 
      collection tool. Data collection will begin in 2005 and continue for up to 6 
      years. Patients of all ages from any country are eligible for inclusion.
FAU - Poon, Man-Chiu
AU  - Poon MC
AD  - University of Calgary, Southern Alberta Hemophilia Clinic, Calgary Health Region, 
      Canada. mcpoon@ucalgary.ca
FAU - Zotz, Rainer
AU  - Zotz R
FAU - Di Minno, Giovanni
AU  - Di Minno G
FAU - Abrams, Zvika S
AU  - Abrams ZS
FAU - Knudsen, Jens B
AU  - Knudsen JB
FAU - Laurian, Yves
AU  - Laurian Y
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Semin Hematol
JT  - Seminars in hematology
JID - 0404514
RN  - 0 (Autoantibodies)
RN  - 0 (HLA Antigens)
RN  - 0 (Hemostatics)
RN  - 0 (Platelet Glycoprotein GPIIb-IIIa Complex)
RN  - 0 (Recombinant Proteins)
RN  - 9001-25-6 (Factor VII)
RN  - AC71R787OV (recombinant FVIIa)
RN  - EC 3.4.21.21 (Factor VIIa)
SB  - IM
MH  - Autoantibodies/immunology
MH  - Autoimmune Diseases/etiology/immunology
MH  - Factor VII/*therapeutic use
MH  - Factor VIIa
MH  - HLA Antigens/immunology
MH  - Hemorrhage/etiology/prevention & control
MH  - Hemostatics/*therapeutic use
MH  - Humans
MH  - Internet
MH  - Multicenter Studies as Topic
MH  - Platelet Glycoprotein GPIIb-IIIa Complex/immunology
MH  - *Platelet Transfusion/adverse effects
MH  - Prospective Studies
MH  - Recombinant Proteins/therapeutic use
MH  - *Registries
MH  - Thrombasthenia/complications/immunology/*therapy
RF  - 13
EDAT- 2006/01/24 09:00
MHDA- 2006/04/13 09:00
CRDT- 2006/01/24 09:00
PHST- 2006/01/24 09:00 [pubmed]
PHST- 2006/04/13 09:00 [medline]
PHST- 2006/01/24 09:00 [entrez]
AID - S0037-1963(05)00216-7 [pii]
AID - 10.1053/j.seminhematol.2005.11.009 [doi]
PST - ppublish
SO  - Semin Hematol. 2006 Jan;43(1 Suppl 1):S33-6. doi: 
      10.1053/j.seminhematol.2005.11.009.