The goal of the Pilot/Demonstration Progam is to conduct early phase research studies what will advance our knowledge about immune mediated disorders after allogeneic hematopoietic cell transplantation (HCT). We propose three pilot studies focused on clinical/translational research. Additional pilot studies may be funded or may replace these studies, depending on evolving knowledge in the field. Pilot 1 focuses on bronchiolitis obliterans syndrome (BOS) and proposes a small (N=40) clinical trial testing a treatment approach targeting the innate and adaptive immune system using a combination of fluticasone, azithromycin and montelukast. All three agents are relatively nontoxic FDA-approved medications with preliminary data suggesting benefit in both the lung transplant and allogeneic HCT BOS settings. Biomarker studies from bronchoalveolar fluid and blood will complement clinical assessment of response using serial pulmonary function tests. Two other pilot studies currently planned for the later half of the funding period focus on the pathophysiology and treatment of late acute graft versus host disease (GVHD). One is a clinical trial potentially testing alefacept, an agent targeted to memory T cells. The other is a laboratory study to evaluate whether effector or memory T cell populations specific for recipient alloantigens are associated with late acute and classic chronic GVHD. Selection of pilot projects and management of the Pilot/Demonstration Program is the responsibility of the Rare Diseases Clinical Research Consortium (RDCRC) PI with input from other members of Consortium. Criteria for pilot project selection emphasize scientific potential and integration/synergy with other RDCRC initiatives. Mechanisms for quality assurance, data management, Data Safety Monitoring Board oversight and interactions with the entire Consortium will mirror the procedures established for the Projects. We will work closely with NIH program officers to ensure that the Pilot Program is fulfilling the funding intention.

Public Health Relevance

A major commitment of the RDCRC is directed at pilot/demonstration projects. Funding is flexible so that new clinical research opportunities in rare diseases can be exploited. All three of our currently proposed pilot studies focus on clinical/translational research. Additional pilot studies may be funded or may replace these studies, depending on new knowledge gained in immune mediated disorders after allogeneic HCT.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZRG1-HOP-Y)
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Fred Hutchinson Cancer Research Center
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Inamoto, Yoshihiro; Martin, Paul J; Storer, Barry E et al. (2014) Association of severity of organ involvement with mortality and recurrent malignancy in patients with chronic graft-versus-host disease. Haematologica 99:1618-23
Inamoto, Yoshihiro; Flowers, Mary E D; Sandmaier, Brenda M et al. (2014) Failure-free survival after initial systemic treatment of chronic graft-versus-host disease. Blood 124:1363-71
El-Jawahri, Areej; Pidala, Joseph; Inamoto, Yoshi et al. (2014) Impact of age on quality of life, functional status, and survival in patients with chronic graft-versus-host disease. Biol Blood Marrow Transplant 20:1341-8
Inamoto, Yoshihiro; Pidala, Joseph; Chai, Xiaoyu et al. (2014) Assessment of joint and fascia manifestations in chronic graft-versus-host disease. Arthritis Rheumatol 66:1044-52
Palmer, Jeanne; Williams, Kirsten; Inamoto, Yoshihiro et al. (2014) Pulmonary symptoms measured by the national institutes of health lung score predict overall survival, nonrelapse mortality, and patient-reported outcomes in chronic graft-versus-host disease. Biol Blood Marrow Transplant 20:337-44
Kitko, Carrie L; Levine, John E; Storer, Barry E et al. (2014) Plasma CXCL9 elevations correlate with chronic GVHD diagnosis. Blood 123:786-93
Inamoto, Yoshihiro; Kim, Dennis D; Storer, Barry E et al. (2014) Application of CIBMTR risk score to NIH chronic GVHD at individual centers. Blood 123:453-5
Allen, Jessica L; Tata, Prasanthi V; Fore, Matthew S et al. (2014) Increased BCR responsiveness in B cells from patients with chronic GVHD. Blood 123:2108-15
Duarte, R F; Greinix, H; Rabin, B et al. (2014) Uptake and use of recommendations for the diagnosis, severity scoring and management of chronic GVHD: an international survey of the EBMT-NCI Chronic GVHD Task Force. Bone Marrow Transplant 49:49-54
Inamoto, Y; Jagasia, M; Wood, W A et al. (2014) Investigator feedback about the 2005 NIH diagnostic and scoring criteria for chronic GVHD. Bone Marrow Transplant 49:532-8

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