Click the "Start Activity" button to indicate you have reviewed the CME information for this activity. Activity Speakers Sergio A. Giralt, MD Program Chair Professor of Medicine Weill Cornell College of Medicine Chief Attending, Adult BMT Service Memorial Sloan...

Aplastic anemia (AA), the myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare diseases that all result in bone marrow failure—the ineffective formation of circulating blood cells—leading to anemia, bleeding, infection, and death in many cases, even with modern therapies. Once thought of as distinct, these three diseases are now believed to be linked by similar pathophysiologic pathways. Of the three bone marrow failure diseases, MDS currently has the largest number of therapeutic drugs available, although none of them is curative. Much of the current confusion and controversy in MDS treatment stems from the lack of consensus on which therapies to use in which patients, and what realistic outcomes might be.

Pre-transplant conditioning can significantly influence post-transplant outcomes. A strong anti-tumor effect achieved by myeloablative conditioning (MAC) is frequently counterbalanced by higher morbidity and non-relapse mortality, particularly in older adults. On the other hand, reduced intensity conditioning (RIC) is associated with lower post-transplant mortality, but may not be sufficient enough to prevent relapse, particularly in patients with persistent and aggressive malignances.

Bloodline Reviews™ presents "Highlights from the 2012 Bone Marrow Failure Scientific Disease Symposium," held March 22-23, 2012 in Bethesda, MD, USA Expert faculty interviews were recorded on March 23, 2012, at the "Bone Marrow Failure Disease Scientific Symposium," a...

Blood and Marrow Transplantation Reviews: Volume 19, Issue 1 Multiple Choice Questions: Treatment Options for Multiple Myeloma and Myelodysplastic Syndromes by John R. Wingard, MD, Editor The therapeutic prospects for multiple myeloma and the myelodysplastic syndromes...

Although the myelodysplastic syndromes (MDS) are not curable without hematopoietic cell transplant (HCT), advances in non-transplant therapies today offer considerable benefit to our patients. Over the years, prognostic algorithms have been developed and validated and these are useful guides to allow us to more accurately predict the likely trajectory of disease progression in a group of syndromes that have a notorious heterogeneity.

The content of this continuing medical education (CME) activity is derived from a symposium, “The Promise of Epigenetic Therapy,” presented at the 48th Annual Meeting of the American Society of Hematology.

The myelodysplastic syndromes (MDS) have often been called the poster child for apoptosis gone awry: in some cases death (to hematopoietic progenitors) comes too infrequently, in other cases it comes too readily. The quest to better understand its basis and to translate this understanding into therapeutics is only beginning, but already several therapies have emerged and others are in development. For now, there is only one curative therapy for MDS: hematopoietic cell transplantation (HCT).

Management of myelodysplastic syndrome has been disappointing for years. The mainstay approach was only supportive care. A malaise settled all around. Today there are new Food and Drug Administration cleared therapies, and novel agents are on the horizon. True progress has been small to date, but with the number of new therapies and new classes of agents undergoing study, the future is hopeful.

Try as hard as it can, the myelodysplastic bone marrow just cannot seem to meet the ordinary day-to-day needs for blood cells. Indeed, failure is destined no matter what we do. Then, when it seems as bad as it can get, it only gets worse: total marrow shutdown or change into leukemia. Effective therapy has been long overdue.