PMID- 38519044
OWN - NLM
STAT- Publisher
LR  - 20240322
IS  - 2567-5761 (Electronic)
IS  - 0720-9355 (Linking)
DP  - 2024 Mar 22
TI  - Use of Vonicog Alpha and Acquired von Willebrand Syndrome, a New Approach: A Case 
      Report.
LID - 10.1055/a-2266-7984 [doi]
AB  - Therapeutic management of acquired von Willebrand syndrome (AVWS) can be 
      challenging, particularly in cases of AVWS associated with monoclonal IgM such as 
      Waldenstrom macroglobulinemia (WM) where several therapeutic options may be 
      ineffective. Here, we describe the case of an 88-year-old patient who developed 
      AVWS during follow-up for WM. The presence of a severe bleeding symptomatology 
      not controlled by several therapies (plasma-derived von Willebrand factor, 
      plasmapheresis) led us to introduce a supplementation with recombinant von 
      Willebrand factor, vonicog alpha (Veyvondi, Takeda, Japan), starting at a dose of 
      50 IU/kg/d. This supplementation allowed clinical (no further bleeding) and 
      biological (hemoglobin level, von Willebrand factor parameters) improvements. 
      Because of the persistence of bleeding risk factors, the treatment was maintained 
      at a prophylactic dose (20 UI/kg three times a week), without recurrence of 
      bleeding events for a period of 9 months.
CI  - Thieme. All rights reserved.
FAU - Blandinieres, Adeline
AU  - Blandinieres A
AD  - Department of Hematology, Public Assistance Hospitals of Paris (AP-HP), Bicetre 
      Hospital, Le Kremlin-Bicetre, France.
AD  - University of Paris-Saclay, INSERM Hemostasis, Inflammation and Thrombosis Unit 
      (HITh) U1176, Le Kremlin-Bicetre, France.
FAU - Combe, Sophie
AU  - Combe S
AD  - Department of Hematology, Public Assistance Hospitals of Paris (AP-HP), Bicetre 
      Hospital, Le Kremlin-Bicetre, France.
FAU - Chanson, Noemie
AU  - Chanson N
AD  - Department of Internal Medicine and Clinical Immunology, Public Assistance 
      Hospitals of Paris (AP-HP), Bicetre Hospital, Le Kremlin-Bicetre, France.
FAU - Lambotte, Olivier
AU  - Lambotte O
AD  - Department of Internal Medicine and Clinical Immunology, Public Assistance 
      Hospitals of Paris (AP-HP), Bicetre Hospital, Le Kremlin-Bicetre, France.
AD  - University of Paris-Saclay, INSERM, CEA, UMR 1184, Le Kremlin-Bicetre, France.
FAU - Lavenu-Bombled, Cecile
AU  - Lavenu-Bombled C
AD  - Department of Hematology, Public Assistance Hospitals of Paris (AP-HP), Bicetre 
      Hospital, Le Kremlin-Bicetre, France.
AD  - University of Paris-Saclay, INSERM Hemostasis, Inflammation and Thrombosis Unit 
      (HITh) U1176, Le Kremlin-Bicetre, France.
LA  - eng
PT  - Journal Article
DEP - 20240322
PL  - Germany
TA  - Hamostaseologie
JT  - Hamostaseologie
JID - 8204531
SB  - IM
COIS- A.B. has received financial support from BMS, Takeda, Sobi, and LFB for 
      registration fees and accommodation for scientific congress. S.C. has received 
      financial support from CSL Behring for registration fees and accommodation for 
      scientific congress. O.L. has received financial supports from MSD, BMS, AbbVie, 
      and Boehringer for registration fees, education, and consultancy fees. C.L-B. has 
      received financial supports from CSL Behring, Sobi, Novo Nordisk, Takeda, 
      Octapharma, and LFB for registration fees and education fees. N.C. declares no 
      conflict of interest.
EDAT- 2024/03/23 05:42
MHDA- 2024/03/23 05:42
CRDT- 2024/03/22 20:33
PHST- 2024/03/23 05:42 [medline]
PHST- 2024/03/23 05:42 [pubmed]
PHST- 2024/03/22 20:33 [entrez]
AID - 10.1055/a-2266-7984 [doi]
PST - aheadofprint
SO  - Hamostaseologie. 2024 Mar 22. doi: 10.1055/a-2266-7984.