Crizanlizumab (SEG101) for Anemia, Sickle Cell

The causes of anemia and sickle cell disease are relatively well understood. These include blood disorders such as thalassemia, and chronic obstructive pulmonary disease; environmental exposures such as lead; iron deficiency; infections; and genetic disorders such as sickle cell disease.

There is no evidence for any cure for sickle cell anemia. Treatment of anemia can be successful in preventing complications, but does not necessarily eliminate the anemia.

Anemia is present in 20-30% of African Americans and is caused by an abnormal production of hemoglobin (the red blood cell). Sickle cell anemia is a defect of the red blood cell and is one form of blood disorder, sickle cell trait. The prevalence of sickle cell anemia in African American blood donors is about 10 times higher than the prevalence of sickle cell trait among African American donors of African, Arab and Middle Eastern descent. African American subjects with sickle cell trait who have no symptoms rarely develop sickle cell anemia. Because sickle cell trait in African Americans is associated with a relatively high prevalence of anemia, anemia is a more common cause of morbidity and mortality than previously reported.

For the second consecutive year, The American College of Physicians has found that anemia, which is a life-threatening disease, affects an estimated 5% of adults in the United States. For most, the condition is manageable. For others - including children and adults in special populations - and for the many more who may not be treated because of lack of access to proper care, anemia is most frequently caused by a combination of factors. That nearly half of individuals diagnosed with anemia are not treated for this disease underscores the need to improve care through more universal preventive and secondary prevention initiatives. The overall number of cases diagnosed with sickle cell disease appears to have slightly increased in the last decades.

A low hemoglobin level should be considered in patients with painful crises without other obvious causes for the acute illness, because they may prove to be sickle cell disease and, if diagnosed too late, could have fatal complications. Physicians must be aware of the frequent combination of symptoms of acute splenic enlargement and low haemoglobin, because these patients may present sickle cell anemia as acute abdominal pain, and, if diagnosed too late, may prove fatal complications.

A wide range of treatments are available to treat anemia, anemia due to sickle cell disease (SCD), and thalassemia. The use of iron therapy alone did not modify the time to anemia resolution in our clinic. Iron therapy did shorten the time to normalization of hemoglobin and to return to pre-treatment values in patients on the anemia treatment only group.

Selexys appears well tolerated in this open-label study, but its safety was dependent on the patient population studied. It demonstrates promise as a treatment for patients with ESCC and other types of solid tumours.

Patients showed an increased rate of anemia in the crizanlizumab group at week 4, but all improvements remained statistically insignificant after 4 weeks. The most frequent adverse effects were pneumonias, headache, infusion site pain, and arthralgia. For safety reasons, it was decided that anemia in an individual should not be used as an endpoint for treatments like crizanlizumab. The trial in this study failed to demonstrate improvements in all endpoints.

Data from a recent study of this study show that crizanlizumab has a positive impact on the QoL of children and adolescents with HSC and anemia. Considering the clinical improvement and the safety profile, we believe crizanlizumab should be considered as the new therapy of choice for patients with SCD and anemia.

There were many possible causes of anemia in these patients, but those with sickle cell disease were probably the most common. The presence of the hemoglobin S allele is associated with sickle cell crises. Sickle cell disease is probably more deadly for adults than for children. Adult patients with sickle cell anemia may require blood transfusions. However, since blood transfusions may be dangerous for all patients, we think it is better to use these medicines cautiously. It is important to talk to your doctor, who may refer you to a specialist anemia clinic. There is no cure for anemia or sickle cell disease, although medical therapies have been successful to treat these disorders.

Anemia remains a major, yet under-recognized comorbidity in pediatric population that can significantly impact both the physical and cognitive health of patients. Future studies are needed to identify the factors associated with anemia, and to determine the clinical and economic implications in health care.