Researchers evaluated characteristics of bleeding patterns in infants and toddlers with von Willebrand disease (VWD) and reported their findings in a new study published in the journal Blood Advances.
“Although VWD can be diagnosed at any age, diagnosis at <2 years of age is difficult, because hemostatic challenges are rare, and bruising of extremities and head trauma are common, especially in toddlers as they learn to crawl and walk,” the researchers explained in their report.
The analysis made use of data obtained through the US Hemophilia Treatment Center Network involving patients of less than 2 years of age with VWD. The researchers analyzed details related to birth, diagnosis, bleeding episodes, and patterns of complications.
A total of 105 patients were evaluated for this study, of whom 58% were male. Type 1 VWD was seen in 63% of patients, with type 2 VWD seen in 28% and type 3 in 9%. Examination of birth and delivery data for the patients in this study showed that 86% were delivered at full term, 82% were of normal weight, and 89% were considered to be of normal length. Vaginal delivery was used with 63% of births, although elective cesarean sections were used more often with mothers who were known carriers of VWD (P =.02).
Patients had a mean age at diagnosis of 7 months, with type 2 VWD identified sooner than types 1 and 3 on average (P =.04). Having a family history of VWD was also linked to an earlier diagnosis, occurring approximately 4 months earlier than in those without such a family history (P <.001). Ultimately a family history involving a bleeding disorder was what led to diagnostic testing for 68% of the population.
Most patients (70%) had a bleeding event. The majority of initial bleeding events (68%) were in the first year of life, and 41% were prior to the first 6 months of life. Initial bleeding events occurred in the oral mucosa in 32% of patients, were related to circumcision in 12% of patients, and were intracranial or extracranial bleeds in 10% of patients. Intracranial hemorrhage occurred in approximately 5% of patients, and each of these events was subdural. Sex was not associated with differences in sites of first bleeding events.
Treatment was given to approximately 64% of patients in this study. This involved plasma-derived von Willebrand factor VIII concentrates in nearly half of the patients (47%). Aminocaproic acid was used in 32% of patients, and 14% of patients had intravenous or intranasal desmopressin.
The researchers considered this study to contribute to knowledge around infants and toddlers with VWD, especially elucidating the influence of family history in diagnosis, the greater rate of cesarean sections, the elevated rates of preterm birth and low birth rate, and patterns of bleeding sites in these patients.
Dupervil B, Abe K, O’Brien SH, et al; US Hemophilia Treatment Center Network. Characteristics, complications, and sites of bleeding among infants and toddlers less than 2 years of age with VWD. Blood Adv. 2021;5(8):2079-2086. doi:10.1182/bloodadvances.2020004141