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Myelodysplastic Syndrome - General
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Pomalidomide Active for Treatment of Anemia of Myelofibrosis (8/21/2009)
Researchers involved in an international trial have reported that pomalidomide is active for the treatment of anemia associated with myelofibrosis. The details of this study appeared in an early online publication in the Journal of Clinical Oncology on August 3, 2009.
Weight-based Dose Escalation of Aranesp® May Improve Responses in Patients with Low- and Intermediate-risk MDS (3/13/2009)
Researchers from Stanford University have reported that an intra-patient weight-based dose escalation scheme of Aranesp® (darbepoetin) may improve responses in patients with low and intermediate risk myelodysplastic syndromes (MDS). The details of this study appeared in the January 2009 issue of the American Journal of Hematology.
Vidaza® Better Than Conventional Care for High-risk MDS (3/4/2009)
Researchers affiliated with the AZA-001 study have reported that Vidaza® (azacitadine) prolongs survival of patients with higher-risk myelodysplastic syndromes (MDS). The final analysis of this randomized study was published in the March 2009 issue of Lancet Oncology, and[1] preliminary results were presented at the 2007 meeting of the American Society of Hematology.
The American Society of Clinical Oncology 2008: Highlights of Treatment of Hematological Malignancies (1/30/2009)
The 2008 annual meeting of the American Society of Clinical Oncology (ASCO), held in Chicago, Illinois, again revealed advances in the treatment of hematologic malignancies. Patients with chronic or acute leukemias, myelodysplastic syndromes, and myeloproliferative disorders continue to be presented with novel, effective options for the treatment of their diseases.
GVAX Tested as Post-allogeneic Stem Cell Transplant Therapy in Patients with Advanced Myeloid Malignancies (1/13/2009)
Researchers from the Dana Farber Cancer Center have reported that GVAX, a cancer vaccine composed of autologous leukemia cells genetically modified to secrete granulocyte macrophage-colony stimulating factor (GM-CSF), may have anti-leukemic effects when administered after a reduced-intensity allogeneic stem cell transplant in patients with advanced myeloid malignancies. The details of this study were presented at the 2008 meeting of the American Society of Hematology on December 9, 2008, in San Francisco.
New Risk Model for MDS Applies to All Stages of Disease (1/12/2009)
Researchers from several U.S. institutions have proposed a new prognostic model for MDS that includes additional variables and applies to all phases of disease. The details of this proposal were presented at the 2008 meeting of the American Society of Hematology on December 8, 2008 in San Francisco.
Older Age No Barrier to Reduced-intensity Allogeneic Stem Cell Transplants for AML and MDS (1/12/2009)
Researchers affiliated the Center for International Blood and Marrow Transplant Research (CIBMTR) reported that elderly patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) have similar outcomes to younger patients following reduced-intensity allogeneic stem cell transplants. The details of this study were presented on December 8 at the 2008 meeting of the American Society of Hematology in San Francisco.
Oral Chelation with Exjade® Effective Treatment for Iron Overload in Patients with MDS (1/9/2009)
At the 2008 meeting of the American Society of Hematology in December, two studies documented the effectiveness of oral chelation with Exjade® (deferasirox) for treating iron overload in patients with myelodysplastic syndromes (MDS).
Nplate® and Vidaza® Effective for Low- and Intermediate-risk MDS (1/5/2009)
Researchers involved in a U.S. multicenter trial have reported that the combination of Nplate® (romiplostim) and Vidaza® (azacytidine) is effective and well tolerated in patients with low- and intermediate-risk myelodysplastic symdrome. The details of this Phase II study were presented on December 8, 2008 at the 2008 meeting of the American Society of Hematology.
Few Patients with Myelodysplastic Syndromes Entered on Clinical Trials (11/18/2008)
Researchers from the Cleveland Clinic, M. D. Anderson Cancer Center, the H. Lee Moffitt Cancer Center, and Amgen Inc. have reported that only 4% of patients with myelodysplastic syndromes (MDS) were treated on clinical trials. The details of this study appeared in the November 5, 2008 issue of the Journal of the National Cancer Institute.
