A study of 736 patients with sickle cell disease (SCD) divided into 3 age groups (0-5; 6-15, and >15 years) who underwent hematopoietic cell transplantation (HCT) with identical sibling donors found that the youngest age group experienced 4-year event-free survival (EFS) of 93%.
Patients underwent transplant between 1986-2017 and the results were reported through the European Blood and Marrow Transplant (EBMT)/Eurocord registries.
HCT outcomes by age group are shown below.
|Outcome||0-5 years||6-15 years||>15 years||p-value|
|Overall Survival – 4 years||100%||95%||88%||0.007|
|EFS – 4 years||93%||89%||81%||0.003|
|Grade II-V acute GVHD||10%||18%||17%||0.047|
The researchers recommend “that with the excellent results for patients younger than 5, when an identical sibling is available, HCT should be proposed early in life before complications occur.”
When discussing treatment options with patients and families, HCT complications such as graft rejection, growth impairment, gonadal dysfunction, infertility and infections need to be taken into account in making the decision to offer HCT to young children with a non-malignant disorder. The authors noted that “the occurrence of these undesirable late effects must be balanced with the potential complications of SCD itself (cognitive deficits, iron overload, asplenism, and low quality of life.)”