Clinical trial NCT05884060: Retrospective Chart Review Screening Algorithm to Assess the Prevalence of PNH-clones
Official title: Retrospective Electronic Chart Review to Assess the Prevalence of PNH-clones Among Patients Identified With PNH Risk Factors
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: PNH
Thrombosis
Keywords: PNH
Thrombosis
anemia
aplastic anemia
myelodysplastic syndrome (MDS)
Lead sponsor: AZ Delta
Sponsor class: OTHER
Collaborators: name: Alexion Pharmaceuticals, Inc.
First posted: 2023-06-01
Enrollment: 568
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: RETROSPECTIVE
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

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.

Eligibility criteria: 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

Primary outcomes:
measure: PNH risk factors translation into query codes that can be interpreted by a computer system for analysis
timeFrame: 2022
measure: Number of patients identified with high risk for PNH clone and per type of screening criteria by developing a computational screening algorithm
timeFrame: 2022
Secondary outcomes:
measure: The number of patients at high risk for PNH, categorized by risk factor, across each medical department
timeFrame: 2023
Other outcomes:
Locations:
city: Roeselare
country: Belgium
facility: AZ Delta
state: West-Vlaanderen
Study officials:
affiliation: AZ Delta
name: Dries Deeren, MD
role: PRINCIPAL_INVESTIGATOR
References: