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Intrathecal chemoprophylaxis after HSCT in children
Johanna Rubin1, Britt-Marie Frost, Johan Arvidson
1Department of Pediatrics, Karolinska University Hospital-Huddinge, Karolinska Institutet, Stockholm, Sweden. johanna.rubin@karolinska.se
Insights
Intrathecal (i.t.) chemotherapy after hematopoietic stem cell transplantation (HSCT) did not significantly reduce central nervous system (CNS) relapses in children with acute leukemia. This study suggests i.t. therapy has limited use and should be carefully considered, especially for high-risk cases.
Area of Science:
- Pediatric Oncology
- Hematology
- Stem Cell Transplantation
Background:
- Limited literature exists on intrathecal (i.t.) chemotherapy's efficacy and risks post-hematopoietic stem cell transplantation (HSCT) in pediatric acute leukemia.
- Practices for reducing central nervous system (CNS) leukemic relapse after HSCT vary across transplant centers.
Purpose of the Study:
- To compare the efficacy of i.t. chemotherapy in preventing CNS relapse in children undergoing HSCT for acute leukemia.
- To evaluate the risks and benefits of i.t. therapy in this patient population.
Main Methods:
- Retrospective comparison of 74 patients receiving i.t. therapy post-HSCT versus 46 patients not receiving i.t. therapy.
- Patients were from two Swedish transplant centers with differing i.t. therapy protocols.
- Primary endpoint: isolated CNS relapses; secondary endpoints: other relapses, death, neurological complications.
Main Results:
- No statistically significant difference in isolated CNS relapse rates between the i.t. therapy and no i.t. therapy groups (p > 0.05).
- I.t. chemotherapy did not reduce overall CNS relapse incidence or mortality.
- No significant protective effect of i.t. therapy was demonstrated.
Conclusions:
- Post-HSCT i.t. chemotherapy appears to have limited utility in treating childhood acute leukemia.
- Given the associated risks, i.t. therapy should be carefully evaluated and reserved for high-risk cases.
Abstract:
At present, the literature on the efficacy and risks of i.t. chemotherapy to children after HSCT is scarce. Current practices to reduce the risk of leukemic relapse in the CNS after HSCT differ between centers of transplantation. We compared 74 patients (56 ALL/18 AML), who received i.t. therapy post-HSCT with 46 patients (36 ALL/10 AML) who did not receive post-HSCT i.t. therapy. The patients were transplanted at the University Children's Hospital, Uppsala or the Karolinska University Hospital, Huddinge, two Swedish transplantation units with different routines concerning i.t. therapy after HSCT. The primary end-point was the number of isolated CNS relapses. Secondary end-points were other types of relapse, death, and neurological complications. There was no statistically significant difference in the incidence of CNS relapses between the groups (p > 0.05). I.t. therapy did not reduce the overall incidence of isolated CNS relapse or mortality. Our study did not demonstrate a protective effect of i.t. therapy indicating that post-HSCT i.t. therapy may only be of limited use in the treatment of acute childhood leukemia. We conclude that with the risks present, i.t. therapy should be carefully evaluated, and only considered in high-risk cases.
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