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

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....

Study Overview

• Status: Completed
• Conditions: Severe Aplastic Anemia

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
• Enrollment (Actual): 173

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Heart, Lung and Blood Institute (NHLBI)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Relapsed SAA Subjects who had a repeat IST

Description

• 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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Relapsed SAA Patients; Patients with Severe Aplastic Anemia who Relapsed after Immunosuppressive Therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of long-term outcomes of relapsed patients who received repeat IST	Characteristics and outcomes of relapsed SAA patients between those who received alemtuzumab versus rATG for repeat IST	Between the Period of 1990 to 2020

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical predictors for the development of relapse	Clinical predictors for the development of relapse such as age, gender, baseline laboratory values, time from diagnosis to initial treatment, and initial treatment received.	Between the Period of 1990 to 2020
Rates of clonal evolution between relapsed patients depending on type of IST received and number of IST rounds required	Compare 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

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): June 15, 2020
• Primary Completion (Actual): March 15, 2023
• Study Completion (Actual): March 15, 2023

Study Registration Dates

• First Submitted: June 17, 2020
• First Submitted That Met QC Criteria: June 17, 2020
• First Posted (Actual): June 18, 2020

Study Record Updates

• Last Update Posted (Actual): March 16, 2023
• Last Update Submitted That Met QC Criteria: March 15, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: Natural History; Alemtuzumab; rATG
• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic
• Other Study ID Numbers: 999920119; 20-H-N119