Veno Occlusive Disease News and Research

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Gene therapy leads to durable transfusion independence in youngest patients with thalassemia

Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age.

22 Feb 2022

Molecular approach can improve diagnosis of pulmonary hypertension

Pulmonary hypertension is a serious problem associated with a wide variety of lung diseases, which can lead to right ventricular dysfunction and death.

15 Jun 2020

Clinical trial to test the efficacy of defibrotide medicine for reducing COVID-19 death rate

The Neurosciences Institute, mixed centre of the Spanish National Research Council and the Miguel Hernández University of Elche, have collaborated on the first clinical trial conducted in the world to test whether the defibrotide medicine, which has already been authorized to treat a severe obstruction of the hepatic sinusoidal veins of people who have undergone a bone marrow transplant, can be used as therapy against COVID-19.

12 May 2020

Merck presents MK-1454 Phase 1 data for treatment of advanced solid tumors or lymphomas

Merck, known as MSD outside the United States and Canada, today announced the first presentation of preliminary data from a Phase 1 clinical trial evaluating MK-1454, an investigational STING (stimulator of interferon genes) agonist, as monotherapy and in combination with KEYTRUDA (pembrolizumab), Merck's anti-PD-1 therapy, in patients with advanced solid tumors or lymphomas.

22 Oct 2018

Merck presents KEYNOTE-057 trial results for patients with high-risk non-muscle invasive bladder cancer

Merck, known as MSD outside the United States and Canada, today announced the first presentation of results from an interim analysis of KEYNOTE-057, a Phase 2 trial evaluating KEYTRUDA, Merck's anti-PD-1 therapy, for previously treated patients with high-risk non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ or CIS plus papillary disease (Cohort A).

22 Oct 2018

CNDA approves KEYTRUDA for treatment of advanced melanoma

Merck, known as MSD outside the United States and Canada, today announced that KEYTRUDA, Merck's anti-PD-1 therapy, has been approved by the China National Drug Administration for the treatment of adult patients with unresectable or metastatic melanoma following failure of one prior line of therapy.

26 Jul 2018

Phase 3 study of KEYTRUDA monotherapy meets primary endpoint

Merck, known as MSD outside the United States and Canada, announced today that the pivotal Phase 3 KEYNOTE-048 trial investigating KEYTRUDA, Merck's anti-PD-1 therapy, for first-line treatment of recurrent or metastatic head and neck squamous cell carcinoma, met a primary endpoint of overall survival as monotherapy in patients whose tumors expressed PD-L1.

25 Jul 2018

Merck announces FDA acceptance of supplemental Biologics License Application for KEYTRUDA

Merck, known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration has accepted for standard review a new supplemental Biologics License Application for KEYTRUDA, Merck's anti-PD-1 therapy, as adjuvant therapy in the treatment of patients with resected, high-risk stage III melanoma and granted a Prescription Drug User Fee Act, or target action, date of February 16, 2019.

25 Jun 2018

FDA approves drug to treat patients with newly diagnosed CD33-positive AML

The U.S. Food and Drug Administration today approved Mylotarg (gemtuzumab ozogamicin) for the treatment of adults with newly diagnosed acute myeloid leukemia whose tumors express the CD33 antigen (CD33-positive AML).

1 Sep 2017

Pfizer receives FDA approval for new therapy to treat adults and children with AML

Pfizer Inc. today announced that the U.S. Food and Drug Administration approved MYLOTARG (gemtuzumab ozogamicin) for adults with newly diagnosed CD33-positive acute myeloid leukemia (AML), and adults and children 2 years and older with relapsed or refractory CD33-positive AML.

1 Sep 2017

Researchers develop quick and easy-to-use test for detecting rare immunodeficiency

Researchers at the University of Basel have developed a test to quickly and reliably diagnose a rare and severe immune defect, hepatic veno-occlusive disease with immunodeficiency. They reported on their findings in the Journal of Clinical Immunology.

22 Aug 2017

$FDA approval provides new, targeted treatment option for adults with relapsed or refractory B-cell ALL$

FDA approval provides new, targeted treatment option for adults with relapsed or refractory B-cell ALL

The U.S. Food and Drug Administration today approved Besponsa (inotuzumab ozogamicin) for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).

