Danazol as first-line therapy for myelodysplastic syndromes

At present, no standard therapy is available for most patients with myelodysplastic syndromes. In this retrospective study, we analyse data from 42 patients with myelodysplastic syndrome treated with low-dose danazol. More than half achieved a response. Results show that danazol remains an attractive option due to its low cost and good safety profile in centers with reduced access to novel therapeutic alternatives.
Source: Clinical Lymphoma, Myeloma and Leukemia - Category: Hematology Authors: Tags: Original Study Source Type: research

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IntroductionChronic graft-versus-host disease (cGVHD) affects up to 50% of the long-term survivors of allogeneic hematopoietic stem cell transplantation (HCT), and is the leading cause of mortality in patients who survive to two years post-transplant. Unlike acute GVHD (aGVHD), the primary pathology in cGVHD is fibrotic, affecting the skin, and lacrimal and salivary glands, and shares many features with the autoimmune conditions Sjogrens syndrome and systemic sclerosis. Certain gastrointestinal microbiota compositions have been associated with these autoimmune conditions, and we thus hypothesized that the configuration of ...
Source: Blood - Category: Hematology Authors: Tags: 722. Clinical Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Biomarkers and the Microbiome Source Type: research
MF is a myeloproliferative neoplasm characterized by abnormal megakaryocytes and elevated proinflammatory cytokines which results in bone marrow fibrosis, progressive hepatosplenomegaly due to extramedullary hematopoiesis, and debilitating constitutional symptoms. Current treatments, including ruxolitinib (the only approved drug for MF), provide symptomatic relief but have limited effects on the underlying disease. Effective therapies with potential MF disease course modification and second line therapies are urgently needed.CPI-0610 has been evaluated in 3 Phase 1 studies in> 140 patients with lymphoma, multiple myelom...
Source: Blood - Category: Hematology Authors: Tags: 634. Myeloproliferative Syndromes: Clinical Source Type: research
Conclusions. ISV is widely used in hematological pts with IFI also in diseases other than acute myeloid leukemia and it is overall well tolerated. ORR to ISV is at least comparable with other antifungal agents. A rec/ref underlying hematological disease impacts both on OS and response to ISV, while having an IFI refractory to other antifungal agents including azoles does not seem to compromise the response to ISV, although this promising result should be confirmed in prospective studies and larger groups of patients.DisclosuresBusca: Gilead: Honoraria, Membership on an entity's Board of Directors or advisory committees, Sp...
Source: Blood - Category: Hematology Authors: Tags: 203. Lymphocytes, Lymphocyte Activation, and Immunodeficiency, including HIV and Other Infections: Poster III Source Type: research
This study analyzes patient and hospital characteristics of patients undergoing BMT, in addition the study describes resource utilization and outcomes of BMT among various hematological cancers.Methods: We performed a retrospective cohort analysis of the National Inpatient Sample 2014 Database (HCUP-NIS). Patients were included in the study if they had a procedure diagnosis of BMT and a medical diagnosis of acute or chronic leukemia (ALL, AML, CLL, CML), lymphoma (HL and NHL), MM, MDS or HLH. HSCT or BMT includes both Allogeneic and Autologous Stem Cell Transplant however we did not differentiate one from the other since B...
Source: Blood - Category: Hematology Authors: Tags: 902. Health Services Research-Malignant Diseases: Poster III Source Type: research
ConclusionsOur results suggest divergent validity of ESMO-MCBS and ASCO-VF when assessing hematologic malignancies, indicating that these frameworks likely measure different constructs of clinical benefit in this setting. Furthermore, the ASCO-VF demonstrated improved inter-rater reliability compared to ESMO-MCBS. It remains unclear which framework is correctly assessing the clinical benefit of hematologic drugs. To better understand if one framework is more accurate in its evaluation, future research that compares these frameworks to other established measures of clinical benefit (such as quality adjusted life years) in t...
Source: Blood - Category: Hematology Authors: Tags: 902. Health Services Research-Malignant Diseases: Poster III Source Type: research
INTRODUCTION: Timely goals of care (GOC) discussions improve end-of-life (EOL) care for patients with solid malignancies. Although timely GOC discussions might also improve EOL care for patients with blood cancers, data are sparse regarding such discussions for this population. We studied the impact of timing and location of GOC discussions on quality of EOL care for a cohort of patients who died of blood cancers.METHODS: We assembled a retrospective cohort of blood cancer decedents from Dana-Farber Cancer Institute, Boston, MA. We included patients diagnosed with a blood cancer, who had ≥ 2 outpatient visits at the stu...
Source: Blood - Category: Hematology Authors: Tags: 902. Health Services Research-Malignant Diseases: Quality Of Life Studies Source Type: research
Conclusion: Combing AI "software" and the human expert "hardware" will allow for the quick delivery of comprehensive information needed for patient care that outperforms what either can achieve individually in the field of hematological disease.Figure1. Comparison of potential driver mutations between human curation and Watson for Genomics. The size of the gene symbol indicates the total number of mutations identifiedDisclosuresNo relevant conflicts of interest to declare.
Source: Blood - Category: Hematology Authors: Tags: 902. Health Services Research-Malignant Diseases: Poster I Source Type: research
ConclusionsThis is a retrospective study from rituximab era. The prognosis of follicular lymphoma is good with the current treatment methods and the risk of secondary hematological malignancy seems to be low. Due to low incidence, it seems, that it is not necessary to avoid chemotherapy in the fear of secondary hematological malignancies. However, multiple lines of treatment are associated with higher risk for secondary hematological malignancies. Therefore, the use of regimens with long remission, like rituximab maintenance, would probably reduce the risk of secondary hematological malignancies.DisclosuresNo relevant conf...
Source: Blood - Category: Hematology Authors: Tags: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma-Clinical Studies Source Type: research
Conclusions: This study provides evidence that: 1) the cumulative incidence of ST is about 1% person-year of follow up in SMF patients; 2) JAK inhibitors given during ET/PV or SMF phase are neutral for ST development within the limit of current follow up; 3) developing SMF in patients with PV or ET does not imply a higher risk of ST. These findings highlight the need of studies aimed at identifying patients at higher risk of ST occurrence.DisclosuresRambaldi: Roche: Consultancy; Celgene: Consultancy; Novartis: Consultancy; Italfarmaco: Consultancy; Omeros: Consultancy; Amgen Inc.: Consultancy; Pfizer: Consultancy. Komrokji...
Source: Blood - Category: Hematology Authors: Tags: 634. Myeloproliferative Syndromes: Clinical: Poster II Source Type: research
There is no standard therapy for patients with myelodysplastic syndromes (MDS) refractory to hypomethylating agents (HMA) and the median overall survival (OS) of HMA-refractory MDS patients with IPSS Intermediate-2/High-risk MDS is ~6 months. Here, we present data from a phase 2 clinical trial of selinexor, an oral, first-in-class Selective Inhibitor of Nuclear Export (SINE) compound that inhibits XPO1, in patients with MDS refractory to HMAs. XPO1 is the major nuclear export protein responsible for shuttling many key cellular regulators out of the nucleus and is overexpressed in many cancers. Preclinical studies of seline...
Source: Blood - Category: Hematology Authors: Tags: 637. Myelodysplastic Syndromes-Clinical Studies: Novel Therapeutics I Source Type: research
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