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Bone marrow transplantation for childhood leukemia: five years' experience in a pediatric hematology center
C Uderzo1, A Locasciulli, A Rovelli
1Clinica Pediatrica dell'Università di Milano, Ospedale San Gerardo, Monza (MI), Italy.
Insights
Pediatric bone marrow transplantation (BMT) for leukemia shows satisfactory outcomes, with improved survival rates and reduced early toxicity due to specialized center experience. Long-term endocrine and growth effects require further study.
Area of Science:
- Pediatric Hematology
- Oncology
- Transplantation Medicine
Background:
- Fifty-three children with leukemia underwent bone marrow transplantation (BMT) at a specialized pediatric center.
- Conditioning regimens varied based on disease type and BMT timing.
- The study evaluated the impact of center experience on BMT-related issues in pediatric patients.
Purpose of the Study:
- To assess the outcomes of bone marrow transplantation in children with leukemia.
- To analyze the influence of a pediatric hematology center's expertise on BMT-related complications.
- To evaluate disease-free survival and various organ functions post-transplantation.
Main Methods:
- Analysis of disease-free survival (DFS).
- Assessment of early BMT-related effects.
- Evaluation of hepatic, cardiac, and respiratory function.
- Monitoring of late endocrine effects using standard tests.
Main Results:
- Overall satisfactory outcomes with a 3-year DFS of 50.1%.
- Improved survival rates for lymphoblastic and acute myeloid leukemia.
- Reduced early morbidity and mortality (14.3% in the last 3 years) attributed to improved management.
- Normal cardiac and respiratory function observed at 3 years post-transplantation.
- Endocrine effects included compensated hypothyroidism and hypogonadism in some patients.
Conclusions:
- Pediatric BMT for leukemia can yield satisfactory outcomes, with survival rates improving over time.
- Specialized center experience significantly reduces early BMT-related toxicity and mortality.
- While major organ functions remain largely preserved, long-term endocrine and growth monitoring is essential.
- Pre-existing hepatitis did not worsen post-BMT hepatotoxicity.
Background:
Fifty-three children (39 male, 14 female, median age 9 years 3 months) with different forms of leukemia underwent bone marrow transplantation (BMT) at our center. Various conditioning regimens were used according to the disease and time of BMT. In this paper we evaluate the impact of the experience of a pediatric hematology center on BMT-related problems in children.
Methods:
We analyzed disease-free survival (DFS), early BMT-related effects, hepatic, cardiac and respiratory function and late endocrine effects as shown by standard instrumental and laboratory tests.
Results And Conclusions:
Outcome (overall median follow-up 34 months) was satisfactory. Three years DFS was 50.1% in all patients, 58.8% in lymphoblastic leukemia in 2nd complete remission (CR), and 50.0% in acute myeloid leukemia (some in 2nd or subsequent CR). Three of four patients with chronic myeloid leukemia were alive at 38 months. Management of the problems causing early post-BMT toxicity contributed to a progressive fall in early morbidity and mortality (14.3% in the last 3 years). Pre-BMT hepatitis in most patients was not associated with increased post-BMT hepatotoxicity. Cardiac function, even in patients given aggressive anthracycline treatment before BMT, remained normal 3 years after transplantation, as did respiratory function, although 8 cases presented subclinical restrictive and/or obstructive alterations. Compensated hypothyroidism was observed in 9 patients. Six boys received replacement treatment for hypogonadism. Severe height impairment was seen in 2 patients. Post-BMT endocrine and growth effects require a longer follow-up for definitive conclusions to be drawn.(ABSTRACT TRUNCATED AT 250 WORDS)