Sickle cell disease represents a major hematological disease affecting millions of individuals of mostly African descent around the world. Patients present with a hemoglobinopathy that requires life-long red blood cell transfusion to minimize the known complications such as acute chest syndrome and cerebrovascular accidents due to red cell sickling in the microvasculature. However, this continuous transfusion requirement can lead to iron overload and all of its associated adverse events, as well as red cell alloimmunization due to the chronic exposure to donated red cells with subsequent increased risk of severe post-transfusion hemolysis. To mitigate the risk of alloimmunization, extended red blood cell antigen matching is recommended but challenging since the donor pool in many western countries is dissimilar to sickle cell patients.
In recent years a great deal of attention has been given to the development of biologicals to treat sickle cell disease. Historically, iron chelation is sometimes not well tolerated by some patients and chronic use of hydroxyurea can also be difficult due to associated adverse effects of chronic exposure. Thus, targeted biologicals with better tolerance will lead to improved compliance. Voxelotor and the monoclonal Crizanlizumab are examples of targeted therapies that modulate hemoglobin’s oxygen affinity and prevent vaso-occlusive crises respectively, have recently gained approval for use in patients with sickle cell disease. Similarly, with increasing use of gene editing technology such as the clustered regularly interspaced short palindromic repeats (CRISPR), the promise of treating sickle cell patients by editing their own hemoglobin genes have been reported in case reports. Indeed, CRISPR approaches to treating diseases could make traditional allogeneic transplantation in sickle cell patients less attractive. In this exciting period of discovery this special issue aims to showcase new areas in sickle cell therapies and improvement of transfusion safety.
For this special issue submissions encompassing new developments in the pathophysiology, diagnosis, and therapeutics of sickle cell disease are encouraged. These can include but not limited to original articles describing new biologicals, new ways of using existing agents, transplantation approaches involving minimal manipulation or graft using gene editing approaches. Likewise, reviews describing therapeutic approaches in the setting of sickle cell disease are also encouraged. Case reports will be considered if the description involves significant findings to merit publication or if a concise review of the topic involving the case is included. These will be considered upon the consensus and discretion of the Editors of the special topic.
Sickle cell disease represents a major hematological disease affecting millions of individuals of mostly African descent around the world. Patients present with a hemoglobinopathy that requires life-long red blood cell transfusion to minimize the known complications such as acute chest syndrome and cerebrovascular accidents due to red cell sickling in the microvasculature. However, this continuous transfusion requirement can lead to iron overload and all of its associated adverse events, as well as red cell alloimmunization due to the chronic exposure to donated red cells with subsequent increased risk of severe post-transfusion hemolysis. To mitigate the risk of alloimmunization, extended red blood cell antigen matching is recommended but challenging since the donor pool in many western countries is dissimilar to sickle cell patients.
In recent years a great deal of attention has been given to the development of biologicals to treat sickle cell disease. Historically, iron chelation is sometimes not well tolerated by some patients and chronic use of hydroxyurea can also be difficult due to associated adverse effects of chronic exposure. Thus, targeted biologicals with better tolerance will lead to improved compliance. Voxelotor and the monoclonal Crizanlizumab are examples of targeted therapies that modulate hemoglobin’s oxygen affinity and prevent vaso-occlusive crises respectively, have recently gained approval for use in patients with sickle cell disease. Similarly, with increasing use of gene editing technology such as the clustered regularly interspaced short palindromic repeats (CRISPR), the promise of treating sickle cell patients by editing their own hemoglobin genes have been reported in case reports. Indeed, CRISPR approaches to treating diseases could make traditional allogeneic transplantation in sickle cell patients less attractive. In this exciting period of discovery this special issue aims to showcase new areas in sickle cell therapies and improvement of transfusion safety.
For this special issue submissions encompassing new developments in the pathophysiology, diagnosis, and therapeutics of sickle cell disease are encouraged. These can include but not limited to original articles describing new biologicals, new ways of using existing agents, transplantation approaches involving minimal manipulation or graft using gene editing approaches. Likewise, reviews describing therapeutic approaches in the setting of sickle cell disease are also encouraged. Case reports will be considered if the description involves significant findings to merit publication or if a concise review of the topic involving the case is included. These will be considered upon the consensus and discretion of the Editors of the special topic.
