Medical Professionals

Abstract

Significance: Sickle cell disease (SCD) is the most common inherited diathesis affecting mostly underserved populations globally. SCD is characterized by chronic pain and fatigue, severe acute painful crises requiring hospitalization and opioids, strokes, multiorgan damage, and a shortened life span. Symptoms may appear shortly after birth, and, in less developed countries, most children with SCD die before attaining age 5. Hematopoietic stem cell transplant and gene therapy offer a curative therapeutic approach, but, due to many challenges, are limited in their availability and effectiveness for a majority of persons with SCD. A critical unmet need is to develop safe and effective novel targeted therapies. A wide array of drugs currently undergoing clinical investigation hold promise for an expanded pharmacological armamentarium against SCD. Recent Advances:Hydroxyurea, the most widely used intervention for SCD management, has improved the survival in the Western world and more recently, voxelotor (R-state-stabilizer), l-glutamine, and crizanlizumab (anti-P-selectin…

Chronic pain is the most common complication affecting adults with sickle cell disease (SCD).1 Pain profoundly affects people’s quality of life, functional ability, and health care utilization. Clinicians are often unsuccessful at addressing chronic pain in SCD, especially among the large number of patients for whom nonopioid analgesics aren’t sufficient and those who have developed opioid tolerance. Why aren’t we doing better?

We believe the medical community is looking at sickle cell pain through the wrong lens — treating it as a hematologic problem, while overlooking the neurologic, psychological, and social aspects of chronic pain. Given the current understanding of the neuropsychology of pain, the health care system has the ability to manage sickle cell pain more effectively. Doing so will require accepting a broader understanding of the experience of pain and devoting adequate resources to addressing its multiple dimensions.

The biopsychosocial model helps capture people’s experience of chronic pain…

Abstract

Sickle cell disease (SCD), a distinctive and often overlooked illness in the 21st century, is a congenital blood disorder characterized by considerable phenotypic diversity. It comprises a group of disorders, with sickle cell anemia (SCA) being the most prevalent and serious genotype. Although there have been some systematic reviews of global data, worldwide statistics regarding SCD prevalence, morbidity, and mortality remain scarce. In developed countries with a lower number of sickle cell patients, cutting-edge technologies have led to the development of new treatments. However, in developing settings where sickle cell disease (SCD) is more prevalent, medical management, rather than a cure, still relies on the use of hydroxyurea, blood transfusions, and analgesics. This is a disease that affects red blood cells, consequently affecting most organs in diverse manners. We discuss its etiology and the advent of new technologies, but the aim of this study is to understand the various…

Dear SCD Partners,

Today, the Department of Health and Human Services announced that two drug manufacturers with Food and Drug Administration-approved gene therapies for sickle cell disease have entered into agreements with the Centers for Medicare & Medicaid Services (CMS) to participate in the Cell and Gene Therapy Access Model (press release attached).

The voluntary model, led by the Center for Medicare and Medicaid Innovation, will test outcomes-based agreements for cell and gene therapies, with the aim to improve health outcomes, increase access to cell and gene therapies, and lower health care costs. These outcomes-based agreements will tie payments to whether the therapy improves health outcomes for people with Medicaid who receive these drugs.