Summary

Eligibility
for people ages up to 24 years (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Alexis Melton, MD, PhD

Description

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 (alloHSCT) or autologous hematopoietic stem cell transplantation (autoHSCT). 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.

Keywords

Hematopoietic Stem Cell Transplant (HSCT) Diffuse Alveolar Hemorrhage Thrombotic Microangiopathies Interstitial Pneumonitis Bronchiolitis Obliterans Bronchiolitis Pneumonia Lung Injury Lung Diseases, Interstitial Hemorrhage

Eligibility

You can join if…

Open to people ages up to 24 years

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

You CAN'T join if...

  • Subjects over 24 years of age.

Locations

  • University of California San Francisco accepting new patients
    San Francisco California 94158 United States
  • Fred Hutchinson Cancer Center accepting new patients
    Seattle Washington 98109 United States

Lead Scientist at UCSF

  • Alexis Melton, MD, PhD
    Associate Professor, Pediatrics, School of Medicine. Authored (or co-authored) 24 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Children's Hospital Medical Center, Cincinnati
ID
NCT04098445
Study Type
Observational
Participants
Expecting 2000 study participants
Last Updated