Active clinical trials for "Leukemia, Myeloid"

To provide access to ivosidenib monotherapy to patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation.

The current understanding of acute myeloid leukemia (AML) is that one site of bone marrow (BM) sampling serves as a window that represents all AML cells distributed throughout the BM, an assumption that has yet to be questioned. Simulation in mice led to inconsistent representation of the full BM, which can incorrectly suggest the absence of leukemic cells. Positron-emission tomography (PET) scan can detect areas of high metabolic activity in the body using for instance a radioactive sugar. In one report, its use in human AML has provided proof-of-principle evidence of unequal distribution of AML cells in BM. Accordingly, the alternative hypothesis is to test if PET scan can demonstrate if BM geography can alter AML cells spread and home them as distinct areas rather than uniform spread as if they are distributed in liquid state.

The purpose of this study is to retrospectively evaluate the treatment patterns and AML-related key healthcare resource use among AML patients, stratified by FLT3 mutation status, intensive chemotherapy (IC) eligibility, and relapsed or refractory (R/R) status.

The purpose of this study is to gather and evaluate additional safety data on the combination of midostaurin and standard of care for adult patients with newly diagnosed Fms-like tyrosine kinase receptor (FLT3) mutated Acute Myeloid Leukemia (AML) who are eligible for standard induction and consolidation chemotherapy and are without satisfactory treatment alternatives prior to the commercial availability* and reimbursement of midostaurin during the regulatory approval process

This is a prospective, multicenter observational study to collect clinically annotated biospecimens in order to assess the correlation between ex vivo data generated by the Notable assay platform and clinical outcome.

Chronic myeloid leukemia (CML) is a model of targeted therapy for human malignancies. Over the past decade, a broad array of drugs designed to selectively inhibit protein tyrosine kinases (PTK) [i.e., tyrosine kinase inhibitors, (TKI)] have emerged as novel therapies for cancer patients. Hence, CML is an hematopoietic stem cell disorder in which a t(9;22) (q34;q11) reciprocal chromosomal translocation gives rise to Philadelphia chromosome (Ph) and generates the BCR-ABL1 fusion gene encoding a constitutively activated PTK. TKIs, such as imatinib by blocking BCR-ABL1 kinase activity, selectively eradicate CML cells and induce durable responses and prolong survival. CML patients treated with TKI are monitored by quantitative RT-PCR to detect leukemic BCR-ABL1 transcript performed from peripheral blood samples (1). Since TKI treated CML patients have a near-normal life expectancy two important issues must be considered in the future: the quality of life and ethical aspects of a lifetime treatment, the budget impact for healthcare providers of treating patients during lifetime. One of the best ways to consider these two points is to ask the question about stopping TKI in good responder patients. We first reported a pilot study where imatinib was withdrawn in 12 CML patients treated and maintained in complete molecular remission (CMR), defined by undetectable residual disease (with sensitivity of 4.5 log) on quantitative RT-PCR, for at least two years. Then, we demonstrated in a multicenter study entitled STIM trial that imatinib could be safely discontinued in patients with CMR for at least 2 years (2). All molecular relapsing patients were sensitive when imatinib was re-challenged (3). Around 40% of these patients remain in a prolonged treatment-free remission (TFR) after treatment cessation (4). Taking into account the cost of imatinib and the number of months without treatment in STIM trial, the savings in France were estimated to be 9 million €. However, since only 40 % of patients are in treatment free remission, a study, assessing the real budget impact of stopping TKI in the eligible population seems necessary as no published study has ever addressed this question in France. Our aim is to assess the budget impact of discontinuing TKI treatment in patients with CML in deep molecular response since at least two years, compared with treatment during lifetime, from the French healthcare system point of view. This budget impact will be expressed as a "net benefit" and will be based on the difference between total costs incurred by this strategy and total costs avoided also. One of the originality of our study is to raise the issue of treatment cessation in the context of a chronic disease from an economic point of view. The other originality of this study is to use a decision model to perform this French budget impact analysis of TKI discontinuation, without setting up another trial. Besides the literature review and meta-analysis; the proposed probabilistic Markov model will use direct costs (including treatment costs and all health care related costs as well as costs related to relapse) extracted mainly from the French Health Insurance Databases.

The purpose of this study is to determine whether a novel standard of care protocol, washing red cell and platelet transfusions for younger patients with acute leukemia, has yielded improved clinical outcomes at Strong Memorial Hospital (Rochester, New York, USA). This standard of care was implemented based upon an earlier randomized trial (BMC Blood Disorders. 2004 Dec 10;4(1):6) The comparator will be historical controls from the medical literature.

Analyze the results of conditioning with once-daily dose intravenous busulfan and fludarabine in patients undergoing HLA identical sibling Allogeneic HSCT for myeloid malignancies.

Chronic myelogenous leukemia (CML) is a chronic myeloproliferative disorder characterized by a translocation between chromosome 9 and 22, leading to a pathogenic tyrosine kinase signal transduction protein. CML can be treated with tyrosine kinase inhibitors (TKIs), which inhibit BCR/ABL kinase, such as imatinib. In about 20% of CML patients who are treated by imatinib, a complete cytogenetic response cannot be achieved. The other two novel TKIs (dasatinib and nilotinib), achieve higher rates of complete cytogenetic response and they are proposed as second-line therapy for imatinib-resistant patients or for those who do not tolerate imatinib. Dasatinib inhibits BCR/ABL kinase in about >300 times in vitro in more than imatinib and also inhibits several other kinases, including the Src family. Src tyrosine kinase is crucial for potassium channel function in human pulmonary arteries. Imatinib and nilotinib do not inhibit the Src. Incident cases of precapillary PH have been reported in patients who have CML treated with the dasatinib. Improvements were usually observed after withdrawal of dasatinib. This study is designed to identify incident cases of dasatinib-associated PH and describe pulmonary vascular changes induced by dasatinib. As comparison population will be patients who receive another second-line TKI (nilotinib).

In adults with acute myeloid leukemia, especially those < 60 years of age, high-dose cytarabine consolidation therapy has been shown to influence survival, but the appropriate dose has not been defined.