Sickle cell disease (SCD) is a monogenic disorder that afflicts approximately 100,000
Americans and millions of people worldwide. It is characterized by hemolytic anemia, vaso …

Chimerism analysis has become an important tool for the peri-transplant surveillance of
engraftment. It offers the possibility to realize impending graft rejection and can serve as an …

Background Neonates and children differ from adults in physiology, pharmacologic
responses to drugs, epidemiology, and long-term consequences of thrombosis. This …

Gene therapy (GT) provides a potentially curative treatment option for patients with sickle
cell disease (SCD); however, the occurrence of myeloid malignancies in GT clinical trials …

Importance Myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) is
curative for children with severe sickle cell disease, but toxicity may be prohibitive for adults …

Background Myeloablative allogeneic hematopoietic stem-cell transplantation is curative in
children with sickle cell disease, but in adults the procedure is unduly toxic. Graft rejection …

Allogeneic hematopoietic stem-cell transplantation (HSCT) is the only curative treatment for
sickle cell disease (SCD); nevertheless, its use has been limited by the risk of …

This chapter about antithrombotic therapy in neonates and children is part of the
Antithrombotic and Thrombolytic Therapy: American College of Chest Physicians Evidence …

Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted
treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with …

Curative therapy for individuals with severe sickle cell disease (SCD) who lack an HLA-
identical sibling donor has been frustratingly elusive. In with the goal of improving …