17 Aug 2017

Experts develop new genomic testing method for pulmonary hypertension caused by genetic mutation

Pulmonology and genetics experts from two Utah healthcare organizations have collaboratively developed a new diagnostic genomic testing method for a rare form of pulmonary hypertension caused by a genetic mutation they discovered three years ago.

9 Aug 2016

Salvage inotuzumab ozogamicin improves ALL outcomes

Compared with standard chemotherapy, treatment with inotuzumab ozogamicin increases the rate of complete remission and allows a higher proportion of patients with relapsed or refractory acute lymphoblastic leukaemia to subsequently receive stem-cell transplantation, research suggests.

22 Jun 2016

FDA approves Defitelio to treat hepatic veno-occlusive disease in adults, children

The U.S. Food and Drug Administration today approved Defitelio (defibrotide sodium) to treat adults and children who develop hepatic veno-occlusive disease (VOD) with additional kidney or lung abnormalities after they receive a stem cell transplant from blood or bone marrow called hematopoietic stem cell transplantation (HSCT).

31 Mar 2016

HCT not precluded by prior second-generation TKI therapy in chronic, advanced phase CML

Allogeneic haematopoietic stem cell transplantation can be considered for chronic- and accelerated-phase chronic myeloid leukaemia patients with prior exposure to second-generation tyrosine kinase inhibitors, say Polish researchers.

17 Aug 2015

HCT not precluded by prior second-generation TKI therapy in chronic, advanced phase CML

14 Aug 2015

Jazz Pharmaceuticals to present defibrotide results for hepatic VOD at BMT Tandem meetings

Jazz Pharmaceuticals plc announced today that researchers will present data on the use of defibrotide, an investigational medicine being studied in the United States (U.S.) for the treatment of hepatic veno-occlusive disease (VOD), a rare, potentially life-threatening, early complication in patients undergoing hematopoietic stem-cell transplantation (HSCT) therapy.

13 Feb 2015

Bayer HealthCare receives FDA approval for Adempas tablets

Bayer HealthCare announced today that the United States Food and Drug Administration has approved Adempas (riociguat) tablets for: (i) the treatment of adults with persistent/recurrent chronic thromboembolic pulmonary hypertension (WHO Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class; and (ii) the treatment of adults with pulmonary arterial hypertension (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening.

9 Oct 2013

Tacrolimus plus sirolimus not better than standard regimen in preventing GVHD

An experimental drug combination for preventing graft-versus-host disease (GVHD) was not significantly better than the standard regimen on key endpoints, according to a report of a phase 3 trial at the American Society of Hematology annual meeting.

11 Dec 2012

Veno Occlusive Disease News and Research

Gene therapy leads to durable transfusion independence in youngest patients with thalassemia

Molecular approach can improve diagnosis of pulmonary hypertension

Clinical trial to test the efficacy of defibrotide medicine for reducing COVID-19 death rate

Merck presents MK-1454 Phase 1 data for treatment of advanced solid tumors or lymphomas

Merck presents KEYNOTE-057 trial results for patients with high-risk non-muscle invasive bladder cancer

CNDA approves KEYTRUDA for treatment of advanced melanoma

Phase 3 study of KEYTRUDA monotherapy meets primary endpoint

Merck announces FDA acceptance of supplemental Biologics License Application for KEYTRUDA

FDA approves drug to treat patients with newly diagnosed CD33-positive AML

Pfizer receives FDA approval for new therapy to treat adults and children with AML

Researchers develop quick and easy-to-use test for detecting rare immunodeficiency

FDA approval provides new, targeted treatment option for adults with relapsed or refractory B-cell ALL

Experts develop new genomic testing method for pulmonary hypertension caused by genetic mutation

Salvage inotuzumab ozogamicin improves ALL outcomes

FDA approves Defitelio to treat hepatic veno-occlusive disease in adults, children

HCT not precluded by prior second-generation TKI therapy in chronic, advanced phase CML

HCT not precluded by prior second-generation TKI therapy in chronic, advanced phase CML

Jazz Pharmaceuticals to present defibrotide results for hepatic VOD at BMT Tandem meetings

Bayer HealthCare receives FDA approval for Adempas tablets

Tacrolimus plus sirolimus not better than standard regimen in preventing GVHD

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