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

AZ Delta

Status: Completed

Conditions: PNH; Thrombosis

Treatments: Other: No intervention

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT05884060

B1172022000017

Details and patient eligibility

About

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.

Full 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.

Enrollment: 568 patients

Sex: All

Ages: 14+ years old

Volunteers: No Healthy Volunteers

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

Trial design

568 participants in 4 patient groups

group 1

Description:

patients with evidence of haemolysis without obvious cause

Treatment:

Other: No intervention

group 2

Description:

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

Treatment:

Other: No intervention

group 3

Description:

patients with thrombosis

Treatment:

Other: No intervention

group 4

Description:

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

Treatment:

Other: No intervention