Retrospective Chart Review Screening Algorithm to Assess the Prevalence of PNH-clones

NCT05884060 | Completed DMC FDA Drug

• 1 other identifier: B1172022000017
• Study Type: observational
• Target: 568
• Locations: 1 country
• Sites: 1

Brief Summary

The present study is a non-interventional retrospective chart review study assessing the prevalence of PNH-clones in patients with PNH risk-factors aged ≥14 years and treated at our hospital. The objective of this study is to develop a PNH screening tool on the hospital Electronic Health Record (EHR) system. An algorithm defining PNH risk groups is developed.

Conditions

PNH; Thrombosis

Keywords

PNH; Thrombosis; anemia; aplastic anemia; myelodysplastic syndrome (MDS)

Outcome Measures

Primary Outcomes (2)

• PNH risk factors translation into query codes that can be interpreted by a computer system for analysis

  Three main PNH risk sub-cohorts were constructed, representing patients exhibiting PNH risk factors such as hemolytic anemia (group 1), bone marrow dysfunction (group 2) and thrombosis (group 3). To build these sub-cohorts, queries were created using a combination of structured and unstructured electronic health record (EHR) data, including lab results, diagnoses, medication, questionnaire data, text from medical and radiology reports, notes, and Internation Classification Codes (ICD-10). These sub-cohorts were validated by two hematologists who reviewed randomly selected patients, resulting in several iterations and query optimizations.

  2022

• Number of patients identified with high risk for PNH clone and per type of screening criteria by developing a computational screening algorithm

  A computational algorithm was employed for a retrospective EHR analysis, to identify high-risk cohorts of potential PNH patients who need treatment from all registered patients, with maximum ability to find relevant cases. Three main PNH risk sub-cohorts were constructed, representing patients exhibiting PNH risk factors such as hemolytic anemia (group 1), bone marrow dysfunction (group 2) and thrombosis (group 3). These sub-cohorts were validated by two hematologists who reviewed randomly selected patients, resulting in several iterations and query optimizations. Sub-cohorts were subsequently merged and refined into high risk cohorts that undergo further analysis and manual review. Two hematologists independently reviewed and rated medical records to achieve a manual risk stratification of the high risk cohorts.

  2022

Secondary Outcomes (1)

• The number of patients at high risk for PNH, categorized by risk factor, across each medical department

  2023

Study Arms (4)

group 1

patients with evidence of haemolysis without obvious cause

Other: No intervention

group 2

patients with evidence of bone marrow dysfunction (AA, MDS, unexplained cytopenia)

Other: No intervention

group 3

patients with thrombosis

Other: No intervention

group 4

patient group that needs to be eliminated from final high risk cohort: patients with cirrhosis, patients wit septic embolisms \& embolisation

Other: No intervention

Interventions

No intervention OTHER

No interventions

group 1; group 2; group 3; group 4

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Eligible patients will be identified by retrospective screening of the hospital's electronic patients records using data search queries based on ICD-10 codes, lab results, keyword search in questionnaires, medical reports, notes, radiology reports \& medical diagnoses corresponding to one of the PNH Risk factors described: 1. Patients with evidence of haemolysis without obvious cause 2. Patients with evidence of bone marrow dysfunction 3. Patients with thrombosis

You may qualify if:

• Patient ≥14 years of age
• At least 1 record encoded in the General Hospital Delta (AZ Delta) patient database (HiX) between 20 April 2018 and 1 March 2022
• A history or presence of at least one PNH risk factors identified following retrospective screening of electronic patient records

You may not qualify if:

• None

Sponsors & Collaborators

• AZ Delta: lead
• Alexion Pharmaceuticals, Inc.: collaborator

Study Sites (1)

AZ Delta

Roeselare, West-Vlaanderen, 8800, Belgium

Location

MeSH Terms

Conditions

Thrombosis; Anemia; Anemia, Aplastic; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Embolism and Thrombosis; Vascular Diseases; Cardiovascular Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Dries Deeren, MD

  AZ Delta

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2023
• First Posted: June 1, 2023
• Study Start: April 20, 2018
• Primary Completion: March 1, 2022
• Study Completion: March 1, 2022
• Last Updated: June 2, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1)