Articles

New and emergent treatment options in sickle cell disease

BJH - volume 12, issue 7, november 2021

V. Labarque MD, PhD

SUMMARY

Sickle cell disease (SCD) is one of the most frequently inherited diseases but it no longer only affects children. More and more patients survive well into adulthood. They experience repeated acute complications and inevitably develop chronic organ damage. For years, hydroxyurea and chronic transfusions were the only disease-modifying options in the treatment of SCD patients. Thanks to a better understanding of the pathophysiology, new components have been and are now being tested. Three of these are already used in clinical practice, namely L-glutamine, crizanlizumab and voxelotor. On the other hand, progress has also been made in the field of haematopoietic stem cell transplantation, through the introduction of alternative donors as well as the use of less toxic conditioning regimens. Finally, hopeful results are being achieved in the first studies of gene therapy in patients with SCD but it has yet to be proven that genetically manipulated stem cells maintain the long-term repopulation potential.

(BELG J HEMATOL 2021;12(7):290–5)

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Sickle cell disease for the adult hematologist

BJH - 2021, issue SPECIAL, january 2021

V. Labarque MD, PhD

In recent years we have witnessed a growing interest in the treatment of patients with sickle cell disease (SCD). Advances in general medical care, early diagnosis and comprehensive treatment have led to substantial improvements in the life expectancy of individuals with SCD in high-income countries. During her lecture at the 2021 annual meeting of the BHS, Prof. Veerle Labarque gave an overview on the current treatment landscape for adult SCD patients.

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Retrospective analysis of the incidence and characteristics of paediatric myelodysplastic syndrome and juvenile myelomonocytic leukaemia in Belgium

BJH - volume 11, issue 6, october 2020

L. De Smaele , M. Hofmans MD, PhD, T. Lammens PhD, A. Van Damme MD, PhD, J. van der Werff ten Bosch MD, PhD, A. Ferster MD, PhD, J. Verlooy MD, C. Chantrain , J. Philippé MD, PhD, N. Van Roy PhD, P. De Paepe MD, PhD, V. Labarque MD, PhD, B. De Moerloose MD, PhD

SUMMARY

Childhood myelodysplastic syndrome (MDS) and juvenile myelomonocytic leukaemia (JMML) are very rare clonal stem cell disorders of early childhood. Paediatric MDS can be further subdivided in refractory cytopenia of childhood (RCC) and high grade MDS, in case of excess blasts. Given their rarity, little is known about the epidemiology of these diseases in Belgium. The aim of this study is to investigate the incidence, characteristics, treatment and prognosis of paediatric MDS and JMML in Belgium. Prospectively collected data of 56 Belgian patients with MDS and JMML were enrolled in the study, of which 41 (73%) with MDS, eleven with JMML (20%) and four (7%) with Noonan syndrome associated myeloproliferative disorder. The incidence rates of MDS and JMML in Belgium were 1.5 and 0.4 per million children per year respectively, with a median age of diagnosis of 9.3 years for RCC, 9.5 years for high grade MDS and 2.6 years for JMML. Monosomy 7 was the most common cytogenetic abnormality and could particularly be found in high grade MDS (33%) and JMML (45%). RCC treatment consisted of immunosuppressive therapy (IST) and haematopoietic stem cell transplantation (HSCT), but in high grade MDS and JMML only HSCT was a valid treatment option. Overall survival was significantly lower in high grade MDS (45.0%) compared to JMML (79.5%) and RCC (80.6%) (log-rank p-value = 0.038), whereas event-free survival (EFS) was comparably low in high grade MDS and JMML (46.7% and 58.4% respectively) due to a high cumulative incidence of relapse (CIR) of 33% and 29.9%, respectively. Outcome was best for RCC patients with highest EFS (76.3%; 57.1% if IST failure was considered as event) and lowest CIR (9.3%). This study highlights that paediatric MDS and JMML are very rare disorders with associated morbidity and mortality, especially in high grade MDS and JMML. Considering the high relapse risk in high grade MDS and JMML, new therapeutic options are required.

(BELG J HEMATOL 2020;11(6):233-9)

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Highlights in red blood cell disorders

BJH - volume 11, issue 1, february 2020

V. Labarque MD, PhD

Summary

In the field of red blood cell disorders, ASH 2019 mainly focused on sickle cell disease (SCD). Current disease-modifying therapeutic options for SCD consist of hydroxyurea and chronic transfusions but none of them can fully prevent complications and both have their drawbacks. A haematopoietic stem cell transplantation is the only curative option to date. Yet, frequently a compatible donor is missing. ASH 2019 featured several abstracts on optimising disease-modifying therapies as well as curative options, such as gene therapy. A selection of abstracts is discussed below.

(BELG J HEMATOL 2020;11(1):21–6)

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BHS clinical guidelines on the management of acute complications in sickle cell disease

BJH - volume 10, issue 4, june 2019

T. van Genechten MD, A. Vanderfaeillie MD, M.A. Azerad MD, D. Kieffer PhD, PharmD, V. Labarque MD, PhD, B. Gulbis MD, PhD, A. Ferster MD, PhD, B. De Wilde MD, PhD

With the increasing prevalence of sickle cell disease patients in Western countries, it is of importance to improve awareness among medical doctors of its complications. To reduce long-term morbidity and mortality, the prompt recognition and treatment of acute complications is important. The existing clinical guideline ‘Follow-up and treatment of patients with sickle cell disease hospitalised for Vaso Occlusive Crisis or infection’, published in 2012 by the Belgian Haematological Society, was revisited to better suit the practical needs of first-line practitioners.

(BELG J HEMATOL 2019;10(4):165–8)

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O09 VISUALIZING DENSE GRANULE FORMATION IN MEGAKARYOCYTES USING ADVANCED MICROSCOPY AND ITS APPLICATION FOR HERMANSKY-PUDLAK SYNDROME

BJH - 2019, issue ?, february 2019

J. Heremans , C. Thys , K. Cludts , V. Labarque MD, PhD, C. Van Geet MD, PhD, K. Freson PhD

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Retrospective study of infant leukaemia in the University Hospitals of Leuven and Ghent

BJH - volume 9, issue 2, march 2018

B. De Moerloose MD, PhD, E. Nauwynck MD, K. Arts MD, L. Willems MD, PhD, V. Labarque MD, PhD, T. Lammens PhD, A. Uyttebroeck MD, PhD

SUMMARY

Infant leukaemia is a rare disease but the 3rd most frequent malignancy in this age group. Both acute lymphoblastic leukaemia and acute myeloid leukaemia in the first year of life have particular clinical and biological characteristics such as B-cell phenotype with co-expression of myeloid markers in acute lymphoblastic leukaemia, FAB M5 or M7 in acute myeloid leukaemia, the presence of extramedullary symptoms and a high frequency of KMT2A rearrangements. Survival rates for infant acute leukaemia are worse than for older children. In this study, the characteristics and outcome of 50 infants with acute lymphoblastic leukaemia and acute myeloid leukaemia treated at the University Hospitals of Ghent and Leuven between 1989 and 2015 were studied and correlated with literature data. With event-free survival and overall survival rates of 44% and 52% for the entire cohort, the outcome of these patients was comparable to those in published clinical trials. In general, the event-free survival and overall survival was superior in acute myeloid leukaemia compared to acute lymphoblastic leukaemia infants and not influenced by age (< or ≥6 months), white blood cell count at diagnosis or presence of a KMT2A rearrangement. For future trials in infant leukaemia, the high number of early deaths, toxic deaths and relapses remain the most challenging problems.

(BELG J HEMATOL 2018;9(2):57–63.)

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