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

Sponsor
National Heart, Lung, and Blood Institute (NHLBI) (NIH)
Overall Status
Completed
CT.gov ID
NCT04436367
Collaborator
(none)
38
1
21
1.8

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase

    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 Design

    Study Type:
    Observational
    Actual Enrollment :
    38 participants
    Observational Model:
    Cohort
    Time Perspective:
    Retrospective
    Official Title:
    Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive Therapy
    Actual Study Start Date :
    Jun 15, 2020
    Actual Primary Completion Date :
    Apr 22, 2021
    Actual Study Completion Date :
    Mar 15, 2022

    Arms and Interventions

    Arm Intervention/Treatment
    SAA patients with Monosomy 7

    Severe Aplastic Anemia Patients who Developed High Risk Clonal Evolution with Chromosome 7 Abnormalities after Immunosuppressive Therapy

    Outcome Measures

    Primary Outcome Measures

    1. Characteristics and outcomes of SAA patients who developed chromosome 7 abnormalities [Between the Period of 1990 to 2020]

      To compare characteristics and outcomes of SAA patients who developed chromosome 7 abnormalities between those who ultimately received HSCT versus those who received chemotherapy alone or supportive care

    Secondary Outcome Measures

    1. Clinical predictors for the development of chromosome 7 abnormalities such as age, gender, baseline laboratory value [Between the Period of 1990 to 2020]

      To identify clinical predictors for the development of chromosome 7 abnormalities such as age, gender, baseline laboratory values, time from diagnosis to initial treatment, relapse, and number of IST treatments

    2. Ascertain the natural history of patients with a chromosome 7 abnormality on karyotype [Between the Period of 1990 to 2020]

      Ascertain the natural history of patients with a chromosome 7 abnormality on karyotype who were surveilled until the development of an overt myeloid neoplasm

    3. Morphological predictors in the bone marrow for progression to an overt myeloid neoplasm [Between the Period of 1990 to 2020]

      To identify morphological predictors in the bone marrow for progression to an overt myeloid neoplasm in those with without an overt myeloid neoplasm at the time of development of chromosome 7 abnormalities

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    • 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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 National Heart, Lung and Blood Institute (NHLBI) Bethesda Maryland United States 20892

    Sponsors and Collaborators

    • National Heart, Lung, and Blood Institute (NHLBI)

    Investigators

    • Principal Investigator: Emma M Groarke, M.D., National Heart, Lung, and Blood Institute (NHLBI)

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    National Heart, Lung, and Blood Institute (NHLBI)
    ClinicalTrials.gov Identifier:
    NCT04436367
    Other Study ID Numbers:
    • 999920118
    • 20-H-N118
    First Posted:
    Jun 18, 2020
    Last Update Posted:
    Jul 7, 2022
    Last Verified:
    Mar 15, 2022
    Keywords provided by National Heart, Lung, and Blood Institute (NHLBI)
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Jul 7, 2022