Goal 3: Advance Translational Research

Bone Marrow Stem Cell Transplant in Peds sibling matched SCD

There is a need to improve accessibility of Bone Marrow Stem Cell Transplantation (BMSCT) for Sickle Cell Disease patients who are most likely to benefit from this treatment option. 1. Building a culture of trust between and among primary care providers, specialists, patients/families, and other stakeholders 2. Consensus building around BMSCT as an acceptable treatment alternative (as opposed to another research endeavor) ...more »

Submitted by (@nhlbiforumadministrator)

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Goal 3: Advance Translational Research

How can we develop more selective immunosuppression for allogeneic hematopoietic cell transplantation?

Graft versus host disease (GVHD) remains the most significant complication of allogeneic hematopoietic stem cell transplantation (HCT). While the use of HCT has grown significantly safer and has demonstrated broad efficacy in the setting of a broad range of blood diseases, immunosuppressive therapy has not dramatically evolved since the introduction of calcineurin inhibitor-based approaches decades ago. The availability ...more »

Submitted by (@kkomanduri)

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Goal 3: Advance Translational Research

Modulating Immune Checkpoints to Enhance Curative Potential of Autologous Transplantation in classical Hodgkin lymphoma (cHL)

PD-L1 and PD-L2, engage PD-1 receptors on activated T cells and induce T-cell exhaustion. Antibody-mediated PD-1 blockade has already been successfully exploited as a therapeutic strategy in solid tumors. PD-L1 is expressed on suppressor immune cells in the tumor microenvironment and in cHL tumors. PD1 blockade has shown clinical activity in cHL. Disease relapse, post autoHCT; however remains the most common cause of ...more »

Submitted by (@mehdihamadani)

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Goal 2: Reduce Human Disease

The role of Extracorporeal Photopheresis (ECP) in the prophylaxis and treatment of acute & chronic Graft Versus Host Disease

In Acute Graft Versus Host Disease (aGVHD), we would like to examine whether early and intensified delivery of ECP as part of standard prophylaxis will decrease overall corticosteroid exposure while preserving expected relapse rates in patients undergoing unrelated donor hematopoietic stem cell transplantation (HSCT). Chronic GVHD (cGVHD) is common after HSCT (30-50% recipients) and is a major contributor to late transplant-related ...more »

Submitted by (@js2745)

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Goal 2: Reduce Human Disease

Lung Transplantation

Although the majority of lung recipients experience significant health improvement, they also frequently face serious symptom distress, impaired physical functioning and poor quality of life due to post-transplant morbidity, such as chronic rejection, infection and multiple side-effects of immunosuppression. a) Conduct clinical trials of interventions designed to maximize clinicians' support of patients' self-management ...more »

Submitted by (@nhlbiforumadministrator)

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Goal 1: Promote Human Health

Host and environmental factors effect on transplantation biology and regenerative medicine

How does inflammation and cell injury induce the immune system during transplant rejection? How do we control immune responses to enable cell- and tissue-regenerative strategies? • How does inflammation and cell injury induce the immune system during transplant rejection? • How do we control immune responses to enable cell and tissue regenerative strategies? • How do we use iPS cell based and gene editing based therapies ...more »

Submitted by (@nhlbiforumadministrator)

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Goal 2: Reduce Human Disease

Can we break the silos at NHLBI? Why are we not working on studiying heart and lung issues in blood cancer survivors?

There is an increasing number of blood cancer survivors in the United States. Many of them have treatment induced heart and lung comorbidities (i.e CHF, pulmonary fibrosis, early aging, etc). However, there does not seem to be a concerted effort by the NHLBI to leverage their relationship with the NCI or the BMT CTN to address this issue. NHLBI should be developing a funding mechanism for cardiopulmonary researchers to ...more »

Submitted by (@giralts)

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Goal 3: Advance Translational Research

Achieving Transplantation Tolerance in Recipients of Heart and Lung Allografts

Despite improvements in the early post-transplant survival of thoracic organs, registry data show that the graft half-life is only 11 years for heart recipients and 5 years for lung recipients. Infection accounts for 33% of cardiac and 40% of lung transplant recipient death between day 31 and one year post transplant. After 5 years, cardiac allograft vasculopathy (30%), and malignancy (23%) cause most cardiac recipient ...more »

Submitted by (@nhlbiforumadministrator)

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