Current Understanding in the Management of Sickle Cell Disease

Abstract

Sickle cell disease (SCD), the commonest monogenetic disorder worldwide, represents a major public health burden because of its significant morbidity and mortality. Advances in molecular and cellular biology have resulted in an accumulation of knowledge on sickle cell pathophysiology and broadened our understanding of the complexity of this molecular disease with heterogeneous manifestations. Natural history studies and clinical trials have provided incremental data on clinical features, complications, and predictors of severity in SCD and, above all, have laid important recommendations for prevention and treatment of complications. Disease modifying therapies that have significantly improved survival of SCD patients have been identified over recent years. Despite increasingly successful therapies and better overall survival, patients continue to die especially with increasing age and health providers caring for SCD patients face major challenges. This article will highlight modern management of SCD and its impact on the lives of affected patients.

A retrospective analysis of the cost of hospitalizations for sickle cell disease with crisis in England, 2010/11

“…Patients and parents of children affected by SCD should also have a good awareness of symptoms and when and how to seek help. 32 Recently, SCD has achieved specialized commissioning status but the current specifications focus mainly on provision of acute care. 33 Links between primary care and community care could be improved through proper discharge planning, multi-disciplinary teams and better communication with primary care.…”

Oral Monomethyl Fumarate Therapy Ameliorates Retinopathy in a Humanized Mouse Model of Sickle Cell Disease

“…S ickle cell disease (SCD) is a common genetic mutation in the United States, principally among African Americans. Millions are affected worldwide (21,45). There is no cure for the disease.…”

“…There is no cure for the disease. Current treatment options are limited in that they are largely supportive in nature, targeting secondary manifestations of the disease that present after patients have already suffered much discomfort and irreversible damage to organs (21,45). Pain medication, repeated blood transfusion, and hospitalization during crises are the present standard for SCD management.…”

“…Pain medication, repeated blood transfusion, and hospitalization during crises are the present standard for SCD management. This reduces the incidence of crises and hospitalization in some, but is ineffective in a high number of SCD patients (1,5,15,21,28,45).…”

Extracellular microvesicle microRNAs in children with sickle cell anaemia with divergent clinical phenotypes

“…Sickle cell anaemia (SCA; i.e. HbSS) is the most common hereditary haemoglobinopathy and results from a unique genetic alteration in the HBB (β‐globin) gene (Inati et al , ; Rees et al , ). Sickling events lead to intravascular haemolysis and recurrent vaso‐occlusion, in association with vascular inflammation and endothelial activation (Hebbel et al , ).…”