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

NCT04436367 | Completed

• 2 other identifiers: 99992011820-H-N118
• Study Type: observational
• Target: 38
• Locations: 1 country
• Sites: 1

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.

Conditions

Severe Aplastic Anemia

Keywords

Cytogenic Abnormality; Natural History

Outcome Measures

Primary Outcomes (1)

• Characteristics and outcomes of SAA patients who developed chromosome 7 abnormalities

  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

  Between the Period of 1990 to 2020

Secondary Outcomes (3)

• Clinical predictors for the development of chromosome 7 abnormalities such as age, gender, baseline laboratory value

  Between the Period of 1990 to 2020

• Ascertain the natural history of patients with a chromosome 7 abnormality on karyotype

  Between the Period of 1990 to 2020

• Morphological predictors in the bone marrow for progression to an overt myeloid neoplasm

  Between the Period of 1990 to 2020

Study Arms (1)

SAA patients with Monosomy 7

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

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

SAA Subjects who developed chromosome 7 abnormalities

* 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.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

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

Study Sites (1)

National Heart, Lung and Blood Institute (NHLBI)

Bethesda, Maryland, 20892, United States

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Emma M Groarke, M.D.

  National Heart, Lung, and Blood Institute (NHLBI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 17, 2020
• First Posted: June 18, 2020
• Study Start: June 15, 2020
• Primary Completion: April 22, 2021
• Study Completion: March 15, 2022
• Last Updated: September 29, 2022

Record last verified: 2022-09

Locations

US (1)