Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy

Sponsor
National Heart, Lung, and Blood Institute (NHLBI) (NIH)
Overall Status
Active, not recruiting
CT.gov ID
NCT04436380
Collaborator
(none)
173
1
32.5
5.3

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 most people with SAA. One is allogeneic hematopoietic stem cell transplant (HSCT). The other is immunosuppressive treatment (IST). For people who are treated with IST, relapse can occur. If this happens, they can have HSCT or be re-treated with IST. The two most common IST regimes used for relapsed SAA are rabbit ATG (rATG) and alemtuzumab. Both rATG and alemtuzumab have similar response rates and survival rates. There is not much long-term data on people who need repeat IST treatment due to relapse. Researchers want to look at data from past studies to learn more.

Objective:

To compare the data of relapsed SAA patients between those who received alemtuzumab versus rATG for repeat IST treatment.

Eligibility:

Adults and children with SAA who were enrolled on NHLBI protocol 12-H-0150, 06-H-0034, 05-H-0242, 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 secure sites.

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 and 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 upfront allogeneic hematopoietic stem cell transplant (HSCT). However, in patients who are treated with IST, relapse remains an issue with a rate of 30-40%. Once relapsed, patients can be either re-treated with IST or undergo HSCT. The two most common IST regimes used for relapsed SAA are rabbit ATG (rATG) and alemtuzumab. Both rATG and alemtuzumab have been shown to have a response rate of approximately 60% in relapsed patients with similar overall survival rates of 83-85%. Previous predictors of relapse include time from diagnosis to IST, and early response to IST. Few long-term data exist on patients requiring repeat IST treatment due to relapse.

    Study Design

    Study Type:
    Observational
    Actual Enrollment :
    173 participants
    Observational Model:
    Cohort
    Time Perspective:
    Retrospective
    Official Title:
    Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy
    Actual Study Start Date :
    Jun 15, 2020
    Anticipated Primary Completion Date :
    Feb 28, 2023
    Anticipated Study Completion Date :
    Feb 28, 2023

    Arms and Interventions

    Arm Intervention/Treatment
    Relapsed SAA Patients

    Patients with Severe Aplastic Anemia who Relapsed after Immunosuppressive Therapy

    Outcome Measures

    Primary Outcome Measures

    1. Assessment of long-term outcomes of relapsed patients who received repeat IST [Between the Period of 1990 to 2020]

      Characteristics and outcomes of relapsed SAA patients between those who received alemtuzumab versus rATG for repeat IST

    Secondary Outcome Measures

    1. Clinical predictors for the development of relapse [Between the Period of 1990 to 2020]

      Clinical predictors for the development of relapse such as age, gender, baseline laboratory values, time from diagnosis to initial treatment, and initial treatment received.

    2. Rates of clonal evolution between relapsed patients depending on type of IST received and number of IST rounds required [Between the Period of 1990 to 2020]

      Compare rates of clonal evolution between relapsed patients depending on type of IST received and number of IST rounds required.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    • This is a retrospective chart review. The investigators will review the medical records of adults and children with severe aplastic anemia (SAA) enrolled on immunosuppressive therapy (IST) treatment protocols in the NHLBI.

    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:
    NCT04436380
    Other Study ID Numbers:
    • 999920119
    • 20-H-N119
    First Posted:
    Jun 18, 2020
    Last Update Posted:
    Jul 7, 2022
    Last Verified:
    Jun 17, 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