The goals of therapy are to control symptoms, improve quality of life, improve overall survival, and decrease progression to AML. The IPSS scoring system can help triage patients for more aggressive treatment (i.e. bone marrow transplant) as well as help determine the best timing of this therapy. Supportive care with blood products and hematopoietic growth factors (e.g. erythropoietin) is the mainstay of therapy. The regulatory environment for the use of erythropoietins is evolving, according to a recent US Medicare National coverage determination. However, no comment on the use of hematopoietic growth factors for<unk>  was made in that document. Agents have been approved by the U.S. Food and Drug Administration (FDA) for the treatment of<unk> : # 5-azacytidine: 21-month median survival # Decitabine: Complete response rate reported as high as 43%. A phase I study has shown efficacy in AML when decitabine is combined with valproic acid. # Lenalidomide: Effective in reducing red blood cell transfusion requirement in patients with the chromosome 5q deletion subtype of<unk>  # Decitabine/cedazuridine (Inqovi) is a fixed-dosed combination medication for the treatment of adults with<unk> s <unk>  and chronic myelomonocytic leukemia (CMML). Chemotherapy with the hypomethylating agents 5-azacytidine and decitabine has been shown to decrease blood transfusion requirements and to retard the progression of<unk>  to AML. Lenalidomide was approved by the FDA in December 2005 only for use in the 5q-<unk> . In the United States, treatment of<unk>  with lenalidomide costs about $9,200 per month. The chemotherapy may be supported by other drugs like all-trans retinoic acid (ATRA), however the evidence of benefit is not clear. HLA-matched allogeneic stem cell transplantation, particularly in younger (i.e. less than 40 years of age) and more severely affected patients, offers the potential for curative therapy. The success of bone marrow transplantation has been found to correlate with severity of<unk>  as determined by the IPSS score, with patients having a more favorable IPSS score tend to have a more favorable outcome with transplantation. If patients receive a stem cell transplant, they can develop a graft-versus-host disease (GvHD). The evidence is very uncertain about the therapeutic effect of mesenchymal stromal cells to treat graft-versus-host diseases after a stem cell transplantation on the all-cause mortality and complete disappear of chronic acute graft-versus-host diseases. Mesenchymal stromal cells may results in little to no difference in the all-cause mortality, relapse of malignant disease and incidence of acute and chronic graft-versus-host diseases if they are used for prophylactic reasons. Patients that receive a stem cell transplant or a chemotherapy as a treatment might have a higher risk for bleeding and may require platelet transfusions. Physical exercises in addition to the standard treatment for adult patients with hematological malignancies may result in little to no difference in the mortality, in the quality of life and in the physical functioning. These exercises may result in a slight reduction in depression. Furthermore, aerobic physical exercises probably reduce fatigue. The evidence is very uncertain about the effect on anxiety and serious adverse events. Iron levels Iron overload can develop in<unk>  as a result of the RBC transfusions, which are a major part of the supportive care for anemic<unk>  patients. A particular risk exists of delayed engraftment and a need for repeated red-cell transfusion following HLA-identical allogeneic stem-cell transplantation if the recipient is blood type O and the stem-cell donor, type A. Although the specific therapies patients receive may alleviate the RBC transfusion need in some cases, many<unk>  patients may not respond to these treatments, thus may develop secondary hemochromatosis due to iron overload from repeated RBC transfusions. Patients requiring relatively large numbers of RBC transfusions can experience the adverse effect of chronic iron overload on their liver, heart, and endocrine functions. The resulting organ dysfunction from transfusional iron overload might be a contributor to increased illness and death in early-stage<unk> . For patients requiring many RBC transfusions, serum ferritin levels, number of RBC transfusions received, and associated organ dysfunction (heart, liver, and pancreas) should be monitored to determine iron levels. Monitoring serum ferritin may also be useful, aiming to decrease ferritin levels to .Currently, two iron chelators are available in the US, deferoxamine for intravenous use and deferasirox for oral use. These options now provide potentially useful drugs for treating this iron overload problem. A third chelating agent is available in Europe, deferiprone, for oral use, but is not available in the US. Clinical trials in the<unk>  are ongoing with iron chelating agents to address the question of whether iron chelation alters the natural history of patients with<unk>  who are transfusion dependent. Reversal of some of the consequences of iron overload in<unk>  by iron chelation therapy has been shown. Both the<unk>  Foundation and the National Comprehensive Cancer Network<unk>  Guidelines Panel have recommended that chelation therapy be considered to decrease iron overload in selected<unk>  patients. Evidence also suggests a potential value exists to iron chelation in patients who will undergo a stem-cell transplant. Although deferasirox is generally well tolerated (other than episodes of gastrointestinal distress and kidney dysfunction in some patients), recently a safety warning by the FDA and Novartis was added to deferasirox treatment guidelines. Following postmarketing use of deferasirox, rare cases of acute kidney failure or liver failure occurred, some resulting in death. Due to this, patients should be closely monitored on deferasirox therapy prior to the start of therapy and regularly thereafter.
Myelodysplastic syndrome
Primary myelofibrosis
Acute myeloid leukemia
Myeloid leukemia
Autoimmune neutropenia
Mixed-phenotype acute leukemia
Childhood leukemia
B-cell leukemia