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Desmopressin: therapeutic limitations in children and adults with inherited coagulation disorders.
Br J Haematol 2000; 109(4):865-9BJ

Abstract

Desmopressin (DDAVP), a synthetic analogue of vasopressin has been successfully used in the treatment of type I von Willebrand's disease (VWD), mild factor VIII (FVIII) deficiency and intrinsic platelet function defects (PFDs) for almost three decades. However, there is limited published data documenting its efficacy and the reliability of circulating plasma FVIII:C as a surrogate marker of response to therapy in VWD. We report the haemostatic response to DDAVP in 133 consecutive patients (91 type I VWD, 20 mild FVIII deficiency and 22 PFDs). Minimal therapeutic response to DDAVP (0.3 microg/kg) was defined by normalization 30 min post- infusion of bleeding time for PFDs, factor VIII:C (FVIII:C) for mild haemophilia A, and von Willebrand factor antigen (VWF:Ag), von Willebrand factor functional activity (VWF:Ac) and FVIII:C for VWD. Nine out of 91 (10%) VWD patients failed to achieve minimal therapeutic response to DDAVP; plasma FVIII:C levels were an unreliable surrogate marker of DDAVP response as 6 out of 9 (67%) of these patients had normal post-infusion FVIII:C levels. Five out of the 20 (25%) patients with mild FVIII deficiency and 5 out of 22 (23%) patients with PFDs failed to achieve a minimal therapeutic response to DDAVP. DDAVP is an effective therapy in the majority of patients with type I VWD, PFDs and mild FVIII deficiency. The significant failure rate associated with this therapy supports the recent recommendations that response should be assessed in all patients at the time of diagnosis. FVIII:C is an unreliable guide of response to DDAVP in patients with VWD and therefore VWF:Ag and VWF:Ac should also be assessed. Failure to demonstrate the response of VWF:Ag, VWF:Ac and FVIII:C to DDAVP in patients with VWD is likely to increase the risk of haemorrhagic complications in patients with bleeding episodes or who are undergoing surgery.

Authors+Show Affiliations

National Centre for Inherited Coagulation Disorders, National Children's Hospital, Tallaght Hospital, St James's Hospital, Trinity College Dublin, Ireland.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

10929043

Citation

Nolan, B, et al. "Desmopressin: Therapeutic Limitations in Children and Adults With Inherited Coagulation Disorders." British Journal of Haematology, vol. 109, no. 4, 2000, pp. 865-9.
Nolan B, White B, Smith J, et al. Desmopressin: therapeutic limitations in children and adults with inherited coagulation disorders. Br J Haematol. 2000;109(4):865-9.
Nolan, B., White, B., Smith, J., O'Reily, C., Fitzpatrick, B., & Smith, O. P. (2000). Desmopressin: therapeutic limitations in children and adults with inherited coagulation disorders. British Journal of Haematology, 109(4), pp. 865-9.
Nolan B, et al. Desmopressin: Therapeutic Limitations in Children and Adults With Inherited Coagulation Disorders. Br J Haematol. 2000;109(4):865-9. PubMed PMID: 10929043.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Desmopressin: therapeutic limitations in children and adults with inherited coagulation disorders. AU - Nolan,B, AU - White,B, AU - Smith,J, AU - O'Reily,C, AU - Fitzpatrick,B, AU - Smith,O P, PY - 2000/8/6/pubmed PY - 2000/9/2/medline PY - 2000/8/6/entrez SP - 865 EP - 9 JF - British journal of haematology JO - Br. J. Haematol. VL - 109 IS - 4 N2 - Desmopressin (DDAVP), a synthetic analogue of vasopressin has been successfully used in the treatment of type I von Willebrand's disease (VWD), mild factor VIII (FVIII) deficiency and intrinsic platelet function defects (PFDs) for almost three decades. However, there is limited published data documenting its efficacy and the reliability of circulating plasma FVIII:C as a surrogate marker of response to therapy in VWD. We report the haemostatic response to DDAVP in 133 consecutive patients (91 type I VWD, 20 mild FVIII deficiency and 22 PFDs). Minimal therapeutic response to DDAVP (0.3 microg/kg) was defined by normalization 30 min post- infusion of bleeding time for PFDs, factor VIII:C (FVIII:C) for mild haemophilia A, and von Willebrand factor antigen (VWF:Ag), von Willebrand factor functional activity (VWF:Ac) and FVIII:C for VWD. Nine out of 91 (10%) VWD patients failed to achieve minimal therapeutic response to DDAVP; plasma FVIII:C levels were an unreliable surrogate marker of DDAVP response as 6 out of 9 (67%) of these patients had normal post-infusion FVIII:C levels. Five out of the 20 (25%) patients with mild FVIII deficiency and 5 out of 22 (23%) patients with PFDs failed to achieve a minimal therapeutic response to DDAVP. DDAVP is an effective therapy in the majority of patients with type I VWD, PFDs and mild FVIII deficiency. The significant failure rate associated with this therapy supports the recent recommendations that response should be assessed in all patients at the time of diagnosis. FVIII:C is an unreliable guide of response to DDAVP in patients with VWD and therefore VWF:Ag and VWF:Ac should also be assessed. Failure to demonstrate the response of VWF:Ag, VWF:Ac and FVIII:C to DDAVP in patients with VWD is likely to increase the risk of haemorrhagic complications in patients with bleeding episodes or who are undergoing surgery. SN - 0007-1048 UR - https://www.unboundmedicine.com/medline/citation/10929043/Desmopressin:_therapeutic_limitations_in_children_and_adults_with_inherited_coagulation_disorders_ L2 - https://onlinelibrary.wiley.com/resolve/openurl?genre=article&sid=nlm:pubmed&issn=0007-1048&date=2000&volume=109&issue=4&spage=865 DB - PRIME DP - Unbound Medicine ER -