mpn-mds eu focus 2021

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MPN-MDS EU Focus 2021 Online International Knowledge & Clinical Practice in 2021

Online Virtual Meeting16-18 of September 2021

ABSTRACTS

MPN-MDS EU Focus 2021 Online International Knowledge & Clinical Practice in 2021

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Internation scientific online virtual meeting 2021

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2 MPN-MDS EU Focus Virtual Meeting 2021

Abstract Book

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Dear Colleagues and Friends,

On behalf of the co-chairs, Prof. Dr. Srdan Verstovsek and Prof. Dr. Alessandro M. Vannucchi, MD Education are proud to announce the 2021 MPN-MDS EU Focus Meeting will be held virtually on 16, 17, and 18 of September.

Featuring 25+ MPN and MDS clinical experts from across Europe and the United States, the theme of the meeting, International Knowledge & Clinical Practice in 2021, reflects the collaboration to advance the research and treatment of patients with MPN & MDS globally.

The 3-day meeting will include presentations, round-table discussions, interactive Q&A with the presenters and poster sessions, all held virtually. Register below for a unique opportunity to garner knowledge, skills, and experience from leading clinicians to assess best practices and state-of-the-art treatments in MDS and MPN for your patients.

Learning Objectives

Upon completion of this event, participants should be better able to:

Recognise the clinical and molecular features of patients suffering with MPN and MDS

Identify the latest available treatment options and of those still in development, or pending approval, to improve patient outcomes.

Understand the rationale behind the use of MPN and MDS drugs

Identify the standard/combination therapy and novel drugs for managing MPN and MDS patients

Prof. Dr. Srdan Verstovsek

Dr. Srdan Verstovsek is a Medical Oncologist and Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, Houston, Texas. USA With research focused on understanding the biology of and developing new therapies for myeloproliferative neoplasms (MPNs) Member as a physician-scientist to The American Society for Clinical Investigation.

Prof. Dr. Alessandro M. Vannucchi

Dr. Alessandro Vannucchi is a Professor of Hematology, a head of Center of Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi, University of Florence, Italy. Director of the Denothe Excellence Center, University of Florence. Chair of the MPN board of GIMEMA, and Principal Investigator of MYNERVA research group, Italy.

Foreword



SUPPORTER ACKNOWLEDGEMENT

This event is supported by an unrestricted educational grant from the following supporters.

Exhibition opens September 16th 2021 - Come and visit our exhibitors at their virtual booth here. You can learn more about the science around current treatment as well as those in the pipeline.

Poster Hall opens September 16th 2021 - Access the Poster Hall here. View and download posters of interest, as well as finding out more about the authors.

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Simple Guideline to Systemic Mastocytosis in 2021 Prof Andreas Reiter University Hospital Mannheim, Germany

Myeloid/Lymphoid Neoplasms with EosinophiliaProf Grzegorz HelbigSilesian Medical University, Katowice, Poland

Reapraisal of Anagrelide in Essential ThrombocythaemiaProf Mary McMullinQueen’s University Belfast, Northern Ireland

Ropeginterferon: for High and Low Risk PV Patients? Prof Heinz GisslingerThe Medical University of Vienna, Austria

This symposium is not part of the CME educational programme

and has been supported by Celgene, A BMS company. Lunch Symposium - Join us at 13:00 CEST Treatment Options for Ruxo Refractory/Resistant Patients Prof Alessandro Vannucchi; Dr Francesca Palandri; Prof Srdan Verstovsek; Prof Martin Grieshammer

Choosing “The Best” Prognostic Score for MF Dr Lucia MasarovaMD Anderson Cancer Center, Houston, Texas, USA

Progress in the Management of Cytopenia in MF Prof Claire HarrisonGuys and St Thomas’ NHS Trust , London, United Kingdom

Accelerated Phase MPN: Management Challenge Prof Srdan VerstovsekMD Anderson Cancer Center, Houston, Texas, USA

How to Get the Most Out of JAK Inhibitors for MFDr Francesca PalandriUniversity of Bologna, Italy

Click for easy navigation to speaker abstracts

Agenda MPN

Thursday, September 16




Beyond JAK Inhibitors for MF Prof Alessandro VannucchiUniversity of Florence, Italy

Recent Developments in SCT for MFProf Juan Carlos Hernández-BoludaUniversity of Valencia, Spain

MPN and MDS/MPN Unclassifiable Diagnostic Criteria and Clinical FeaturesProf Umberto GianelliUniversita degli Studi Di Milano, Italy

Primer on Atypical CML Associate Prof Guillermo Montalban BravoMD Anderson Cancer Center, Houston, Texas, USA

CMML: Dysplastic vs Poliferative Subtype, Does it Matter?Prof Raphael ItzyksonUniversity Paris Diderot, France

The Spectrum of Clonal Hematopoiesis: from CHIP to MDS Associate Prof Matteo Della PortaUniversity of Pavia, Italy

Diagnostic Challenge: MDS with Fibrosis Prof Hans Michael KvasnickaUniversity of Witten/Herdecke, Germany

Challenges in Risk Stratification of MDSProf Uwe PlatzbeckerUniversity Hospital Leipzig, Germany

Update on Lenalidomide for MDSProf Rajko KušecUniversity of Zagreb, Croatia

Progress in the Management of Cytopenia in MDSProf Jaroslav ČermákInstitute of Hematology and Blood Transfusion, Czech Republic

Agenda MDS

Friday, September 17




Recent Developments in SCT for MDS Prof Uday PopatMD Anderson Cancer Center, Houston, Texas, USA

Novel Therapies for MDSDr Magnus TobiassonThe Karolinska University Hospital, Stockholm, Sweden

Managing Unfit Patients with AML Dr Tapan KadiaMD Anderson Cancer Center, Houston, Texas, USA

How I Treat FLT3-Mutated AMLDr Musa YilmazMD Anderson Cancer Center, Houston, Texas, United States

Venetoclax for AML Prof Charles CraddockUniversity of Birmingham, UK

New Strategies for Treating Relapsed AMLProf Farhad Ravandi-KashaniMD Anderson Cancer Center, Houston, Texas, USA

The Role of MRD Monitoring in AML Prof Jacqueline CloosAmsterdam University Medial Center, Netherlands

Agenda MDS

Friday, September 17

Agenda AML

Saturday, September 18


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Prof Andreas ReiterUniversity Hospital Mannheim, Germany Dr. Andreas Reiter is an Associate Professor and a Consultant of Hematology and Oncology at the University Medical Centre Mannheim in Germany. Dr. Reiter’s main research focus lies in the pathogenesis and treatment of chronic myeloid neoplasms. He has been involved in numerous clinical trials, most recently particularly focusing on diagnosis, prognosis and treatment of myelofibrosis, eosinophilia-associated MPN and systemic mastocytosis with tyrosine kinase inhibitors. Dr. Reiter has established a German registry on disorders of eosinophils and mast cells.

