NHLBI State of the Science Symposium in Transfusion Medicine and Hemostasis/Thrombosis

External Panel for Transfusion Medicine

Summary and Recommendations

On September 14 – 15, 2009, the National Heart, Lung and Blood Institute convened a State of the Science Symposium in Transfusion Medicine and Hemostasis/Thrombosis. The aims of this symposium were to identify Phase II and III clinical trials that could have a significant impact in advancing transfusion therapies or treatment for hemostatic or thrombotic disorders and to provide a forum for investigators to discuss clinical research needs. Oversight committees were established for both transfusion medicine and hemostasis/thrombosis areas. The transfusion medicine group identified seven clinical research areas and the hemostasis/thrombosis group identified six clinical research areas. Subcommittees were formed to explore the clinical research needs and develop clinical trial concepts for each identified area. In addition, two External Panels, one for transfusion medicine and one for hemostasis/thrombosis, were established to evaluate the proposed clinical trial concepts and provide NHLBI with recommendations to develop future clinical research programs. During the first one and a half day open session of the symposium forty-three clinical trial concepts were presented by the thirteen subcommittees. After the open session the two External Panels met separately and each provided recommendations to NHLBI.

The seven Transfusion Medicine subcommittees (Pediatric and Neonatal; Platelet Products: ICU/Trauma; Surgery; Red Blood Cell Conservation; Medical; and Therapeutic Apheresis) prepared 24 clinical trial concepts which are listed at the end of this summary. The Transfusion Medicine External Panel (EP) met, after the presentations, to review the clinical trial concepts and provide recommendations regarding the potential impact of the clinical trials. The clinical trial concepts were evaluated in regard to scientific merit, clinical importance, feasibility, and clinical study environment. NHLBI plans to convene a follow-up meeting to discuss the infrastructure required for these trials.

The summary and recommendations from the External Panel for the Thrombosis and Hemostasis portion of the symposium can be found at: Part I ) The EP positioned the proposals into three tiers: top, middle, and low. In some instances, proposals prepared by different Subcommittees, but with similar themes, were considered jointly:

1. The top tier (n = 10) included clinical trial studies of red cell (TM-105) and platelet (TM-104) indications in at-risk neonates. There are limited data in this area and the studies proposed were important and mature. Studies of prophylactic vs. therapeutic platelet transfusion (TM-101), red cell transfusion indications in the setting of acute coronary syndromes (TM-109, TM-110), pediatric ICU patients (TM-103), and cardiac surgery (TM-108) were placed into this tier. Studies of PI platelets (TM-102, TM-120) were likewise in this tier as was the massive transfusion protocol trial (TM-112). The former studies were considered candidates for industry and the Department of Defense (DOD) support or for partnering, and the latter trial concept of particular interest to the DOD as well.
2. The middle tier (n = 5) included clinical trial studies of FFP transfused for mildly elevated INR for interventional studies in hospitalized patients and particularly in the ICU (TM-106, TM-113). These studies were recognized as important, but in need of more deliberation with particular attention to feasibility issues. Plasma exchange in sepsis (TM-115), and studies of acute normovolemic hemodilution (TM-119), and blood salvage for the cancer patient (TM-117) were also rated as middle tier.
3. In the lower tier (n = 9) were the donor trials referred to above (TM-122, TM-123) as well as studies of hematopoietic growth factors (TM-121), and the trial of moderate brain trauma in patients on anticoagulants or agents that compromise hemostasis (TM-111). There was concern with the latter study that the current agents would no longer be in widespread use by the time any such trial was completed. Also in the lower tier were the clinical trials of FFP in the surgery setting (TM-107), use of cryoprecipitate (TM-114) and P2Y12 inhibitors (TM-118) in cardiac surgery patients, use of platelet stimulating agents in patients with liver disease (TM-124), and use of extracorporeal photopheresis to treat chronic graft-versus-host disease in the hematopoietic stem cell transplant setting (TM-116).

LIST OF PROPOSED TM CLINICAL TRIALS.

Platelet Products Sub-committee

Pediatric and Neonatal Sub-committee

Surgery Sub-committee

ICU/Trauma Sub-committee

Therapeutic Apheresis, Plasma, Cryoprecipitate Sub-committee

Red Blood Cell Conservation and Management Sub-committee

Medical Sub-committee

Last updated: May 4, 2010