Vidaza® Improves Survival of Higher-Risk Myelodysplastic Syndromes (MDS) (1/2/2008)
Researchers affiliated with the AZA-001 study have reported that Vidaza (azacitadine) prolongs survival of patients with higher risk myelodysplastic syndrome (MDS). The details of this randomized study were presented at the 2007 meeting of the American Society of Hematology in Atlanta, Georgia in December.
Romiplostim (AMG 531) for Treatment of Thrombocytopenia in MDS (12/27/2007)
Researchers affiliated with The AMG 531 Myelodysplastic Syndrome Study Group have reported that romiplostim (AMG531) can reduce bleeding and transfusion events in patients with myelodysplastic syndromes (MDS). The details of this study were presented at the 2007 meeting of the American Society of Hematology (ASH), December 8-11, 2007 in Atlanta, Georgia.
Umbilical Cord Blood Transplantation with Reduced Intensity Regimen Effective (11/14/2007)
Researchers from the University of Minnesota have reported that adults with hematological diseases have a three year survival of almost 50% following umbilical cord blood transplantation after a reduced intensity treatment regimen. The details of this study appeared in the October 15, 2007 issue of Blood.
Vidaza® Significantly Improves Survival in MDS (8/6/2007)
Researchers involved in a multi-center international trial have reported that Vidaza (5-azacitadine) improves survival in patients with myelodysplastic syndromes (MDS). The preliminary details of this study were announced at a news conference on 8/3/07 and details can be accessed on the Pharmion website. The final report of these data will be submitted for presentation at the 2007 meeting of the American Society of Hematology in December of 2007.
AMG 531 Maybe Effective in Treating Thrombocytopenia in Patients with MDS (6/11/2007)
Researchers affiliated with The AMG 531 in Myelodysplastic Syndrome Study Group have reported that AMG 531 can reduce bleeding and transfusion events in patients with myelodysplastic syndromes (MDS). The details of this study were presented at the 2007 meeting of the American Society of Clinical Oncology in June.
Dacogen™ Has Significant Activity in Chronic Myelomonocytic Leukemia (2/8/2007)
Researchers from M.D. Anderson have reported a 58% complete remission rate and a 19-month median survival for patients with chronic myelomonocytic leukemia (CMML) treated with Dacogen (decitabine).
Mylotarg® and Vidaza® Effective for Elderly with AML or MDS (1/3/2007)
Researchers from Loyola University have reported that the combination of Mylotarg® (gemtuzumab ozogamicin) and Vidaza® (azacitidine) has a high rate of response in elderly patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The details of this study were presented at the 2006 meeting of the American Society of Hematology.
Revlimid® Confirmed Effective for Low and Average Risk MDS (10/5/2006)
Results from a multi-center phase II study indicate that Revlimid (lenalidomide) is highly effective in the treatment of transfusion-dependent myelodysplastic syndromes (MDS) associated with chromosome 5q31 deletion.
Peg Interferon Produces Molecular Responses in Polycythemia Vera (9/14/2006)
Researchers in France have reported that patients with polycythemia vera (PV) respond well to treatment with pegylated interferon alfa-2a including a reduction in the V617FJAK2 (V617F) mutation from an average of 49% to an average of 27%.
Optimal Dacogen™ Regimen for MDS and Chronic Myelomonocytic Leukemia Defined (8/18/2006)
Researchers from M.D. Anderson Cancer Center have reported that a 5 day intravenous schedule of Dacogen (decitabine) was optimal for the treatment of patients with myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML).
Vidaza®/Thalomid® Effective in MDS and AML (6/9/2006)
Results from a pilot study evaluating low doses of Vidaza in combination with Thalomid (thalidomide) provide promising results in the treatment of myelodysplastic syndromes (MDS) and acute myelogenous leukemia (AML). These results were presented at the 42nd annual meeting of the American Society of Clinical Oncology (ASCO).
