Long-term outcomes with single-agent BRAF inhibitor therapy in Erdheim-Chester disease.

IF 21 1区医学 Q1 HEMATOLOGY

Blood Pub Date : 2025-03-07 DOI:10.1182/blood.2024028032

Gaurav Goyal, Anne S Reiner, Dana Bossert, Allison M Sigler, Mario E Lacouture, Veronica Rotemberg, Jasmine H Francis, Ronald S Go, Raajit K Rampal, Eli L Diamond

引用次数: 0

Abstract

Among 64 patients with Erdheim-Chester disease treated with a BRAF-inhibitor (median follow up 4 years), we found high response rates (85%) but frequent discontinuations (61%), primarily due to adverse events. Additionally, patients experienced persistently poor health-related quality of life.

查看原文本刊更多论文

求助全文

约1分钟内获得全文求助全文

来源期刊

Blood 医学-血液学

CiteScore

23.60

自引率

3.90%

发文量

955

审稿时长

1 months

期刊介绍： Blood, the official journal of the American Society of Hematology, published online and in print, provides an international forum for the publication of original articles describing basic laboratory, translational, and clinical investigations in hematology. Primary research articles will be published under the following scientific categories: Clinical Trials and Observations; Gene Therapy; Hematopoiesis and Stem Cells; Immunobiology and Immunotherapy scope; Myeloid Neoplasia; Lymphoid Neoplasia; Phagocytes, Granulocytes and Myelopoiesis; Platelets and Thrombopoiesis; Red Cells, Iron and Erythropoiesis; Thrombosis and Hemostasis; Transfusion Medicine; Transplantation; and Vascular Biology. Papers can be listed under more than one category as appropriate.