Life after Recovery in Adult Thrombotic Thrombocytopenic Purpura (TTP): Let's Hear It from the Survivors

成人血栓性血小板减少性紫癜 (TTP) 康复后的生活：让我们听听幸存者的心声

基本信息

批准号：
10438374
负责人：
Deirdra R Terrell
金额：
$ 3.8万
依托单位：
UNIVERSITY OF OKLAHOMA HLTH SCIENCES CTR
依托单位国家：
美国
项目类别：
财政年份：
2017
资助国家：
美国
起止时间：
2017-04-18 至 2023-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10438374
关键词：
18 year old Acute Address Adult Affect Area Assessment tool Autoimmune Child Clinical Cognition Development Plans Disease Disease remission Epidemiologist Epidemiology Fatigue Focus Groups Foundations Funding Future General Population Geography Goals Guidelines Health Health Sciences Hearing Hematologist Impaired cognition Impairment Individual Information Systems Interview Knowledge Lead Life Life Expectancy Long-Term Care Major Depressive Disorder Measurement Measures Mental Depression Methodology Methods Minor Minority Morbidity - disease rate Ohio Oklahoma Outcome Outcome Assessment Paper Participant Patient Outcomes Assessments Patient Preferences Patient-Focused Outcomes Patients Plasma Exchange Population Prevalence Provider Publishing Qualitative Methods Questionnaires Rare Diseases Recovery Registries Reporting Research Residual state Sampling Science Severities Support Groups Survivors Symptoms Testing Thrombosis Thrombotic Thrombocytopenic Purpura Training Training Activity United States United States National Institutes of Health Universities Woman base black women career career development child bearing clinical care clinical decision-making clinical remission clinically significant computerized dissemination research experience health related quality of life implementation barriers implementation research insight instrument professor provider-level barriers recruit relapse risk routine care theories thrombotic

项目摘要

PROJECT SUMMARY/ABSTRACT. Acquired autoimmune thrombotic thrombocytopenic purpura (TTP) is a rare disorder characterized by acute episodes of systemic microvascular thrombosis. TTP is more common in women, occurs during the child-bearing years, and rarely occurs in children <18 years of age. With early recognition and plasma exchange treatment, 78-90% of patients will survive their acute episode. Long-term adverse health outcomes in adults following recovery from TTP are under recognized. We have documented in the Oklahoma TTP Registry that survivors have an increased prevalence of major depression, decreased health-related quality of life, minor cognitive impairment, and a decreased life expectancy compared to the general population. However, current guidelines on the management of TTP focus on the long-term risk for relapse with no mention of patient-reported outcomes (PROs). We propose to utilize rigorous qualitative methods to determine from survivors the most important long-term outcomes that impact their daily routines during remission (Aim 1), which fills an existing gap by identifying specific PROMIS® PROs that should be evaluated following recovery. Currently, there are no published studies on PROMIS® instruments or the preferred mode of administration in TTP patients; therefore, we will determine the feasibility of administering PROMIS® instruments online in a TTP population, including recruitment of TTP patients from the United States Thrombotic Microangiopathy Consortium (Aim 2). Understanding the TTP patient preference will assist in integrating these PROMIS® instruments into routine clinical care. Finally, although it is becoming more common to integrate PRO assessments in routine clinical care, a barrier to successful integration is that clinicians are often unsure how to interpret PRO results in a clinically-meaningful way. Clinical severity cut-points such as `mild', `moderate' or `severe' may be more easily understood in a clinical setting than a comparison to a normative population. We propose to create clinical threshold levels for PROMIS® scores (Aim 3) which fills an existing gap by reducing an implementation barrier for hematologists using PROMIS® assessments to manage long-term outcomes. The rationale for the proposed study is that it is expected to yield important new insights into impairments during clinical remission of TTP and extend clinical interpretability of PROMIS® measures to include a new disease area (TTP). Dr. Terrell is an Assistant Professor of Epidemiology at the University of Oklahoma Health Sciences Center. Her additional training proposed under the current career development plan will enhance her ability to become an independent NIH-funded clinical epidemiologist with training and experience in qualitative methodology and the theory and application of the measurement science of PROs.