Vidaza® Provides High Rates of Transfusion Independence in Myelodysplastic Syndromes (6/8/2006)
Retrospective analysis from three studies evaluating Vidaza (azacitadine) have demonstrated high rates of transfusion independence among patients diagnosed with myelodysplastic syndromes (MDS). These results were presented at the 42nd annual meeting of the American Society of Clinical Oncology (ASCO).
Aranesp® Effective for Treating Anemia Associated with ‘Low-Risk’ MDS (5/12/2006)
Researchers from France have reported that Aranesp (darbepoetin) produces an erythroid response in approximately 70% of patients with lower risk myelodysplastic syndrome (MDS).
Aranesp® Effective in Low and Intermediate Risk Myelodysplastic Syndromes (12/7/2005)
Researchers from Italy have demonstrated that long-acting Aranesp (darbepoetin alfa) is as effective as Procrit® (epoetin alfa) for the treatment of patients with low and intermediate risk myelodysplastic syndrome (MDS).
Erythropoietin and Granulocyte-Colony Stimulating Factor Improve Anemia in Patients with MDS. (7/22/2005)
Researchers affiliated with the Nordic Myelodysplastic Syndrome Group have reported that approximately 40% of patients with myelodysplastic syndrome(MDS) treated with Procrit® (epoetin alfa) and granulocyte-colony stimulating factor (G-CSF, Neupogen®) have partial or complete correction of anemia without an increase in the incidence of leukemic transformation or a decrease in survival.
Revlimid™ Highly Effective in Transfusion-Dependent Myelodysplastic Syndromes (5/17/2005)
Results from a multi-center phase II study indicate that Revlimid (lenalidomide) is highly effective in the treatment of transfusion-dependent myelodysplastic syndromes (MDS). These results were presented at a plenary session of the 2005 annual meeting of the American Society of Clinical Oncology (ASCO).
Myeloproliferative Disorders Associated with Tyrosine Kinase Mutation (3/25/2005)
Researchers from the UK have identified an acquired single point mutation as the probable cause of more than half the cases of myeloproliferative disorders. The details of this study appeared in the March 19, 2005 issue of the Lancet.
Revlimid™ Effective for Myelodysplastic Syndromes (2/25/2005)
A multi-center US trial has reported hematologic activity of Revlimid™ (lenalidomide, CC5013) in patients with myelodysplastic syndromes. The details of this report appeared in the February 10, 2005 issue of the New England Journal of Medicine.
Aranesp® Effective in Low-Intermediate Risk MDS (2/16/2005)
Researchers from Italy have reported that Aranesp® (darbepoetin alfa) improves anemia in patients with myelodysplastic syndromes. The details of this report appeared in the January 2005 issue of the British Journal of Hematology.
Zarnestra™ Effective in Poor-Risk MDS (1/31/2005)
A multi-institutional international trial has reported “clinical activity with durable responses and limited toxicity is confirmed for” Zarnestra™ (R115777) in patients with poor-risk myelodysplastic syndromes. These data were presented at the 2004 meeting of the American Society of Hematology in San Diego December 4-7.
Vidaza™: First Drug Approved for Myelodysplastic Syndromes (1/14/2005)
In May of 2004, the U.S. Food and Drug Administration (FDA) approved Vidaza™ (azacitadine) for the treatment of myelodysplastic syndromes (MDS). Vidaza™ is the first agent to ever be approved specifically for the treatment of MDS.
Dacogen™ Shows Promise for Treatment of “Poor Risk” MDS (1/10/2005)
A multicenter trial demonstrated that treatment with the hypomethylating agent Dacogen™ (decitabine) was superior to supportive care of poor risk patients with MDS.
EPO plus Neupogen® Effective for MDS (1/5/2005)
Researchers affiliated with the Eastern Cooperative Oncology Group (ECOG) and the Canadian Leukemia Study Group (CLSG) have reported that the combination of EPO and Neupogen “were effective in EPO non-responsive or refractory patients” with MDS. The results of this study were reported at the 2004 meeting of the American Society of Hematology in San Diego, December 4-7.
