Treatment of acquired von Willebrand syndrome in childhood

Abstract

A 3-1/2-year-old male with no personal or family history of bleeding disorders presented with abdominal distension, epistaxis, and anemia (hemoglobin 8.2 g/dL). A magnetic resonance imaging scan of the abdomen demonstrated a mass arising from the left kidney. Preoperative laboratory studies revealed a prolonged activated partial thromboplastin time of 49.2 seconds, a normal prothrombin time of 12.4 seconds, and a platelet count of 230 000/mL. Further testing revealed factor VIII (FVIII) activity of 16%, factor IX (FIX) activity of 74%, von Willebrand factor (VWF) activity of 12%, VWF antigen activity of 31%, and decreased high-molecular-weight VWF multimers consistent with acquired von

Acquired von Willebrand syndrome: von Willebrand factor propeptide to von Willebrand factor antigen ratio predicts remission status

“…In certain anti-VWF-mediated AVWS cases, DDAVP was able to increase VWFpp levels by 5-to sevenfold, 12,15 so that DDAVP may be used therapeutically. 16,17 In our patient, there was no VWF:Ag increment and only a slight increase in VWFpp (1.33-fold) after DDAVP administration. The lower VWFpp at RL1 and less-than-expected increase in VWFpp in response to DDAVP may potentially be a result of an exhausted VWF/VWFpp pool resulting from chronic low-grade endothelial activation secondary to For personal use only.…”

An 11‐year experience of acquired von Willebrand syndrome in children diagnosed with Wilms tumour in a tertiary referral centre

“…Except in case 5, where the results were only marginally abnormal, all children received haemostatic cover for interventional procedures. Desmopressin acetate (DDAVP) was not used as previously described . This was due to both the age of the patients and potential for hyponatraemic convulsions and concerns regarding reliable response.…”

“…Acquired von Willebrand syndrome (avWS) is a rare bleeding disorder that results from structural and functional abnormalities of the von Willebrand factor (vWF) antigen . It has been described in the context of autoimmune disease, structural cardiac abnormalities, hypothyroidism and malignancy, including Wilms tumour (WT) .…”

“…Acquired von Willebrand syndrome (avWS) is a rare bleeding disorder that results from structural and functional abnormalities of the von Willebrand factor (vWF) antigen . It has been described in the context of autoimmune disease, structural cardiac abnormalities, hypothyroidism and malignancy, including Wilms tumour (WT) . The underlying pathophysiology differs in these patient cohorts and includes reduced synthesis of the antigen, increased proteolysis of high molecular weight multimers, autoantibody‐mediated clearance, and adsorption on to tumour cells …”

Perioperative intravenous immunoglobulin treatment in a patient with severe acquired von Willebrand syndrome: case report and review of the literature

“…Due to the heterogeneous etiology, as well as the limited number of cases and consequent absence of prospective studies, a standard medical procedure for the treatment and prevention of bleeding complications in AVWS has not yet been developed . Treatment options for AVWS include desmopressin (preferred), factor VIII:C/VWF substitution, and activated factor VII (eptacog alfa) for acute bleeding, as well as the application of intravenous immunoglobulins (IVIG) typically acting within a few days . Furthermore, long‐term treatment includes plasmapheresis, the use of immunosuppressants and antifibrinolytics in monotherapy or in combination.…”