Charles Parker, MD,[1] Keith Hoots, MD,[2] Armand Keating, MD [3]

2012-09-01

1. Professor of Medicine, Division of Hematology and Hematologic Malignancies, University of Utah School of Medicine

2. Director, Division of Blood Diseases and Resources, National Heart, Lung, and Blood Institute

3. ASH President, Professor of Medicine, Director, Division of Hematology, Epstein Chair in Cell Therapy and Transplantation; Professor, Institute of Biomaterials and Biomedical Engineering, University of Toronto

Is “benign hematology” in the United States a subspecialty that deals with diagnosis and management of a loose collection of uncommon/rare diseases (e.g., thalassemias, hemoglobinopathies, and inherited bleeding disorders), or one that covers those diseases together with some of the country’s most common disorders (e.g., complications of arterial and venous thromboembolism and anemia)? This is one of many questions that was addressed at a workshop convened by the National Heart, Lung, and Blood Institute (NHLBI) on June 8, 2012. The title of the workshop was “Planning for the Training Future of the Workforce in Hematology.” The roster was composed of 28 health professionals from throughout the United States (both pediatric and adult hematology were represented) and 19 National Institutes of Health (NIH) participants. Dr. Keith Hoots, director, Division of Blood Diseases and Resources (DBDR), NHLBI, and Dr. Donna DiMichele, deputy director, DBDR, hosted the meeting that was held on the NIH campus. Dr. Armand Keating, ASH president, Martha Liggett, Esq., ASH executive director, and Mila Becker, Esq., ASH senior director, Government Relations, Practice, and Scientific Affairs, dutifully represented the Society.

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The impetus for the meeting is the perception that the number of physicians whose clinical and research focus is non-malignant hematology is declining. In his introductory remarks, Dr. Hoots presented data in support of that perception. In addition to the problems that plague all areas of academic medicine (the steady decline in NIH appropriations since 2003, the low success rate of grant funding, the high burden of educational debt), Dr. Hoots showed that the number of principal investigators funded through DBDR has declined by more than 50 percent over the past 10 years (Figure 1). What accounts for this precipitous decline in funded investigation in the field? This question generated a wideranging discussion, but there was a consensus that the field’s growth has been stymied by a paucity of career opportunities for those who invest in training in benign hematology.

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According to the model envisioned by Dr. Gil White of the BloodCenter of Wisconsin (Figure 2), entry into the training “pipeline” is discouraged, because, at the other end, career opportunities in benign hematology are limited almost exclusively to academic positions, and those positions must be leveraged through acquisition of highly competitive extramural grant funding as clinical revenue alone is insufficient to support those positions. But most of those who enter hematology/oncology training programs eventually take jobs in private practice, and, currently, positions in the private sector devoted exclusively or even predominately to benign hematology are rare. The idea that this shortage of career opportunities constrained growth in the field resonated with the group and produced a number of suggestions aimed at addressing this critical issue. Much of the discussion focused on how to create job opportunities in the private sector. This issue has been championed by ASH, and Dr. Keating presented a vision of how the unique skills of an expertly trained benign hematologist could be utilized by hospitals to improve patient care while reducing costs through improved efficiency. The position of a “systems hematologist” is seen as somewhat analogous to that of the hospital-based infectious disease specialist who acts in a number of capacities that utilize his/her special skills, including serving as the hospital’s infection control officer and infectious disease epidemiologist and providing oversight on the use of antimicrobial agents. Similarly, the systems hematologist would provide expertise in the use of anticoagulant drugs; oversee the utilization of recombinant prohemostatic drugs; interface with the pathologist in transfusion medicine, laboratory medicine, and bone marrow analysis; and provide support in fetal/maternal hematology and critical care hematology in intensive care units and emergency rooms.

Looking to the future, one can easily imagine the systems hematologist providing expertise in interpretation of the results of whole-genome and whole-exome sequencing, in implementation of cell and gene therapy, and in appropriate use of regenerative medicine technology. Successful implementation of the systems hematologist concept centers on the following factors: 1) delineation of the scope of the specialty and the duties of the specialist; 2) development of comprehensive training programs (incorporating a “cradle-to-grave” component that supports longitudinal management of patients from the newborn period through adulthood); 3) financial and organizational support for the position within target institutions; 4) appropriate reimbursement for services by federal and third-party payers; 5) support from other specialists including pathologists, intensive care specialists, emergency room physicians, and surgeons; 6) and planning for changes that may result from implementation of the Affordable Care Act.

Clearly, research funding will continue to play a critical role in maintaining the vibrancy of the field, as it is the excitement of discovery that attracts the best trainees. Dr. Barry Coller, of Rockefeller University and past-president of ASH, and Dr. David Ginsburg, of the University of Michigan, articulated concern that the current NIH funding situation had changed the profile of those who are most involved in the training component of the pipeline (Fig. 2). Following the law of unintended consequences, the low success rate of NIH funding is removing physician/scientists from the clinic. In order to maintain grant support, physician/scientists must be relieved of clinical responsibilities so that they can devote their energy and imagination to the ever-increasing demands of the laboratory. Consequently, trainees have less exposure to the laboratory-based physician/scientist. For many of our generation, it was that experience of working in the clinic or on the inpatient service with an enthusiastic, creative physician/scientist that inspired us to try our own hand at the bench. That critical element of the training experience is being lost as the percentage of funded grant applications falls into the single digits.

In response to this observation, a proposal was made that consideration be given to structuring a grant program that would be available to those with a substantial clinical commitment (e.g., 20-25%). Such a program would have the added benefit of increasing the pool of physician/scientists that supports NIH’s “K” award programs of mentored research (Fig. 2). Many investigators are lost both during the challenging “K” to “R” transition from mentored trainee to newly independent investigator and the treacherous “R” to “R” transition from newly independent investigator to established investigator. These crucial periods have been recognized within the academic establishment including by ASH and NIH, and creative approaches that support deserving trainees and junior faculty members during these anxious times are under development.

In 1925, Cooley and Lee described five children with skeletal abnormalities, hepatomegaly, splenomegaly, icterus, and “mongoloid” faces.¹ All had anemia with striking morphologic abnormalities observed in the peripheral blood. Even today, in our experience, it is rare that review of the blood film of a patient with thalassemia doesn’t elicit a “wow” from a medical student or house officer sitting with us at the microscope. It’s inspiring to trace the arc of discovery that began with the observations of Cooley and Lee more than 85 years ago to the elucidation of the chemical structure of hemoglobin in the mid- to late-1950s, to the delineation of the molecular biology of thalassemia in the late 1970s and early 1980s, to the present-day discovery of the mechanisms underlying hemoglobin switching that suggest a novel approach to the treatment of two of the world’s most common and most deadly diseases, β-thalassemia and sickle cell disease.²

The field of benign hematology remains vigorous, exciting, and essential, but it must be supported by the infusion of new investigators and new practitioners who have an opportunity to use their special skills. Developing a vision for the field’s future is critical, and DBDR/NHLBI in conjunction with ASH and other public and private stakeholders is providing the leadership and resources that are crucial for moving beyond rhetoric to implementation of meaningful change. Does the management of “benign” diseases such as aplastic anemia, thrombotic thrombocytopenic purpura, and catastrophic anti-phospholipid syndrome benefit from the input of a highly trained specialist? Just ask the patient.

1. Cooley TB, Lee P. A series of cases of splenomegaly in children with anemia and peculiar bone changes. Trans Am Pediatr Soc. 1925;37:29-34.

2. Xu J, Bauer DE, Orkin SH. Targeting regulators of hemoglobin F. The Hematologist: ASH News and Reports. 2011;8:5.

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