Showing 4 ideas for tag "transplant"

Goal 3: Advance Translational Research

Dissemination & Implementation of new treatments and therapies in sickle cell disease

Are current advances in gene editing, new drug therapies and less restrictive BMT criteria being explained and rolled out to the sickle cell community in an effective and timely manner? When can people living with sickle cell disease experience a better quality of life on more permanent based on the treatments we already have?

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

Bone marrow transplant criteria has become less restrictive yet there has not been a steep increase in procedures. My understanding is that a sibling or child with the trait can be a donor. At some point this treatment needs to become more widely accessible and discussed with all patients by their doctors.

Name of idea submitter and other team members who worked on this idea Sickle Cell Warriors, Inc.

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Goal 3: Advance Translational Research

Don’t ask, don’t tell; the hidden costs of heart, lung & stem cell transplantation:Financial burden on transplant survivors

Thousands of patients get cured of their diseases with organ and/or hematopoietic stem cell transplantation (HSCT), but what happens after that to their social and financial life? Drug costs, multiple tests, travel to transplant centers, and many other factors cost a fortune to the patients (and their insurers). Can the NHLBI provide systems based approach longitudinally to alleviate the long term cost burden on patients... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

The number and indications for heart and lung transplant is increasing in the US. Number and indications for HSCTs in the US are increasing at an unprecedented rate with currently more than 20000 HSCTs being performed annually in the US alone. Most of the heart and lung transplant patients are on immunosuppressants for life which are responsible for significant out of pocket costs as a direct result or due to the complications of the drug therapy. Most of the patients with steroid refractory GVHD are also on immunosuppressants for many years post HSCT. Many patients file bankruptcy. QOL is significantly affected by severe impact of financial drainage.

The billed charges for heart, single lung, double lung and allogeneic stem cell transplant (2014 estimates) were $1,242,000, $785,000, $1,037,700 and $930,600 respectively (Milliman 2014 report). Now these figures widely differ from the published costs of transplantation, a figure some may quote to the patients’ e.g. the cost of allogeneic HSCT being $200,000 or more. To address the deficiencies in these transparencies is a big challenge since many factors play a role. These costs are for the first few months of transplants and represent only tip of the iceberg. Longitudinal assessment of both indirect and direct costs in transplant survivors is the first step towards devising strategies which can prevent financial toxicities in patients and as an indirect effect, would have a deep impact on private insurers and Medicare.

Feasibility and challenges of addressing this CQ or CC

It is feasible to evaluate the long term financial burden on transplant recipients by well conducted prospective studies. BMT-CTN which is funded by NHLBI has already started a clinical trial (1101) in which ancillary Cost-Effectiveness Analysis data is being assimilated, and out of pocket costs, productivity, and QOL data is being collected. Such a study is imperative to do in transplant arena where costs are climbing at an unparalleled rate with no check points and patients continues to suffer. This would indicate the health care burden and the specific issues in transplant survivors which can help in formulating systems based practice both at individual and health policy level.

Name of idea submitter and other team members who worked on this idea Shahrukh Hashmi

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Goal 3: Advance Translational Research

Developing animal models of lung transplantation.

Lung transplantation as a cure for terminal lung disease has seen little improvement in outcomes for more than 20 years. The field remains highly challenging, in part, because of an absence of robust animal models which are technically- feasible and reproducible across centers. Further, models have limited relevance to clinical (chronic) airway remodeling, the leading problem in pulmonary allografts. In the absence of... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

The critical challenge is to address the leading problems facing lung transplant patients through the creative application of multiple technological platforms employing all available pre-clinical models by multiple NIH investigators. With greater focus on the deeper development of existent models, the delineation of their strengths and limitations and importantly, cooperation between Academic Centers, efforts can be optimized to improve outcomes for a condition that has enjoyed little benefit from basic research.

Feasibility and challenges of addressing this CQ or CC

The siloed nature of much clinical and experimental lung transplantation research limits progress and broader initiatives. With specific respect to developing animal models of lung transplantation, the general lack of consensus about the suitability of the techniques employed at different institutions stifles progress. A strategic vision, guided by leaders across the field, highlighting benefits and limitations of current animal models can be coupled with a consensus statement about the most pressing issues in lung transplantation worthy of increased investigation.

Name of idea submitter and other team members who worked on this idea Mark Nicolls

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Goal 3: Advance Translational Research

Translational Research for HIV/AIDS and HLB Health and Diseases

What are the best inroads for the NHLBI to support innovative approaches in the next 5-10 years, especially blood cell therapies based on hematopoietic stem cell and novel gene therapy approaches to control or even cure HIV infection?

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

HIV control or possibly even HIV cure could result from developing novel cell therapies, especially hematopoietic stem cell (HSC) transplants, and might also result from early use of antiretroviral therapy in acutely HIV-infected individuals.
• Transplantation of HSC including engineered cells has the potential to eradicate HIV reservoirs for HIV cure: the Berlin patient treated with HSC transplant remains free of HIV and is still the only patient cured of HIV infection as of today;
• Identification of acute HIV infections through routine blood donor screening and early anti-retroviral therapy for identified HIV-infected donors can limit or even prevent the establishment of HIV reservoirs.

Feasibility and challenges of addressing this CQ or CC

• The Berlin patient has provided the proof of concept that HIV infection can be eradicated, that is, sterilizing cure can be achieved, through HSC transplantation in combination with other therapies;
• Recent studies have shown that early identification of HIV infection and treatment of infected individuals with anti-retroviral therapy as soon as possible can significantly limit the size of the HIV reservoirs even if such early treatment may not be able to completely prevent the establishment of HIV reservoirs; routine blood donor screening for both anti-HIV antibodies and HIV RNA among blood donors offers unique opportunities to identify acute HIV infections.

 

For HIV cure, the challenges include:

• Generation of HIV-resistant HSCs in adequate quantity for transplantation;

• Efficiency of homing and expansion of HIV-resistant HSC transplants;

• Efficiency in replacing HIV-infected cells, including CD4+ resting cells as the major HIV reservoirs, with HIV-resistant HSCs following transplantation;

• Efficiency in immune reconstitution by HSC transplants;

• Safety of HSC transplantation with needed GVHD to eliminate HIV-infected resting T cells while avoiding irreversible damage to the host.

Name of idea submitter and other team members who worked on this idea NHLBI Staff

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