Although allogeneic hematopoietic cell transplantation (HCT) is a potential cure for hemoglobinopathies such as sickle cell disease (SCD) and thalassemia major, avoiding complications and mortality caused by the underlying disease or transplant requires anticipation, early recognition, and adequate medical support. HCT recipients require systematic monitoring, comprehensive follow up, and clinical intervention when indicated.
An international panel of HCT experts has updated late effects screening guidelines for pediatric transplantation for hemoglobinopathies, focusing on sickle cell disease and thalassemia, which include specific recommendations for long-term follow-up care that take into consideration both disease- and transplant-related effects.
The consensus report includes guidelines on:
- Follow-up recommendations after HCT for thalassemia
- SCD transplant-specific follow-up
- Neurocognitive follow-up
- HCT-related complications
- Gonadal function and fertility preservation
- Assessment of health-related quality of life (HRQOL)
- Assessment of health care utilization
The expert panel members note that the guidelines may also be applicable to novel curative therapies such as autologous HCT after hematopoietic progenitor stem cell gene modification and other emerging curative interventions.