Clinical trial NCT04098445: TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Official title: TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hematopoietic Stem Cell Transplant (HSCT)
Diffuse Alveolar Hemorrhage
Thrombotic Microangiopathies
Interstitial Pneumonitis
Bronchiolitis Obliterans
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2019-09-23
Enrollment: 2000
Sex: ALL
Maximum age (years): 24
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study participants will include eligible patients undergoing allogeneic or autologous HSCT at participating institutions.

Brief 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, 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.


Eligibility criteria: Inclusion Criteria:

* Subjects ≤ 24 years of age undergoing allogeneic or autologous HSCT.

Exclusion Criteria:

* Subjects over 24 years of age.

Primary outcomes:
measure: Incidence of Lung Injury after HSCT
timeFrame: 24 months after HSCT
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Boston
country: United States
facility: Dana-Farber Cancer Institute/Boston Children's Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: Baylor College of Medicine/Texas Children'S Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children'S Hospital
state: Washington
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Center
state: Washington
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Stella Davies, MBBS, PhD, MRCP
role: PRINCIPAL_INVESTIGATOR
References: