Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7Abnormalities After Immunosuppressive Therapy

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.

Estimated Enrollment

38

Start Date

June 15, 2020

Completion Date

January 30, 2024

Primary Completion Date

April 22, 2022

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

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

April 30, 2021