Simple Guideline to Systemic Mastocytosis in 2021Systemic mastocytosis (SM) is a rare myeloid neoplasm characterized by multifocal infiltration and accumulation of various tissues by neoplastic mast cells (MC). Advanced SM (AdvSM) comprises SM with an associated hematologic neoplasm (SM-AHN), aggressive SM (ASM), and MC leukemia (MCL), the median survival is 3-4 years. A common feature of AdvSM is the heterogenous multilineage involvement of the KIT D816V mutation in both MC (>90%) and diverse non-MC (>70-80%) lineages, morphologically diagnosed as chronic myelomonocytic leukemia, chronic eosinophilic leukemia, acute myeloid leukemia and others. Similar to other myeloid neoplasms, prognostic scores include age, cytopenias and additional somatic mutations, e.g. in SRSF2, ASXL1 or RUNX1. For many years, off-label treatment with the purine analogue cladribine has been the most widely used first-line option for patients with AdvSM. In 2016/2017, the multikinase-inhibitor midostaurin received approval for front-line treatment by FDA/EMA. Avapritinib, a highly selective and potent inhibitor of KIT D816V is currently being investigated in a phase I (EXPLORER) and phase II (PATHFINDER) trial. Independent of subtype and prior treatment, preliminary reports revealed high overall response rates with profound reductions in mast cell burden and KIT D816V variant allele fraction (VAF) and improvements in patient symptoms.

MPN abstract


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Prof Grzegorz HelbigSilesian Medical University, Katowice, Poland Grzegorz Helbig, M.D., Ph.D., is Professor of Medicine in the Department of Hematology and Bone Marrow Transplantation of Silesian Medical University in Katowice, Poland. He received his medical degree in Silesian Medical University. He received his Ph.D. on connective tissue metabolism in patients undergoing allogeneic stem cell transplantation. Since 2014 he is a professor of medicine. In 2017 he took his current position as Chief Department in the biggest transplant center in Poland. Moreover, he works as vice-dean in Faculty of Medcine in Katowice. Prof. Helbig’s research interests are in eosinophilia-associated disorders, myeloproliferative neoplasms and post- transplant complications. He is an author of >150 full-text manuscripts in peer-reviewed journals, he serves as reviewer for many international medical journals (e.g. Blood, New England Journal of Medicine, Haematologica). He delivers lectures during domestic and international conferences.

MPN abstract

Myeloid/Lymphoid Neoplasms with Eosinophilia

Myeloid/lymphoid neoplasms with eosinophilia (M/Leo) and tyrosine kinase (TK) fusion genes constitute a separate category within the 2016 WHO classification. All these arecharacterized by blood or tissue eosinophilia and the presence of a unique genetic abnormality. M/Leo may have diverse clinical manifestations with variable response to TKinhibitors (TKI). The PDGFRA-rearranged neoplasms (usually with detectable FIP1L1- PDGFRA) are found to be extremely sensitive to low dose of imatinib (IM at 100 mg daily) with nearly 100% hematological complete response rate. Moreover, >90% of IM treated patients may achieve long-term molecular response. IM discontinuation may result insustained remission in about 50%-60% of patients. An exquisite response to IM (but at 400 mg/day) was also demonstrated for patients with PDGFRB rearrangements, but trials on IM cessation were not attempted. The FGFR1-rearranged neoplasms are associated with an aggressive disease course and allogeneic stem cell transplantation (allo-SCT) is the only potentially curative approach. Recently, pemigatinib was found to be effective in a proportion of FGFR1-rearranged individuals. An aggressive outcome with rapid blast transformation is also characteristic for the JAK2-rearranged neoplasms. These patients should be included into clinical trials or attempted with ruxolitinib or fedratinib as a “bridge” to allo-SCT. A new category of neoplasms with eosinophilia and FLT3 and ABL1 rearrangements has not yet been incorporated into the WHO 2016 classification. The prognosis is poor with a tendency to evolve into resistant acute leukemia. The treatment includes TKI with known activity against FLT3/ABL1 followed by allo-SCT.


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Prof Mary McMullinQueen’s University Belfast, Northern Ireland Professor Mary Frances McMullin graduated from QUB in 1980 and trained in Haematology in N. Ireland and The Royal Postgraduate Medical School, Hammersmith hospital, London. In 1991, she was appointed senior lecturer in haematology in QUB and consultant haematologist. Since 2001 she has delivered a specialised service in the Belfast City Hospital in adult acute leukaemia and myeloproliferative neoplasms. She proceeded to Professor of Haematology in 2006. Her research interests include trials in myeloproliferative neoplasms and acute myeloid leukaemia and investigation of rare congenital erythrocytosis.

MPN abstract

Reapraisal of Anagrelide in Essential Thrombocythaemia

Anagrelide in a synthetic quinazolone derivative which was discovered to reduce platelet aggregation and to decrease megakaryocyte production and thus reduce the platelet count. As such it has utility in the treatment of essential thrombocythaemia in risk groups where cytoreduction is required to attempt to reduce the incidence of thromboembolic events as it is effective in reducing the platelet count. In randomised controlled trials, on occasion, it has been shown to be non-inferior to hydroxyurea in the treatment of this disorder. However, arterial thrombosis, bleeding and progression to myelofibrosis were found to occur more frequently with anagrelide than with hydroyurea. In contrast venous thrombosis was a more frequent occurrence with hydroxyurea use. The side effect profile of anegrelide includes cardiovascular side effects and termination of treatment because of side effects, occurs more frequently with anagrelide than hydroxyurea. Prolonged release formulations of anagrelide are equally affective and require less frequent dosing and therefore may be preferable.

In conclusion, anagrelide is an effective therapy for essential thrombocythaemia in a first or second line setting with an acceptable side effect profile. Choice of agent used depends on patient age and co-morbidities and patient preference.


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Prof Heinz GisslingerThe Medical University of Vienna, Austria Heinz Gisslinger from Medical University of Vienna, is a pioneer in treating MPN patients with Interferon (IFN). He treated the first patient with Interferon 30 years ago. He graduated at the University of Vienna (M.D.), thereatier, fellowships at theinstitute of clinical pathology and at the department of internalmedicine. 1988 awarded degree as specialist for internal medicine and 1996 for hemato-oncology.1993 tenue with the degree of venia docendi, since 1998 Professor of Medicine.

