Request for Information (RFI): Soliciting input for future research at the NHLBI regarding Late complications after allogeneic hematopoietic stem cell transplantation or autologous gene modified stem cell transplant for non-malignant blood disorders

Notice Number: NOT-HL-18-659

Key Dates
Release Date: October 23, 2018
Response Date: December 17, 2018

Related Announcements
None

Issued by
National Heart, Lung, and Blood Institute (NHLBI)

Purpose

HSCT has been increasingly used to treat non-malignant blood disorders (>2600 transplants/year).  Examples include disorders of red cells and platelets such as sickle cell disease, thalassemia and congenital amegakaryocytosis; disorders of the hematopoietic stem cells such aplastic anemia or other bone marrow failure syndromes, myelodysplastic disorders and myeloproliferative neoplasms; and disorders of the immune system such as severe combined immune deficiencies and Wiskott Aldrich syndrome. Transplantation strategy in these patients includes allogeneic HSCT or autologous gene-modified stem cell transplants. Progressive advances in standard of care have led to an increase in early survival (first year) post-transplant. Unfortunately, survivors often experience late complications (>1 year post transplant) in the cardiac, pulmonary, vascular and/or hematopoietic  system(s) that-, over time, -contribute to increased morbidity and mortality when compared to that of non-transplanted individuals. Additionally, hematopoietic and non-hematopoietic malignancies may occur post-transplant. The underlying  mechanisms of these complications are poorly understood and further research is needed to understand the underlying pathophysiology and develop novel therapies that will help decrease the development of late complications (or their severity). This research would help ensure that the growing population of patients with non-malignant blood disorders who receive a transplant receive optimal care going forward.

Information Requested

NHLBI seeks comments on any, or all of the following topics but not limited to:

  • Nature of the strategies and resource(s) needed to foster research in this area

  • Availability and nature of longitudinally collected institutional data and biospecimens on donors, recipients, and transplant procedures, and continued access to relevant study populations [ patients with non-malignant blood diseases post (>1 yr) transplant as well as appropriate control populations ]

  • Data and biospecimen sharing ability that would allow for the establishment of retrospective cohorts encompassing information from multiple centers and that include patients who have been followed and characterized  for various amounts of time (at least >1 year) post-transplant

  • Scientific research opportunities that could be addressed through the establishment of retrospective cohorts and nature of further prospective characterization to advance research into the pathophysiology underlying the development of cardiac, pulmonary, vascular, hematologic and/or sleep complications  

  • Requirements for the collection, timing and frequency, verification, standardization, storage, quality control metrics, sharing and testing/analysis of data and biospecimens that were retrospectively collected

  • Requirements for the collection, timing and frequency, verification, standardization, storage, quality control metrics, sharing and testing/analysis of data and biospecimens that could potentially be collected prospectively to supplement retrospectively collected information/biospecimens

  • Challenges, resources and partners required for successfully establishing retrospective longitudinally-followed cohorts of transplanted patients
  • Transplant community outreach, engagement and communication strategies
  • The types of data (e.g., clinical, EMR, imaging, PRO) and biospecimens most relevant to the evaluation of the pathophysiology underlying heart, lung, blood and sleep late complications including immune reconstitution and premature aging
  • Screening tests recommended in this population to identify late complications
  • Types of currently available assays, models and associated functional analyses that are amenable to the study of late post HSCT complications, and which would advance our understanding of the role of the genome, proteome, microbiome, epigenome, metabolome, and premature aging in the development of those complications
  • Bioethical considerations in subject recruitment, study design and sharing of data and biospecimens
  • Consideration of demographics, psychosocial, behavioral,  emotional , gender, socio-economic and QOL factors

Submitting a Response

All responses must be submitted via email to PostHSCTDBDR@nhlbi.nih.gov by December 17th, 2018.  Please include the Notice number, NOT-HL-18-659, in the subject line.  The submitted information will be reviewed by NIH staff.

Responses to this RFI are voluntary.  Please do not include any proprietary, classified, confidential, or sensitive information in your response.  NIH will use information submitted in response to this RFI at its discretion and will not provide comments to any responder’s submission.  The collected information will be reviewed by NIH staff, may appear in reports, and may be shared publicly on an NIH web site.

The government reserves the right to use any non-proprietary technical information in summaries of the state of the science, and any resultant solicitation(s).  The NIH may use information gathered by this RFI to inform development of future funding opportunity announcements.

This RFI is for information and planning purposes only and should not be construed as a solicitation or an obligation on the part of the Federal Government, the National Institutes of Health (NIH), or individual NIH institutes and Centers.  NIH does not intend to make any awards based on responses to this RFI or to otherwise pay for preparation of any information submitted or for the Government’s use of such information.  No basis for claims against the U.S. Government shall arise as a result of a response or from the Government’s use of such information.

Inquiries

Please direct all inquiries to:

National Heart, Lung, and Blood Institute (NHLBI)
Telephone: 301-435-0065
Email:  PostHSCTDBDR@nhlbi.nih.gov