598 research outputs found
New Trends in Iron Chelation: Impacting Outcomes
No full textThe availability of the effective, well-tolerated, and convenient, once-daily oral iron chelation therapy, deferasirox, has the potential to change clinical practice in a number of ways. It is possible that the outcome of patients who are currently receiving chelation therapy could be improved by freeing them from the burden of frequent, intravenous infusions of deferoxamine (DFO) and thereby improving long-term compliance. In addition, the ease of treatment may make treatment accessible to patients who currently do not receive iron chelation therapy. The potential of deferasirox therapy is illustrated in two typical clinical cases. One outlines a patient with β-thalassemia who is struggling to comply with DFO or DFO-deferiprone combination therapy and is consequently failing therapy and susceptible to the serious clinical sequelae of iron overload. The other is a patient with myelodysplastic syndrome who is transfusion-dependent and chelation-naïve, initiating therapy for the first time. © 2007 Elsevier Inc. All rights reserved.Alessandrino EP, 2002, HAEMATOLOGICA, V87, P1286; BRITTENHAM GM, 1994, NEW ENGL J MED, V331, P567, DOI 10.1056-NEJM199409013310902; Cappellini MD, 2005, BLOOD, V106; Cappellini MD, 2006, BLOOD, V107, P3455, DOI 10.1182-blood-2005-08-3430; Davis BA, 2000, BLOOD, V95, P1229; Gattermann N., 2005, HEMATOL ONCOL CLI S1, V19, P13; Gattermann N, 2005, HEMATOL ONCOL CLI S1, V19, P18; Greenberg Peter L, 2006, J Natl Compr Canc Netw, V4, P58; Kattamis C, 2005, BLOOD, V106; Malcovati L, 2005, J CLIN ONCOL, V23, P7594, DOI 10.1200-JCO.2005.01.7038; Piga A, 2006, HAEMATOL-HEMATOL J, V91, P873; TAHER A, 2005, BLOOD, V106; VICHINSKY E, 2005, BLOOD, V10622
Nilotinib is effective in patients with chronic myeloid leukemia in chronic phase after imatinib resistance or intolerance: 24-month follow-up results
No full textNilotinib is a potent selective inhibitor of the BCR-ABL tyrosine kinase approved for use in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CMLCP), and in CML-CP and CML-accelerated phase after imatinib failure. Nilotinib (400 mg twice daily) was approved on the basis of the initial results of this phase 2 open-label study. The primary study endpoint was the proportion of patients achieving major cytogenetic response (CyR). All patients were followed for > 24 months or discontinued early. Of 321 patients, 124 (39%) continue on nilotinib treatment. Overall, 59% of patients achieved major CyR; this was completeCyR(CCyR) in 44%. Of patients achieving CCyR,56% achieved major molecular response. CyRs were durable, with 84% of patients who achieved CCyR maintaining response at 24 months. The overall survival at 24 months was 87%. Adverse events were mostly mild to moderate, generally transient, and easily managed. This study indicates that nilotinib is effective, with a manageable safety profile, and can provide favorable long-term benefits for patients with CML-CP after imatinib failure. This trial was registered at www.clinicaltrials.gov as #NCT00109707.Hagop M. Kantarjian, Francis J. Giles, Kapil N. Bhalla, Javier Pinilla-Ibarz,Richard A. Larson, Norbert Gattermann, Oliver G. Ottmann, Andreas Hochhaus, Giuseppe Saglio, Timothy P. Hughes, Giovanni Martinelli, Dong-Wook Kim, Yaping Shou, Neil J. Gallagher, Rick Blakesley, Michele Baccarani, Jorge Cortes and Philipp D. le Coutr
Prognostic factors in treatment-related Myelodysplastic syndromes (t-MDS) and acute myeloid leukemia (t-AML).
No full textPrognostic factors in treatment-related Myelodysplastic syndromes (t-MDS) and acute myeloid leukemia (t-AML).
No full textResults of a phase 2 study of valproic acid alone or in combination with all-trans retinoic acid in 75 patients with myelodysplastic syndrome and relapsed or refractory acute myeloid leukemia
No full textValproic acid (VPA) inhibits historic deacetylase activity and induces differentiation of acute myeloid leukemia (AML) blasts in vitro. We observed clinical responses to VIA in patients with myelodysplastic syndrome (MDS) and AML. Here, we report follow-up data on 75 patients. Of these, 66 were started on VPA monotherapy, with later addition of all-trans retinoic acid (ATRA) in patients who did not respond or relapsed. Nine patients were treated with VIA + ATRA from the start. Median treatment duration was 4 months for VIA and 2 months for ATRA. Hematological improvement, according to international working group criteria for MDS, was observed in 18 patients (24%). Median response duration was 4 months. ATRA exerted no additional effect in patients receiving the combination from the start or benefited primary VPA nonresponders. However, of ten VIA responders who relapsed, four achieved a second response after addition of ATRA. Response rates were strongly dependent on disease type according to WHO classification. We found a response rate of 52% in MDS patients with a normal blast count (refractory sideroblastic anemia, refractory cytopenia with multilineage dysplasia, and refractory sideroblastic cytopenia with multilineage dysplasia). The response rate was 6% in refractory anemia with excess blasts (I + II), 16% in AML, and 0% in chronic myelomonocytic leukemia. Bone marrow blast count was the only variable that predicted responses. We conclude that VPA is clinically useful in low-risk MDS. For patients with high-risk MDS, VPA may be combined with chemotherapy or demethylating drugs. If patients relapse after an initial response to VPA, ATRA has the potential to induce a prolonged second response
Overview of guidelines on iron chelation therapy in patients with myelodysplastic syndromes and transfusional iron overload
No full textA Phase II Study of Valproic Acid and Lenalidomide Combination Therapy in Patients with Myelodysplastic Syndrome (MDS) and a Favorable Risk Profile: Final Results of the Valena Trial
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Valproic acid (VPA) achieves high response rates in patients with low-risk myelodysplastic syndromes.
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