Blast crisis is the terminal phase of CML and clinically behaves like an acute leukemia. Drug treatment will usually stop this progression if started early. One of the drivers of the progression from chronic phase through acceleration and blast crisis is the acquisition of new chromosomal abnormalities (in addition to the Philadelphia chromosome). Some patients may already be in the accelerated phase or blast crisis by the time they are diagnosed. Chronic phase
Approximately 85% of patients with CML are in the chronic phase at the time of diagnosis. During this phase, patients are usually asymptomatic or have only mild symptoms of fatigue, left side pain, joint and/or hip pain, or abdominal fullness. The duration of chronic phase is variable and depends on how early the disease was diagnosed as well as the therapies used. In the absence of treatment, the disease progresses to an accelerated phase. Precise patient staging based on clinical markers and personal genomic profile will likely prove beneficial in the assessment of disease history with respect to progression risk. Accelerated phase
Criteria for diagnosing transition into the accelerated phase are somewhat variable; the most widely used criteria are those put forward by investigators at M.D. Anderson Cancer Center, by Sokal et al., and the World Health Organization.
Efgartigimod alfa, sold under the brand name Vyvgart, is a medication used to treat myasthenia gravis.The most common side effects include respiratory tract infections, headache, and urinary tract infections.Efgartigimod alfa is a neonatal Fc receptor blocker and is a new class of medication. It is an antibody fragment that binds to the neonatal Fc receptor (FcRn), preventing FcRn from recycling immunoglobulin G (IgG) back into the blood. The medication causes a reduction in overall levels of IgG, including the abnormal acetylcholine receptor (AChR) antibodies that are present in myasthenia gravis. It was approved for medical use in the United States in December 2021, and in the European Union in August 2022. Medical uses
Efgartigimod alfa is indicated for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody positive. History
The safety and efficacy of efgartigimod alfa were evaluated in a 26-week clinical study of 167 participants with myasthenia gravis who were randomized to receive either efgartigimod alfa or placebo. The study showed that more participants with myasthenia gravis with antibodies responded to treatment during the first cycle of efgartigimod alfa (68%) compared to those who received placebo (30%) on a measure that assesses the impact of myasthenia gravis on daily function. More participants receiving efgartigimod alfa also demonstrated response on a measure of muscle weakness compared to placebo. Pharmacodynamics
Efgartigimod alfa as a drug is an antibody fragment that binds to the neonatal Fc receptor. When this binding happens, the IgG recycling process is blocked.