Quality of life (healthcare)

EMA and European Organisation for Research and Treatment of Cancer (EORTC) workshop: How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions?, Online, European Medicines Agency, Amsterdam, t[...]

Retrieved on: 
Sunday, March 10, 2024

This workshop will bring together international experts from academia, learned societies, patients, regulatory agencies, health technology assessment bodies, and industry.

Key Points: 
  • This workshop will bring together international experts from academia, learned societies, patients, regulatory agencies, health technology assessment bodies, and industry.
  • The aims of the workshop are to:
    - Understand the current global landscape on the use of PROs and HRQOL for evaluation of anti-cancer treatments across different stakeholder groups.
  • - Understand the use of validated PRO questionnaires to measure core HRQOL outcomes and disease-related symptoms; and PRO Item Libraries in the development of trial-specific/treatment-specific item lists.
  • The recording of this meeting will be made available after the event.

Mallinckrodt Presents Data on Real-World Outcomes with THERAKOS™ CELLEX™ Photopheresis System Treatment at the 2024 Tandem Meetings

Retrieved on: 
Wednesday, February 21, 2024

DUBLIN, Feb. 21, 2024 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, today announced a poster presentation of findings from a systematic literature review and meta-analysis of the safety, efficacy, and real-world outcomes of extracorporeal photopheresis (ECP) treatment for patients with steroid-refractory chronic graft-versus-host disease (SR-cGvHD).1 An analysis of 47 studies reporting on the THERAKOS™ CELLEX™ Photopheresis System showed that treatment of SR-cGvHD with ECP was associated with improvements in patients' overall survival (OS), failure-free survival (FFS), and overall response rate (ORR).1 The results will be shared in a poster presentation at the 2024 Tandem Meetings, the combined Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood and Marrow Transplant Research (CIBMTR) taking place February 21-24, 2024 in San Antonio, TX.

Key Points: 
  • Skin-specific response at Months 2-3 and at Months 4-6 were 34.86% (95% CI: 13.26-65.21) and 54.22% (95% CI: 35.67-71.67), respectively.
  • Outcomes may be influenced by therapies not evaluated in the study and the clinical/health economics outcomes may not be solely attributable to THERAKOS ECP.
  • THERAKOS™ Photopheresis is contraindicated in:
    THERAKOS™ Photopheresis treatments should always be performed in locations where standard medical emergency equipment is available.
  • Please refer to the THERAKOS™ CELLEX™ Photopheresis System Operator Manual for a complete list of warnings and precautions and adverse events.

LEO Pharma Announces Positive Phase 3 Head-to-head Data Results from DELTA FORCE Trial Comparing Delgocitinib Cream With Alitretinoin Capsules in Adults With Severe Chronic Hand Eczema (CHE)

Retrieved on: 
Wednesday, January 24, 2024

LEO Pharma A/S, a global leader in medical dermatology, today announced positive results from the DELTA FORCE trial.

Key Points: 
  • LEO Pharma A/S, a global leader in medical dermatology, today announced positive results from the DELTA FORCE trial.
  • Delgocitinib cream achieved the primary outcome measure of the trial by demonstrating a superior reduction in Hand Eczema Severity Index (HECSI) score from baseline to Week 12 compared to alitretinoin capsules.
  • Furthermore, patients receiving delgocitinib cream had a lower number of treatment-emergent adverse events (AEs) compared to those receiving alitretinoin capsules.
  • Detailed results from DELTA FORCE are planned to be submitted for scientific presentation and publication at a later date.

Latest Odronextamab Data in Relapsed/Refractory Follicular Lymphoma Showed Compelling Responses and Overall Maintenance of Patient-Reported Outcomes

Retrieved on: 
Monday, December 11, 2023

TARRYTOWN, N.Y., Dec. 10, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive data for odronextamab in patients with relapsed/refractory (R/R) follicular lymphoma (FL) from a pivotal Phase 2 trial (ELM-2). These data – which include updated efficacy, safety and patient-reported outcomes (PROs) – were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition from December 9 to 12 in San Diego, CA. Odronextamab is an investigational CD20xCD3 bispecific antibody designed to bridge CD20 on cancer cells with CD3-expressing T cells to facilitate local T-cell activation and cancer-cell killing.

Key Points: 
  • Odronextamab is an investigational CD20xCD3 bispecific antibody designed to bridge CD20 on cancer cells with CD3-expressing T cells to facilitate local T-cell activation and cancer-cell killing.
  • “As clinicians, our focus must remain patients’ wellbeing, along with favorable outcomes.
  • For odronextamab, it is particularly encouraging to see the unprecedented clinical results complemented by patient-reported outcomes that show quality of life and functional measures are maintained overall.
  • A replay of the conference call and webcast will be archived on the company's website for at least 30 days.