MPN abstract

Ropeginterferon: for High and Low Risk PV Patients?

Polycythemia vera (PV) is associated with a risk for thrombosis, for transformation to myelofibrosis and for transformation to acute leukemia. Risk factors for a worse outcome are older age and an advanced stage of disease represented by a history of thrombosis, an elevated leukocyte count, higher Jak2 allele burden and/or a high-risk gene expression profile. Patients with PV require life-long treatment to prevent thromboembolic events and minimize the risk of progression. Interferon alpha (IFN) ultimately modifies the natural history of PV by selectively targeting the malignant cell clone. The knowledge about the beneficial effect of Interferon alpha in PV stems, besides from numerous phase II trials, from two prospective, randomized trials and retrospective long-term analyses of IFN treatment in PV. In the recently published low-PV trial, designed as a multicenter, randomised phase 2 trial, Ropeginterferon alfa-2b (RopegIFN) was compared to phlebotomy in low-risk PV patients. In the prospective phase III PROUD-PV/CONTINUATION-PV studies, long-term treatment with RopegIFN was compared to the standard cytoreductive therapy hydroxyurea, regarding thromboembolic and other adverse events as well as hematologic and molecular parameters over five years. In the randomized controlled setting, RopegIFN effectively controlled hematocrit and phlebotomy rate and minimized the occurrence of thromboembolic events in patients with earlier staged PV in the cohort with a need for cytoreduction (PROUD-PV/CONTINUATION-PV studies) patients were significantly more likely to be phlebotomy-free in the 4th and 5th year of treatment than patients receiving the control treatment (BAT).Disease progression was rare during long-term IFN treatment in comparison to BAT-therapy, which might reflect a change in the natural history of PV due to deep and durable molecular responses to IFN.


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Dr Lucia MasarovaMD Anderson Cancer Center, Houston, Texas, USA Dr. Lucia Masarova is an Assistant Professor at the Department of Leukemia, the University of Texas MD Anderson Cancer Center in Houston, Texas, USA.

Dr. Masarova’s primary research interest lies in the myeloid neoplasms and particularly, myeloproliferative neoplasms, their pathogenesis, molecular background and therapies. Dr. Masarova has been trained and mentored by Prof. Dr. Srdan Verstovsek at the same institution where she gained broad experience in treating patients with these rare disorders.

She has initiated and has been involved in numerous research projects and clinical trials focused on myeloid neoplasms and authored or co-authored many publications in related topics.

MPN abstract

Choosing “The Best” Prognostic Score for MF

Patients with myelofibrosis have variable life expectancy, ranging from months to decades. Proper disease prognostication is paramount to patients’ outcomes, especially in patients with advanced disease who are potentially eligible for curative stem cell transplantation (SCT). Older, mostly clinical models, such as IPSS and DIPSS/Plus, remain powerful to detect higher risk disease often requiring immediate attention. Molecular features and cytogenetic are important drivers of disease behaviorand are implemented in novel models, such as MIPSS70+ v2.0. Every molecular information, even without the entire analysis required for complete prognostication by novel models, shall be taken into prognostic consideration. Every patient with higher risk disease (using any score), shall beevaluated for stem cell transplantation and individual decisions depend on patient risks and fitness.Repeated assessment of disease using all available prognostic tools allows timely treatment decisions and positively impacts patients’ outcome.


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Prof Claire HarrisonGuys and St Thomas’ NHS Trust , London, United Kingdom Prof. Claire Harrison graduated from Oxford University Medical School and became a consultant at the Guy’s and St Thomas Hospital in 2001, where she is now clinical director of haematology, cellular pathology, and palliative care. The focus of her clinical work is myeloproliferative neoplasms (MPN), for which she has a national and international reputation. She has a strong interest in patient advocacy andfounded the UK MPN groupwww.mpnvoice.org.uk

MPN abstract

Progress in the Management of Cytopenia in MF

Cytopenias are a frequent and often difficult clinical problem in patients with myelofibrosis (MF). Anemia is present at diagnosis in approximately 30% of patients with PMF and eventually develops in the majority. Severe thrombocytopenia (platelets <50 x 109/L) in patients with MF is less common but frequently coincides with anaemia and both are well-recognized poor prognostic factors. Importantly, on-target anemia and thrombocytopenia are frequently observed during therapy with the JAK1/2 inhibitor ruxolitinib, especially during the first 12-24 weeks on therapy. Careful dose adjustment of ruxolitinib without premature discontinuation is critical while managing these cytopenias, as spleen responses to ruxolitinib are dose-dependent and correlate with survival in patients with advanced MF. In this presentation we will examine these factors in more detail and the newer options for management.

http://www.mpnvoice.org.uk


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Prof Srdan VerstovsekMD Anderson Cancer Center, Houston, Texas, USA Prof. Srđan Verstovsek is a medical oncologist and professor in the Department of Leukaemia at The University of Texas MD Anderson Cancer Center, Houston, Texas, USA. He obtained his medical and doctoral degree at the University of Zagreb, Zagreb,Croatia.

Dr. Verstovsek’s clinical and translational research is focused on understanding the biology of and developing new therapies for myeloproliferative neoplasms (MPNs). He is Director of theClinical Research Center for MPNs at MD Anderson. More than 50 related clinical trials and more than 400 peer-reviewedmanuscripts.

MPN abstract

Accelerated Phase MPN: Management Challenge

Myeloproliferative neoplasms (MPN) can transform into MPN in the blast phase (MPN-BP), which typically transitions through the accelerated phase (MPN-AP), defined by ≥10-19% blasts in the peripheral blood/bone marrow. MPN-AP/BP have poor prognosis with no established standard of care. Allogeneic hematopoietic cell transplantation (allo-HCT) is the sole modality conferring long-term survival and is recommended after intensive chemotherapy/other treatments in judiciously chosen patients. Treatment of elderly MPN-AP/BP patients with ruxolitinib combined with hypomethylating agents (decitabine, azacitidine) is a viable option that may provide control of the disease sign and symptoms for 1-1.5 years. Given that IDH1/2 mutations are more common in MPN-AP/BP, such inhibitors have sporadically been used in the clinic: retrospective studies on small cohorts of IDH1/2-mutated patients with MPN-AP/BP who were treated with IDH1/2-inhibitor based combinations showed positive outcomes, including complete hematological/molecular remissions and extended survivals. Furthermore, combination treatment of enasidenib/ruxolitinib is evaluated in clinical trial, in IDH2-mutated patients with MPN in chronic phase (4-9% blasts) and MPN-AP/BP in a phase 2 trial (NCT04281498); this trial is strongly supported by preclinical results showing synergism. Venetoclax in combination with hypomethylating agent may be a viable, short-term option for select patients in MPN-AP/BP that have a donor available and are ready to undergo allo-HCT; otherwise, therapy with this combination leads to increased toxicity/death rate, and has no influence on overall survival.


