Current and emerging therapies in primary myelofibrosis.

Current and emerging therapies in primary myelofibrosis. Cardiovasc Hematol Disord Drug Targets. 2012 Sep;12(1):6-20 Authors: Ayyappan S, Janakiram M, Raghupathy R Abstract Primary myelofibrosis is a clonal hematopoietic disorder characterized by ineffective hematopoiesis and progressive bone marrow fibrosis. Patients with high risk myelofibrosis as determined by their advanced age, degree of anemia, leukocytosis, constitutional symptoms and high percentage of circulating blasts have a very short median survival of 2 years. In addition quality of life is significantly compromised due to cytokine induced constitutional symptoms, frequent transfusion for cytopenias and bulky splenomegaly. Progression to myelogenous leukemia occurs in about 20% of patients within 10 years of diagnosis and is often fatal. Allogeneic hematopoietic transplantation is the only curative therapy but is limited by patient eligibility, transplant related mortality and graft versus host disease. Androgens, erythropoietin analogues, hydroxyurea, alkylators and spleen directed therapies have all been used with limited efficacy and no curative potential. The discovery of mutations in the hematopoietic progenitors of patients with myelofibrosis, including the JAK2 V617F mutation and others has greatly improved our understanding of the disease and facilitated development of newer targeted therapies. Our article will review new discoveries in the pathogenesis of myelofibrosis and focus on ...
Source: Cardiovascular and Hematological Disorders Drug Targets - Category: Drugs & Pharmacology Tags: Cardiovasc Hematol Disord Drug Targets Source Type: research

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ConclusionWe suggest a one ‐compartment population model with first‐order elimination to capture the pharmacokinetic profile for basiliximab for this patient population.
Source: Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy - Category: Drugs & Pharmacology Authors: Tags: ORIGINAL RESEARCH ARTICLE Source Type: research
The evolution of Allogeneic Haematopoietic Stem Cell Transplant (allo-HSCT) as a treatment modality has witnessed the cure of several haematological conditions such as leukaemia, lymphoma, multiple myeloma, myelodysplastic syndromes, thalassaemia and aplastic anaemia [1]. The many advances made in this domain, and the availability of excellent post transplant care have ensured increased longevity in recipients of allo-HSCT. It has been demonstrated in previous studies that patients who are disease-free at five years after HSCT have a 10-year survival rate that exceeds 80% [2].
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
The evolution of allogeneic hematopoietic stem cell transplant (allo-HSCT) as a treatment modality has witnessed the cure of several hematologic conditions such as leukemia, lymphoma, multiple myeloma, myelodysplastic syndromes, thalassemia, and aplastic anemia [1]. The many advances made in this domain and the availability of excellent post-transplant care have ensured increased longevity in recipients of allo-HSCT. It has been demonstrated in previous studies that patients who are disease free at 5 years after HSCT have a 10-year survival rate that exceeds 80% [2].
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
A 45-year-old woman with Usher syndrome, associated congenital deafness, progressive blindness due to retinitis pigmentosa, and latent autoimmune diabetes presented to the emergency department with malaise, dizziness, and pelvic pain following removal of an intrauterine device. A posterior vaginal wall mass was found on examination. Laboratory values demonstrated anemia, thrombocytopenia, and an elevated white blood cell count, raising concern for infection and potential onset of diabetic ketoacidosis. This prompted a peripheral blood smear review, which showed 60% monocytic blasts. A subsequent vaginal mass biopsy showed ...
Source: Pathology Case Reviews - Category: Pathology Tags: Case Reviews Source Type: research
Granulocyte colony-stimulating factor (G-CSF), a growth factor for neutrophils, has been successfully used for stem cell mobilization and T cell immune tolerance induction. The establishment of G-CSF-primed unmanipulated haploidentical blood and marrow transplantation (The Beijing Protocol) has achieved outcomes for the treatment of acute leukemia, myelodysplastic syndrome, and severe aplastic anemia with haploidentical allografts comparable to those of human leukocyte antigen (HLA)-matched sibling donor transplantation. Currently, G-CSF-mobilized bone marrow and/or peripheral blood stem cell sources have been widely used ...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Prognostic refinement in Fanconi anemia (FA) is needed, especially when considering allogeneic hematopoietic stem cell transplantation (HCT). We studied 20 children with FA and bone marrow failure from a single center. According to Hôpital Saint-Louis risk classification for FA, patients were classified in stage A (no or mild cytopenia/dysplasia), B (single non–high-risk cytogenetic abnormality), C (severe cytopenia and/or significant dysplasia and/or high-risk cytogenetic abnormality), and D (myelodysplastic syndrome with excess of blasts/acute myeloid leukemia) in 4, 2, 13, and 0 cases, respectively. Nine pat...
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Original Articles Source Type: research
Acute myeloid leukemia (AML) represents 80% of adult leukemias and 15–20% of childhood leukemias. AML are characterized by the presence of 20% blasts or more in the bone marrow, or defining cytogenetic abnormalities. Laboratory diagnoses of myelodysplastic syndromes (MDS) depend on morphological changes based on dysplasia in peripheral blood and bone marrow, including peripheral blood smears, bone marrow aspirate smears, and bone marrow biopsies. As leukemic cells are not functional, the patient develops anemia, neutropenia, and thrombocytopenia, leading to fatigue, recurrent infections, and hemorrhage. The genetic b...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
stic Anemia Working Party of the European Society for Blood and Marrow Transplantation Abstract This follow-up study of the randomized prospective trial included 192 patients with newly diagnosed severe aplastic anemia receiving Antithymoglobulin and Cyclosporine, with and without G-CSF. We aimed to evaluate the long-term effect of G-CSF on overall survival, event-free survival, probability of secondary myelodysplastic syndrome or acute myeloid leukemia, clinical paroxysmal nocturnal hemoglobinuria, relapse, avascular osteonecrosis and chronic kidney disease. The median follow-up was 11.7 years (10.9-12.5). Overal...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
In October 2015, a 58-year-old male was presented to Hematology, Oncology and Stem Cell Transplantation Research Center; Tehran University of Medical Sciences, Tehran, Iran, with complaints of bone pain and decreased urine volume about 2 months prior to admission. Initial laboratory findings demonstrated a normocytic, normochromic anemia with a hemoglobin of 8.2 g/dL, a white blood cell count (WBC) of 6,500 cell/mm3, and a platelets count of 245,000/mm. He had a creatinine of 4.2 mg/dL, calcium of 12.5 mg/dL, albumin of 3.7 g/dL, total protein of 11.7 g/dL, Beta-2 microglobulin of 13.8mg/L, and an erythrocyte sedimentation...
Source: Clinical Lymphoma, Myeloma and Leukemia - Category: Hematology Authors: Source Type: research
Advances in multiple myeloma (MM) treatment, including novel agents and autologous stem cell transplant, have led to improved outcomes in survival. However, these treatment regimens are time intensive, complex and length of treatment is indefinite. Supportive care for complications including bone disease, hypercalcemia and anemia is frequently needed. Given this treatment burden, access to care may be influential in survival.
Source: Clinical Lymphoma, Myeloma and Leukemia - Category: Hematology Authors: Source Type: research
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