Patients

Abstract

BACKGROUND

Hemoglobin SC (HbSC) is a common sickle hemoglobinopathy that causes acute complications, chronic organ damage, and early death with no established disease-modifying treatment. In this trial, we examined the safety and efficacy of hydroxyurea treatment in patients with HbSC.

METHODS

Prospective Identification of Variables as Outcomes for Treatment (PIVOT) was a double-blind, randomized, placebo-controlled, non-inferiority phase 2 trial in which we assigned children and adults with HbSC in Ghana to 12 months of hydroxyurea or placebo. The primary end point was hematologic dose-limiting toxicities (DLTs), including cytopenias or elevated hemoglobin levels during 12 months of blinded treatment. Clinical end points included vaso-occlusive pain events, acute chest syndrome, hospitalizations, transfusions, and malaria. Quality-of-life measures, organ function assessments, and rheological measurements were also collected.

RESULTS

Abstract

Sickle cell disease (SCD), which affects approximately 100,000 individuals in the USA and more than 3 million worldwide, is caused by mutations in the βb globin gene that result in sickle hemoglobin production. Sickle hemoglobin polymerization leads to red blood cell sickling, chronic hemolysis and vaso-occlusion. Acute and chronic pain as well as end-organ damage occur throughout the lifespan of individuals living with SCD resulting in significant disease morbidity and a median life expectancy of 43 years in the USA. In this review, we discuss advances in the diagnosis and management of four major complications: acute and chronic pain, cardiopulmonary disease, central nervous system disease and kidney disease. We also discuss advances in disease-modifying and curative therapeutic options for SCD. The recent availability of L-glutamine, crizanlizumab and voxelotor provides an alternative or supplement to hydroxyurea, which remains the mainstay for disease-modifying therapy. Five-year event-free and overall survival rates remain high…

A new type of bone marrow transplant can cure sickle cell disease with only half of the donor's cell proteins matching, according to new clinical trial results published in the New England Journal of Medicine.

Why it matters: The procedure would greatly expand the pool of potential donors, in addition to costing less than one-quarter of the price of innovative gene therapies for the condition that have earned Food and Drug Administration approval in recent years.

Context: Sickle cell anemia is considered a rare disease, but it's the most common inherited blood disorder. It affects about 100,000 people in the U.S. and 8 million people across the world.

• The disease changes the shape of red blood cells in a way that can block blood flow to the rest of the body. Sickle cell anemia is associated with extreme pain and other health complications including strokes and organ damage.

What they…

Welcome to our group My Site Group! A space for us to connect and share with each other. Start by posting your thoughts, sharing media, or creating a poll.