Early Detection of Infectious and Noninfectious Lung Diseases Following Allogeneic Hematopoietic Stem Cell Transplantation

A Prospective Approach for the Early Detection of Infectious and Noninfectious Lung Diseases Following Allogeneic Hematopoietic Stem Cell Transplantation

Background:

Stem cell transplants (called hematopoietic stem cell transplantation, or HSCT) are used to treat various diseases. But when the cells for this procedure are donated by someone other than the person who receives the HSCT ( allogeneic HSCT ), the recipient has an increased risk of lung inflammation and scarring. This happens when their immune cells attack healthy lung cells. In this natural history study, researchers will look for the best ways to detect developing lung inflammations earlier after an HSCT.

Objective:

To see if certain tests can detect early signs of lung inflammation in people after HSCT.

Eligibility:

People aged 5 to 70 years who will have HSCT as part of another NIH study.

Design:

Participants will undergo these tests prior to their HSCT. These tests will then be repeated regularly for 2 years:

Ultra-low dose computed tomography (CT) scans. Participants will lie on a table that slides through a machine; the machine uses X-rays to get pictures of the inside of the body. This type of scan uses less radiation than normal CT scans.

Bronchoscopy with lavage: Participants will be sedated. A flexible tube will be inserted through the mouth and into the airways. Salt water will be squirted into the lung, then sucked out to collect cells and fluids from the lung. Another tube with a camera may be inserted into the airways to take pictures.

Blood tests. Blood will be drawn every 2 to 4 weeks.

Pulmonary function tests. Participants will breathe into a machine to test their lung function. They will see how far they can walk in 6 minutes.

Study Overview

• Status: Recruiting
• Conditions: Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Detailed Description

Study Description:

The primary goal of this proposal is to use preemptive pulmonary imaging, biomarker measurement with surveillance bronchoscopy to improve the early detection of inflammatory injury after allogeneic hematopoietic stem cell transplantation (HSCT). These data may provide early identification of patients at risk for infectious and noninfectious lung diseases and accelerate the development of targeted therapies.

Objectives:

Primary Objective:

To use preemptive assessments with sequential chest imaging and bronchoalveolar lavage (BAL) measures to provide earlier identification of lung injury due to infection or alloreactive lung syndromes associated with hematopoietic stem cell transplantation

Secondary Objectives:

To use high density proteomic, transcriptomic and metagenomic analysis of specimens from the lung and blood to define mechanisms of altered lung function associated with HSCT.

Endpoints:

Primary Endpoint: To assess whether surveillance chest tomography and bronchoscopy with lavage detects incipient lung infection and noninfectious lung injury prior to and following HSCT.

Secondary Endpoints: To determine if biomarkers from surveillance proteomic, transcriptomic or metagenomic analysis will provide early identification of lung injury prior to the development chronic progressive and irreversible lung disease.

• Study Type: Observational
• Enrollment (Estimated): 40

Contacts and Locations

• Study Contact: Name: Gloria Y Pastor, R.N.; Phone Number: (301) 402-3484; Email: gloria.pastor@nih.gov
• Study Contact Backup: Name: Anthony F Suffredini, M.D.; Phone Number: (301) 496-9320; Email: asuffredini@mail.nih.gov

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • Recruiting
      • National Institutes of Health Clinical Center
      • Contact: NIH Clinical Center Office of Patient Recruitment (OPR); Phone Number: TTY dial 711 800-411-1222; Email: ccopr@nih.gov
      • Contact: Gloria Pastor; Phone Number: (301) 402-3484; Email: gloria.pastor@nih.gov

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Men or women, 18 - 70 years of age & children 5 - 17 years , who are being treated with allogeneic HSCT for a hematologic malignancy, blood, or immune system disorder at the NIH Clinical Center

Description

• INCLUSION CRITERIA:

In order to be eligible to participate in this study, an individual must meet all of the following criteria:

1. Stated willingness to comply with all study procedures and availability for the duration of the study.
2. Male or female, aged 5 - 70 years of age
3. Are to be treated with allogeneic HSCT for a hematologic malignancy, blood, or immune system disorders at the NIH Clinical Center.
4. Ability of subject to understand and the willingness to sign a written informed consent document.

EXCLUSION CRITERIA:

An individual who meets any of the following criteria will be excluded from participation in this study:

1. Pregnancy or lactation

a. HSCT is not available to pregnant women or lactating women in the intramural program. For women of reproductive potential, have a negative serum pregnancy test during the screening period. Women of reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy, or tubal occlusion; or who have not been naturally postmenopausal (i.e., who have not menstruated at all for at least the preceding 1 year prior to signing informed consent unrelated to hormonal contraception).

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
allogeneic HSCT; Up to 30 men or women, 18 - 70 years of age, who are being treated with allogeneic HSCT for a hematologic malignancy, blood, or immune system disorder at the NIH Clinical CenterUp to 10 children 5 - 17 years of age who are being treated with allogeneic HSCT for a hematologic malignancies, blood, or immune system disorder at the NIH Clinical CenterNIH Clinical Center

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To use preemptive assessments with sequential chest imaging and BAL measures to provide earlier identification of lung injury due to infection or alloreactive lung syndromes associated with HSCT.	To assess whether surveillance chest tomography and bronchoscopy with lavage detects incipient lung infection and noninfectious lung injury prior to and following HSCT	30 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To use high density proteomic, transcriptomic and metagenomic analysis of specimens from the lung and blood to define mechanisms of altered lung function associated with HSCT.	To determine if biomarkers from surveillance proteomic, transcriptomic or metagenomic analysis will provide early identification of lung injury prior to the development chronic progressive and irreversible lung disease	30 months

Collaborators and Investigators

• Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Collaborators: National Institutes of Health Clinical Center (CC)
• Investigators: Principal Investigator: Anthony F Suffredini, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Publications and helpful links

Helpful Links

• NIH Clinical Center Detailed Web Page

Study record dates

Study Major Dates

• Study Start (Actual): April 8, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2029

Study Registration Dates

• First Submitted: October 20, 2023
• First Submitted That Met QC Criteria: October 20, 2023
• First Posted (Actual): October 23, 2023

Study Record Updates

• Last Update Posted (Actual): April 9, 2026
• Last Update Submitted That Met QC Criteria: April 8, 2026
• Last Verified: April 6, 2026

More Information

Terms related to this study

• Keywords: allogeneic hematopoietic stem cell transplantation (HSCT); early detection of inflammatory injury
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases
• Other Study ID Numbers: 10001742; 001742-H

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Refer to the DMSP
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No