Abou-Ismail Mouhamed Yazan, Rodgers George M, Bray Paul F, Lim Ming Y
Division of Hematology and Hematologic Malignancies Department of Internal Medicine University of Utah Salt Lake City UT USA.
Res Pract Thromb Haemost. 2021 Feb 17;5(2):356-365. doi: 10.1002/rth2.12481. eCollection 2021 Feb.
Acquired von Willebrand syndrome (AVWS) has been associated with monoclonal gammopathy of undetermined significance (MGUS), with limited data on its management.
We conducted a systematic literature search in Medline (Ovid), Embase, and Scopus up to September 11, 2019, for studies reporting on the management of AVWS associated with MGUS (AVWS-MGUS). Data on patient characteristics, laboratory parameters at presentation, and clinical and laboratory outcomes were extracted.
To describe the clinical presentation and outcomes of different therapeutic approaches.
Seventy-five studies were included in the final review, for a total of 137 patients. Most patients had von Willebrand factor ristocetin cofactor activity <30 IU/dL (86.6%) and factor VIII levels <50 IU/dL (91.8%). Bleeding severity ranged from no bleeding (16.1%) to minor bleeding (46.4%) and major bleeding (37.5%). The overall clinical success rates for 1-deamino-8-D-arginine vasopressin (DDAVP), factor replacement therapy, and intravenous immunoglobulin (IVIG) were 43.8%, 33.3%, and 85.4%, respectively. The laboratory response rates for DDAVP, factor replacement therapy, and IVIG were 39.0%, 62.9%, and 88.6%, respectively. Several other treatments were also reported in small numbers, out of which myeloma-directed therapies, plasma exchange, recombinant factor VIIa, and antifibrinolytics appeared most successful, while immunosuppressive agents were largely ineffective.
IVIG appears to be an effective treatment for AVWS-MGUS bleeding, conferring a high clinical success rate with measurable laboratory outcomes; albeit temporary. DDAVP and factor replacement therapy may be partially successful in controlling minor bleeds, but not major bleeds. Other less commonly used agents may be effective in certain cases, although data are limited.
获得性血管性血友病综合征（AVWS）与意义未明的单克隆丙种球蛋白病（MGUS）相关，但其治疗方面的数据有限。
我们在截至2019年9月11日的Medline（Ovid）、Embase和Scopus中进行了系统的文献检索，以查找关于与MGUS相关的AVWS（AVWS-MGUS）治疗的研究。提取了患者特征、就诊时的实验室参数以及临床和实验室结果的数据。
描述不同治疗方法的临床表现和结果。
最终综述纳入了75项研究，共137例患者。大多数患者的血管性血友病因子瑞斯托霉素辅因子活性<30 IU/dL（86.6%），因子VIII水平<50 IU/dL（91.8%）。出血严重程度从无出血（16.1%）到轻度出血（46.4%）和重度出血（37.5%）不等。1-去氨基-8-D-精氨酸加压素（DDAVP）、因子替代疗法和静脉注射免疫球蛋白（IVIG）的总体临床成功率分别为43.8%、33.3%和85.
