What could explain failure to luspatercept in patients with transfusion-dependent beta thalassemia?
1 Answer
Mednet MemberInvited Expert
Pediatric Hematology/Oncology · Weill Cornell Medical College
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Luspatercept works by trapping TGF beta ligands (GDF 11 and 15) and optimizing maturation and differentiation of erythroid precursors, thus facilitating increased output of red cells.
In individuals who have beta zero-beta zero thalassemia, this must mean that the cells coming out would contain hemoglobin F or A2, since they cannot make HbA. Factors which influence the expression of the gamma and delta genes could play a role in response to luspatercept in these situations. In individuals who hav...
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