Nov 23, 2021

Historically, data on infants and toddlers (ITs) with von Willebrand disease (VWD), particularly relevant to bleeding patterns, has been lacking. To address this absence of data, a team of researchers from the U.S. Hemophilia Treatment Center Network (USHTCN) and the Centers for Disease Control and Prevention conducted retrospective analysis of HTC patients with VWD who are less than two years of age. The data were obtained through the USHTCN. The results were published in the journal Blood Advances.

A total of 105 VWD patients two years of age or less were ultimately included in the analysis: of those, 63% were type I VWD, 28% were type II VWD, and 9% were type III VWD. Investigators focused primarily on birth characteristics, bleeding episodes, and complications.

A review of birth and delivery data showed that 86% were delivered at full term, 82% were of normal weight, and 89% were considered to be of normal length. 63% of the births were done vaginally, while elective cesarean sections were utilized more often with mothers who were known carriers of VWD.

An examination of diagnosis data showed that patients with type 2 VWD were identified sooner, on average, than types 1 and 3. Patients had a mean age at diagnosis of seven months, with little variation by sex. A family history of VWD was also associated with an earlier diagnosis, occurring approximately four months earlier than in those without such a history. In all, family history of a bleeding disorder prompted diagnostic testing for 68% of this population.

The majority of the patients (70%) experienced a bleeding event, with 68% of those having their initial bleed in the first year of life. Initial bleeds most often occurred in the oral mucosa (mucous membrane lining the inside of the mouth), with 32% of patients experiencing this symptom.

The second and third most common bleeds were circumcision-related (12%) and intracranial/extracranial bleeding (10%), respectively. Approximately 5% of patients suffered an intracranial hemorrhage, though none was associated with delivery at birth. Treatment with bleeding disorder therapies was utilized in approximately 64% of the patients in the study, with nearly half (47%) of these receiving plasma-derived von Willebrand factor VIII concentrates. Aminocaproic acid was used in 32% of patients, while 14% received intravenous or intranasal desmopressin.

The authors highlight the central role HTCs play in mitigating risk and ensuring the best possible outcomes for these patients.

“Other studies have recommended a multidisciplinary care approach to provide early diagnosis and optimal care for this population. Specialized HTCs are uniquely positioned to offer such multidisciplinary care, including genetic counselors throughout the prepartum period who work to increase expectant mothers’ understanding of the risks associated with having a child with VWD, and adult and pediatric hematologists, obstetrician-gynecologists, genetic counselors, nurses, and social workers throughout the pre- and postpartum period who seek to optimize outcomes and disease management,” conclude the authors.

Source: Hematology Advisor, October 4, 2021

Stay connected with us! Subscribe to receive the latest news on treatments, clinical trials, advocacy issues, and NHF events.