PubMed Journals Articles About "FEDRATINIB Best Available Therapy Primary Myelofibrosis" RSS

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Showing "FEDRATINIB Best Available Therapy Primary Myelofibrosis" PubMed Articles 1–25 of 20,000+

The evolving understanding of prognosis in post-essential thrombocythemia myelofibrosis and post-polycythemia vera myelofibrosis vs primary myelofibrosis.

Myelofibrosis (MF) is the most aggressive of the classic Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs). In some patients with essential thrombocytopenia or polycythemia vera, which are relatively benign MPNs, MF develops as a natural evolution of their disease, resulting in post-essential thrombocythemia myelofibrosis (PET-MF) or post-polycythemia vera myelofibrosis (PPV-MF). Presenting with the same clinical features, including debilitating symptoms and signs of bone marrow failure, ...

Reduced intensity allogeneic stem cell transplantation for younger patients with myelofibrosis.

Allogeneic stem cell transplantation (alloSCT) is a curative procedure for myelofibrosis. Elderly people are mainly affected, limiting the feasibility of myeloablative regimens. The introduction of reduced-intensity conditioning (RIC) made alloSCT feasible for older patients. Nevertheless, the incidence of myelofibrosis is not negligible in young patients, who are theoretically able to tolerate high-intensity therapy. Very few data are available about the efficacy of RIC-alloSCT in younger myelofibrosis pat...

Splenectomy following JAK1/JAK2 inhibitor therapy in patients with myelofibrosis undergoing allogeneic stem cell transplantation.

Allogeneic hematopoietic stem cell transplantation (alloHSCT) is the only treatment option with curative potential in patients with myelofibrosis (MF). The aim of our study was to evaluate the safety of splenectomy before alloHSCT in MF patients who failed to achieve significant spleen response after ruxolitinib therapy.

Intestinal Behçet's Disease with Primary Myelofibrosis Involving Trisomy 8.

Behçet's disease (BD) is a disorder characterized by systemic inflammation of multiple organs, including the intestines. Several studies have reported a relationship between myelodysplastic syndrome and BD, and trisomy 8 was frequently seen, especially in intestinal BD. However, the association of BD with primary myelofibrosis (PMF) has not been well documented. A 58-year-old Japanese female was diagnosed with PMF in 2014. The symptoms of PMF resolved with ruxolitinib. However, she developed fever and inte...

Clinical and molecular features of patients with prefibrotic primary myelofibrosis previously diagnosed as having essential thrombocythemia in Japan.

Prefibrotic/early primary myelofibrosis (pre-PMF) and essential thrombocythemia (ET) exhibited different features of bone marrow, however, this is not always easy to judge objectively, making pathologists' distinction often suboptimal. In the WHO 2008 criteria, pre-PMF was not defined as a subgroup of PMF; therefore, affected patients were at a higher risk of misdiagnosis with ET. In this study, we examined the prevalence of pre-PMF patients among those previously diagnosed with ET in Japan.

A case of gout secondary to primary myelofibrosis.

A 52-year-old man was referred to our department with a 2-year history of polyarthritis. He was diagnosed as gout due to acute arthritis of bilateral feet dorsum 2 years ago,but he didn't receive any standard treatment. 1 year ago,there were more and more joints evolved during the gout attack, and many subcutaneous nodules occurred. When he presented to our clinic 1 month ago,the urate acid level was as high as 715 μmol/L. Moreover, we could find bone erosion in the X rays of his hand and foot,as well as s...

Homozygous CALR Mutation in Primary Myelofibrosis and Its Effect on Disease Phenotype: A Case Report and Review of the Literature.

Somatic mutations in gene have been reported in 60%-88% of patients with essential thrombocythemia (ET) and primary myelofibrosis (PMF) who are negative for and mutations. Most of the mutations analyzed to date are heterozygous mutations in exon 9 of the gene. Homozygosity in gene is rarely reported, and its association with clinical behavior of disease and impact on outcome of patients is not studied so far. We herein report a case of intermediate-2 risk PMF (according to IPSS) diagnosed with homozygo...

