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Desmopressin Use in Type 2B von Willebrand Disease in North America, a Survey of Members of the Hemophilia Research Society of North America



Desmopressin Use in Type 2B von Willebrand Disease in North America, a Survey of Members of the Hemophilia Research Society of North America



Blood 100(11): Abstract No 2750



The role of desmopressin (DDAVP) in the management of Type 1 von Willebrand disease (VWD) is well established. However, its use in Type 2B vWD is uncertain given the observation of worsening thrombocytopenia with increased release of abnormal von Willebrand factor. There has also been a concern of inducing thrombosis with platelet aggregate formation. Some published cases, primarily from Europe, have reported clinical efficacy of DDAVP given for prophylaxis of bleeding from operative procedures (hysterectomy, cholecystectomy) and dental extraction. Objective: To accrue further reports on the use of DDAVP in clinical practice in North America and to examine related laboratory variables in hopes of reaching a consensus regarding the feasibility of DDAVP use in Type 2B. Patients and Methods: A two page data collection form regarding laboratory and clinical items visa a vie DDAVP use in Type 2B approved by the Hemophilia Research Society (HRS) of North America was sent to all members. Results: 38/148 (25%) HRS members responded to date; 128 patients with Type 2B vWD were identified. Two patients had a DDAVP trial only: 5/128 (4%) Type 2B patients were administered DDAVP for either active bleeding or prophylaxis with subsequent grading of clinical response: cntdot epistaxis-2 patients with "good" response cntdot trauma- 1 patient with "excellent" response (Pre-DDAVP Platelet count 249,000/mulfwdarwPost-DDAVP Platelet count 156,000/mul, Pre-DDAVP FVIII 44%fwdarwPost-DDAVP FVIII 184%) cntdot dental prophylaxis-1 patient with "poor" response with fall in platelet count from 84,000/mul Pre-DDAVPfwdarw16,000/mul Post-DDAVP cntdot surgical prophylaxis (given pre-operatively for trans-urethral prostate resection)- "poor" response with continued hematuria necessitating Humate P infusions There were no thrombotic events. Conclusions: As expected, only a small proportion of Type 2B patients have received DDAVP. The clinical response can be adequate but there is a risk of severe thrombocytopenia. However, no thrombotic events were observed. To answer the question of whether certain subsets of Type 2B patients may selectively be given DDAVP safely and effectively based on DDAVP trial results, the baseline platelet level or the specific clinical bleeding situation will necessitate pooling of data worldwide.

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