Brief Title
Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive Therapy
Official Title
Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive Therapy
Brief Summary
Background: Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for SAA. One is allogeneic hematopoietic stem cell transplant (HSCT). The other is immunosuppressive treatment (IST). In most cases, HSCT or IST works. But for some people, clonal evolution occurs after IST. One of the most common forms of clonal evolution is chromosome 7 abnormalities. These have a poor prognosis. HSCT can be used to treat them. Researchers do not know why clonal evolution happens. They want to look at data from past studies to learn more. Objective: To compare the data of people with SAA who developed chromosome 7 abnormalities between those who ultimately received HSCT versus those who received chemotherapy alone or supportive care. Eligibility: Adults and children with SAA who were enrolled on NHLBI protocol 12-H-0150, 06-H-0034, 03-H-0249, 03-H-0193, 00-H-0032, or 90-H-0146 Design: This study uses data from past studies. The participants in those studies have allowed their data to be used in future research. Researchers will review participants medical records. They will collect clinical data, such as notes, test results, and imaging scans. They will also collect the research data gathered as part of the original study. Researchers will enter the data into an in-house database. It is password protected. All data will be kept in secure network drives or in sites that comply with NIH security rules. Other studies may be added in the future.
Detailed Description
Severe aplastic anemia (SAA) is a form of bone marrow failure in most cases is the result of a cytotoxic T cell attack on the marrow stem cell. It is effectively treated in most patients with either immunosuppressive treatment (IST) or allogeneic hematopoietic stem cell transplant (HSCT). However, after IST, 'clonal evolution' is a significant complication in about 15% of patients, presenting as either a new cytogenetic abnormality or morphological evidence of myeloid malignancy. In particular, the development of chromosome 7 abnormalities is considered high risk and is associated with poor prognosis. The optimal treatment of chromosome 7 abnormalities following SAA is not defined though HSCT is widely offered.
Study Type
Observational
Primary Outcome
Characteristics and outcomes of SAA patients who developed chromosome 7 abnormalities
Secondary Outcome
Clinical predictors for the development of chromosome 7 abnormalities such as age, gender, baseline laboratory value
Condition
Severe Aplastic Anemia
Study Arms / Comparison Groups
SAA patients with Monosomy 7
Description: Severe Aplastic Anemia Patients who Developed High Risk Clonal Evolution with Chromosome 7 Abnormalities after Immunosuppressive Therapy
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
38
Start Date
June 15, 2020
Completion Date
March 15, 2022
Primary Completion Date
April 22, 2021
Eligibility Criteria
- Subjects will not be recruited for this study. This is a retrospective chart review. Patients who opted out of future use of data on their prior studies will be excluded from this study.
Gender
All
Ages
2 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Emma M Groarke, M.D., ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04436367
Organization ID
999920118
Secondary IDs
20-H-N118
Responsible Party
Sponsor
Study Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study Sponsor
Emma M Groarke, M.D., Principal Investigator, National Heart, Lung, and Blood Institute (NHLBI)
Verification Date
March 15, 2022