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Dr Francesca PalandriUniversity of Bologna, Italy

Dr Francesca Palandri is the Head of the Clinical Research Unit of Philadelphia-negative Chronic Myeloproliferative Neoplasms at the Institute of Hematology “L. and A. Seragnoli”, University of Bologna in Italy.

Elected member of the MPN board of GIMEMA, and Principal Investigator of several phase Ib-IV clinical studies on MPNs. Author of more than 150 peer reviewed publications and book chapters on MPNs.

MPN abstract

How to Get the Most Out of JAK Inhibitors for MF

Ruxolitinib is the first JAK1/2 inhibitor approved for treatment of splenomegaly and symptoms related to MF. Results of the COMFORT studies demonstrated that RUX provided rapid reductions in splenomegaly/symptoms, and that response correlates with ruxolitinib dose. Longer follow-up of prospective studies and real-world data showed also that earlier RUX start may achieve better responses and that abrupt discontinuation may result in potentially severe rebound of symptoms. Anemia/thrombocytopenia, infections and non-melanoma skin cancers represent the main expected toxicity. To optimize ruxolitinib therapy it is important to start the drug with no delay, to use the maximum tolerated dose, to monitor the patient for main adverse events and to avoid abrupt drug discontinuation.Fedratinib is a JAK2-selective inhibitor that has been approved for MF treatment based on the results of two prospective clinical trials JAKARTA-2 (phase 2) and JAKARTA (phase 3). In these studies, it demonstrated significant clinical activity both front-line and after ruxolitinib failure. Hematological toxicity and gastrointestinal disturbances represent the main clinical toxicities; also, fedratinib was placed on temporary hold in Nov 2013 due to suspected Wernicke encephalopathy related to thiamine deficit. To optimize fedratinib therapy it is important to make a smooth transition from ruxolitinib, to assess thiamine levels at baseline and periodically, discontinuing the drug if neurological symptoms occur, to proactively manage GI symptoms, and to monitor hematological values. Since a fedratinib discontinuation syndrome can not be excluded, it is recommended to avoid abrupt drug discontinuation.


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Prof Alessandro VannucchiUniversity of Florence, Italy

Prof. Alessandro Vannucchi is aFull Professor of Haematology, a head of Center of Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi, University of Florence, Italy.

Director of the Denothe Excellence Center, University of Florence. Chair of the MPN board of GIMEMA, and Principal Investigator of MYNERVA research group, Italy. More than 400 peer-reviewed publications.

Principal Investigator on several phase Ib-IV clinical studies in the field of myeloproliferativeneoplasms.

MDS abstract

Beyond JAK Inhibitors for MF

The use of ruxolitinibn, and more recently fedratinib, as approved therapeutic agents for myelofibrosis has revolutioned the therapeutic landscape of this myeloproliferative neoplasms, producing valuable improvements in splenomegaly, quality of life, and also resulting in survival advantage. However, about 50% of patients lose response to ruxolitinib after three years, as the result of clonal progression; refractory and relapsing (R/R) patients have short life expectancy, and represent an unmet need. Novel JAKi are also in clinical trials, that may be more suitable for cytopenic patients, compared to ruxolitinib and fedratinib. The discovery of the complexity of molecular landscape of myelofibrosis has brought the attention to several possible targets for therapeutic other than JAKi, and a variety of such novel agents are currently in clinical trials. These include molecules targeting epigenetic regulation, signaling/regulatory pathways, drugs acting on cells/mechanisms of fibrosis, and regulators of apoptosis. Most of these trials include (R/R) patients, are designed as single agents or combination with ruxolitinib, and are actively enrolling worldwide. It can be reasonably expected that results of at least some of these studies will contribute to improve therapeutic options for “difficult “patients with myelofibrosis.


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Prof Juan Carlos Hernández-BoludaUniversity of Bologna, Italy

Dr Juan Carlos Hernández-Boluda was trained in the Hematology Service of the Hospital Clínic of Barcelona. Since 2001, he has been a consultant at the Hematology Department of the Hospital Clínico Universitario of Valencia, working primarily in the fields of hematopoietic stem cell transplantation and the chronic myeloproliferative neoplasms (MPN).

He is an Associate Professor at the Medicine Department of the University of Valencia. Since April 2018, he is Vice-chair of the MPN subcommittee of the Chronic Malignancies Working Party of the EBMT. He is the President of the Grupo Español de Neoplasias Mieloproliferativas Filadelfia Negativas (GEMFIN). As a result of his scientific activity, he has published around 150 papers in peer-review international journals.

MDS abstract

Recent Developments in SCT for MF

Allogeneic hematopoietic cell transplantation (allo-HCT) constitutes the only curative treatment for myelofibrosis (MF), but the advanced age of MF patients and the significant transplant-related mortality have historically limited the application of this procedure to less than 10% of MF patients. In this presentation, I will review the recent advances in the field of allo-HCT in MF. Data from international registries show that the activity of allo-HCT in MF is increasing over time, with older recipients and more frequent use of reduced-intensity conditioning (RIC) and unrelated donors. Despite this trend, a significant proportion of patients decide not to receive this potential life-saving therapy due to concerns about its toxicity. According to a recent study from the EBMT and the Spanish MF Registry addressing the role of allo-HCT in elderly MF patients, males seem to benefit more than females from transplant, mainly due to their worse prognosis with non-transplant approaches. Another EBMT study has shown that a large splenomegaly is associated with increased non-relapse mortality, mainly in RIC transplants. Moreover, splenectomized patients had better survival than those with progressive disease and massive splenomegaly at transplant. Accumulating evidence suggests that the benefit of pre-transplant ruxolitinib may be circumscribed to patients who remain in spleen response at transplantation. Based on retrospective data, a myeloablative conditioning regimen seems the most appropriate option for younger individuals suitable for such an approach due to a trend toward less graft failure and disease relapse. Finally, a second allo-HCT is a potential useful approach for patients failing first allo-HCT for MF.