WT1 gene is overexpressed in myeloproliferative neoplasms, especially in myelofibrosis.

Classical Philadelphia-negative myeloproliferative neoplasms include Polycythemia Vera (PV), Essential Thrombocythemia (ET) and Primary Myelofibrosis (PMF). They are characterized by the presence of driver mutations of JAK2, CALR or MPL genes. Overexpression of WT1 is used as a marker of minimal residual disease in acute myeloid leukemia, especially after allogeneic stem cell transplantation (SCT). We investigated WT1 expression at diagnosis in 152 MPN patients and showed that the WT1 transcript was overexp...

Phenotypic characterization of malignant progenitor cells in patients with idiopathic myelofibrosis.

Idiopathic myelofibrosis (IM) is a clonal hematological malignancy originating from pluripotent hematopoietic stem cells (HSC). HSC are very rare potent cells that reside in the bone marrow (BM) and at a lower level in peripheral blood (PB). Previous studies showed that IM PB CD34 cells contain not only BM repopulating cells belonging to the malignant clone but also residual normal HSC.

Second primary malignancies in postpolycythemia vera and postessential thrombocythemia myelofibrosis: A study on 2233 patients.

Patients with myeloproliferative neoplasms (MPN) are known to have higher incidence of nonhematological second primary malignancies (SPM) compared to general population. In the MYSEC study on 781 secondary myelofibrosis (SMF) patients, the incidence of SPM after SMF diagnosis resulted 0.98/100 patient-years. When including non-melanoma skin cancers (NMSC), the incidence arose to 1.56/100 patient-years. In SMF, JAK inhibitor treatment was associated only with NMSC occurrence. Then, we merged the MYSEC cohort...

Massive megakaryocyte clustering in triple-negative primary myelofibrosis associated with an unusual cytogenetic aberration.

GATA-1 a potential novel biomarker for the differentiation of essential thrombocythaemia and myelofibrosis.

The BCR-ABL negative myeloproliferative neoplasms, polycythaemia vera, essential thrombocythaemia (ET) and myelofibrosis (MF) are characterised by mutations in JAK2, CALR or MPL. However, a yet unknown factor drives the precise disease phenotype. The haematopoietic transcription factor GATA-1 and its downstream targets NFE2 and FLI1 are responsible for determining erythroid and megakaryocyte lineages during haematopoietic stem cell differentiation. Previous studies have demonstrated low level of GATA-1 expr...

Corrigendum to "Phase 1/2 trial of glasdegib in patients with primary or secondary myelofibrosis previously treated with ruxolitinib" Leukemia Res. 79 (2019) 38-44.

Phase 1/2 trial of glasdegib in patients with primary or secondary myelofibrosis previously treated with ruxolitinib.

Glasdegib is a potent and selective oral inhibitor of the Hedgehog pathway. We report data from the single-arm, lead-in cohort of an open-label phase 1b/2 trial of glasdegib in patients with primary/secondary myelofibrosis (MF) previously treated with at least one Janus kinase inhibitor (JAKi). Patients received glasdegib 100 mg orally once daily until there was no further clinical benefit. Primary endpoints included adverse events (AEs). Secondary endpoints included patients with spleen volume reduction ...

Osteolytic Lesions in Primary Myelofibrosis and Effect of Ruxolitinib Therapy: Report of a Case and Literature Review.

Here, we report the case of a young female affected by primary myelofibrosis (PMF) who developed an osteolytic lesion of the humerus during the follow-up, and the possible efficacy of ruxolitinib in controlling this rare event. After 26 years of follow-up, the patient reported onset of acute pain at the proximal region of the left upper limb. An X-ray revealed an osteolytic bone lesion in the proximal third of the humeral shaft, which was then confirmed by magnetic resonance imaging. A biopsy of the lytic l...

Ruxolitinib treatment reduces monocytic superoxide radical formation without affecting hydrogen peroxide formation or systemic oxidative nucleoside damage in myelofibrosis.

