Ruxolitinib in patients with graft versus host disease (GvHD): findings from a compassionate use program

Author:

Pattipaka ThirupathiORCID,Sarp Séverine,Nakhaei Peyman,Güneş SibelORCID

Abstract

AbstractThe ruxolitinib compassionate use (CU) program offered ruxolitinib to patients ≥2 years of age with confirmed steroid-resistant acute or chronic graft-versus-host disease (aGvHD and cGvHD, respectively). Data from 1180 patients (n = 775, 370 and 35 with cGvHD, aGvHD, and non-specified GvHD, respectively) were analyzed. Most patients had severe cGvHD (56%) or stage III/IV aGvHD (70%) disease and had previously received corticosteroids ( > 80%); ruxolitinib was requested primarily as a second-/third-line option. Patients <12 and ≥12 years old most often received the recommended ruxolitinib doses (5 mg twice daily [BID] and 10 mg BID, respectively); however, 23% and 30% of ≥12 year olds with cGvHD and aGvHD, respectively, received the lower dose of 5 mg BID. Notably, corticosteroid usage decreased with ruxolitinib treatment; at the initial ruxolitinib request, 81% and 91% of patients with cGvHD and aGvHD, respectively, were receiving corticosteroids whereas at resupply, 62% and 64%, respectively, were receiving corticosteroids. Eighty two percent of evaluable patients with cGvHD had a complete or partial response to treatment and 56% of evaluable patients with aGvHD had a best response of grade 0/I. These findings demonstrate the rapid and positive effects of ruxolitinib in patients with GvHD in a real-world setting.

Funder

Novartis Pharma AG, Basel, Switzerland

Publisher

Springer Science and Business Media LLC

Reference43 articles.

1. Zeiser R, Blazar BR. Acute Graft-versus-Host Disease - Biologic Process, Prevention, and Therapy. N. Engl J Med. 2017;377:2167–79.

2. Lee SJ, Kim HT, Ho VT, Cutler C, Alyea EP, Soiffer RJ, et al. Quality of life associated with acute and chronic graft-versus-host disease. Bone Marrow Transplant. 2006;38:305–10.

3. Kurosawa S, Oshima K, Yamaguchi T, Yanagisawa A, Fukuda T, Kanamori H, et al. Quality of Life after Allogeneic Hematopoietic Cell Transplantation According to Affected Organ and Severity of Chronic Graft-versus-Host Disease. Biol Blood Marrow Transplant. 2017;23:1749–58.

4. Hamad N, Lachance S, De Courcy J, Rowaichi L, Zuurman M, Mohty M. Patient-Reported Outcomes in Acute Graft-Vs-Host Disease: Quality-Of-Life Findings from a Real-World Study. Bone Marrow Transplant. 2021;56:85–7.

5. Lachance S, Hamad N, De Courcy J, Gibson G, Zuurman M, Mohty M. Impact of Chronic Gvhd Severity and Steroid Response on the Quality of Life in Patients Following Allogeneic Stem Cell Transplantation: Findings from A Real-World Study. Bone Marrow Transplant. 2021;56:83–4.

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3