Patient Quality of Life (QOL) In Nonsplenectomized Immune Thrombocytopenia (ITP) Patients Receiving Romiplostim or Medical Standard of Care (SOC)

Author:

Rummel Mathias J.1,Kuter David J.2,Mandanas Romeo3,Giagounidis Aristoteles4,Wang Xuena5,Mathias Susan D.6,Deuson Robert5

Affiliation:

1. Klinikum der Justus-Liebig-Universität, Giessen, Germany,

2. Massachusetts General Hospital, Boston, MA, USA,

3. Integris Cancer Institute of Oklahoma, Oklahoma City, OK, USA,

4. Med. Klinik 2, St. Johannes Hospital, Duisburg, Germany,

5. Amgen Inc., Thousand Oaks, CA, USA,

6. Health Outcomes Solutions, Winter Park, FL, USA

Abstract

Abstract Abstract 569 Introduction: Romiplostim, a novel peptibody that increases platelet production, is approved for the treatment of adult chronic ITP. Positive results from a previous study suggest the potential that romiplostim could improve QOL (Kuter, ASH 2009, #679). Immunosuppressive therapies for ITP may adversely affect patient QOL. We compared QOL between SOC- and romiplostim-treated pts from this study, and examined changes among subgroups of pts. Methods: This was an open-label study of nonsplenectomized ITP pts who were randomized to receive either once-weekly subcutaneous romiplostim or SOC. SOC treatments were prescribed according to standard institutional practices or therapeutic guidelines. QOL was assessed using the ITP patient assessment questionnaire (ITP-PAQ; consisting of 10 scales scored from 0–100); assessments were taken at baseline and every 12 weeks to week 52. Scores were also assessed for subgroups of pts with the following favorable outcomes: (1) did not receive blood transfusions or rescue medications, (2) did not experience bleeding ≥ grade 2, (3) did not experience a platelet count < 20 × 109/L. Mean and change from baseline scores were computed, and differences between treatment groups and among subgroups were assessed. Clinical significance was determined from the minimal important difference (MID), which is the smallest difference in QOL considered clinically meaningful (Mathias et al., CMRO 375-83) and corresponds to an 8–15 point improvement depending on the scale. The time to MID was also computed for Symptoms, Bother, Activity and Fatigue. Results: In total, 157 pts were randomized to receive romiplostim and 77 to receive SOC. At baseline, no statistically significant differences were found between the romiplostim and the SOC group on any of the scales. At 52-weeks, change scores for both the romiplostim group and the SOC group showed improvements that exceeded the MID with the exception of Fatigue in both arms and Activity in the SOC arm (Table). In comparison to the SOC group, the romiplostim group showed statistically significantly greater improvements from baseline for all scales except Fatigue. The differences between treatment groups did not exceed the MID for any of the scales. The time to MID was significantly shorter for the romiplostim group vs the SOC group on the Symptoms and Bother (p<0.0001), and Fatigue (p=0.0068) scales. Among subgroups with favorable clinical outcomes, statistically significantly greater improvements were seen in the romiplostim group compared with the SOC group in many of the scales. The difference between treatment groups exceeded the MID only once: romiplostim-treated pts in the subgroup who did not experience platelet counts < 20 × 109/L had a 12.6 point greater improvement in Activity score compared to SOC pts. Conclusions: Nonsplenectomized ITP pts receiving romiplostim had greater improvements in QOL than pts receiving SOC. These improvements were maintained even among subgroups of pts with favorable clinical outcomes. However, the open-label study design limits our ability to make conclusions, and the clinical significance of the QOL improvements in romiplostim-treated pts relative to those receiving SOC remains uncertain. Disclosures: Kuter: Amgen Inc.: Consultancy, Research Funding; GlaxoSmithKline: Consultancy, Research Funding; ONO: Consultancy; Pfizer: Consultancy, Research Funding; Shionogi: Consultancy, Research Funding. Mandanas:Celgene: Honoraria; Genentech/Roche: Honoraria; Amgen Inc.: Honoraria; GlaxoSmithKline: Speakers Bureau; Pfizer: Speakers Bureau; Genomic Health: Speakers Bureau. Giagounidis:Celgene: Consultancy, Honoraria. Wang:Amgen Inc.: Employment, Equity Ownership. Mathias:Amgen Inc.: Consultancy. Deuson:Amgen Inc.: Employment, Equity Ownership.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Cited by 1 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

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