Endocrinological late complications after hematopoietic SCT in children

Cohen, Amnon; Békássy, Albert N.; Gaiero, Alberto; Faraci, Maura; Zecca, S; Thewis, André; Dini, Giorgio

doi:10.1038/bmt.2008.54

Cited by 82 publications

(79 citation statements)

References 31 publications

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“…Besides the well-known damage to puberty and gonadal function, 3 we confirm a consequent impact on growth, as previously noted. 28 Moreover, BU increased widely the risk of overweight (OR ¼ 3.9).…”

Section: Health Status After Hct F Bernard Et Alsupporting

confidence: 72%

“…This increased morbidity has often been attributed to the late toxicity of preparative regimens. 3,4 A detailed knowledge of the latter is essential: first, for physicians to develop lessening strategies, whether by adapting preparative regimens or by careful monitoring of patients, then for adult survivors to receive accurate information and care and finally for researchers to investigate physiopathologic pathways. The great majority of myeloablative conditioning in children includes either TBI or BU.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Health status of childhood leukemia survivors who received hematopoietic cell transplantation after BU or TBI: an LEA study

Bernard

Auquier

Herrmann

et al. 2014

Bone Marrow Transplant

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The purpose of this multicenter study was to compare the long-term impact of a preparative regimen with either BUBU or TBI on health status and quality of life (QoL) in childhood acute leukemia survivors treated with hematopoietic SCT (HSCT). Two-hundred and forty patients were included. Sixty-six had received BU, while 174 had received TBI. Median follow-up from HSCT was 10.1 years. Multivariate analyses were performed to assess the occurrence of late effects according to treatment. QoL was assessed in 130 adults using SF-36 questionnaires. Patients developed fewer late complications after BU (2.35 vs 3.01, P ¼ 0.03) while the risk to present with at least one complication was equivalent in both groups (87.9% after BU and 93.1% after TBI, P ¼ 0.66). Detailed multivariate analyses revealed a lower risk of height growth failure (OR ¼ 0.2), cataract (OR ¼ 0.1) and iron overload (OR ¼ 0.2) after BU, and an increased risk of overweight (OR ¼ 3.9) and alopecia (OR ¼ 11.2). SF-36 mental and physical composite scores were similar in both treatment groups and proved significantly lower than French norms. Late effects induced by BU might differ from those experienced after TBI. Although less frequent, they are still of considerable importance and may affect patients' QoL.

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Section: Health Status After Hct F Bernard Et Alsupporting

confidence: 72%

Section: Introductionmentioning

confidence: 99%

Health status of childhood leukemia survivors who received hematopoietic cell transplantation after BU or TBI: an LEA study

Bernard

Auquier

Herrmann

et al. 2014

Bone Marrow Transplant

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“…This is consistent with the reports of most studies in the field, which describe peripheral hypothyroidism as the most common thyroid disorder following treatment in leukemia survivors. 5,25,29,35 However, Sanders et al reported 74 cases of central hypothyroidism among 791 patients, 17 which may be due to a high number of single doses of total body irradiation. It was previously shown that single-dose total body irradiation is associated with increased toxicity and notably greater thyroid toxicity.…”

Section: Discussionmentioning

confidence: 99%

Late thyroid complications in survivors of childhood acute leukemia. An L.E.A. study

Oudin¹,

Auquier²,

Bertrand³

et al. 2016

Haematologica

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T hyroid complications are known side effects of irradiation. However, the risk of such complications in childhood acute leukemia survivors who received either central nervous system irradiation or hematopoietic stem cell transplantation is less described. We prospectively evaluated the incidence and risk factors for thyroid dysfunction and tumors in survivors of childhood acute myeloid or lymphoid leukemia. A total of 588 patients were evaluated for thyroid function, and 502 individuals were assessed for thyroid tumors (median follow-up duration: 12.6 and 12.5 years, respectively). The cumulative incidence of hypothyroidism was 17.3% (95% CI: 14.1-21.1) and 24.6% (95% CI: 20.4-29.6) at 10 and 20 years from leukemia diagnosis, respectively. Patients who received total body irradiation (with or without prior central nervous system irradiation) were at higher risk of hypothyroidism (adjusted HR: 2.87; P=0.04 and 2.79, P=0.01, respectively) as compared with transplanted patients who never received any irradiation. Patients transplanted without total body irradiation who received central nervous system irradiation were also at higher risk (adjusted HR: 3.39; P=0.02). Patients irradiated or transplanted at older than 10 years of age had a lower risk (adjusted HR: 0.61; P=0.02). Thyroid malignancy was found in 26 patients (5.2%). Among them, two patients had never received any type of irradiation: alkylating agents could also promote thyroid cancer. The cumulative incidence of thyroid malignancy was 9.6% (95% CI: 6.0-15.0) at 20 years. Women were at higher risk than men (adjusted HR: 4.74; P=0.002). In conclusion, thyroid complications are frequent among patients who undergo transplantation after total body irradiation and those who received prior central nervous system irradiation. Close monitoring is thus warranted for these patients.

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“…It has been increasingly used in conditioning protocols as an alternative to TBI, which is often poorly tolerated in children. [3][4][5] Busulfan has a narrow therapeutic index: low drug exposure is associated with increased risk of rejection and disease relapse in transplant recipients, 6,7 whereas high drug exposure is associated with toxicity (that is, veno-occlusive disease (VOD)), fatalities and overall poor outcome. [8][9][10] The exposure to busulfan is measured by the area under the concentration-time curve (AUC).…”

Section: Introductionmentioning

confidence: 99%

Should busulfan therapeutic range be narrowed in pediatrics? Experience from a large cohort of hematopoietic stem cell transplant children

Philippe

Goutelle

Guitton

et al. 2015

Bone Marrow Transplant

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Busulfan, the corner stone of hematopoietic stem cell transplantation regimens, has a narrow therapeutic window. Therapeutic drug monitoring (TDM)-guided dosing to reach the conventional area under the concentration-time curve (AUC) target range of 900-1500 μmol min/L is associated with better outcomes. We report our experience with busulfan TDM in a large cohort of children. The aims were to investigate the relevance of using a more restricted therapeutic range and investigate the association between busulfan therapeutic range and clinical outcome. This study includes 138 children receiving 16 doses of intravenous busulfan, with the first dose assigned based on weight and doses adjusted to a local AUC target range of 980-1250 μmol min/L. Busulfan TDM combined with model-based dose adjustment was associated with an increased probability of AUC target attainment, for both target range: 90.8% versus 74.8% for the conventional target range and 66.2% versus 43.9% for the local target range (P o 0.001). The median follow-up was 56.2 months. Event-free survival was 88.5%, overall survival was 91.5% and venoocclusive disease occurred in 18.3% of patients. No difference was observed for clinical outcomes depending on the selected target range. Pharmacokinetic monitoring and individualization of busulfan dosage regimen are useful in improving target attainment, but using a restricted target range has no impact on clinical outcomes.

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Endocrinological late complications after hematopoietic SCT in children

Cited by 82 publications

References 31 publications

Health status of childhood leukemia survivors who received hematopoietic cell transplantation after BU or TBI: an LEA study

Health status of childhood leukemia survivors who received hematopoietic cell transplantation after BU or TBI: an LEA study

Late thyroid complications in survivors of childhood acute leukemia. An L.E.A. study

Should busulfan therapeutic range be narrowed in pediatrics? Experience from a large cohort of hematopoietic stem cell transplant children

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