PNHNCT05884060
Retrospective Chart Review Screening Algorithm to Assess the Prevalence of PNH-clones
An observational study of PNH and Thrombosis, run by AZ Delta at 1 site in Belgium.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- PNH, Thrombosis
- How many people
- 568 participants
- Where
- 1 siteBelgium
- Study dates
- 20 Apr 2018to 1 Mar 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 years and older
- All sexes
- Child, Adult, Older adult
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
Who may be able to join
- 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
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Retrospective Electronic Chart Review to Assess the Prevalence of PNH-clones Among Patients Identified With PNH Risk Factors
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.
Read the 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.
- Conditions
- PNH, Thrombosis
- Being tested
- No intervention
- Keywords
- PNH, Thrombosis, anemia, aplastic anemia, myelodysplastic syndrome (MDS)
- Medical subject headings
- Thrombosis, Anemia, Anemia, Aplastic, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
No interventions
| Study group | Role | What they receive |
|---|---|---|
| group 1 | patients with evidence of haemolysis without obvious cause Receives: Other: No intervention | |
| group 2 | patients with evidence of bone marrow dysfunction (AA, MDS, unexplained cytopenia) Receives: Other: No intervention | |
| group 3 | patients with thrombosis Receives: 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 Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 2022
Secondary outcomes (1)
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
Measured: 2023
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2023
- Study start20 Apr 2018
- Primary completion1 Mar 2022
- Study completion1 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PNHStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 568 participants (actual)
Sponsor and investigators
- Lead sponsor
- AZ Delta
- Sponsor type
- University, hospital or other
- Collaborators
- Alexion Pharmaceuticals, Inc.
- Responsible party
- Sponsor
- Investigators
- Dries Deeren, MD, Principal investigator, AZ Delta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05884060
- Sponsor's protocol ID
- B1172022000017
- Status last verified
- May 2023
- First submitted
- 10 May 2023
- Last updated
- 2 Jun 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Electronic Chart Review to Assess the Prevalence of PNH-clones Among Patients Identified With PNH Risk Factors
- ClinicalTrials.gov ID
- NCT05884060
- Acronym / sponsor ID
- B1172022000017
- Conditions
- PNH, Thrombosis
- Keywords
- PNH, Thrombosis, anemia, aplastic anemia, myelodysplastic syndrome (MDS)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 568 participants (actual)
- Lead sponsor
- AZ DeltaUniversity, hospital or other
- Collaborators
- Alexion Pharmaceuticals, Inc.
- Study start
- 20 Apr 2018 (actual)
- Primary completion
- 1 Mar 2022 (actual)
- Study completion
- 1 Mar 2022 (actual)
- First posted
- 1 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| group 1 | – | Other: No interventionpatients with evidence of haemolysis without obvious cause |
| group 2 | – | Other: No interventionpatients with evidence of bone marrow dysfunction (AA, MDS, unexplained cytopenia) |
| group 3 | – | Other: No interventionpatients with thrombosis |
| group 4 | – | Other: No interventionpatient group that needs to be eliminated from final high risk cohort: patients with cirrhosis, patients wit septic embolisms \& embolisation |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- 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
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionNo interventions | Other | group 1, group 2, group 3, group 4 |
Primary outcome
PNH risk factors translation into query codes that can be interpreted by a computer system for analysis
Time frame: 2022
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 algorithm
Time frame: 2022
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.
1 secondary outcome
The number of patients at high risk for PNH, categorized by risk factor, across each medical department
Time frame: 2023
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2023.
- Individual participant dataSharing planned: No
Related trials for PNH
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Active, not recruitingPhase 3
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria
RecruitingObservational study
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Enrolling by invitationObservational study
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
Bone Marrow Failure Disorders, VEXAS Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dries Deeren, MD principal investigator
AZ Delta
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of PNH and Thrombosis, run by AZ Delta at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PNH risk factors translation into query codes that can be interpreted by a computer system for analysis (time frame: 2022). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jun 2023.