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Bleeding symptoms in patients diagnosed as type 3 von Willebrand disease: Results from 3WINTERS‐IPS, an international and collaborative cross‐sectional study
Author(s) -
Tosetto Alberto,
Badiee Zahra,
Baghaipour MohammadReza,
Baronciani Luciano,
Battle Javier,
Berntorp Erik,
Bodó Imre,
Budde Ulrich,
Castaman Giancarlo,
Eikenboom Jeroen C. J.,
Eshghi Peyman,
Ettorre Cosimo,
Goodeve Anne,
Goudemand Jenny,
Hay Charles Richard Morris,
Hoorfar Hamid,
Karimi Mehran,
Keikhaei Bijan,
Lassila Riitta,
Leebeek Frank W. G.,
Lopez Fernandez Maria Fernanda,
Mannucci Pier Mannuccio,
Mazzucconi Maria Gabriella,
Morfini Massimo,
Oldenburg Johannes,
Peake Ian,
Parra Lòpez Rafael,
Peyvandi Flora,
Schneppenheim Reinhard,
Tiede Andreas,
Toogeh Gholamreza,
Trossaert Marc,
Zekavat Omidreza,
Zetterberg Eva M. K.,
Federici Augusto B.
Publication year - 2020
Publication title -
journal of thrombosis and haemostasis
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.947
H-Index - 178
eISSN - 1538-7836
pISSN - 1538-7933
DOI - 10.1111/jth.14886
Subject(s) - von willebrand disease , medicine , cross sectional study , von willebrand factor , gastroenterology , pathology , platelet
Background Type 3 von Willebrand's disease (VWD) patients present markedly reduced levels of von Willebrand factor and factor VIII. Because of its rarity, the bleeding phenotype of type 3 VWD is poorly described, as compared to type 1 VWD. Aims To evaluate the frequency and the severity of bleeding symptoms across age and sex groups in type 3 patients and to compare these with those observed in type 1 VWD patients to investigate any possible clustering of bleeding symptoms within type 3 patients. Methods We compared the bleeding phenotype and computed the bleeding score (BS) using the MCMDM‐1VWD bleeding questionnaire in patients enrolled in the 3WINTERS‐IPS and MCMDM‐1VWD studies. Results In 223 unrelated type 3 VWD patients, both the BS and the number of clinically relevant bleeding symptoms were increased in type 3 as compared to type 1 VWD patients (15 versus 6 and 5 versus 3). Intracranial bleeding, oral cavity, hemarthroses, and deep hematomas were at least five‐fold over‐represented in type 3 VWD. A more severe bleeding phenotype was evident in patients having von Willebrand factor antigen levels < 20 IU/dL at diagnosis in the two merged cohorts. In type 3 patients, there was an apparent clustering of hemarthrosis with gastrointestinal bleeding and epistaxis, whereas bleeding after surgery or tooth extraction clusters with oral bleeding and menorrhagia. Conclusions In the largest cohort of type 3 VWD patients, we were able to describe a distinct clinical phenotype that is associated with the presence of a more severe hemostatic defect.