项目摘要/摘要获得性自身免疫性血栓性血小板减少性紫癜 (TTP) 是一种以全身微血管血栓形成急性发作为特征的罕见疾病在 TTP 中更为常见。女性，发生在育龄期，很少发生在<18岁的儿童中。通过识别和血浆置换治疗，78-90% 的患者可以在急性发作后存活下来。我们对 TTP 恢复后成人的不良健康结果的认识不足。俄克拉荷马州 TTP 登记处显示，幸存者的重度抑郁症患病率增加，与健康相关的生活质量、轻微的认知障碍以及与正常人相比预期寿命的缩短然而，目前 TTP 管理指南重点关注的是 TTP 的长期风险。复发，但没有提及患者报告的结果（PRO）。我们建议利用严格的定性方法来确定幸存者最重要的长期目标影响缓解期间日常生活的结果（目标 1），通过识别来填补现有空白恢复后应评估的具体 PROMIS® PRO 目前尚未发表研究。因此，我们将确定 PROMIS® 仪器或 TTP 患者的首选给药方式；在 TTP 人群中在线管理 PROMIS® 仪器的可行性，包括招募 TTP 来自美国血栓性微血管病联盟的患者（目标 2）。患者偏好将有助于将这些 PROMIS® 仪器整合到常规临床护理中。尽管将 PRO 评估纳入常规临床护理变得越来越普遍，但成功整合的关键在于，忠诚者常常不确定如何以具有临床意义的方式解释 PRO 结果诸如“轻度”、“中度”或“重度”之类的临床严重程度分界点可能更容易理解。我们建议为临床环境创建临床阈值水平，而不是与正常人群进行比较。 PROMIS® 评分（目标 3）通过减少血液学家的实施障碍来填补现有空白使用 PROMIS® 评估来管理长期结果拟议研究的基本原理是：预计将对 TTP 临床缓解期间的损伤产生重要的新见解，并扩展临床 PROMIS® 测量的可解释性包括新的疾病领域 (TTP)。特雷尔博士是俄克拉荷马大学健康科学中心流行病学助理教授。她根据当前职业发展计划提出的额外培训将增强她成为一位由 NIH 资助的独立临床流行病学家，在定性方法和方法方面接受过培训并具有经验 PRO测量科学的理论与应用。

项目成果

期刊论文数量（21）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

Management of antithrombotic therapy in adults with immune thrombocytopenia (ITP): a survey of ITP specialists and general hematologist-oncologists.

成人免疫性血小板减少症 (ITP) 的抗血栓治疗管理：对 ITP 专家和普通血液肿瘤学家的调查。

DOI：
发表时间：
2018-07
期刊：
影响因子：
4
作者：
Pishko, Allyson M;Misgav, Mudi;Cuker, Adam;Cines, Douglas B;George, James N;Vesely, Sara K;Terrell, Deirdra R
通讯作者：
Terrell, Deirdra R

Thrombotic thrombocytopenic purpura patients' attitudes toward depression management: A qualitative study.

血栓性血小板减少性紫癜患者对抑郁症治疗的态度：一项定性研究。

DOI：
发表时间：
2019-10
期刊：
影响因子：
2
作者：
Terrell, Deirdra R;Tolma, Eleni L;Stewart, Lauren M;Shirley, Erin A
通讯作者：
Shirley, Erin A

Health following recovery from immune thrombotic thrombocytopenic purpura: the patient's perspective.

免疫性血栓性血小板减少性紫癜恢复后的健康状况：患者的观点。

DOI：
发表时间：
2023-05-09
期刊：
影响因子：
7.5
作者：
Kelley, Rachel A;Cheney, Marshall K;Martin, Clare M;Cataland, Spero;Quick, Lauren B;Keller, San;Vesely, Sara K;Llaneza, Amanda J;Khawandanah, Mohamad O;Journeycake, Janna M;Panepinto, Julie A;Terrell, Deirdra R
通讯作者：
Terrell, Deirdra R

The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: key findings at enrollment until 2017.

国际遗传性血栓性血小板减少性紫癜登记处：截至 2017 年登记时的主要发现。

DOI：
发表时间：
2019
期刊：
影响因子：
10.1
作者：
van Dorland, Hendrika A;Taleghani, Magnus Mansouri;Sakai, Kazuya;Friedman, Kenneth D;George, James N;Hrachovinova, Ingrid;Knöbl, Paul N;von Krogh, Anne Sophie;Schneppenheim, Reinhard;Aebi;Bütikofer, Lukas;Largiadèr, Carlo R;Cer
通讯作者：
Cer

The Prevalence of Pelvic Floor Hematoma After Vaginal Delivery.

阴道分娩后盆底血肿的患病率。

DOI：
发表时间：
2021
期刊：
影响因子：
0
作者：
Denson, Lindsay E;Terrell, Deirdra R;Vesely, Sara K;Peck, Jennifer D;Quiroz, Lieschen H;Shobeiri, S Abbas
通讯作者：
Shobeiri, S Abbas