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Late effects in children after bone marrow transplantation: a review
F Locatelli1, G Giorgiani, A Pession
1Department of Pediatrics, University of Pavia, IRCCS Policlinico San Matteo, Italy.
Insights
Long-term survivors of pediatric bone marrow transplants (BMT) face significant side effects, primarily from conditioning regimens like total body irradiation (TBI). These effects impact growth, endocrine function, and organ systems, requiring vigilant pediatric follow-up.
Area of Science:
- Pediatric Hematology/Oncology
- Transplantation Medicine
- Long-term Survivorship Research
Background:
- Increasing numbers of pediatric bone marrow transplant (BMT) survivors necessitate understanding long-term sequelae.
- Pediatricians face challenges in monitoring unique long-term health issues due to extended life expectancy post-BMT.
- Previous studies highlight the need for comprehensive follow-up to detect adverse effects.
Purpose of the Study:
- To review and analyze frequently observed long-term side effects in pediatric BMT survivors.
- To identify the primary causes of these sequelae, focusing on conditioning regimens.
- To inform pediatricians about potential complications affecting growth, endocrine, ocular, and pulmonary systems.
Main Methods:
- Review of existing literature on long-term side effects in pediatric BMT recipients.
- Analysis of sequelae affecting specific organ systems including lungs, eyes, brain, and endocrine system.
- Identification of conditioning agents, particularly radiotherapy, as major contributors to toxicity.
Main Results:
- Conditioning regimens, especially total body irradiation (TBI) and cranial irradiation, are linked to most long-term toxicities.
- Growth impairment, growth hormone deficiency, and delayed puberty are common, particularly after TBI.
- Thyroid dysfunction (30-40%), gonadal failure, skin/mucosal abnormalities, cataracts, dry eyes, and lung function issues are frequently reported.
Conclusions:
- Radiotherapy is a key factor in the development of long-term side effects following pediatric BMT.
- Comprehensive monitoring for endocrine, growth, ocular, and pulmonary complications is crucial for pediatric BMT survivors.
- Further research and clinical vigilance are needed to manage and mitigate these late effects.
Abstract:
Since the number of children receiving a bone marrow transplantation (BMT) and becoming long-term survivors continues to increase, more attention has to be paid to detect long-term side effects in these unique patients. Follow-up studies to timely identify these untoward sequelae are a matter of particular concern for pediatricians due to the longer life expectancy of children cured by BMT. The more frequently recognized sequelae affecting lung, eyes, brain and the endocrine system have been analyzed in this review. The majority of long-term side effects could be related to the conditioning regimens employed to prepare children before marrow transplantation and radiotherapy has been indicated as the most important agent determining deleterious toxicities. Most children receiving BMT present a decreased growth velocity and this growth impairment is especially observed in patients receiving total body irradiation (TBI) and prophylactic cranial irradiation prior to marrow transplant. Growth hormone deficiency could be demonstrated in the majority of patients with a reduced growth rate, even though an impairment of liver somatomedin production or a direct radiation-induced skeletal dysplasia could not be excluded. Overt and compensated hypothyroidism have been reported after TBI and patients given single dose radiotherapy are at greater risk with an overall incidence of thyroid function abnormalities approaching 30-40%. Delayed puberty development was reported in boys and girls after a TBI-containing conditioning regimen, whereas patients given BMT for severe aplastic anaemia presented a normal puberty. The absence of pubertal growth spurt contributes to the growth impairment of prepubertal children. In post-pubertal patients amenorrhea, azoospermia and gonadal failure can be observed after radiotherapy and several patients can require hormonal substitutive therapy. Skin and mucosal abnormalities referred to teguments involvement by chronic graft-versus-host disease (GVHD). Moreover, alopecia or abnormal pigmentation of the skin are observed in patients given busulfan as part of their myeloablative therapy. Cataracts are a well recognized complication of children receiving ionizing radiations and chronic steroid therapy. Again, posterior subcapsular cataracts occur more frequently in patients given TB1 as single exposure. Decreased lacrimal gland function, with impairment of tear production is another late effect of irradiation to the eye. Lung function abnormalities are not rare after transplant and may cause late mortality and morbidity.(ABSTRACT TRUNCATED AT 400 WORDS)