The role of excess reactive oxygen species (ROS) with consequent DNA/RNA damage is now recognized as a hallmark of cancer. In JAK2V617F mutated myeloproliferative neoplasms, ROS have been suggested to be important factors in disease initiation and progression. Ruxolitinib is the most widely used drug for myelofibrosis, because it improves symptom-score. However, both the anti-clonal potential and improvement in overall survival are limited. We investigated the impact of ruxolitinib on formation of superoxid...

Times Not to Forget Radiotherapy When Treating Patients With Lymphoma.

Radiation therapy remains an important component of lymphoma treatment. It has evolved with improvements in technology and a better understanding of how to successfully integrate it into lymphoma treatment. There are specific clinical presentations where omission of radiation therapy could adversely affect patient outcome and should not be overlooked. Radiation therapy may serve an important role as primary treatment, as a component of combined modality therapy, as adjuvant therapy to maximize local control...

Reduced occurrence of appropriate therapy for ventricular arrhythmias after beta-blocker uptitration following implant of a primary prevention CRT-defibrillator.

Absence of beta-blocker use independently predicts appropriate therapy. Following cardiac resynchronisation therapy (CRT) implant, reverse remodelling and protection against bradycardia allows for beta-blocker dose uptitration. The differential dosing effects on the occurrence of a first episode of appropriate therapy in primary prevention CRT-defibrillator (CRT-D) patients remains unstudied.

Clinical Profile, Response to Therapy, and Outcome of Children with Primary Intestinal Lymphangiectasia.

Intestinal lymphangiectasia (IL; primary or secondary) is an important cause of protein-losing enteropathy. We evaluated the clinicolaboratory profile, response to therapy, complications, and outcome of children with primary IL (PIL).

Allogeneic stem cell transplantation for myelofibrosis patients aged ≥ 65 years.

Myelofibrosis (MF) is a disease of elderly with median age of 65 years at diagnosis. Allogeneic stem cell transplantation (ASCT) currently is the only potentially curative option, although associated with treatment-related morbidity and mortality. Development of reduced intensity conditioning (RIC) regimens enabled transplant to be performed successfully in older patients.

Efficacy and safety of ruxolitinib and hydroxyurea combination in patients with hyperproliferative myelofibrosis.

Ruxolitinib is the only commercially available JAK1/2 inhibitor approved for the treatment of myelofibrosis-related splenomegaly and symptoms. During treatment, as rare conditions, leukocytosis and/or thrombocytosis could develop and the management of these situations is not well established. We report here 53 myelofibrosis patients that received a combination of hydroxyurea and ruxolitinib because of uncontrolled myeloproliferation. Both drugs were administered outside clinical trials. At 48 weeks, a sign...

Results of neuroprotective therapy in primary open-angle glaucoma.

To determine the relationships between the changes in functional parameters of vision that occur in primary open-angle glaucoma (POAG) patients on neuroprotective therapy.

Assessment of Quality of Life Following Allogeneic Stem Cell Transplant for Myelofibrosis.

Patient reported outcomes (PRO) for patients with myelofibrosis (MF) have been well characterized, however, little is known about quality of life (QoL) following allogeneic stem cell transplantation (alloSCT).

Implementation of a medication therapy management program in a primary care clinic.

This study examined the use of a medication therapy management (MTM) program by clinic-embedded pharmacists within a primary care clinic primarily by evaluating the impact on patient identification for pharmacy services. Secondary outcomes included characterizing intervention type, targeted drugs and disease states, and barriers to successful interventions, SETTING: Primary care clinic.

An atypical presentation of Sweet's syndrome in a myelofibrosis patient.

A 46-year-old man with no significant medical history presented to haematology with symptoms of fatigue, dyspnoea on exertion and weight loss. Physical examination revealed a lesion on the right shin and splenomegaly. Labs were significant for leucocytosis with immature components, thrombocytosis and 3% peripheral blasts on smear. A bone marrow biopsy confirmed a diagnosis of myelofibrosis (MF). Dynamic International Prognosis Scoring system was 2. He was started on ruxolitnib, with decitabine added subsequ...

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