A number of commentaries have been published over the past 4 years by the present author on the manifest flaws in the reference case value assessment framework of the Institute for Economic and Clinical Review. The recent release of the evidence report on sickle cell disease continues ICER's commitment to what has been described as the creation of imaginary worlds to support value assessment. The purpose of the present commentary is to continue the critiques that have been presented for earlier evidence reports. This is important because of the apparent willingness to take ICER's recommendations at face value rather than a critical review of the value assessment framework. The case presented here points to a number of weaknesses in the ICER framework: (i) the fabrication of imaginary constructs with a lifetime cost-per-incremental QALY framework; (ii) the consequent failure to meet the standards of normal science; (iii) the illogical reliance of assumptions drawn from the literature to create future scenarios; (iv) the rejection of hypothesis testing in favor of 'approximate information' and (v) a belief that in the construction of QALYS that the EQ-5D-3L utility scale has ratio properties. This last point is demonstrably false which means that the ICER value assessment framework collapses. It is impossible mathematically, a failure to meet the axioms of fundamental measurement, for an ordinal utility scale to be combined with time spent in a disease state. The result is that the pricing and access recommendations for Crizanlizumab, Voxelotor and L-glutamine in sickle cell disease (SCD) are complete nonsense and should be rejected.
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http://dx.doi.org/10.24926/iip.v11i2.3123 | DOI Listing |
Free Radic Biol Med
January 2025
University of Colorado, Anschutz Medical Campus School of Medicine| Translational research laboratory of Red Blood Cell Diseases and Hypoxia related illnesses| Cardiovascular Pulmonary Research (CVP) group, Pediatrics. Electronic address:
Lung tissue from human patients and murine models of sickle cell disease pulmonary hypertension (SCD-PH) show perivascular regions with excessive iron accumulation. The iron accumulation arises from chronic hemolysis and extravasation of hemoglobin (Hb) into the lung adventitial spaces, where it is linked to nitric oxide depletion, oxidative stress, inflammation, and tissue hypoxia, which collectively drive SCD-PH. Here, we tested the hypothesis that intrapulmonary delivery of hemopexin (Hpx) to the deep lung is effective at scavenging heme-iron and attenuating the progression of SCD-PH.
View Article and Find Full Text PDFJ Clin Med
January 2025
Department of Cardiovascular & Thoracic Anaesthesia and Critical Care, University Hospital of Martinique, F-97200 Fort de France, France.
Anesthesiology, the medical specialty that deals with the management of vital functions in patients undergoing surgery, has played an important role in the successful development of cardiac interventions worldwide. Tracing the historical roots of cardiac anesthesia and critical care from its inception in the late 1950s, a paradigm shift in perioperative care has been driven by a better understanding of the mechanisms of organ dysfunction in stressful conditions and technological advances regarding surgical approach, patient monitoring, and organ protection. Although progress in cardiac anesthesia and critical care lagged a little behind in Caribbean territories, successful achievements have been accomplished over the last forty years.
View Article and Find Full Text PDFMolecules
January 2025
Faculty of Science, Pavol Jozef Šafárik University in Košice, Park Angelinum 19, 040 01 Košice, Slovakia.
Hemoglobin is an oxygen-transport protein in red blood cells that interacts with multiple ligands, e.g., oxygen, carbon dioxide, carbon monoxide, and nitric oxide.
View Article and Find Full Text PDFMedicina (Kaunas)
January 2025
Department of Thalassemia Unit, Hatay Education and Research Hospital, Hatay 31027, Turkey.
This study aimed to identify asymptomatic brain lesions in patients with β-thalassemia major (TM) and sickle cell anemia (SCA) and evaluate the correlation of these lesions with factors such as splenectomy, thrombocytosis, and blood transfusions. A total of 26 patients with thalassemia major and 23 patients with sickle cell anemia were included. Ischemic lesions were categorized as lacunar, small vessel, or multifocal.
View Article and Find Full Text PDFGenes (Basel)
January 2025
Mnazi Mmoja Hospital (MMH), Kaunda Road, Vuga Street, Zanzibar 71102, Tanzania.
Background: This study aimed to describe Sickle Cell Disease (SCD) phenotypes, sociodemographic characteristics, healthcare, and clinical outcomes of patients with SCD attending Mnazi Mmoja Hospital (MMH) in Zanzibar.
Methods: Individuals who visited MMH between September 2021 and December 2022 and were known or suspected to have SCD were enrolled in the clinic. Sociodemographic characteristics and clinical features were documented, and laboratory tests were performed.
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