Beta thalassemias are a group of inherited blood disorders caused by reduced or absent synthesis of the beta chains of hemoglobin. This defect results in chronic anemia and reduced quality of life that negatively impacts patients’ physical, social, and emotional well-being. Repeated blood transfusions can cause severe problems associated with iron overload for example.
Recently, erythroid maturation agents have been developed to treat patients who have serious blood disorders marked by these late-stage defects in the maturation of red blood cells. A recombinant fusion protein can now be used in adults with transfusion dependent beta-thalassemia (approved in the US and EMA in 2020), and has shown to reduce the transfusion burden.
In this context, AplusA has set up a proprietary tracking program to understand the market situation and continuously monitor the evolution of the management of these patients. This research is a fully syndicated patient chart study to measure key behavioral metrics (prescription by patient type) (note: in some smaller EU countries individual patient record forms may be replaced by stated caseload questions).
Typically 70 Onco-Haematologists initiating beta-thalassemia anemia treatment answer per country, and provide 3 patient charts on average per physician. Questions include patient characteristics, treatments, transfusions status, iron overload status.
- Gender / Age
- Year of diagnosis
- BThal Mayor, Intermediate
- Anemia status (anemia patients requiring treatment)
- Hb and Serum Ferritin levels
- Thresholds for diagnosing and treating iron overload
- Bone Marrow Transplant (yes/no)
- # of different treatments received for anemia
- Current and previous treatments
- Treatment goal(s)
- Reasons and modalities
- First RBC-T received
- Year of transfusion dependence
- Number of RBC-T events in past 6 months
- Number of units in past 6 months
- Date of last transfusion
- RBC units during last transfusion
- Change of frequency of RBC-T compared to 6 months ago
- Change of RBC-T units compared to 6 months ago
- Satisfaction with response to RBC-T