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Prof Umberto GianelliUniversita degli Studi Di Milano, Italy

Prof. Umberto Gianelli MD, is full professor of Anatomic Pathology at the University of Milan and chair of the Hematopathology section, Division of Pathology of the Fondazione IRCCS Ca’ Granda – Ospedale Maggiore Policlinico di Milano, Italy. His research field spreads from myeloid (particularly MPN and MDS) to lymphoid neoplasms. It is a co-author for the WHO classification in the chapters concerning Essential Thrombocythaemia and Primary Myelofibrosis.

MDS abstract

MPN and DS/MPN Unclassifiable Diagnostic Criteria and Clinical FeaturesMPN-U are cases with clinical, morphological and molecular features of a myeloproliferative neoplasm but failing to meet the diagnostic criteria of a specific entity or presenting with features that overlap between two or more MPNs. Three types of MPN-U are most frequent:

1) Early-stage MPN in which the characteristic features are not yet fully developed;

2) Advanced-stage MPN with myelofibrosis and osteosclerosis that mask the underlying disorder;

3) MPN with coexisting neoplastic or inflammatory disorder that obscure some of the usual diagnostic clinical and/or morphological features.

MPN-U diagnosis requires the exclusion of the diagnostic criteria for the other MPN and of reactive conditions. It can be supported by di identification of driver mutations (JAK2, CALR, or MPL), of other mutations associated with MPN (ASXL1, EZH2, TET2, IDH1, IDH2, SRSF2, SF3B1 and others) or of cytogenetic abnormalities.

Myelodysplastic / Myeloproliferative neoplasm, Unclassifiable (MDS/MPN-U) are neoplasms with mixed myeloproliferative and myelodysplastic features at onset not meeting the WHO criteria for the diagnosis of other MDS/MPN characterized by < 20% of blasts without PDGFRA, PDGRFB or FGFR1 rearrangement and no PCM1-JAK2. According to their molecular profile, they can be classified in five categories (CMML-like, aCML-like, MDS/MPN-RS-T, TP53, “other”) with clinical and prognostic implications.


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Assoc. Prof Guillermo Montalban BravoMD Anderson Cancer Center, Houston, Texas, USA

MDS abstract

Primer on Atypical CML

Abstract coming soon


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Prof Raphael ItzyksonUniversity Paris Diderot, France

Hematology Department, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris, FranceINSERM U944, Saint-Louis Institute for Research Raphael Itzykson is Professor of Hematology at Université de Paris, affiliated to the Hematology and Immunology Department at Hôpital Saint-Louis in Paris, France. He studied biology at École Normale Supérieure in Paris and received his medical training at University Paris Descartes. He completed a PhD thesis under the direction of Pr. Eric Solary in Gustave Roussy Institute in Paris.His research focuses on the treatment of CMML and on the use of precision medicine tools in AML. Pr Itzykson has published more than a hundred articles in peer-reviewed journals such as theJournal of Clinical Oncology, Cancer Discovery, Blood, Haematologica and Leukemia. He is an active member of the GFM (Groupe Francophone des Myelodysplasies) and ALFA (AcuteLeukemia French Association) cooperative groups. He heads the “Hematology Oncogenesisand Biotherapies” PhD Program at Université de Paris.

MDS abstract

CMML: Dysplastic vs Poliferative Subtype, Does it Matter?

Chronic myelomonocytic leukemia (CMML) is the most frequent overall MDS/MPN myeloid malignancy with overall poor outcome (median survival of 3 years). The World Health Organization defines myeloproliferative CMML based on a WBC count greater than 13 x109/L, as opposed to the myelodysplastic subset of CMML, but also proposes to segregate CMML subsets on the basis of peripheral and bone marrow blast counts. After reviewing WHO criteria for CMML diagnosis, we discuss the pathophysiology of the disease and the possible biological underpinnings of such a classification for CMMLs. We then propose a pragmatic tool to prioritize treatment goals in CMML based on overall disease risk and predominant clinical symptoms, then review current therapeutic options, with an emphasis on allogeneic stem cell transplantation, cytoreduction and hypomethylating agents.


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Assoc. Prof Matteo Della PortaUniversity of Pavia, Italy

Head, Leukemia Unit - Cancer Center - IRCCS Humanitas Research Hospital Professor of Hematology - Humanitas UniversityVia Manzoni, 113 - 20089 Rozzano - Milan, Italywww.humanitas.it www.hunimed.eu

Current research interests mainly concern myeloid malignancies (myelodysplastic syndromes, MDS acute myeloid leukemias AML, myeloproliferative neoplasms, MPN)

Scientific publications include more than 150 peer-reviewed articles in journals covered by the JCR®. Matteo G Della Porta’s H-index is equal to 70 (Google Scholar).

MDS abstract

The Spectrum of Clonal Hematopoiesis: from CHIP to MDS

Clonal hematopoiesis of indeterminate potential (CHIP) is associated with increased risk of cancers and inflammation-related diseases. This phenomenon becomes very common in oldest-old individuals (aged 80y or older), in whom the implications of CHIP are not well defined. We performed a mutational screening in 1794 oldest-old individuals enrolled in two population-based studies and investigate the relationships between CHIP and associated pathologies. Clonal mutations were observed in one third of oldest-old individuals and were associated with reduced survival. Mutations in JAK2 and splicing genes, multiple mutations (DNMT3A, TET2, ASXL1 with additional genetic lesions) and variant allele frequency ≥0.096 had positive predictive value for myeloid neoplasms. Combining mutation profiles with abnormalities in red blood cell indices improved the ability of myeloid neoplasm prediction. On this basis, we defined a predictive model that identifies 3 risk groups with different probabilities of developing myeloid neoplasms. Mutations in DNMT3A, TET2, ASXL1 or JAK2 (most occurring as single lesion) were associated with coronary heart disease and rheumatoid arthritis. Cytopenia was a common finding in oldest-old population, the underlying cause remaining unexplained in 30% of cases. Among individuals with unexplained cytopenia, the presence of highly-specific mutation patterns was associated with myelodysplastic-like phenotype and a probability of survival comparable to that of myeloid neoplasms. Accordingly, 7.5% of oldest-old subjects with cytopenia had presumptive evidence of myeloid neoplasm. In conclusion, specific mutational patterns define different risk of developing myeloid neoplasms vs. inflammatory-associated diseases in oldest-old population. In individuals with unexplained cytopenia, mutational status may identify those subjects with presumptive evidence of myeloid neoplasms.

https://www.humanitas.it/

https://www.hunimed.eu/


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Prof Hans Michael KvasnickaUniversity of Witten/Herdecke, Germany

Prof Dr Hans-Michael Kvasnika is Chair at Institute of Pathology and Molecular Pathology, Helios University Clinic Wuppertal, University of Witten/Herdecke. He has been ChiefConsultant and Deputy Director at the Senckenberg Institute of Pathology, University of Frankfurt, in Frankfurt, Germany, and Head of Hematopathology and TumorpathologySection and Professor of Pathology. He is a consultant in a number of international trials and several World Health Organization-associated clinicopathological studies. He has published impressive list of peer-reviewed papers and has served as a co-author in the WHO Classification of Tumours of Haematopoietic and Lymphoid Tissues.