Aranesp® Produces Erythroid Responses in “Low-Risk” Patients with MDS (1/3/2005)
Researchers affiliated with the Groupe Francais des Myelodysplasies (GFM) have reported that the “results obtained with darbepoietin alfa alone on the anemia of low-risk MDS may be superior to those obtained with conventional EPO alone, and the drug is well tolerated.” This study was presented at the 2004 meeting of the American Society of Hematology in San Diego December 4-7.
Myelodysplastic Syndromes Treated Successfully with Reduced-Intensity Regimens and Allogeneic Stem Cell Transplantation (8/14/2003)
Researchers from England reported a two-year, event-free survival of 65% for older patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia treated with reduced-intensity regimens and allogeneic stem cell transplants. These results were published in the August 15, 2003 issue of the
Journal of Clinical Oncology.
Unrelated Umbilical Cord Transplants Highly Successful for Adults with MDS (6/16/2003)
Japanese researchers have reported that over 75% of patients with myelodysplastic syndrome (MDS) appear to be cured with unrelated umbilical cord transplants. They reported these results in the June 15, 2003 issue of
Blood.
Rituxan® and Campath® can be Safely Given Together with Encouraging Responses in Refractory Lymphoid Malignancies Expressing CD20 and CD52 (4/22/2003)
Researchers from the MD Anderson Cancer Center reported improved response rates in patients with refractory CLL or other B-cell malignancies that coexpressed CD20 and CD52 when treated with combined Rituxan® (rituximab) and (Campath®) alemtuzumab for the treatment of patients with refractory chronic lymphocytic leukemia (CLL) or other B-cell malignancies. These reported favorable results were reported in the May 1, 2003 issue of
Blood.
Long-Term Outcomes for Aplastic Anemia Treated with Immunosuppression Defined (3/12/2003)
Aplastic anemia is a life-threatening disease which is best treated in younger individuals by allogeneic stem cell transplantation. However, the majority of patients with this disease are older or do not have a suitable stem cell source available for transplantation. The most common treatment regimen for patients with aplastic anemia who are not transplanted consists of anti-thymocyte globulin (ATG), prednisolone and cyclosporine. This combination has been associated with a higher response rate than for patients getting ATG or cyclosporine alone. At the present time, it is not clear what factors are associated with an improved survival and what is the ultimate long-term outcome for patients with aplastic anemia treated with immunsuppression. In the March 5, 2003 issue of the Journal of the American Medical Association researchers from the Hematology Branch and the Office of Biostatistics Research of the National Heart, Lung, and Blood Institute reported long term follow-up of patients with severe aplastic anemia treated with cyclosporine and ATG.
Canadian Study Confirms Better Results with Peripheral Blood Stem Cells Compared to Bone Marrow in Recipients of Allogeneic Transplants for Myeloid Malignancies (8/20/2002)
Over the past decade, peripheral blood stem cells have gradually replaced bone marrow as the source of stem cells for allogeneic transplantation. Randomized studies have consistently shown faster engraftment, similar incidences of acute graft-versus-host disease and, usually, an increased incidence of chronic graft-versus- host disease. To date, the only survival advantage has been in patients transplanted for advanced malignancies. In the September 1, 2002 issue of the journal
Blood, researchers affiliated with the Canadian Bone Marrow Transplant Group have published data that supports the above findings, but in addition, they found a survival advantage for patients transplanted for chronic myeloid leukemia in the chronic phase. However, they also found an increase in the incidence of severe chronic graft-versus-host disease. There were no subgroups who appeared to be harmed by receiving peripheral blood stem cells rather than bone marrow.
