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Intensive conditioning regimen for bone marrow transplantation in children with high-risk haematological malignancies
C H Cole1, S Pritchard, P C Rogers
1Department of Paediatric Oncology/Haematology, British Columbia Children's Hospital, Vancouver, Canada.
Insights
This study found that a conditioning regimen of total body irradiation, etoposide, and cyclophosphamide is well-tolerated in pediatric bone marrow transplantation (BMT) for advanced hematological malignancies, showing mild regimen-related toxicity.
Area of Science:
- Pediatric Hematology Oncology
- Stem Cell Transplantation
- Cancer Treatment Regimens
Background:
- Advanced hematological malignancies in children often require intensive treatment.
- Bone marrow transplantation (BMT) is a critical therapeutic option for these conditions.
- Standardized conditioning regimens are essential for successful BMT outcomes.
Purpose of the Study:
- To evaluate the safety and efficacy of a specific conditioning regimen for pediatric BMT.
- To assess regimen-related toxicity (RRT) and engraftment times in children undergoing BMT.
- To analyze survival rates and causes of mortality in this patient cohort.
Main Methods:
- A cohort of 21 children (10 months to 15 years) received BMT between 1987 and 1991.
- The conditioning regimen included total body irradiation (TBI), etoposide, and cyclophosphamide.
- Patients underwent either allogeneic or autologous BMT, with marrow purged by 4-hydroperoxycyclophosphamide in the latter.
Main Results:
- Median myeloid engraftment was faster in allogeneic BMT (19 days) versus autologous BMT (28 days) (P < .01).
- Mucositis was the primary RRT; other toxicities included GI issues, and veno-occlusive disease of the liver.
- Ten patients survived with a median follow-up of 44 months; deaths were due to toxicity or relapsed disease.
Conclusions:
- The TBI, etoposide, and cyclophosphamide conditioning regimen is well-tolerated in pediatric BMT.
- Regimen-related toxicities were generally mild and reversible.
- The regimen demonstrates acceptable survival rates for children with advanced hematological malignancies.
Abstract:
Between September 1987 and May 1991, 21 children aged 10 months to 15 years (median 9 years) underwent bone marrow transplantation (BMT) for advanced haematological malignancies using a conditioning regimen consisting of total body irradiation (TBI), etoposide 1.8 g/m2 by continuous infusion, and cyclophosphamide 2 g/m2 on 3 consecutive days. The patients included 14 with acute lymphoblastic leukaemia (ALL), 1 with chronic myeloid leukaemia (CML), 1 with juvenile CML, 4 with non-Hodgkin's lymphoma and 1 with acute nonlymphocytic leukaemia. Eleven had an allogeneic BMT from an HLA-matched sibling, and 1 from an unrelated donor. Nine patients received 4-hydroperoxycyclophosphamide purged autologous marrow. Median time to myeloid engraftment (ANC > 500/microliters) was 19 days in allogeneic BMT patients and 28 days in autologous BMT patients (P < .01). Mucositis was the major regimen-related toxicity (RRT). GI toxicity in the form of diarrhoea affected ten patients and five had veno-occlusive disease of the liver. Two patients had mild bladder toxicity and one died of renal toxicity. There was no CNS or cardiac toxicity. There was no significant difference in the incidence of toxicity according to the type of BMT (autologous or allogeneic), total dose, or sequence of TBI. With a median follow-up of 44 months, ten patients are alive (6/12 allogeneic BMT patients and 4/9 autologous BMT patients). Of the 11 deaths, four were related to toxicity (2 aspergillus, 1 haemorrhage following liver biopsy, and 1 from haemolytic-uraemic syndrome), and 4/12 allogeneic and 4/9 autologous BMT patients died from relapsed disease. This conditioning regimen is well tolerated in children, demonstrating mild and reversible RRT.