Biological and clinical response to desmopressin (DDAVP) in a retrospective cohort study of children with low von Willebrand factor levels and bleeding history

Abstract

The diagnosis and management of von Willebrand disease (VWD) in paediatrics is challenging. Our aim was to review patient's characteristics related to biological and clinical response to DDAVP in children with low von Willebrand factor (VWF) levels and bleeding history from a single institution. We included a retrospective cohort of 221 children (median age 11 years; 137 females): 27 type 1 (VWF levels within 15-30 IU dL-1) and 194 possible type 1 (VWF levels within 31-49 IU dL-1). The DDAVP infusion-test was performed in 214/221 children, 93.4% of whom showed good response. Patients with type 1 were at higher risk of DDAVP-test failure: 9/26 (34.6%) vs. 18/188 (9.6%) with possible type 1 (RR 3.44, 1.75-6.79; p= 0.002, Fisher's exact test). In 68 children, the clinical response to DDAVP was evaluated 87 times: i) to stop bleeding: menorrhagia (13), mucocutaneous (12), haemarthrosis (1); and ii) to prevent surgical bleeding: adenotonsillectomy (17), major (15) and minor surgery (10); and dental procedures (19). No major adverse events or bleeding were observed. The treatment was effective with one single dose of DDAVP in almost all patients, without antifibrinolytic or local therapy, except in a girl with severe haemorrhage during menarche who required replacement therapy. In conclusion, patients with VWD type 1 were at higher risk of no response to DDAVP infusion-test. In this series, one dose of DDAVP proved effective and safe for children with VWD. Since this is a safe, effective and affordable therapy, we consider that a wider use should be promoted, especially in developing countries.

A phase II dose-escalation trial of perioperative desmopressin (1-desamino-8-d-arginine vasopressin) in breast cancer patients

“…Blood was drawn within 7 days prior to surgery to obtain a baseline, and postoperatively 90–120 min after the first and the second dose of dDAVP. All laboratory assays were performed by investigators blinded to the clinical data, as described elsewhere (Sanchez-Luceros et al 2010 ). The vWF antigen (vWF:Ag) was measured by ELISA.…”

The utility of the DDAVP challenge test in children with low von Willebrand factor

“…(Castaman, et al 2008, Revel-Vilk, et al 2003, Sánchez-Luceros, et al 2010) Sanchez-Luceros et al (2010) used a less conservative response criteria of a two-fold increase in post-VWF:RCo and FVIII:C or post values > 50 iu/dl to define DDAVP response . Using these criteria, 99·4% of the children reported in this study had a complete response to DDAVP.…”

“…In this patient's hospital record, it is noted that the patient had no facial flushing and that the providers did not feel the patient received a full dose. Thus, if the criteria of Sanchez-Luceros et al (2010) were applied to this study population, all of the children with low VWF would have had a complete response after receiving an appropriate dose of DDAVP.…”

Administration of the vasopressin analog desmopressin for the management of bleeding in rectal cancer patients: results of a phase I/II trial

“…Blood was drawn immediately before and 60 minutes after the first intravenous infusion of dDAVP. All laboratory assays were performed by investigators blinded to the clinical data, as described [ 22 ]. The VWF antigen (VWF:Ag) was measured by ELISA.…”