Clinical trial · Observational
Identifying Characteristics of Bone Marrow Failure Syndromes
Screening Protocol and Longitudinal Study of Bone Marrow Failure Syndromes and Cytopenias
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Bone marrow failure syndromes (BMFS) are rare disorders characterized by dysfunctional hematopoietic stem cells, which give rise to all red and white blood cells. The deficiency of blood cells, or cytopenia, caused by this malfunction leads to an assortment of diseases and disorders, all of which are characterized as BMFS. Because these diseases are rare, conducting research on them is difficult, and standards of treatment for most BMFS have yet to be developed. This study will collect clinical and laboratory data from people with BMFS to identify the characteristics and biological markers associated with these diseases over time. This information will assist doctors and researchers to develop better therapies and diagnostic tests that will help improve the management of BMFS and cytopenias.
Conditions
Conditions (7)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Anemia, Aplastic | — | UNRESOLVED | — |
| Bone Marrow Failure Syndromes | — | UNRESOLVED | — |
| Hemoglobinuria, Paroxysmal | — | UNRESOLVED | — |
| Leukemia, Lymphocytic | Lymphoid Leukemia | ALIAS | 0.90 |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
| Purpura, Thrombocytopenic | — | UNRESOLVED | — |
| Red-Cell Aplasia, Pure | — | UNRESOLVED | — |
Interventions
Interventions (0)
Data not yet available
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 11 Years
Show eligibility criteria text
Inclusion Criteria: * Diagnosis of one of the following diseases: aplastic anemia; myelodysplastic syndrome; paroxysmal nocturnal hemoglobinuria; idiopathic pure red cell aplasia; amegakaryocytic thrombocytopenia purpura; or large granular lymphocyte leukemia Exclusion Criteria: * N/A
References
Publications (5)
- BACKGROUNDYoung NS. Immunosuppressive treatment of acquired aplastic anemia and immune-mediated bone marrow failure syndromes. Int J Hematol. 2002 Feb;75(2):129-40. doi: 10.1007/BF02982017. PMID 11939258
- BACKGROUNDPlasilova M, Risitano A, Maciejewski JP. Application of the molecular analysis of the T-cell receptor repertoire in the study of immune-mediated hematologic diseases. Hematology. 2003 Jun;8(3):173-81. doi: 10.1080/1024533031000107505. PMID 12745651
- BACKGROUNDFrickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H; German Aplastic Anemia Study Group. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003 Feb 15;101(4):1236-42. doi: 10.1182/blood-2002-04-1134. Epub 2002 Oct 10. PMID 12393680
- BACKGROUNDMolldrem JJ, Leifer E, Bahceci E, Saunthararajah Y, Rivera M, Dunbar C, Liu J, Nakamura R, Young NS, Barrett AJ. Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes. Ann Intern Med. 2002 Aug 6;137(3):156-63. doi: 10.7326/0003-4819-137-3-200208060-00007. PMID 12160363
- BACKGROUNDSloand E, Kim S, Maciejewski JP, Tisdale J, Follmann D, Young NS. Intracellular interferon-gamma in circulating and marrow T cells detected by flow cytometry and the response to immunosuppressive therapy in patients with aplastic anemia. Blood. 2002 Aug 15;100(4):1185-91. doi: 10.1182/blood-2002-01-0035. PMID 12149196