Transplant-related intracranial hemorrhage occurred in 4.7% of pediatric thalassemia major patients after bone marrow transplant and was associated with significantly worse overall survival (51% vs. 88%), disease-free survival (37% vs. 76%), and higher transplant-related mortality, with age at transplant as the main positive predictor.
Key Findings
Results
The incidence of transplant-related intracranial hemorrhage (ICH) in pediatric thalassemia major patients after bone marrow transplant was 4.7%.
28 patients out of 595 total patients developed ICH
Data collected retrospectively from four participating centers in the subcontinent from 2010 to 2023
All patients underwent first matched-related BMT
Mean age of the patient population at transplant was 8.32 ± 4.09 years
Results
ICH presented predominantly with headache and hypertension, most commonly in the early post-transplant period.
Headache was present in 60% of ICH patients
Hypertension was present in 57% of ICH patients
Most ICH events occurred in the early post-transplant period, defined as less than 28 days post-BMT
Results
Overall survival and disease-free survival were significantly worse in patients who developed ICH compared to those who did not.
Overall survival in ICH patients was 51% compared to 88% in non-ICH patients
Disease-free survival in ICH patients was 37% compared to 76% in non-ICH patients
These outcomes were based on 28 patients with ICH versus the remaining 567 patients
Results
Transplant-related mortality was higher in patients who developed ICH than in patients who did not.
The paper reports transplant-related mortality was higher in the ICH group compared to non-ICH patients
There was no statistically significant difference in rejection between the two groups
CNS complications after BMT were noted to have 'a major impact on overall survival, disease-free survival, and transplant-related mortality'
Results
Age at time of bone marrow transplant was identified as the main positive predictor of developing ICH after BMT.
Age at transplant was described as 'the positive predictor of developing ICH after BMT'
Age at transplant was described as 'the main predictor that emerged at the time of developing ICH'
The mean age of the overall patient population at transplant was 8.32 ± 4.09 years
No other statistically significant risk factors were reported as independent predictors
What This Means
This research examined how often bleeding in the brain (intracranial hemorrhage, or ICH) occurs in children with thalassemia major who receive a bone marrow transplant, and what happens to those children afterward. Looking at 595 children treated across four centers in South Asia over 13 years, the researchers found that about 1 in 20 children (4.7%) developed this serious complication. It most often appeared within the first month after transplant and typically came with symptoms like headache and high blood pressure.
Children who developed brain bleeding had dramatically worse outcomes than those who did not. Their overall survival rate was 51% compared to 88% in children without ICH, and their disease-free survival was 37% compared to 76%. Transplant-related deaths were also more common in the ICH group. The researchers found that older age at the time of transplant was the main factor that predicted which children were more likely to develop this complication.
This research suggests that intracranial hemorrhage is a serious but relatively uncommon complication of bone marrow transplantation in children with thalassemia major, and that it substantially worsens survival outcomes. The finding that older age at transplant increases risk may support the existing clinical rationale for performing transplants earlier in life when possible. Early recognition of warning signs like headache and high blood pressure in the first month after transplant could be important for prompt diagnosis and management.
Batool A, Khalid S, Ainur Z, Naghmi N, Niazi M, Reddy M, et al.. (2026). Incidence, Risk Factors, and Outcome of Transplant-Related Intra-Cranial Hemorrhage in Pediatric Thalassemia Major Patients.. Pediatric transplantation. https://doi.org/10.1111/petr.70350