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

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: TCR Vbeta Repertoire and PNH Clones in Children With Refractory Cytopenia (RC). An Open Nonrandomised Multi-Center Prospective Study

Brief 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.


Detailed Summary:

OBJECTIVES:

Primary

  • To evaluate the value of TCR V beta repertoire analysis for the determination of autoimmunity in refractory cytopenia (RC).
  • To evaluate which immunophenotypic hematopoietic subclones are associated with oligoclonal T-cell expansion in RC.
  • To evaluate the presence of paroxysmal nocturnal hemoglobinuria (PNH) clones in RC.

Secondary

  • To compare the molecular response with the hematologic response in patients with RC after treatment with immunosuppressive therapy (IST).
  • To compare the molecular response with human leukocyte histocompatability antigen (HLA) expression in patients with RC after treatment with IST.

OUTLINE: This is an open-label, multicenter, nonrandomized, prospective study.

Patients undergo biopsy, bone marrow, and blood sample collection periodically for immunological studies. Samples are analyzed for TCR V beta repertoire and paroxysmal nocturnal hemoglobinuria (PNH) clone analysis via PCR heteroduplex analysis and immunophenotyping of CD14, CD16 , CD55, CD59, and CD24 expression via flow cytometry.


Sponsor: University Hospital Freiburg

Current Primary Outcome:

  • Number of patients with TCR V beta oligoclonality at diagnosis [ Time Frame: 96 months ]
  • Immunophenotype of patients with oligoclonal T-cell expansion [ Time Frame: 96 months ]
  • Number of patients with glycophosphatidylinositol (GPI) deficient clones [ Time Frame: 96 months ]


Original Primary Outcome:

Current Secondary Outcome:

  • Number of patients with molecular response as compared to hematological response after IST [ Time Frame: 96 months ]
  • Number of patients with HLA-DR15 antigen expression and molecular response as compared to number of patients with other HLA-DR antigens and molecular response [ Time Frame: 96 months ]
  • Overall survival [ Time Frame: 96 months ]
  • Failure-free survival [ Time Frame: 96 months ]


Original Secondary Outcome:

Information By: University Hospital Freiburg

Dates:
Date Received: July 10, 2007
Date Started: January 2007
Date Completion:
Last Updated: January 15, 2015
Last Verified: January 2015