Some Patients with Myelodysplastic Syndrome (MDS) Respond to Antithymocyte Globulin (ATG) (8/13/2002)
Patients with MDS have a wide spectrum of marrow dysfunction and pre-leukemia states. The etiology of MDS is probably varied, but in some instances immunosuppression with agents such as cyclosporin appear to have activity. Most patients, however, have been treated with recombinant growth factors, differentiating agents and stem cell transplantation. The role of immunosuppression with more potent immunosuppressive agents such as ATG has not been systematically explored for patients with MDS. However, ATG is the best immunosuppressive agent for the treatment of patients with severe aplastic anemia, with results rivaling those of stem cell transplantation.
Results of Allogeneic Stem Cell Transplants for Patients with Myelodysplastic Syndromes (MDS) May Be Improving With Targeted Method of Busulfan Dosing (8/13/2002)
Allogeneic stem cell transplantation is currently the only curative treatment for patients with MDS. Myelodysplastic syndromes are characterized by variable defects in hematopoietic stem cells, which lead to abnormal production of blood cells and eventually to leukemia. The degree of severity is ascertained by a classification system that includes refractory anemia (RA), RA with ringed sideroblasts (RARS), RA with excess blasts and RAEB in transformation (RAEB-T). In addition to this classification system the International Prognostic Scoring System (IPSS) is also used. The IPSS includes clonal chromosomal abnormalities and the number of peripheral blood cytopenias. Younger patients with more advanced MDS are often considered for allogeneic stem cell transplantation from a related or an unrelated donor, while older patients or those with less advanced disease are treated with a variety of palliative measures. Following allogeneic stem cell transplants, a significant number of patients will die of treatment-related causes and progression to leukemia. Previous studies have shown that approximately one-third of patients with MDS will become long-term survivors following allogeneic stem cell transplants. In the August 15, 2002 issue of the journal
Blood, researchers from the Fred Hutchinson Cancer Research Center report relapse-free survivals of over 50% following allogeneic stem cell transplantation in 109 patients with MDS, which is probably an improvement over previous studies.
Cancer Survivors With Low Sperm Counts Are Candidates for Assisted Reproduction Techniques (8/8/2002)
Chemotherapy, especially with alkylating agents, and radiation therapy cause significant damage to the testes. Sperm production is as sensitive to damage as bone marrow and mucosal cells because of the rapid turnover of cells. Such treatments result in low or absent sperm counts. In men with absent sperm, there is nothing that can currently be done to make them fertile. However, many men will have low sperm counts and can theoretically become fathers by assisted reproduction techniques, especially intracytoplasmic sperm injection (ICSI).
Prolonged Erythropoietin (Epo) Administration May Be Necessary For Optimal Response in Myelodysplastic Syndromes (MDS) (7/16/2002)
Patients with myelodysplasia sometimes respond to erythropoietin with an increase in red blood cell production and sometimes become transfusion dependent. The overall response rate of MDS patients is approximately 20%, but response is related to the specific type of MDS and whether or not the syndrome has progressed to a state where increased blast cells are present. Most patients with MDS receive a relatively short trial of Epo to determine responsiveness. Now, researchers affiliated with the Greek MDS Study Group report prolonged administration of Epo therapy may be necessary to evaluate a response. They reported their findings in the July 2002 issue of the
British Journal of Haematology.
Erythropoietin (EPO) and All-Trans Retinoic Acid (ATRA) Produces Sustained Responses in Patients with Myelodysplastic Syndromes (MDS) (4/1/2002)
Italian researchers have reported in the March 1 issue of
Blood that the combination of intermittent oral ATRA and subcutaneous EPO is an effective and well-tolerated treatment for patients with low- and intermediate-risk MDS. This study was based on in vitro studies suggesting that ATRA synergizes with EPO for the stimulation of hematopoiesis in patients with MDS.
Allogeneic Blood Cell Transplantation Following Reduced-Intensity Conditioning is Effective Therapy for Older Patients with Myelofibrosis with Myeloid Metaplasia (3/13/2002)
Researchers from the Blood and Marrow Transplantation Program, University of Illinois College of Medicine, have reported that reduced-intensity conditioning is effective treatment for older patients with myelofibrosis with myeloid metaplasia. These results were reported in a recent issue of the journal
Blood.
