This trial is evaluating whether Plerixafor will improve 1 primary outcome in patients with Anemia, Sickle Cell. Measurement will happen over the course of 2 days.
This trial requires 15 total participants across 2 different treatment groups
This trial involves 2 different treatments. Plerixafor is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.
There were several clinical considerations that led us to feel there were enough anemic pediatric SSC patients that might benefit from such an evaluation. These were: 1) Anemic SSC patients present in a range of ages, including newborn babies. 2) A large number of SSC patients have anemia from other causes. 3) Many SSC patients present with other chronic medical conditions, and their enrollment criteria might be more appropriate for these studies or the treatment of which they are enrolled.
There are many types of treatment used for the management of SCD, including transfusions and medications, and the effect of aspirin on the process, can be variable. The most successful treatment is to give patients the optimal levels of oxygen that would support their red blood cell production and a regular check-up to monitor the patients for health problems that might be related to sickle cell anemia. A common treatment for all patients is giving daily folic Acid supplements to support red blood cell production and can prevent anemia and its related health complications. It is critical to consider the medical history of the patients when managing with anemia therapy and to make use of the available information to develop an effective plan to achieve desired treatment results.
Although genetic factors are involved in the development of anemia, they only account for a minority of anemia cases. Rather, the most likely causes of anemia, sickle cell disease or iron deficiency, affect hundreds of millions of people, and are potentially preventable with proper nutrition at a population level.
If an anemia is present, it is advisable to take folinic acid (10 micrograms/d) to increase red cell production because it reduces the sickling of red blood cells. (This can cause symptoms of anemia in those already malnourished or malnourished, for example, due to the effects of vitamin deficiency). With folinic acid, there is an increased iron absorption in stool, so it is advisable to give this to those with anemia for this reason. Vitamin A supplementation is also required. Patients treated for anemia are often iron deficient, because erythrocytes (red blood cells) have a shorter life span in those who are iron deficient.
Most common signs and symptoms of anemia are fatigue, shortness of breath, dizziness, paleness, tiredness and headache. However, signs and symptoms of anemia are not specific to anemia, sickle cell. Because of this, if you cannot pinpoint the cause of your symptoms, you should see a medical professional for further work-up.
There are several types of anemia. The common manifestation in children that are mostly caused by a reduction in hemoglobin levels. Children with sickle cell anemia are much more prone toward abnormal development such as strokes. The use of oral sirolimus as a first line therapy to treat children with sickle cell anemia has been approved by a number of major national and international bodies like the ECMO International Clinical Practice Guidelines.
About 5.2 million US adults are diagnosed with hemoglobinopathies. This makes up 13.4% of US adults. Anemia or sickle cell disease affects over 12 million Americans.
In a small group of patients with hemolysis and/or anemia, treatment with PTK improves HRQOL, and this effect is sustained at 12 weeks following cessation of PTK treatment.
Plerixafor is a synthetic type II CXCR4 ligand, which can affect the adhesion of various hematological cell types. It possesses very different biological activities including anti-tumor, anti-inflammatory, myelosuppressive, neurotropic and immunosuppressive activities. This compound often functions as anti-adhesion and anti-tumor activity for these hematological cells, and plays similar roles with related chemokines for immune cells including T and B cells. We believe the use of plerixafor and its related chemokines for cancer treatment could be a more effective and safe therapy for multiple myeloma and leukemia.
Anemia has been and remains an important cause of mortality among adults in the US. The seriousness of anemia remains unknown and warrants further study. Anemia, and the resultant increased risk for morbidity and mortality, warrants increased efforts to treat it.
Approximately 60% of HbF individuals have no identifiable cause, but when patients have anemia from other identifiable causes, the primary cause is usually from an unrecognized cause, such as Vitamin B12 deficiency.
There is evidence that regular NSAIDs may increase the risk of blood clots, so one may want to consult their physician before taking them. In a review article in Annals of Internal Medicine, Dr. Siegel describes the risks that ibuprofen may have. Although it appears that this risk is not a concern for NSAIDs, it is prudent to consult the drug information (DI) for each anti-injury drug before taking them. If one wishes to use an NSAID to reduce pain, then it is important to understand the risk of blood clots and consult with one's physician. The risk of bleeding of a bloodletting drug is also extremely low.