Center for Postgraduate Education and Training, National Center for Child Health and Development (NCCHD), Tokyo, Japan.
Division of Haematology, NCCHD, Tokyo, Japan.
Br J Haematol. 2024 Jun;204(6):2400-2404. doi: 10.1111/bjh.19484. Epub 2024 Apr 22.
MYH9-related disorder (MYH9-RD) is characterized by congenital macrothrombocytopenia and granulocyte inclusion bodies. MYH9-RD is often misdiagnosed as chronic immune thrombocytopenia. In this study, we investigated age at definitive diagnosis and indicative thrombocytopenia in 41 patients with MYH9-RD from the congenital thrombocytopenia registry in Japan. Our cohort comprises 54.8% adults over 18 years at confirmed diagnosis. We found a significant difference (p < 0.0001) between the median age at definitive diagnosis of 25.0 years and for indicative thrombocytopenia it was 9.0 years. Our findings strongly suggest diagnostic delay of MYH9-RD in Japan. Our registry system will continue to contribute to this issue.
MYH9 相关疾病(MYH9-RD)的特征为先天性巨血小板减少症和粒细胞包涵体。MYH9-RD 常被误诊为慢性免疫性血小板减少症。本研究中,我们调查了日本先天性血小板减少症登记处的 41 名 MYH9-RD 患者的确诊年龄和提示性血小板减少症。我们的队列包括 54.8%的确诊年龄超过 18 岁的成年人。我们发现确诊年龄的中位数为 25.0 岁和提示性血小板减少症的中位数为 9.0 岁之间存在显著差异(p<0.0001)。我们的研究结果强烈表明,日本存在 MYH9-RD 的诊断延迟。我们的登记系统将继续为此问题做出贡献。