Articles

Iron overload in haematopoietic stem cell transplantation children and relation to organ damage and survival

BJH - volume 9, issue 5, september 2018

V. Liberton MD, R. Colman , G. Laureys MD, PhD, V. Bordon MD, PhD, C. Dhooge MD, PhD

SUMMARY

Adult patients with high serum ferritin have an increased risk of organ toxicity and iron chelation before stem cell transplant might be an option. We report our experience in 58 paediatric patients (excluding patients with haemoglobinopathy and hyper-transfused patients) undergoing allogeneic stem cell transplant between 2007 and 2012. Serum ferritin pre-transplant was highly variable (mean: 932 µg/L) and related to a number of PRC transfusions. Eighteen of 58 patients had ferritin level >1000 µg/L before transplant. Eight patients suffered from transplant-related mortality. We found no correlation between transplant-related mortality and pre-transplant serum ferritin (p=0.67). Seven patients developed veno-occlusive disease, reversible in all cases. We did not find a correlation between serum ferritin and veno-occlusive disease, graft-versus-host disease or relapse. The evolution of ferritin post-transplant shows a spontaneous lowering of ferritin in the first two years after haematopoietic stem cell transplantation to normal range. An association between serum ferritin and elevated AST/ALT at 12 and 24 months was noted and follow-up concerning possible liver damage in patients with a persistently high serum ferritin is recommended. This study concludes that high serum ferritin has no influence on transplant-related mortality in children, chronically poly-transfused patients excluded. Although chelation is already used in paediatric HSCT, there is insufficient, current evidence to do so.

(BELG J HEMATOL 2018;9(5):182–7)

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PP3.4 Incidence of Cryptosporidium after autologous and allogeneic hematopoietic stem cell transplantation

BJH - volume 7, issue Abstract Book BHS, january 2016

V. Bordon MD, PhD, C. Dhooge MD, PhD, E. Claerebout , S. Casaert , G. Laureys MD, PhD

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Fatal Myceliophthora thermophila infection in a boy with acute myeloid leukaemia and cord blood stem cell transplantation

BJH - volume 5, issue 2, june 2014

A. Piette MD, B. De Moerloose MD, PhD, P. Schelstraete MD, PhD, V. Bordon MD, PhD, T. De Baere PhD, G. Claeys MD, PhD

Introduction

We report a Myceliophthora thermophila infection in a boy who underwent haematopoietic (cord blood) stem cell transplantation as treatment for relapsed acute myeloid leukaemia. Despite neutrophil engraftment and maximal supportive treatment, the child died of multi-organ failure. Infections with environmental moulds are rare, but potentially disastrous in immunocompromised patients. Rapid diagnosis and early treatment is of upmost importance.

(BELG J HEMATOL 2014;5(2):60–3)

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P.71 Swachman-Diamond Syndrome: Frequent misdiagnosis as Jeune Syndrome and other peculiarities

BJH - 2013, issue BHS Abstractbook, january 2013

I. Meyts , H. Schaballie , F. Haerynck MD, PhD, L. Sevenants , C. Vermylen , V. Bordon MD, PhD, X. Bossuyt , A. Corveleyn , A. Uyttebroeck MD, PhD, M. Renard

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P.72 Successful Autologous Stem Cell Transplantation in a Girl with Neuropsychiatric Systemic Lupus Erythematosus

BJH - 2013, issue BHS Abstractbook, january 2013

V. Bordon MD, PhD, P. Verloo , S. Verbeek , C. Dhooge MD, PhD

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Less veno-occlusive disease after intravenous versus oral busulfan for autologous haematopoietic stem cell transplantation: the Belgian paediatric experience

BJH - volume 3, issue 2, june 2012

S. Huybrechts MD, Y. Beguin MD, PhD, V. Bordon MD, PhD, MF. Dresse , S. Dupont MD, A. Ferster MD, PhD, G. Laureys MD, PhD, I. Meyts , M. Renard , C. Vermylen

Summary

Busulfan is commonly used in preparative conditioning regimens prior to haematopoietic stem cell transplantation in children and young adults for malignant and non-malignant disorders. For many years busulfan was only available in oral form, resulting in large inter- and intra-patients variability in plasma exposure, associated with higher graft failure rate as well as higher toxicity such as veno-occlusive disease. With the development of an intravenous formulation of busulfan, a more accurate control of both the inter- and intra-patient variability has been provided. The goal of this study was to evaluate the use and efficacy of intravenous busulfan in comparison with the oral formulation in children undergoing an autologous transplantation after conditioning with busulfan. Despite the small number of patients, this study confirmed the apparent benefit of intravenous busulfan in children undergoing an autologous HSCT. The use of a five-level dose schedule defined by body weight resulted in an efficient engrafitment with marked reduction in the incidence of veno-occlusive disease compared with oral busulfan. In terms of disease-free outcome, survival and event-free survival, similar results have been obtained in both groups. The choice of this formulation of busulfan should therefore be considered.

(BELG J HEMATOL 2012;3:34–40)

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