Articles

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, 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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Leukaemic stem cells in AML: where are we now? An update on recent findings and detection

BJH - volume 11, issue 6, october 2020

B. Depreter PhD, PharmD, B. De Moerloose MD, PhD, J. Philippé MD, PhD, T. Lammens PhD

SUMMARY

Ample evidence was provided these past decades that leukaemic stem cells (LSC) play a role in the outcome of adult and paediatric acute myeloid leukaemia (AML) patients. Although it is generally accepted that the CD34+/ CD38- compartment is most LSC-enriched, novel data have emerged illustrating a distinct biology between CD34+ and CD34- AML. In this review, we discuss the main LSC phenotypes in CD34+ and CD34- AML , as they are of utmost importance for the development of broadly applicable LSC-targeted strategies. The leukaemia-initiating capacity of these cells upon xenografting is still considered to be the gold standard for LSC detection. However, more feasible techniques have been researched to allow the implementation of LSC measurements into clinical practice. Here, we summarise the current state-of-the-art methodologies using flow cytometry and molecular detection, and emphasise their relevance in terms of prognosis and targeted drug therapy.

(BELG J HEMATOL 2020;11(6):246-52)

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P.14.2 KIT D816 mutation in myeloid neoplasms: look out for systemic mastocytosis associated hematologic neoplasms (SM-AHN)

BJH - volume 11, issue Abstract Book BHS, february 2020

E. Linskens , S. Van Landeghem , K. Vandepoele PhD, dr. K. Maes , I. Moors MD, J. Van Dorpe , C. Bonroy , K. Devreese , J. Philippé MD, PhD, B. Denys MD

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O.4 An in-depth investigation of the causes of treatment failure in AML

BJH - volume 11, issue Abstract Book BHS, february 2020

S. Bonte , S. Van Gassen , A. Couckuyt , V. Janda , I. Moors MD, dr. A. Delie MD, S. Kennes MD, J. Philippé MD, PhD, Y. Saeys , T. Kerre MD, PhD

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PP01 Exploring the long non-coding RNA landscape in Juvenile Myelomonocytic Leukemia

BJH - 2018, issue Abstract Book BHS, february 2018

M. Hofmans MD, T. Lammens PhD, S. Bresolin , H. Cavé , C. Flotho , H. Hasle , H. Helsmoortel PhD, M. Van den Heuvel-Eibrink , C. Niemeyer , J. Stary , N. Van Roy PhD, P. Van Vlierberghe PhD, J. Philippé MD, PhD, B. De Moerloose MD, PhD

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PP21 The light chain IgLV3-21 defines a new poor prognostic subgroup in Chronic Lymphocytic Leukemia: results from a multicenter study

BJH - 2018, issue Abstract Book BHS, february 2018

B. Stamatopoulos , T. Smith , E. Crompot , K. Pieters , R. Clifford , M. Mraz , P. Robbe , A. Burns , A. Timbs , D. Bruce , P. Hillmen , N. Meuleman MD, PhD, P. Mineur MD, R. Firescu , M. Maerevoet MD, V. De Wilde MD, PhD, A. Efira MD, J. Philippé MD, PhD, B. Verhasselt MD, PhD, F. Offner MD, PhD, A. Heger , D. Sims , H. Dreau , A. Schuh

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P09 A case of chronic eosinophilic leukemia with secondary transformation to acute myeloid leukemia

BJH - 2018, issue Abstract Book BHS, february 2018

M. Hofmans MD, dr. A. Delie MD, K. Vandepoele PhD, N. Van Roy PhD, J. Van der Meulen , J. Philippé MD, PhD, I. Moors MD

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