Machine-learning Based Prediction Model in Primary Immune Thrombocytopenia

• ClinicalTrials.gov Identifier: NCT05116423
• Recruitment Status: Recruiting
• First Posted: November 11, 2021
• Last Update Posted: February 8, 2022
• Sponsor: Peking University People's Hospital
• Collaborators: The Affiliated Zhongshan Hospital of Dalian University; Jiangsu Provincial People's Hospital; Qilu Hospital of Shandong University; Shanghai Zhongshan Hospital
• Information provided by (Responsible Party): Xiao Hui Zhang, Peking University People's Hospital

Study Description

Brief Summary:

This study developed the first prediction model for risk of critical ITP bleeds for ITP inpatients using a novel machine learning algorithm. This model has been implemented as a web-based model so that clinicians can obtain the estimated probability of critical ITP bleeds for ITP inpatients. The objective of this study is to prospectively and externally validate the risk of critical ITP bleeds in newly admitted ITP patients.

Condition or disease
Immune Thrombocytopenia; ITP

Detailed Description:

Primary immune thrombocytopenia (ITP) is a common acquired autoimmune disease characterized by reduced platelet production and increased platelet destruction due to autoimmune disorders, as patients present with low platelet counts and a high risk of bleeding. Although most ITP patients present a good prognosis, the rare but important critical ITP bleeds events are the threatening-life complication to ITP patients, severely affecting their prognosis, quality of life and treatment decisions.

More recently, the development of clinical prediction models has provided powerful tools for precision diagnosis and early intervention of diseases, especially the application of machine learning methods. Machine learning approaches can overcome some of the limitations of current risk prediction analysis methods by applying computer algorithms to large data sets with numerous multidimensional variables, capturing the high-dimensional nonlinear relationships between clinical features to produce data, drive outcome prediction.

It suggests an unmet need for personalized patient management strategies and an urgent need for effective tools to predict the risk of critical ITP bleeds in hospitalized patients in medical practice.

Here, we aim to integrate clinical and laboratory data based on a nationwide multicenter study in China to build a clinical prediction model. In particular, we also perform external and prospective validation with large sample sizes to improve the robustness and utility of our models.

It is a simple and convenient tool to quickly assess newly admitted ITP patients and achieve early identification and intervention for those at high risk of life-threatening bleeding events, thus reducing disability and mortality rates in the future.

Study Design

• Study Type: Observational
• Estimated Enrollment: 100 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Personalized Machine-Learning Based Prediction Model for Bleeding in Immune Thrombocytopenia: a Nationwide Representative Data Study
• Actual Study Start Date: November 10, 2021
• Estimated Primary Completion Date: March 1, 2022
• Estimated Study Completion Date: June 30, 2022

Groups and Cohorts

Group/Cohort
ITP inpatients; The study population included nonsplenectomized primary ITP inpatients 18 years of age or older. Patients who had a diagnosis of connective tissue disease, cancer (solid tumor or leukemia), or primary immune deficiency were excluded.

Outcome Measures

Primary Outcome Measures :

1. Performance of model [ Time Frame: 3 months ]
   Area under receiver operating characteristic curve (AUC) of the model in predicting critical ITP bleeds in patients with ITP.

Secondary Outcome Measures :

1. Comparison between different machine learning algorithms used in the model [ Time Frame: 3 months ]
   Comparison of sensitivity and specificity of different machine learning algorithms used in the model.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Probability Sample

Study Population

The study population included nonsplenectomized primary ITP inpatients 18 years of age or older. Patients who had a diagnosis of connective tissue disease, cancer (solid tumor or leukemia), or primary immune deficiency were excluded.

Criteria

Inclusion Criteria:

1. Confirmed ITP diagnosis;

Exclusion Criteria:

1. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
2. Current HIV infection or hepatitis B virus or hepatitis C virus infections;
3. Maligancy;
4. Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy
5. Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
6. Patients who are deemed unsuitable for the study by the investigator.

Contacts and Locations

Contacts

Contact: Xiao-Hui Zhang, MD; +8615010638916; zhangxh100@sina.com

Contact: Zhuo-Yu An, MD; +8615010638916; anzhuoyu@pku.edu.cn

Locations

China, Beijing

Peking University Insititute of Hematology, Peking University People's Hospital; Recruiting

Beijing, Beijing, China, 100010

Contact: Xiao-Hui Zhang, MD zhangxh100@sina.com

Contact: Zhuo-Yu An, MD 15010638916 anzhuoyu@pku.edu.cn

Sponsors and Collaborators

Peking University People's Hospital

The Affiliated Zhongshan Hospital of Dalian University

Jiangsu Provincial People's Hospital

Qilu Hospital of Shandong University

Shanghai Zhongshan Hospital

Investigators

• Principal Investigator: Xiao-Hui Zhang, MD; Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Collaborative Innovation Center of Hematology

More Information

• Responsible Party: Xiao Hui Zhang, Vice President of Peking University Institute of Hematology, Peking University People's Hospital
• ClinicalTrials.gov Identifier: NCT05116423
• Other Study ID Numbers: PKU-ITP031
• First Posted: November 11, 2021
• Last Update Posted: February 8, 2022
• Last Verified: January 2022

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Xiao Hui Zhang, Peking University People's Hospital:

Immune Thrombocytopenia; Prediction Model

Additional relevant MeSH terms:

Thrombocytopenia; Immune System Diseases; Purpura, Thrombocytopenic, Idiopathic; Blood Platelet Disorders; Hematologic Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Hemorrhagic Disorders; Autoimmune Diseases; Hemorrhage; Pathologic Processes; Skin Manifestations