Key Points TTP in Jehovah’s Witness patients has been managed successfully without PEX. This experience, plus new TTP treatments, may make it possible for patients who are not Jehovah’s Witnesses to avoid PEX in the future.
Chronic lymphocytic leukemia is the most common leukemia in the United States. It is a slowly progressive disease, with an 82% five-year survival rate. The treatment strategies are highly individualized with patients in the early and stable stages typically not requiring treatment. However, those with progressive or clinically advanced disease will require treatment. Cytotoxic drugs, such as the alkylating agents, purine nucleoside antagonists, and immunotherapeutic agents, have been the mainstay of chemotherapeutic treatment in CLL. However, given the lack of therapeutic specificity, these medications (especially older ones) have limited tolerability due to side effects. In this paper, we will discuss the data on the use of phosphatidylinositol 3 kinase inhibitor Idelalisib in the management of patients with chronic lymphocytic leukemia. The preclinical and clinical data thus far demonstrate that Idelalisib produces a dramatic and durable response in patients with chronic lymphocytic leukemia and without causing significant toxicity. Moving forward, the ongoing clinical trials will help address the various questions currently being raised regarding the long-term application and safety of Idelalisib. With greater clinical experience following more widespread use of Idelalisib, we will be able to determine the optimal combination therapies in treatment-naïve and relapsed/refractory patients, resulting in more individualized therapeutic strategies for patients with chronic lymphocytic leukemia.
The relationship between CAG repeat length and age at nursing home (NH) admission and age at percutaneous endoscopic gastrostomy (PEG) was examined in 47 residents of a specialized Huntington's disease long-term care facility who were observed for a median of 3 years (range, 1 to 11 years). CAG repeat length was inversely correlated with time to NH admission and PEG (both p < 0.001), independent of age at onset. CAG repeat length may influence disease progression to late-stage outcomes in addition to age at onset.
Primary myelofibrosis (PMF) is the most aggressive type of chronic myeloproliferative neoplasm, characterized by a disarray of hematopoietic stem cells and bone marrow fibrosis. The estimated incidence is 1.5 per 100,000 individuals per year with a median survival of less than six years. This statistic can vary by risk category, primarily based on clinical and cytogenetic features. Death can result from many causes, including leukemic transformation, cachexia, vascular events, and infection. Currently, allogeneic hematopoietic cell transplant is the only curative method for those at high risk. Unfortunately, only about 10% are eligible for this therapy. JAK2 kinase inhibitors are commonly used for high-risk patients with symptomatic splenomegaly or systemic symptoms from PMF. In clinical trials, the major endpoint is a reduction of spleen size by 35%. Secondary endpoints have included amelioration of symptomatic PMF and overall survival, which can be difficult to determine because of frequent co-morbid conditions. Current Food and Drug Administration (FDA)-approved JAK2 inhibitors have not shown increased survival or reduced risk of leukemic transformation. In relapsed or refractory disease, there is currently no standard of care. In this paper, we discuss the role of a new anti-apoptotic B cell leukemia 2 (Bcl-2) inhibitor, Navitoclax, for the treatment of myelofibrosis. The clinical data thus far for Navitoclax, especially in synergistic combination with traditional JAK2 inhibitors, have been promising for those with a refractory or relapsing disease on prior therapies. Following the encouraging results of phase II trials, ongoing phase III trials will primarily evaluate splenic size reduction versus the standard of care and evaluate secondary endpoints such as symptom reduction and overall survival. These studies may establish a new standard of care for refractory or relapsed myelofibrosis.
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