Autologous hematopoietic stem cell transplantation (aHSCT) is a well-established treatment for hematologic malignancies such as multiple myeloma (MM) and non-Hodgkin lymphoma (NHL). Various changes in the field over the past decade, including the frequent use of tandem aHSCT in MM, the advent of novel therapies for the treatment of MM and NHL, plus the addition of new stem cell mobilization techniques, have led to the need to reassess current stem cell mobilization strategies.
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.
Hemophilia is a sex-linked genetic disorder characterized by the deficiency or absence of one of the clotting proteins in plasma. Severe deficiency results in spontaneous bleeding into the joints and recurrent bleeding which, in turn, leads to hemophilic arthropathy, disability, and reduced quality of life. Currently, there is no known cure for hemophilia, so treatment goals include preventing bleeding, recognizing bleeding episodes, and providing prompt treatment and intervention to prevent complications.