Diagnosis, treatment, surgical practices and review of the literature in rare coagulation factor deficiencies
2025

Diagnosis and Treatment of Rare Bleeding Disorders

Sample size: 19 publication 10 minutes Evidence: moderate

Author Information

Author(s): Solgun Hüseyin Avni

Primary Institution: Pediatric Hematology and Oncology, SBU Sisli Hamidiye Etfal Training and Research Hospital, Istanbul, Turkey

Hypothesis

This study aimed to evaluate the diagnosis, genetics, treatment, prophylaxis features and surgical experiences of patients with rare bleeding disorders.

Conclusion

Data-based service is required for early diagnosis and follow-up of rare bleeding disorders, along with effective genetic counseling to improve patient outcomes.

Supporting Evidence

  • 14 patients were boys (74%) and 5 patients were girls (26%).
  • 10 of the patients (52%) had FVII deficiency.
  • Skin and soft tissue bleedings were reported in 52% of cases.

Takeaway

This study looked at kids with rare bleeding problems and found that knowing their family history and getting the right treatment can help them stay healthy.

Methodology

Demographic data and clinical symptoms of 19 patients diagnosed with rare bleeding disorders were recorded and evaluated retrospectively.

Limitations

The study is limited by its small sample size and the lack of comprehensive genetic testing for all patients.

Participant Demographics

The average age of participants was 11.2 years, with 74% being boys and 26% girls.

Digital Object Identifier (DOI)

10.1186/s13052-024-01806-7

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