MDS abstract

Diagnostic Challenge: MDS with Fibrosis

Myelodysplastic syndromes (MDS) represent a variety of bone marrow (BM) neoplasms which are characterized by ineffective hematopoiesis and variable prognosis. BM histology is an essential tool for diagnosis of MDS and facilitates the evaluation of bone marrow BM cellularity and interstitial changes such as fibrosis. BM fibrosis (BMF), defined as increased deposition of reticulin in the BM, is observed in 10% to 20% of patients diagnosed with MDS. In general, BM fibrosis identifies a distinct subgroup of MDS (MDS-F) with multilineage dysplasia, high transfusion requirement, and poor prognosis and represents an independent prognostic factor that may be useful in clinical decision making. The presence of grade 3 BMF is associated with reduced overall survival independent from IPSS-R; however, BMF grade does not affect response to hypomethylating agent or lenalidomide treatment. TP53 and SETBP1 mutations occur with greater frequency among patients with severe fibrosis. Furthermore, the presence of CD34 cell clusters is an independent risk factor for progression to acute leukemia. In addition, MDS/MPN overlaps and MPN (especially triple-negative primary myelofibrosis) may share clinical and morphological features with MDS-F. In these cases genomic classification and careful BM evaluation according to the WHO criteria may help in the differential diagnosis and prognostic assessment of patients.


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Prof Uwe PlatzbeckerUniversity Hospital Leipzig, Germany

Since 1998, I have been working at the Department of Hematology and Oncology at the University Hospital “Carl Gustav Carus” in Dresden, Germany. Between 2001 and 2003 completed a 2-year postdoctoral fellowship provided by the Humboldt-foundation at the Fred Hutchinson Cancer Research Center in Seattle, USA. From 2012 to 2018 I held a position of Professor of Hematology at the University Hospital “Carl Gustav Carus” in Dresden and was the head of the section of Hematology as well as chair of the medical ethics committee of the TU Dresden. In 2015 I was appointed as Transcampus Professor of Hematology at the Faculty of Life Sciences and medicine at King’s College London. Since October 2018 I am heading the Hematology program at the University Hospital in Leipzig as Director of the Medical Clinic I, Hematology and Cell Therapy. I am a steering committee member of the SAL group, speaker of the German MDS study group, I have co-founded the European Myelodysplastic Syndromes Cooperative Group (EMSCO) in 2012 and I am chairing the MDS SWG of the EHA.

MDS abstract

Challenges in Risk Stratification of MDS

The heterogeneity is a disease-defining nature of myelodysplastic syndromes (MDS), a clonal disorder of hematopoietic stem and progenitor cells (HSPCs). During the last decade, significant progress has been made in better understanding the diversity of clinical, molecular, cellular and immunological factors, which are bound to outcome and prognosis of MDS patients. The holistic integration of novel, high impact individual risk parameters like patient-reported outcomes (PRO) or mutational and immunological data into conventional risk stratification systems will hopefully further refine patient subgroups, improve predictive power for survival and provide a next-generation classification and prognosis system for MDS patients. This may be also complemented by the definition of granular cohorts of patients with myeloid neoplasms and a druggable target (i.e. IDH 1/2 mutations) across conventional blast thresholds.


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Prof Rajko KušecUniversity of Zagreb, Croatia

Dr. Kusec is consultant hematologist atDepartment of hematology Universityhospital in Zagreb and is full professor of medicine. After postgraduate studiesin hematology at the Medicial University of Vienna, where he is visiting professor, he worked on the genetic basis of MDS at Nuffield department of Cellular science at Oxford University. His interests are in clinical aspects of diagnosis and treatment of MDS and MPN.

MDS abstract

Update on Lenalidomide for MDS

Lenalidomide, a derivative of thalidomide, is standard targeted therapy for del(5q) MDS with full erythroid recovery and cytogenetic remissions in up to 70% of patients. Its current official marketing authorization is for patients with transfusion dependent anemia of del(5q) MDS. Lenalidomide is being investigated in pre-transfusion dependent phase of del(5q) MDS; recent interim report of SITNRA-REV trial (Lopez Cadenas F, et al. ASH 2020) trial showed low dose (5mg/D) lenalidomide to significantly delay transfusion onset (75.7 vs 25.9 months), with 72,5% HI-E hemoglobin response and 70% CCyR. In clinical practice, time to response is short (1-3 months) with reported median duration from two to >6 years. Recent molecular genomic analyses of MDS patients revealed multiple subclonal myeloid mutations to possibly influence lenalidomide therapy outcomes, e.g. favorable response with the presence of DNMT3A in del(5q) MDS, and unfavorable response (resistance) with the presence of TET2 or SF3B1 in non-del(5q) MDS; presence of TP53 is unfavorable in both groups. Low-risk anemic and symptomatic patients with non-del(5q) MDS are candidates for lenalidomide after failing ESA or GFs, but with lower response (23.1%) than in del(5q). Better therapeutic effect in del(5q) occurs in part due to 5q deleted, haploinsufficient RPS14, CSNK1A1 and CAST genes that participate in the Cereblon protease triggered apoptosis mechanism. Immunomodulation of functional T- and B-cell state and changes in the composition of bone marrow microenvironment are additional important aspects of lenalidomide biological activity. Combinations with azacytidine for higher-risk MDS have not provided therapeutic benefit. Other combinations are being studied, e,g, with novel erythropoiesis stimulating agents, like luspatercept (trial NCT04539236).


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Prof Jaroslav ČermákInstitute of Hematology and Blood Transfusion, Czech Republic

Jaroslav Cermak is a professor of Internal Medicine at the Institute of Hematology and Blood Transfusion in Prague. He works as a head of Center for the rare diseases of hematopoiesis in the institute and as a head of Czech MDS Cooperative Group and of the Department for Postgraduate Education in Hematology in Czech Republic. He is a member of ASH, EHA, MDS Foundation, European LeukemiaNet and EuroBloodNet. His main topics of interest are MDS, PNH and rare disorders of erythropoiesis. He is an author or co-author of more than 100 articles in international journals.

