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Clinical Studies

5401: Screening Protocol and Longitudinal Study of Bone Marrow Failure Syndromes and Cytopenias

Status: Recruiting

Summary:

Please Note: The Rare Diseases Clinical Research Network will make every effort to enroll all the patients we can, but we cannot make any guarantees that we will be able to enroll everyone in a particular study who wants to participate.

The purpose of this study is to characterize patients with Bone Marrow Failure Syndromes (BMFS) and to track the clinical course of patients with these diseases over time. An exact and comprehensive diagnosis at presentation is essential. Due to the sporadic nature of bone marrow failure syndromes (aplastic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuria, pure red cell aplasia, amegakaryocytic thrombocytopenic purpura and large granular lymphocyte leukemia) most diseases have not undergone systematic long-term outcome studies. Individual diseases frequently overlap and evolve from each other. The frequency of complications, associations with other diseases, and genetic factors are not well studied.

Target Enrollment:

Population to be studied includes patients with one or more BMFS diagnosis of:

  • Aplastic Anemia (AA)
  • Myelodysplastic Syndrome (MDS)
  • Paroxysmal nocturnal hemoglobinuria (PNH)
  • Pure red cell aplasia (PRCA)
  • Amegakaryocytic thrombocytopenic purpura (ATP)
  • Large granular lymphocyte leukemia (LGL leukemia)

How to Participate:

In order to participate in a study, you must personally contact the study coordinator of any of the participating institutions by phone or by e-mail. Please use the information below to inquire about participation.

Join the: Contact Regsitry