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Characteristics of Hypophosphatasia in Adult Patients in Rheumatology

Characteristics of Hypophosphatasia in Adult Patients in Rheumatology

Recruiting
18 years and older
All
Phase N/A

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Overview

With hypophosphatasia still being frequently overlooked and misdiagnosed, the primary aim of this prospective observational study is to determine the prevalence of hypophosphatasia in adult patients in rheumatology, and beyond that to establish an algorithm that promotes early hypophosphatasia detection in clinical practice.

Description

Hypophosphatasia (HPP) is a rare genetic disorder (1-3/300,000 severe cases in Europe) caused by one or more mutations in the alkaline phosphatase (ALP) gene. Hypomineralization results in symptoms such as arthralgias, insufficiency fractures, and poor dental status beginning in childhood. A fatal outcome is conceivable in circumstances of early infancy first presentation. In consistency with the musculoskeletal complaint pattern, HPP is far more common in the rheumatology patient population than in the general population.

However, HPP is still frequently misdiagnosed as some other form of bone disease (e.g., rickets, osteomalacia, or osteoporosis). Therefore, implementation of a clinically applicable algorithm for early hypophosphatasia detection is needed.

The primary aim of this prospective observational study is to determine the prevalence of hypophosphatasia in adult patients in rheumatology. Moreover, a further goal is to establish an algorithm that reliably separates adult HPP patients from other, rheumatologic and bone diseases.

Eligibility

Inclusion Criteria:

  • Written Informed consent
  • Age > 18 years
  • Clinical suspicion of hypophosphatasia
  • Evidence of a pathological ALP value within the clinical routine screening

Exclusion Criteria:

  • Failure to meet the inclusion criteria listed above

Study details
    Hypophosphatasia

NCT06161142

University of Bonn

27 January 2024

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