Pathobiology of Sickle Cell Disease Vaso-occlusion and Targeted Therapies

Analgesic management of uncomplicated acute sickle-cell pain crisis in pediatrics: a systematic review and meta-analysis

“… 20 , 23 Given these breakthroughs, the pathophysiological comprehension of VOC has sparked curiosity in the scientific literature. These three components (intravenous magnesium, arginine, and inhaled nitric oxide) have therefore revolutionized this in-depth understanding, as well as promoting new therapeutic approaches, 9 , 10 , 25 , 43 , 58 but they require more evidence.…”

“…4 , 5 , 6 , 7 Change to the polymer under a deoxygenated state of hemoglobin defines the hallmark of the condition that induces sickling formation; hence vaso-occlusive crisis (VOC), which is the label of SCD causing jammed microvascular beds, and leading to tissue ischemia. 8 , 9 , 10 Acute pain crisis is a key feature of the external expression of VOC, varying from mild to burdensome and debilitating pain which may continue from hours to weeks. Most often this is the reason to seek medical care in the emergency department (ED), representing approximately 70% of children with SCD, and is the result of increased readmission rates in hospitals.…”

Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

“…Newborn screening, penicillin prophylaxis, and vaccinations have contributed significantly to reduction in early childhood mortality (Lanzkron et al , ); however, few therapeutic options currently exist that manage the complications of the disease. While there are numerous targeted therapies under investigation aimed at disease modulation (Morrone et al , ), haematopoietic stem cell transplantation (HSCT) remains the only available curative option for the few patients with a matched sibling donor (Walters et al , ; Hsieh et al , ; Hsieh et al , ), with transplantation from alternative donors and gene therapy being investigated in multiple clinical trials.…”