Blood Res.  2023 Mar;58(1):42-50. 10.5045/br.2023.2022218.

Acquired von willebrand syndrome in patients with Philadelphia-negative myeloproliferative neoplasm

• Song IC
• Kang S
• Lee MW
• Ryu H
• Lee HJ
• Yun HJ
• Jo DY

• KMID: 2541064
• DOI: http://doi.org/10.5045/br.2023.2022218

Abstract

Background
Acquired von Willebrand syndrome (AVWS) has not been investigated in Korean patients with Philadelphia chromosome-negative myeloproliferative neoplasm.
Methods
This study analyzed the prevalence at diagnosis and clinical features of AVWS in patients with essential thrombocythemia (ET), polycythemia vera (PV), prefibrotic/early primary myelofibrosis (pre-PMF), or overt PMF (PMF) diagnosed between January 2019 and December 2021 at Chungam National University Hospital, Daejeon, Korea. AVWS was defined as below the lower reference limit (56%) of ristocetin cofactor activity (VWF:RCo).
Results
Sixty-four consecutive patients (36 with ET, 17 with PV, 6 with pre-PMF, and 5 with PMF; 30 men and 34 women) with a median age of 67 years (range, 18‒87 yr) were followed for a median of 25.1 months (range, 2.6‒46.4 mo). AVWS was detected in 20 (31.3%) patients at diagnosis and was most frequent in ET patients (41.4%), followed by patients with pre-PMF (33.3%) and PV (17.6%) patients. VWF:RCo was negatively correlated with the platelet count (r=0.937; P =0.002). Only one episode of minor bleeding occurred in a patient with ET and AVWS. Younger age (＜50 yr) [odds ratio (OR), 7.08; 95% confidence interval (CI), 1.27‒39.48; P =0.026] and thrombocytosis (＞600×10 ⁹ /L) (OR, 13.70; 95% CI, 1.35‒138.17; P =0.026) were independent risk factors for developing AVWS.
Conclusion
AVWS based on VWF:RCo was common in patients with ET and pre-PMF, but less common in patients with PV in the Korean population. Clinically significant bleeding is rare in these patients.

Keyword

Myeloproliferative neoplasm; Essential thrombocythemia; Polycythemia vera; Primary myelofibrosis; Acquired von Willebrand syndrome

Figure

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  Fig. 1 Correlation between von Willebrand factor ristocetin cofactor activity (VWF:RCo) and platelet count.

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  Fig. 2 Representative changes with time in platelet count and von Willebrand factor ristocetin cofactor activity (VWF:RCo) following hydroxyurea treatment in a patient with essential thrombocythemia.

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  Fig. 3 Changes in platelet count after normalization of von Willebrand factor ristocetin cofactor activity following cytoreductive treatment in patients with acquired von Willebrand syndrome.

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