Campath-1H and Fludara® Show Promise for Refractory Chronic Lymphocytic Leukemia (3/12/2002)
Researchers in England have evaluated the combination of Campath-1H and Fludara® for treatment of refractory chronic lymphocytic leukemia (CLL). Campath-1H (alemtuzumab) produces response rates of 33% to 70% in B-cell CLL. They treated 6 patients with a median of 8 courses of Fludara® (range of 4-10 courses) and 16 weeks of Campath-1H (range of8-32 weeks). Five patients responded, including one who had a complete response. The responses observed were better from a combination of Campath-1H and Fludara® than responses after each agent used singly. Complete morphologic bone marrow responses were seen in 3 patients, including eradication of disease measured by sensitive flow cytometry in 2 patients. They concluded that Campath-1H combined with Fludara® is a highly promising novel therapy for refractory CLL.
Some Patients with Myelodysplastic Syndromes (MDS) Benefit from Unrelated Donor Marrow Transplantation (3/12/2002)
Allogeneic hematopoietic stem cell transplantation is the only curative therapy available for patients with MDS. The cure rate for bone marrow transplantation (BMT) from HLA-matchedsiblings is 40 to 50%. The main obstacles limiting the success of BMT in these patients have been disease relapse and treatment-related mortality (TRM). Unfortunately, only one third of patients with MDS that are eligible for allogeneic BMT have a suitable HLA-matched related donor. The post-transplantation outcomes of the first 510 unrelated donor BMTs performed as therapy forMDS using marrow from volunteer donors recruited through the National Marrow Donor Program (NMDP) were reported in a recent issue of
Blood.
Low-Dose Interleukin-11 Produces Responses in Patients with Bone Marrow Failure Including Those with Myelodysplastic Syndrome (2/12/2002)
Researchers at M.D. Anderson Cancer Center have evaluated low doses of IL-11 for treatment of patients with bone marrow failure syndromes. This is the first study of IL-11 for patients in bone marrow failure states. The usual dose of IL-11 is 50 µg/kg/d which is associated with significant toxicities in patients with bone marrow failure. They used a relatively low dose of IL-11, starting at 10 µg/kg/d. Patients received at least two courses of IL-11 with each course consisting of 2 weeks of daily IL-11 (10 µg/kg/d SC), followed by a 2-week rest period. After the first two courses (8 weeks), patients who showed any evidence of response could continue receiving maintenance therapy. The length of the courses versus the rest period and the dose of IL-11 could be individualized during the maintenance period in accordance with patient response and side effects. In particular, dosing would be adjusted to maintain platelet counts between 150 and 450,000. They treated 16 patients with bone marrow failure who had a platelet count less than 50,000. The diagnoses of the patients included refractory anemia (RA) (n = 7), refractory anemia with ringed sideroblasts (RARS) (n = 1), refractory anemia with excess blasts (RAEB) (n = 5), severe aplastic anemia (n = 4), and bone marrow failure after autologous bone marrow transplantation (n = 1). Six patients had diploid cytogenetics; the others had a variety of chromosomal abnormalities. Six (38%) of 16 patients showed a platelet response to IL-11, and two had a multilineage response (to IL-11 alone, n = 1; to IL-11 plus G-CSF and erythropoietin, n = 1). The median increase in peak platelet counts was 95,000 above baseline in the responders (range, increase of 55,000 to 130,000 above baseline). Responders included five of 11 patients with myelodysplasia and one of four patients with aplastic anemia. Response durations were 12, 13, 14+, 25, 30, and 30+ weeks. In four patients, significant increases in bone marrow megakaryocytes accompanied the platelet responses. Side effects of IL-11 were mild (peripheral edema, n = 7; conjunctival injection, n = 7; myalgia, n = 1; all grade 1). Seven patients had no side effects. It was concluded that low dose IL-11 produces increases in platelet counts without significant toxicity in selected thrombocytopenic patients with bone marrow failure.
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