Ruxolitinib for Lung Dysfunction Post Stem Cell Transplant
(HSCT Trial)
Trial Summary
What is the purpose of this trial?
Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy, and lung injury affects as many as 25% of children receiving HSCT. Improved transplant techniques and major improvements in survival mean that HSCT is being more widely used, and more mismatched grafts are being used. Bronchiolitis obliterans (BO) is a major limitation of pediatric HSCT success as BO is commonly diagnosed late in children, when lung injury is irreversible, leading to long term morbidity or even death. Currently, there are major gaps in our knowledge regarding incidence, etiology and optimal treatment of BO following HSCT, and important diagnostic limitations specific to children. Diagnosis of BO is usually based on performance of pulmonary function tests, which is usually impossible in ill children under 10. Even older children who feel unwell or un-cooperative may be unable to produce interpretable data. These deficiencies in diagnosis mean that BO is commonly diagnosed late, meaning fibrosis has occurred and lesions are irreversible. The hypothesis for this interventional trial is that early treatment with standard Flovent/montelukast and steroids plus ruxolitinib will reverse lung injury and reduce the frequency of chronic pulmonary impairment or florid BO.
Will I have to stop taking my current medications?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are taking investigational agents for GVHD, you must stop them at least 30 days before starting the trial treatment.
What data supports the effectiveness of the drug Ruxolitinib for lung dysfunction after stem cell transplant?
Ruxolitinib has shown effectiveness in reducing symptoms and improving outcomes in patients with myelofibrosis undergoing stem cell transplants, and it has been used successfully to treat lung issues in transplant patients when reintroduced after discontinuation. This suggests it may help with lung dysfunction post-transplant by reducing inflammation and improving overall transplant outcomes.12345
Is Ruxolitinib generally safe for humans?
Ruxolitinib has been used in various conditions and is generally considered safe, but it can cause side effects like anemia (low red blood cell count) and thrombocytopenia (low platelet count), which are usually manageable. There have been reports of infections and breathing problems when stopping the drug, so careful monitoring is important.24678
How is the drug Ruxolitinib unique for treating lung dysfunction after stem cell transplant?
Ruxolitinib is unique because it is an oral medication that inhibits the Janus kinase (JAK) pathway, which is involved in immune function and inflammation. This mechanism may help manage lung dysfunction after stem cell transplant, although it is primarily used for conditions like myelofibrosis and can increase the risk of infections.29101112
Research Team
Kasiani Myers, MD
Principal Investigator
Children's Hospital Medical Center, Cincinnati
Eligibility Criteria
This trial is for people aged 5 to 60 who've had a bone marrow transplant and are showing early signs of lung problems. They need good blood, kidney, and liver function. It's not for those allergic to the study drug, pregnant or breastfeeding individuals without birth control, or anyone treated with other experimental drugs for graft-versus-host disease in the last month.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive early treatment with Flovent/montelukast, steroids, and ruxolitinib to reverse lung injury
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Ruxolitinib
Ruxolitinib is already approved in United States, European Union for the following indications:
- Intermediate or high-risk myelofibrosis
- Polycythemia vera
- Steroid-refractory acute graft-versus-host disease
- Chronic graft-versus-host disease
- Vitiligo
- Intermediate or high-risk myelofibrosis
- Polycythemia vera
- Steroid-refractory acute graft-versus-host disease
- Chronic graft-versus-host disease
- Non-segmental vitiligo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital Medical Center, Cincinnati
Lead Sponsor