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

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Brief Title

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

Official Title

Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed 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 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....
    

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 Type

Observational


Primary Outcome

Assessment of long-term outcomes of relapsed patients who received repeat IST

Secondary Outcome

 Clinical predictors for the development of relapse

Condition

Severe Aplastic Anemia


Study Arms / Comparison Groups

 Relapsed SAA Patients
Description:  Patients with Severe Aplastic Anemia who Relapsed 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

173

Start Date

June 15, 2020

Completion Date

January 30, 2024

Primary Completion Date

April 22, 2022

Eligibility Criteria

        -  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.
      

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

NCT04436380

Organization ID

999920119

Secondary IDs

20-H-N119

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