Hematopoietic cell transplantation has become a cornerstone in the management of patients with lymphoma. The use of autologous transplant in Hodgkin’s and non-Hodgkin’s lymphoma has significantly improved the survivals of patients with relapsed disease.
Although myelofibrosis (MF) is the least common of the myeloproliferative neoplasms, it is the most lethal with a median survival of only 3-5 years. Cytopenias and leukemic transformation are the major causes of death. For many years palliation of symptoms with alkylating agents or hydroxyurea was the only therapy available and this had little or no effect on the survival of patients with MF.
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.
During the last two decades, the treatment of multiple myeloma (MM) has produced significant improvements in overall survival and quality of life. This can be attributed to the use of autologous stem cell transplantation, novel drugs in combination with old drugs, bisphosphonates, improved supportive care, and, importantly, dissemination of knowledge about the disease and treatments through multiple societies and medical centers.
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.
In a remarkable Editorial in BLOOD more than 60 years ago, Dameshek coined the term “Myeloproliferative Syndromes” (MPS) to characterize an apparently diverse group of hematologic disorders characterized by myeloid hyperplasia with maturation. Based on the relative degree of erythroid, megakaryocytic and granulocytic proliferation all four of the major myeloproliferative neoplasms (MPN) come into view. Equally remarkable is the description of both primary (PMF) and secondary myelofibrosis (SMF) with speculation regarding a pre-fibrotic stage of PMF.
Mantle cell lymphoma (MCL) and peripheral T-cell lymphomas (PTCL) are rare subtypes of non-Hodgkin’s lymphoma that together comprise <5% of all cases of NHL in adults. Both diseases are associated with poor outcomes with standard front line NLH chemotherapy. Additionally, investigations into new therapies for MCL and PTCL have been historically difficult due to their rarity.
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.
According to the American Cancer Society, it was estimated in 2011 there were 44,600 cases of leukemia, 66,360 cases of non-Hodgkin lymphoma, 8,830 cases of Hodgkin lymphoma, and 20,520 cases of myeloma diagnosed for the first time. In addition, the Leukemia and Lymphoma Society estimates that one person is diagnosed with a blood cancer approximately every four minutes in the U.S. The outcome for patients with hematologic malignancies has dramatically changed with the use of novel therapies.