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

Completed
Conditions
PNH
Thrombosis
Interventions
Other: No intervention
Registration Number
NCT05884060
Lead Sponsor
AZ Delta
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.

Detailed Description

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a life-threatening hematological disorder, but with an effective therapy. Prevalence is estimated between 1-5 per million people, often manifested by cardiovascular, gastrointestinal, neurological or haematological symptoms. Referral is therefore typically to several specialists, resulting in PNH underdiagnosis.

This chart review study consists primarily of developing an algorithm to identify a high-risk cohort of potential PNH patients who need treatment from all registered patients, with maximum ability to find relevant cases. Secondly, this cohort will be manually reviewed by clinicians for final screening. The challenge hence is maximizing the ability to find all relevant PNH patients yet limiting the number to ensure manual review is possible.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
568
Inclusion Criteria
  • 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
Exclusion Criteria
  • None

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
group 1No interventionpatients with evidence of haemolysis without obvious cause
group 2No interventionpatients with evidence of bone marrow dysfunction (AA, MDS, unexplained cytopenia)
group 3No interventionpatients with thrombosis
group 4No interventionpatient group that needs to be eliminated from final high risk cohort: patients with cirrhosis, patients wit septic embolisms \& embolisation
Primary Outcome Measures
NameTimeMethod
PNH risk factors translation into query codes that can be interpreted by a computer system for analysis2022

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.

Number of patients identified with high risk for PNH clone and per type of screening criteria by developing a computational screening algorithm2022

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.

Secondary Outcome Measures
NameTimeMethod
The number of patients at high risk for PNH, categorized by risk factor, across each medical department2023

To increase awareness of PNH risk factors by medical departments that need to consider PNH lab testing

Trial Locations

Locations (1)

AZ Delta

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Roeselare, West-Vlaanderen, Belgium

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