RecruitingNCT02964494

The Congenital Dyserythropoietic Anemia Registry (CDAR)


Sponsor

Children's Hospital Medical Center, Cincinnati

Enrollment

10,000 participants

Start Date

Aug 29, 2016

Study Type

OBSERVATIONAL

Conditions

Summary

The investigators have created and maintain a comprehensive registry for patients with the diagnosis of Congenital Dyserythropoietic Anemia (CDA) in North America. The goal of this registry is to collect long-term confidential data on patients with CDA in the US, Canada, and Mexico and maintain a bio-repository of de-identified patient blood and bone marrow specimens as a tool for the investigation of epidemiology, natural history, biology, and molecular pathogenetic mechanisms of CDA.


Eligibility

Inclusion Criteria5

  • Diagnosis of Congenital Dyserythropoietic Anemia (CDA), whether a genetic mutation is identified or not
  • Evidence of congenital anemia/jaundice or a positive family history
  • Evidence of ineffective erythropoiesis
  • Typical morphological appearance of bone marrow erythroblasts
  • All ages (ages 0-99)

Exclusion Criteria5

  • Diagnosis of cancer
  • Myelodysplasia
  • Secondary dyserythropoiesis: e.g.; vitamin B12 deficiency or drug-related.
  • Note1: Patients with rare band 3 (SLC4A1) mutations recently described to be associated with dyserythropoiesis will be eligible since the mechanisms appear to involve direct participation of band 3 in the erythroblast mitosis and cytokinesis.
  • Note2: Siblings, parents, and family members of patients with confirmed CDA diagnosis are encouraged to participate in the study.

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Locations(1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

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NCT02964494


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