An observational study in PNH and Thrombosis, sponsored by AZ Delta. Completed at 1 site in Belgium. Open to participants aged 14 Years and older. Per ClinicalTrials.gov, last updated 2023-06-02.
Sponsored by AZ Delta · Observational
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.
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.
955 studies on the registry are indexed under Thrombosis; 171 are open to participants now.
This study's enrollment of 568 is above the median of 173 across 384 observational studies indexed under Thrombosis.
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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:
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
patients with evidence of haemolysis without obvious cause
Other: No intervention
patients with evidence of bone marrow dysfunction (AA, MDS, unexplained cytopenia)
Other: No intervention
patients with thrombosis
Other: No intervention
patient group that needs to be eliminated from final high risk cohort: patients with cirrhosis, patients wit septic embolisms \& embolisation
Other: No intervention
No interventions
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.
Time frame: 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.
Time frame: 2022
The number of patients at high risk for PNH, categorized by risk factor, across each medical department
To increase awareness of PNH risk factors by medical departments that need to consider PNH lab testing
Time frame: 2023
Plan to share: No
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This study is completed, as verified in May 2023. You cannot join it, but the record below documents what was studied.
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