Long-term Follow-up Data From bluebird’s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling Adult Hemoglobin and Resolution of Vaso-Occlusive Events

Retrieved on: 
Saturday, December 9, 2023

The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.

Key Points: 
  • The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.
  • Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with the most patients treated and longest follow-up in the field.
  • As of February 13, 2023, 59 patients were treated across the entire clinical development program with follow-up beyond 8 years in the earliest treated patients.
  • In sickle cell disease, hemolysis happens too quickly due to the fragility of sickled red blood cells, resulting in hemolytic anemia.

Mirum Pharmaceuticals Presents New Data at The Liver Meeting®

Retrieved on: 
Monday, November 13, 2023

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.
  • “PFIC is a chronic, debilitating cholestatic liver disease characterized by elevated bile acids and pruritus so severe that patients can be listed for liver transplant even in the absence of disease progression.
  • These data suggest overall improved liver health with maralixibat treatment in patients with PFIC that can be maintained long-term.
  • No clinically meaningful changes in liver enzyme levels or hematology parameters were observed after volixibat treatment.

EMA and European Organisation for Research and Treatment of Cancer (EORTC) workshop: How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions?, European Medicines Agency, Amsterdam, the Nethe[...]

Retrieved on: 
Monday, December 18, 2023

Date

Key Points: 
  • Date
    - Thursday, 29 February 2024
    Location
    - European Medicines Agency, Amsterdam, the Netherlands
    Event summary
    The European Medicines Agency (EMA) and the European Organisation for Research and Treatment of Cancer (EORTC) are jointly organising a workshop on how patient-reported outcomes (PRO) as well as health-related quality of life (HRQoL) data can inform regulatory decisions (please see the draft agenda attached).
  • This workshop will bring together international experts from academia, learned societies, patients, regulatory agencies, health technology assessment bodies, and industry.
  • The aims of the workshop are to:
    - Understand the current global landscape on the use of PROs and HRQOL for evaluation of anti-cancer treatments across different stakeholder groups.
  • - Understand the use of validated PRO questionnaires to measure core HRQOL outcomes and disease-related symptoms; and PRO Item Libraries in the development of trial-specific/treatment-specific item lists.

ISHLT Supports Research Funding for Advanced Heart and Lung Disease: Applications Open Now

Retrieved on: 
Tuesday, November 7, 2023

The ISHLT Foundation funds research, including grants like these, to improve treatment options and patient outcomes around the world.

Key Points: 
  • The ISHLT Foundation funds research, including grants like these, to improve treatment options and patient outcomes around the world.
  • The Foundation also funds improved access to care by funding professional education opportunities around the world.
  • ISHLT/ICCAC VAD Coordinator Career Development Grant: Providing support to a VAD coordinator for a quality improvement and/or research project.
  • ISHLT/OH Frazier Grant in MCS Translational Research: Supporting mechanical circulatory support (MCS) research.

Attralus Announces Publication of Two Studies Evaluating Iodine (124I) Evuzamitide (AT-01) using PET/CT in the Journal of American College of Cardiology (JACC CV Imaging)

Retrieved on: 
Tuesday, November 7, 2023

124I-evuzamitide may be more sensitive than standard diagnostics, with cardiac uptake shown in ATTR patients who had negative 99mTc-PYP scans.

Key Points: 
  • 124I-evuzamitide may be more sensitive than standard diagnostics, with cardiac uptake shown in ATTR patients who had negative 99mTc-PYP scans.
  • The manuscripts from Brigham and Women’s Hospital (BWH) and from the University of Tennessee Graduate School of Medicine were published in the Journal of American College of Cardiology – Cardiovascular Imaging (JACC CV Imaging).
  • The manuscripts, entitled “Cardiac Amyloid Quantification Using 124I-Evuzamitide (124I-P5+14) Versus 18F-Florbetapir” and “Cardiac Amyloid Detection by PET/CT Imaging of Iodine (124I) Evuzamitide (124I-p5+14): A Phase 1/2 Study,” have been published online.
  • These studies suggest that 124I-evuzamitide is a promising novel radiotracer to detect and quantify cardiac amyloid in multiple types of amyloidosis.

bluebird bio to Present New and Updated Data from Gene Therapy Programs in Sickle Cell Disease and Beta-Thalassemia at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition

Retrieved on: 
Thursday, November 2, 2023

The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.

Key Points: 
  • The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.
  • lovo-cel treatment regimen largely reflects known side effects of hematopoietic stem cell collection and busulfan conditioning regimen and underlying sickle cell disease.
  • Updated iron management outcomes demonstrating sustained improvements in iron burden, with the majority of patients able to stop iron chelation therapy, will also be presented.
  • Safety of beti-cel treatment largely reflects the known side effects of hematopoietic stem cell collection and busulfan conditioning regimen.