Haematopoietic stem cell transplantation trends in children over the last three decades: a survey by the paediatric diseases working party of the European Group for Blood and Marrow Transplantation

Miano, Maurizio; Labopin, Myriam; Hartmann, Olivier; Angelucci, Emanuele; Cornish, Jacqueline; Gluckman, Éliane; Locatelli, Franco; Fischer, Alain; Egeler, R. Maarten; Or, Reuven; Peters, Christina; Ortega, Juan José López; Veys, Paul; Bordigoni, Pierre; Iori, Anna Paola; Niethammer, D.; Rocha, Vanderson; Dini, Giorgio

doi:10.1038/sj.bmt.1705550

Cited by 106 publications

(103 citation statements)

References 35 publications

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“…1 The cumulative burden of treatment-related morbidity after childhood HSCT is considerable [2][3][4][5] and seems to increase over time. 6 Conditioning with TBI is a risk factor for late effects when compared with chemotherapy-based conditioning.…”

Section: Introductionmentioning

confidence: 99%

Adverse health events and late mortality after pediatric allogeneic hematopoietic SCT—two decades of longitudinal follow-up

Wilhelmsson

Vatanen

Borgström

et al. 2015

Bone Marrow Transplant

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Treatment-related late toxicities after pediatric allogeneic hematopoietic SCT (allo-HSCT) are increasingly important as long-term survival has become an expected outcome for many transplanted children and adolescents. In a retrospective cohort study, we assessed long-term health outcomes in 204 allo-HSCT survivors transplanted in childhood or adolescence (o 20 years) between 1978 through 2000 after a median follow-up time of 12 (range 4-28) years. Data on conditioning regimen, adverse health events (AE) and growth and hormonal substitutions (hormone replacement therapies (HRTs)) were obtained from medical records. AEs were graded retrospectively according to Common Terminology Criteria for Adverse Events v3.0. Late deaths (⩾48 months after allo-HSCT) were evaluated separately. Multivariate analysis demonstrated that chronic GVHD (P o0.000) and longer follow-up time (P o0.05) correlated with AEs, whereas CY-based conditioning was inversely correlated (P o 0.002). TBI and longer follow-up duration predicted more severe AEs (P o 0.001 and P o 0.001, respectively). HRTs were more frequent after TBI. Diabetes type II, dyslipidemia and hypertension were detected in 9, 7 and 7% of the survivors, respectively. Late deaths (n = 22) were most frequently due to pulmonary failure (n = 7), followed by secondary malignancy (n = 5). The occurrence of AEs after pediatric allo-HSCT is high and likely to increase during extended follow-up, particularly in patients who have received TBI.

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Section: Introductionmentioning

confidence: 99%

Adverse health events and late mortality after pediatric allogeneic hematopoietic SCT—two decades of longitudinal follow-up

Wilhelmsson

Vatanen

Borgström

et al. 2015

Bone Marrow Transplant

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“…The yearly minimum of 10 allogeneic transplants must be respected. 2 Keeping in mind the main goals of our EBMT Paediatric Working Party, we decided to organize the first European School of Haematology (ESH)-EBMT Training Course on Blood and Marrow Transplantation in children to promote and facilitate access to cutting edge knowledge in blood and marrow transplantation at the European level. For this purpose, the leading European and international experts operating in the field of HSCT in children gathered in Genova at the Badia Benedettina complex, a magnificent training facility that comprises a former convent, the historic Villa Quartara and a large park sloping down towards the sea.…”

Section: Introductionmentioning

confidence: 99%

The EBMT Paediatric Diseases Working Party and the first ESH–EBMT training course on blood and marrow transplantation in children

Dini

2008

Bone Marrow Transplant

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“…19,20 Therefore, it is a major goal of our trial to apply a well-standardized and risk-adapted GvHD prophylaxis and therapy. In MSD, GvHD prophylaxis consists of CsA; in MD, additional short MTX and ATG-F are administered.…”

Section: Gvhd Prophylaxis and Therapymentioning

confidence: 99%

Allogeneic hematopoietic SCT in children with ALL: current concepts of ongoing prospective SCT trials

Schrauder

Stackelberg

Schrappe

et al. 2008

Bone Marrow Transplant

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The definition of indications for allogeneic SCT in children with high-risk (HR) ALL in the first remission or after the first or subsequent relapse depends on biological features, response to treatment and survival after chemotherapy alone. As the results of frontline and relapse protocols are improving over time, there is a strong need for prospective SCT trials, ensuring a well-standardized procedure regarding all relevant components that are potentially responsible for heterogeneity in post-SCT outcome. Therefore, in 2003, the ALL-BFM and the ALL-REZ BFM Study Group initiated a prospective, international, multicenter trial (ALL- SCT-BFM 2003). This trial will now be extended to a larger consortium, trial ALL-SCT-BFM-international (ALL-SCT-BFMi). Strict rules define HLA-typing, donor selection, conditioning regimen, GvHD prophylaxis and therapy as well as standards of supportive care to reduce treatmentrelated mortality and establish an early GVL effect. Moreover, comprehensive and closely reviewed documentation and serious adverse event reporting shall ensure high study quality. Case-by-case discussions of any fatal or critical course during annual meetings will improve the culture of failure management and lead to modifications of guidelines of supportive care. Finally, the results of these prospective trials will determine the current potential of the different SCT procedures in HR or relapsed childhood ALL.

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Haematopoietic stem cell transplantation trends in children over the last three decades: a survey by the paediatric diseases working party of the European Group for Blood and Marrow Transplantation

Cited by 106 publications

References 35 publications

Adverse health events and late mortality after pediatric allogeneic hematopoietic SCT—two decades of longitudinal follow-up

Adverse health events and late mortality after pediatric allogeneic hematopoietic SCT—two decades of longitudinal follow-up

The EBMT Paediatric Diseases Working Party and the first ESH–EBMT training course on blood and marrow transplantation in children

Allogeneic hematopoietic SCT in children with ALL: current concepts of ongoing prospective SCT trials

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