January 29 – 8am-5pm
January 30 – 8am-3pm

Join us for an exclusive, hybrid workshop where multidisciplinary experts come together to explore groundbreaking research, spark collaboration, and chart the future of hematopoietic stem cell transplantation (HCT) and gene therapy in sickle cell disease. This workshop is an opportunity to be at the forefront of scientific discovery and innovation, helping shape the treatment and care of patients experiencing pulmonary complications.

Objectives:

1. Showcase the latest research breakthroughs funded under NOT-HL-20-761, focusing on pulmonary complications of HCT in childhood.
2. Foster collaboration to expand the scientific scope to include adults, devices, advanced analytics, and experimental human studies under NOT-HL-23-116.
3. Identify knowledge gaps to guide future research on pulmonary complications linked to HCT and gene therapy in sickle cell disease.

Need and Justification: 

With approximately 20,000 HCT procedures performed annually in the USA, and the potential widespread availability of gene therapy for sickle cell disease, pulmonary complications pose a serious challenge across all age groups. Notably, HCT in children often results in severe pulmonary complications, the leading cause of mortality, whereas adults face respiratory insufficiency and ARDS, contributing to long-term pulmonary morbidity.

The urgency to investigate pulmonary diseases is underscored by their high incidence and impact on treatment outcomes. Understanding acute chest syndrome and pulmonary hypertension in adults with sickle cell disease is crucial. Our collaboration aims to tackle these complex issues, leveraging cross-disciplinary expertise for improved outcomes and quality of life.

Who Should Attend:

• Pulmonologists, intensivists, transplant physicians, hematologists, imaging experts, and biomedical scientists specializing in acute and late pulmonary complications post-HCT and sickle cell disease.
• Trainees awarded K and F grants, focusing on HCT, gene therapy, bioinformatics, and hematological diseases.
• Researchers in bioinformatics, device development, gene therapy, and human studies.

Format: 

Hybrid workshop with select in-person and virtual participation, fully accessible to the public by NIH videocast.