MDS abstract

Progress in the Management of Cytopenia in MDS

Main focus of new treatment approaches in patients with lower-risk MDS who are not indicated for stem cell transplantion (SCT) is an improvement in profound cytopenia with correction of anemia and life threatening thrombocytopenia. Erythropoiesis stimulating agents (ESA) represent a standard approach to anemic MDS patients who met the criteria for EPO treatment. Lustpatercept is aninhibitor of GDF 11 mediated defective erythropoiesis and restores normal differentiation of erythroid progenitors. In a phase III. study, 54% of patients with RARS non responding to ESA achieved transfusion indpendence and 70% of patients had an increase of Hb ≥ 15 g/l after luspatercept administration. Imetelstat – a telomerase inhibitor and Roxadustat - a HIF stabilizator represent another promising agents affecting anemia in MDS patients. Thrombopoetin receptor agonists ( romiplostim , etrombopag ) has been shown effecive in correction of thromobytopenia in more than 40% of lower risk MDS patients. A recent long-term analysis did not confirm previous suspicion of an adverse effect of these drugs on disease progression. Currently, a lot of new compounds affecting different molecular nechanisms in pathogenesis of MDS are under investigation, however, SCT still remains the only curative treatment for all subgroups of MDS patients.


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Prof Uday PopatMD Anderson Cancer Center, Houston, Texas, USA

Dr. Popat is a Professor in the department of Stem Cell Transplantation and Cellular Therapy at The University of Texas MD Anderson Cancer Center, Houston, TX. After graduating from medical school, he trained in Internal medicine, Hematology, medical oncology and bone marrow transplantation at various institutions in India, UK, and USA including Royal Free Hospital, Baylor College of Medicine and The University of Texas MD Anderson Cancer Center. His research interests include stem cell transplantation for myeloid malignancies and transplantation in older patients and he has extensively published in these fields.

MDS abstract

Recent Developments in SCT for MDS

Hematopoietic cell transplantation (HCT) cures patients with myelodysplastic syndrome. It should be considered in all suitable patients with intermediate, high, or very high-risk disease by revised international prognostic scoring system (R-IPSS). Selected patients with low-risk disease with adversegenetic risk factors or severe cytopenias or therapy-related disease should also be considered for HCT.

Older patients beween 50-75 have better survival with reduced-intensity transplantation than standard thearpy. Likewise, improving results with haploidentical donors has extended the donor pool, making a donor available to almost all patients. Thus, HCT is a potential treatment option for all fit patients ages 75 years or younger with intermediate, high, or very high-risk diseases.

Though pretransplant cytoreduction has not been proven to be beneficial, it is generally done until logistics of HCT have been worked out. Maintance therapy is an active area of research to reduce relapse.

In future, personalized prediction models may be used to determine prognosis after transplant. Finally, myeloablative fractionated busulfan based conditioning regimen with post transplant cyclophosphamide graft versus host disease prophylaxis is a promising new approach to improvetransplant outcomes.


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Dr Magnus TobiassonThe Karolinska University Hospital, Stockholm, Sweden

Dr Magnus Tobiasson is a clinical hematologist focusing on myeloid leukemia and in particular the myelodysplastic syndromes (MDS). He is working as a senior consultant at the department of Hematology, Karolinska Hospital.

He wrote his thesis in 2015 on clinical and molecular effects of Azacitidine in MDS

His research group at the HERM lab at Karolinska Institute, is focusing on clinical and translational aspects of MDS, with a specific focus on allogeneic stem cell transplantation for MDS patients. He is an active member of the Nordic MDS group collaboration and is acting as PI for clinical trials within this collaboration.

MDS abstract

Novel Therapies for MDS

Dr Tobiasson will discuss new emerging treatments for MDS by presenting completed and ongoing clinical trials. He will also briefly discuss mechanism of action for these drugs. In lower-risk MDS there are some interesting new drugs e.g. luspatercept, roxadustat and imetelstat which can improve erythroid function. In higher risk, there are several new concepts e.g. targeted treatments: ivosidenib and enasidenib, immune checkpoint inhibitors: sabatolimab and magrolimab, pro-apoptotic drugs: venetoclax and p53 stabilizing drugs: eprenetapopt. Dr Tobiasson will also give some future perspectives.


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Dr Tapan KadiaMD Anderson Cancer Center, Houston, Texas, USA

Dr. Tapan Kadia is an Associate Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center in Houston, Texas. He is actively involved in clinical and translational research for the treatment of patients with leukemia. His particular focus is in developmental therapeutics in acute leukemia, including individualized frontline therapy, biologically rational targeted therapy, and longer term maintenance strategies in AML and ALL. He is primary investigator on numerous trials in acute myeloid leukemia, T-cell leukemias, bone marrow failure states, and is a leader in these. He has received numerous academic and clinical honors and awards for his studies and clinical research. Additionally, he’s authored over 270 peer-reviewed articles, numerous abstracts, and has been invited nationally and internationally for presentation of his research.

AML abstract

Managing Unfit Patients with AML

Abstract coming soon


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Dr Musa YilmazMD Anderson Cancer Center, Houston, Texas, USA

Dr. Musa Yilmaz has published extensively in the field of leukemia, and particularly, on prospective phase I and II trials of novel therapeutics. He also serves as the Principal Investigator for multiple clinical trials exploring combination therapies in FLT3 mutated AML. Dr. Yilmaz’ primary, research interests have been the role of early response assessment, particularly MRD (Minimal Residual Disease), in patients with acute leukemias and how this information can be used to identify patients at highest risk for relapse and death. He has also authored several other papers on the role of MRD in acute leukemias.

AML abstract

How I Treat FLT3-Mutated AML

Internal tandem duplication and tyrosine kinase domain mutations in FLT3 gene occur in 20-30% of the patients with newly diagnosed acute myeloid leukemia (AML) and result in a higher risk of relapse and inferior overall survival (OS). Following the discovery of FLT3 mutations and understanding their implications in AML, there has been a significant interest in developing drugs that target FLT3. Midostaurin, a first generation FLT3 inhibitor, became the first FLT3 inhibitor (in combination with intensive chemotherapy) to improve OS in younger adult patients with FLT3 mutated AML. However, the addition of midostaurin or other first generation FLT3 inhibitor, sorafenib, to low-intensity chemotherapy in elderly provided no significant OS benefit; approximately 8 months of median OS in the frontline setting. Although preliminary, more encouraging results were reported with the second generation FLT3 inhibitor-based combination regimens. Phase II and III clinical trials evaluating second generation FLT3i-based combinations in older adults are ongoing. After demonstrating a median OS of 15 months in older patients with newly diagnosed AML, therapy with venetoclax and hypomethylating agent has emerged as a new standard of care. However, the expected median OS in FLT3 mutated patient with frontline HMA plus venetoclax is still less than a year. In this review, we discussed current treatment approaches and future novel therapeutics in FLT3 mutated AML.


