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.
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.
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.
For those who did not practice hematopoietic cell transplantation (HCT) at the time, it may be difficult to appreciate the impact that mobilized peripheral blood stem cells (PBSC) have had on the pace of hematologic recovery. In the days before mobilized PBSC, count recovery before day +21 was uncommon, and prolonged neutropenia resulted in a risk of death from infection that exceeded 5%.