Cognitive Status, Fatigue and Inflammation in Patients With Immune Thrombocytopenia (ITP)

Cognitive Status, Fatigue, and Inflammation in Patients With Immune Thrombocytopenic Purpura (ITP)

This is a multi-center, hospital based, cross-sectional study based on data from patient medical records, including laboratory results. The study will include adult patients with chronic (> 1 year duration) primary Immune Thrombocytopenia (ITP) only. Patients will be identified based on a laboratory confirmed diagnosis of ITP in the medical records, where there is also a physician-confirmed diagnosis of primary ITP. If patients consent to take part in the study, a routine clinical visit will also serve as the study visit. Patients who do not have a routine visit scheduled during the study period but who would otherwise be eligible may be contacted by the site. In such cases, clinicians may use their discretion to schedule a visit specifically for study purposes.

All study participants will have a routine blood test as part of their standard clinical care at the study visit, and this same procedure will be used to collect supplementary blood samples to assess a variety of biomarkers.

Data will be collected using a combination of chart review, clinical outcome assessment administration, and laboratory results through blood tests.

Study Overview

• Status: Completed
• Conditions: Primary Immune Thrombocytopenia; Chronic Immune Thrombocytopenia; Adult Immune Thrombocytopenia
• Intervention / Treatment: Other: No intervention

• Study Type: Observational
• Enrollment (Actual): 97

Contacts and Locations

Study Locations

• United Kingdom
  • London, City of
    • London, London, City of, United Kingdom, W12 0HS
      • Investigational Site Number: 8260001
• United States
  • California
    • Los Angeles, California, United States, 90033
      • USC Norris Comprehensive Cancer Center- Site Number : 8400002
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital- Site Number : 8400001
  • Washington
    • Seattle, Washington, United States, 98109
      • University of Washington-Fred Hutchinson Cancer Center_Site Number: 8400003

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

The source population for this study will be patients treated for primary chronic Immune Thrombocytopenia at one of the four study centers in two countries (3 centers in the USA, 1 center in the UK).

Description

Inclusion Criteria:

• Patient diagnosed with chronic (ITP with > 1 year duration) primary ITP living in the USA or the UK:

  • On ITP pharmacologic treatment, , including on-demand treatment strategy, regardless of last platelet count (below or above 100 x 10^9 counts/L);
  • Patients with their most recent platelet count >50 x 109 counts/L) and without any ITP-specific treatment for 1 year or less.
• Adult patient who are 18 years or older at index date

• Patient who has received at least one initial first line ITP therapy (CS/ IVIg/ANTI d) with initial response as of index date and indicated by one of the following:

  • Platelet count ≥ 50 x 10^9 counts /L; OR
  • Platelet count ≥ 30 x 10^9 counts /L with at least a two-fold increase from baseline within 1 month since start of treatment, as per ASH 2019 guidelines; OR
  • A medical record of initial response as determined by clinical judgement, considering documented platelet count along with overall patient condition and relevant medical factors.
• Patients whose medical history related to the study objectives are available from the site covering 12 months prior to index date.
• Patient provided informed consent to participate in the study.

Exclusion Criteria:

• Secondary ITP.
• Patients with their most recent platelet count >50 x 10^9 counts/L and without any ITP-specific treatment for more than 1 year.
• Patients with recent or active infection recorded 14 days or less before index date; patients with uncontrolled chronic infections or who were recently diagnosed with a chronic infection (≤14 days of index date).
• Patients with vaccination in 28 days before index date.
• Patients treated with rilzabrutinib on or before index date.
• Patients who participated in an interventional clinical trial for any investigational drug in the 180 days prior to index date.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Prevalence of impaired cognitive status among adult Immune Thrombocytopenia patients	Impaired cognitive status is defined as a CBB (CogState Brief Battery) z-score of -1 or less on at least one of 2 composite domains (attention/psychomotor composite and memory/learning composite domains). The CBB is a computerized outcome measure of cognitive function in human subjects aged 6 to 99 years and has been previously used in ITP. The CBB test for psychomotor function, attention, visual learning and working memory.	Day 1 (Index date)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Age, sex, ethnicity, educational attainment		Day 1 (Index date)
Smoking, alcohol, marijuana, cocaine use		Day 1 (Index date)
Comorbidities at index categorical		Day 1 (Index date)
Current medications	ITP-specific treatments, antiplatelet,anticoagulant, psychiatric and pain medications	Day 1 (Index date)
ITP-PAQ	A validated, disease-specifictool to measure health-related quality of life HRQoL in adult ITP patients	Day 1 (Index date)
ITP Bleeding Scale (IBLS)		Day 1 (Index date)
Laboratory characteristics	Complete Blood Count	Day 1 (Index date)
Assessment of key inflammatory biomarkers	Transferrin, Ferritin, Iron, Prothrombin fragment, Fibrinogen, D dimer, Interleukin (IL)-1 beta, IL-6, IL-10, IL-18, TNF alpha, Gamma Interferon, high-sensitivity C-reactive protein (hsCRP), MCP1, NETosis markers (cell-free deoxyribonucleic acid (DNA), neutrophil elastase (NE), myeloperoxidase (MPO)), antinuclear antibodies (ANA), Lupus anticoagulant, Anticardiolipin antibodies (ACA), thrombopoietin level, soluble P-selectin, L-selectin, ICAM-1; whole blood RNA sequencing (transcriptome), C1q, sC5b-9	Day 1 (Index date)
FACIT (Functional Assessment of Chronic Illness Therapy) - Fatigue score	The FACIT-Fatigue is a 13-item tool that measures self-reported fatigue and its impact on daily life. Each question is rated by the participant on a Likert-like scale of 0 ('not at all') to 4 ('very much'). A higher FACIT-Fatigue score represents greater quality of life (QoL).	Day 1 (Index date)
Medical history	Family history of dementia, family history of cognitive impairment, ITP medication and treatment history, age of ITP diagnosis, duration of ITP, history of clinically relevant or severe bleeding complications, history of thromboembolism complications, history of brain injury or neurological sequalae, history of COVID-19, brain imaging results, platelet counts results	Day 1 (Index date)

Collaborators and Investigators

• Sponsor: Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): January 13, 2025
• Primary Completion (Actual): November 26, 2025
• Study Completion (Actual): November 26, 2025

Study Registration Dates

• First Submitted: October 10, 2024
• First Submitted That Met QC Criteria: October 29, 2024
• First Posted (Actual): October 30, 2024

Study Record Updates

• Last Update Posted (Actual): January 26, 2026
• Last Update Submitted That Met QC Criteria: January 23, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: OBS18052; U1111-1299-2041 (Other Identifier: ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No