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Prof Charles CraddockUniversity of Birmingham, UK

Charles Craddock is Academic Director of the Centre for Clinical Haematology, Queen Elizabeth Hospital, Birmingham, UK, and Professor of Haemato-oncology, at the University of Birmingham. He trained in haematology at the Hammersmith Hospital, London, the Institute of Molecular Medicine at the University of Oxford and the University of Washington, Seattle, USA.

Professor Craddock is a recent President of the British Society of Blood and Marrow Transplantation and Chair of the UK Stem Cell Strategic Oversight Committee. He is Director of the Centre for Clinical Hematology at the Queen Elizabeth Hospital and was Transitional Director of the £24 million Birmingham Institute of Translational Medicine which opened in 2015. He was Medical Director of the Anthony Nolan from 2010-2014. His main research interests include the development of novel drug and transplant therapies in myeloid leukaemias and he leads the Bloodwise UK Trials Acceleration Programme.

AML abstract

Venetoclax for AML

Treatment options in adults with newly diagnosed acute myeloid leukemia (AML) who are unfit for intensive chemotherapy (IC) have historically been very limited consisting primarily of a hypomethylating agent, either azacitidine (AZA) or decitabine, or low dose cytosine arabinsoide (araC). At the same time IC fails to deliver long-term disease-free survival particularly patients withadverse genetics or older patients with co-morbidities. Venetoclax (VEN) is a selective Bcl2 inhibitor which, in combination with either a hypomethylating agent or low dose araC, delivers a high complete remission (CR) rate in adults with newly diagnosed AML with a favourable toxicity profile. In the recent VIALE-A trial VEN, in combination with AZA resulted in a statistically significant increase in both Cr/CRi rate and overall survival compared with AZA monotherapy, with an acceptable safety profile, in adults with newly diagnosed AML deemed unfit for IC. These data establish VEN/AZA as a new standard of care in patients unfit for IC. At the same time, they raise the possibility that a VEN based regimen may be preferable to IC in selected fit patients with newly diagnosed AML and the results of ongoing prospective randomised trials are awaited. VEN may also represent an an important maintenance strategy after IC or allogeneic stem cell transplantation.


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Prof Farhad Ravandi-KashaniMD Anderson Cancer Center, Houston, Texas, USA

Dr. Farhad Ravandi is Janiece and Stephen A. Lasher Professor of Medicine and Chief of Section of Acute Myeloid Leukemia in the Department of Leukemia at the University of Texas – M. D. Anderson Cancer Center. He graduated from the University of London, England and undertook residency and fellowship training at the Baylor College of Medicine and The University of Texas M.D. Anderson Cancer Center. During this time he specialized in the management of hematological malignancies and stem cell transplantation. He then joined the University of Illinois at Chicago as the director of leukemia program and the interim director of stem cell transplant program for three years until he joined the Leukemia department of M. D. Anderson in 2003. Dr. Ravandi is board certified in Internal Medicine, Hematology and Medical Oncology. His main areas of interests are therapy of acute myeloid leukemia, as well as rare leukemias including Philadelphia positive acute lymphoblastic leukemia, hairy cell leukemia and T-cell leukemias. He has authored several book chapters and many articles in peer-reviewed journals. He is a member of several professional societies including the American Society of Hematology, American Society of Clinical Oncology, and Society of Hematological Oncology and is a member of editorial board of several journals.

AML abstract

New Strategies for Treating Relapsed AML

Relapse in AML continues to be a major obstacle in improving patients’ outcomes. Advances in understanding the molecular biology of leukemogenesis have led to the development of new, effective agents that have been mostly evaluated in the relapsed setting. Some of these agents such as gilteritinib, enasidenib and ivosidenib have already been approved for treating relapsed disease. Others such as venetoclax combinations have demonstrated significant efficacy in early reports. Many other agents are in early development and undergoing clinical evaluation, some with great promise. Although the outcomes of patients with relapsed AML have improved modestly using these agents, better frontline strategies that would reduce the likelihood of relapse followed by monitoring measurable residual disease and developing agents to eradicate it, are the ways that we can further improve outcomes of patients with AML in the future.


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Prof Jacqueline CloosAmsterdam University Medial Center, Netherlands

The work of Jacqueline Cloos is dedicated to improving diagnostics, treatment strategies and ultimately outcome of acute myeloid (AML) leukemia patients. She focuses on measurable residual disease (MRD) that remains after therapy. MRD is the major cause of relapse in leukemia and her research group assesses MRD using flow cytometry in large clinical trials. Her research group participates in the AML MRD working party of the European Leukemia Network (ELN) to join forces in improving MRD to be used for the individual patient. She will summarize the current view of using MRD for clinical decision making in AML treatment.

AML abstract

The Role of MRD Monitoring in AML

An important factor of treatment failure for AML patients is the development of relapse even after a seemingly successful induction chemotherapy treatment. The majority of patients reach a complete remission, but still leukemia cells may remain. These residual cells are thought to be relatively drug resistant and when they have leukemia initiating properties (such as leukemia stem cells), these cells are prone to cause a relapse. So, the best way to improve patient outcome is the circumvention of a relapse. By identification of those patients at risk for relapse the intensity of the treatment can be tailored accordingly. Risk classification at diagnosis is not sufficient for a good risk estimation, while MRD measured by flow cytometry or molecular techniques above a certain threshold is a very strong prognostic factor, which is established for patients treated with intensive chemotherapy. Besides selecting the best treatment option, MRD is under investigation for other clinical implementations as well such as guiding stem cell transplantation, monitoring disease and as surrogate endpoint for future studies. Current several collaborative initiatives are ongoing (e.g., European Leukemia network, Harmony) to improve both the technical quality of MRD assessments but also relevant guidelines for the clinical implementation of MRD.


MPN MDS & AML US Focus 2022

Online International Knowledge & Clinical Practice in 2022

MPN MDS & AML US Focus 2022

Online International Knowledge & Clinical Practice in 2022

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