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Growth and endocrine function in children with acute myeloid leukaemia after bone marrow transplantation using
Z Afify1, P J Shaw, A Clavano-Harding
1Department of Oncology, The New Children's Hospital, (Royal Alexandra Hospital for Children), Sydney, Australia.
Insights
Children with acute myeloid leukemia (AML) undergoing bone marrow transplantation (BMT) with busulfan and cyclophosphamide (BUCY) experience no significant growth issues but frequently develop gonadal dysfunction, especially girls.
Area of Science:
- Pediatric Oncology
- Hematology
- Endocrinology
Background:
- Longitudinal data on growth and endocrine function in pediatric patients with acute myeloid leukemia (AML) undergoing bone marrow transplantation (BMT) are limited.
- Busulfan and cyclophosphamide (BUCY) is a chemotherapy regimen used in BMT for AML.
Purpose of the Study:
- To evaluate the long-term growth and endocrine outcomes in children with AML who received BUCY-based BMT.
- To identify potential risk factors for impaired growth and endocrine dysfunction post-BMT.
Main Methods:
- A cohort of 23 children with AML surviving at least 2 years post-BMT with BUCY was studied.
- Growth (height standard deviation score) and endocrine function (gonadotropins, sex steroid replacement) were assessed annually for up to 10 years post-transplant.
- Data were analyzed to determine changes in height and the prevalence of gonadal dysfunction.
Main Results:
- The mean height standard deviation score (HtSDS) showed a slight decrease from diagnosis to BMT but remained stable thereafter.
- No significant relationship was found between age at BMT and subsequent change in HtSDS.
- Gonadal dysfunction was prominent, with 5 of 6 girls requiring sex steroid replacement and 6 of 12 boys exhibiting abnormal gonadotropins.
Conclusions:
- Pediatric AML patients undergoing BUCY-based BMT do not typically experience significant growth impairment.
- Gonadal dysfunction is a common and significant long-term complication, particularly in female patients.
- Regular endocrine monitoring is crucial for survivors of BUCY-based BMT for AML.
Abstract:
Longitudinal studies of growth and endocrine function of children with AML transplanted with BUCY are limited. We report a cohort of 23 children with AML transplanted (15 autologous and eight allogeneic) following a single chemotherapy protocol and surviving at least 2 years after BMT. Busulfan was given as a single daily dose. Growth and endocrine function was evaluated yearly from one up to 10 years post transplant (median 4.9 years). The mean height standard deviation score (HtSDS) of the entire group decreased from 0.01 (s.e.m. +/- 0.25) at diagnosis to -0.38 (+/- 0.28) at BMT (P = 0.001). There was no statistically significant difference between HtSDS at BMT and yearly HtSDS from 1 to 5 years post BMT. There was no significant relationship between age at BMT and subsequent change in HtSDS. To date, five of six girls have needed sex steroid replacement. Six of 12 evaluable boys had abnormal gonadotrophins, but none required sex steroid replacement. Children with AML who undergo BMT with BUCY show no significant growth impairment, but gonadal dysfunction is prominent, particularly in girls. Bone Marrow Transplantation (2000).