Hematopoietic Stem Cell Transplant (HSCT)NCT04098445
TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
An observational study of Hematopoietic Stem Cell Transplant (HSCT) and Diffuse Alveolar Hemorrhage, run by Children's Hospital Medical Center, Cincinnati at 9 sites in United States.
Trial summary
Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy and pulmonary morbidity affects as many as 25% of children receiving transplant. Early pulmonary injury includes diffuse alveolar hemorrhage (DAH), thrombotic microangiopathy (TMA) interstitial pneumonitis (IPS) and infection, while later… Read more about this study
- Who is it for?
- Ages Up to 24 years
- Conditions the study is for
- Hematopoietic Stem Cell Transplant (HSCT), Diffuse Alveolar Hemorrhage, Thrombotic Microangiopathies
- How many people
- 2,000 participants
- Where
- 9 sitesUnited States
- Study dates
- 8 Sep 2021to Sep 2033
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 24 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The study participants will include eligible patients undergoing allogeneic or autologous HSCT at participating institutions.
Who may be able to join
- Subjects ≤ 24 years of age undergoing allogeneic or autologous HSCT.
Who cannot join
- Subjects over 24 years of age.
Trial locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
9 sites in 1 country
United States9 sites, 9 recruiting
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Sonya Belimezova, 720-777-5987, Sonya.Belimezova@childrenscolorado.org
Hesham Eissa, MD
- Recruiting
Dana-Farber Cancer Institute/Boston Children's Hospital
Boston, Massachusetts, 02215
Mona Li, 617-632-5727, Mona_Li@DFCI.HARVARD.EDU
Leslie Lehmann, MD
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Sara Loveless, BSN, RN, 513-803-7656, Sara.Loveless@cchmc.org
Stella Davies, MBBS, PhD, MRCP
- Recruiting
Baylor College of Medicine/Texas Children'S Hospital
Houston, Texas, 77030
Martha Arredondo, 832-824-1201, Martha.Arredondo@bcm.edu
Erin Doherty, MD
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
Tamara Griffin, BSN, 612-301-0164, griffint@umn.edu
Phil Lacher, 612-626-2977, placher@umn.edu
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
David Flynn, 718-619-6663, flynnd6@chop.edu
Lisa Young, MD
- Recruiting
University of California San Francisco
San Francisco, California, 94158
Sana Noori, 415-353-1248, sana.noori@ucsf.edu
Gabriel Salinas Cisneros, MD
- Recruiting
Seattle Children'S Hospital
Seattle, Washington, 98105
Sheri Ballard, MPH, 206-667-4222, sballard@fredhutch.org
Matthew Abts, MD
- Recruiting
Fred Hutchinson Cancer Center
Seattle, Washington, 98109
Sheri Ballard, MPH, 206-667-4222, sballard@fredhutch.org
Scott Baker, MD, MS
About this study
Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy and pulmonary morbidity affects as many as 25% of children receiving transplant. Early pulmonary injury includes diffuse alveolar hemorrhage (DAH), thrombotic microangiopathy (TMA) interstitial pneumonitis (IPS) and infection, while later, bronchiolitis obliterans is a complication of chronic GVHD associated with severe morbidity and mortality. Improved diagnosis and treatment of pulmonary complications are urgently needed as survival after HSCT improves, and as HSCT is increasingly used for non-malignant disorders such as sickle cell disease. Currently, there are large and important gaps in the investigator's knowledge regarding incidence, etiology and optimal treatment of pulmonary complications. Moreover, young children unable to perform spirometry are often diagnosed late, and strategies for monitoring therapeutic response are limited.
This is a prospective multi-institutional cohort study in pediatric patients undergoing allogeneic hematopoietic stem cell transplantation (alloHSCT). Assembly of a large prospective uniformly screened cohort of children receiving HSCT, together with collection of biological samples, will be an effective strategy to identify mechanisms of lung injury, test novel diagnostic strategies for earlier diagnosis, and novel treatments to reduce morbidity and mortality from lung injury after transplant.
- Conditions
- Hematopoietic Stem Cell Transplant (HSCT), Diffuse Alveolar Hemorrhage, Thrombotic Microangiopathies, Interstitial Pneumonitis, Bronchiolitis Obliterans
- Medical subject headings
- Thrombotic Microangiopathies, Lung Diseases, Interstitial, Bronchiolitis Obliterans
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Pediatric and young adult HSCT recipients | Prospective multi-institutional cohort study in pediatric patients undergoing allogeneic (alloHSCT) or autologous hematopoietic stem cell transplantation (autoHSCT). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Lung Injury after HSCT
Participants will be assessed for lung injury at 24 months after HSCT
Measured: 24 months after HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2019
- Study start8 Sep 2021
- Primary completionSep 2033 (estimated)
- Study completionSep 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hematopoietic Stem Cell Transplant (HSCT)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Stella Davies, MBBS, PhD, MRCP, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04098445
- Sponsor's protocol ID
- 2019-0001C
- Status last verified
- Jun 2026
- First submitted
- 19 Sep 2019
- Last updated
- 22 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Goldfarb SB, Cooper R, Koo J, Myers KC, Walkup L, Woods J, Cook E, Hils B, Lane A, Allen J, Davies SM, Towe C. Detection of Post- Hematopoietic Stem Cell Transplant Bronchiolitis Obliterans Syndrome (BOS)-The Use of Airway Oscillometry. Pediatr Pulmonol. 2026 Sep;61(9):e71831. doi: 10.1002/ppul.71831.
PubMed 42747153Koo J, Cooper R, Edwards SL, Lane A, Loveless SK, Strecker L, Lake KE, Myers KC, Towe C, Patti J, Walkup LL, Wikenheiser-Brokamp KA, MacMillan ML, Lacher P, Griffin T, Tekman M, Cisneros GS, Wu K, Zinter MS, Urrego FA, Baker KS, Abts MF, Ballard S, Freedman JL, Caraballo A, Young LR, Josephson MB, Allen JL, Camburn DM, Doherty EE, Azamian MS, Arredondo M, Silva-Carmona M, Lehmann LE, Wong W, Gaffin JM, McAlpine W, Li M, Goldfarb SB, Woods JC, Davies SM. High Prevalence of Abnormal Baseline Lung Function in Pediatric and Young Adult Hematopoietic Stem Cell Transplant Recipients: A Report from the TRANSPIRE Study. Pediatr Blood Cancer. 2025 Oct;72(10):e31916. doi: 10.1002/pbc.31916. Epub 2025 Jul 21.
PubMed 40692216