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Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

NCT00499070 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 119

Last updated 2015-01-16

No results posted yet for this study

Summary

RATIONALE: Studying biopsy, bone marrow, and blood samples from patients with cytopenia that did not respond to treatment may help doctors learn more about the disease and plan the best treatment.

PURPOSE: This laboratory study is assessing immune function in young patients with cytopenia that did not respond to treatment.

Conditions

  • Dyskeratosis Congenita
  • Fanconi Anemia
  • Myelodysplastic Syndromes
  • Pearson Marrow-pancreas Syndrome
  • Shwachman-diamond Syndrome

Interventions

GENETIC

polymerase chain reaction

OTHER

flow cytometry

For analyzing GPI deficient clones full blood will be analyzed by phenotyping using flowcytometry. For that purpose CD14, CD16 and CD24 expression will be evaluated in CD45 positive cells. Erythroid cells will be evaluated for CD55 and CD59 expression searching for clear populations with a lack of GPI-linked molecules. In addition, immunophenotyping using flowcytometry will be performed to evaluate which differentiation stages of the major hematopoietic lineages in BM and PB are associated with TCRVβ repertoire skewing. Comparison between BM and PB will identify which is the optimal compartment to analyze the responsible hematopoietic clones.

OTHER

immunologic technique

PROCEDURE

biopsy

Sponsors & Collaborators

  • University Hospital Freiburg

    lead OTHER

Principal Investigators

  • Marry M. Van Den Heuvel-Eibrink, MD, PhD · Erasmus MC-Sophia Children's Hospital

Eligibility

Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-01-31
Primary Completion
2012-08-31
Completion
2012-08-31

Countries

  • Austria
  • Belgium
  • Czechia
  • Denmark
  • Germany
  • Ireland
  • Italy
  • Netherlands
  • Spain
  • Switzerland

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00499070 on ClinicalTrials.gov