TAILIEUCHUNG - Chapter 099. Disorders of Hemoglobin (Part 14)

Deferasirox is a promising oral iron-chelating agent. Single daily doses of 20 or 30 mg deferasirox produced reductions in liver iron concentration comparable to desferoxamine in chronically transfused adult and pediatric patients. Deferasirox produces some elevations in liver enzymes and slight but persistent increases in serum creatinine, without apparent clinical consequence. Other toxicities are similar to those of desferoxamine. Its toxicity profile is acceptable, although long-term effects are still being evaluated. Bone Marrow Transplantation, Gene Therapy, and Manipulation of HbF Bone marrow transplantation provides stem cells able to express normal hemoglobin; it has been used in a large number of patients. | Chapter 099. Disorders of Hemoglobin Part 14 Deferasirox is a promising oral iron-chelating agent. Single daily doses of 20 or 30 mg deferasirox produced reductions in liver iron concentration comparable to desferoxamine in chronically transfused adult and pediatric patients. Deferasirox produces some elevations in liver enzymes and slight but persistent increases in serum creatinine without apparent clinical consequence. Other toxicities are similar to those of desferoxamine. Its toxicity profile is acceptable although long-term effects are still being evaluated. Bone Marrow Transplantation Gene Therapy and Manipulation of HbF Bone marrow transplantation provides stem cells able to express normal hemoglobin it has been used in a large number of patients with 0-thalassemia and a smaller number of patients with sickle cell anemia. Early in the course of disease before end-organ damage occurs transplantation is curative in 80-90 of patients. In highly experienced centers the treatment-related mortality is 10 . Since survival into adult life is possible with conventional therapy the decision to transplant is best made in consultation with specialized centers. Gene therapy of thalassemia and sickle cell disease has proved to be an elusive goal. Uptake of gene vectors into the nondividing hematopoietic stem cells has been inefficient. Lentiviral-type vectors that can transduce nondividing cells may solve this problem. Reestablishing high levels of fetal hemoglobin synthesis should ameliorate the symptoms of 0-thalassemia. Cytotoxic agents such as hydroxyurea and cytarabine promote high levels of HbF synthesis probably by stimulating proliferation of the primitive HbF-producing progenitor cell population . F cell progenitors . Unfortunately no regimen has yet been identified that ameliorates the clinical manifestations of 0-thalassemia. Butyrates stimulate HbF production but only transiently. Pulsed or intermittent administration has been found to sustain HbF .

• Y học thường thức
• Disorders of Hemoglobin
• bệnh học và điều trị
• bài giảng bệnh học
• tài liệu học ngành y
• Harrisons Internal Medicine
• BMC Musculoskeletal Disorders
• Intertrochanteric fractures
• Unstable fractures
• Hemoglobin drop
• Perioperative complications
• AO classification
• medical books
• pharmacology
• epidemiologic
• Obstetrics
• anatomy
• Preventing Morbidity
• Multidisciplinary Care
• Total knee arthroplasty
• Sedative hypnotics
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• Locomotive syndrome risk
• Musculoskeletal organs
• Glycated hemoglobin
• Total protein
• Tranexamic acid
• Intravenous injection
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• Articular cartilage
• Synovial cells
• Thrombospondin motifs
• Aggrecan degradation
• Early stage osteoarthritis