Myelodysplastic Syndromes: Determining Risk, Tailoring Therapy, Supporting Patients

79 Pages·2021·2.831 MB·English

by  Dyer, Peter, Arellano, Martha L.

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Myelodysplastic Syndromes
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9 Epidemiology and etiology la
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20 Pathogenesis and presentation
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31 Diagnosis and classification d
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51 Management
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68 Supporting patients with MDS
ISBN 978-3-318-06872-6
Determining risk, tailoring therapy, supporting patients
FAST FACTS
9 783318 068726
KARGER.COM

FAST FACTS
Myelodysplastic Syndromes
Peter Dyer MBChB BSc MRCP FRCPath PGCMedEd
Consultant Haematologist
University Hospitals of North Midlands NHS Trust
North Midlands, UK
Martha L Arellano MD
Associate Professor of Hematology and Oncology
Interim Associate Director, Hematology Division
Hematology and Medical Oncology Fellowship Program Director
Winship Cancer Institute of Emory University
Atlanta, Georgia, USA
With additional contributions from Nicki H Lawrence BSc(Hons) MSc CSci, Principal
Biomedical Scientist and Advanced Practitioner in Morphology, Royal Stoke University
Hospital, North Midlands, UK.
Declaration of Independence
This book is as balanced and practical as we can make it.
Ideas for improvement are always welcome: [email protected]
Fast Facts: Myelodysplastic Syndromes
First published 2021
Text © 2021 Peter Dyer, Martha L Arellano
© 2021 in this edition S. Karger Publishers Ltd
S.Karger Publishers Ltd, Elizabeth House, Queen Street, Abingdon,
Oxford OX14 3LN, UK; Tel: [phone]
Book orders can be placed by telephone or email, or via the website.
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The publisher and the authors have made every effort to ensure
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A CIP record for this title is available from the British Library.
ISBN 978-3-318-06873-3
Dyer P (Peter)
Fast Facts: Myelodysplastic Syndromes/
Peter Dyer, Martha L Arellano
Cover image shows dysplastic changes in erythroid cells and immature/
dysplastic forms of myeloid cells in a bone marrow sample from a patient with
a myelodysplastic syndrome.
Typesetting by Amnet, Chennai, India.
Printed in the UK with Xpedient Print.
Made possible by a contribution from Takeda Pharmaceutical Company
Limited. Takeda did not have any influence on the content and all items were
subject to independent peer and editorial review.
List of abbreviations 5
Introduction 7
Epidemiology and etiology 9
Pathogenesis and presentation 20
Diagnosis and classification 31
Management 51
Supporting patients with MDS 68
Useful resources 74
Index 75

List of abbreviations
AML: acute myeloid leukemia IST: immunosuppressive therapy
ATG: antithymocyte globulin LR-MDS: low-risk MDS
AUC: area under the curve MDS: myelodysplastic syndromes
CHIP: clonal hematopoiesis of MDS-EB: MDS with excess blasts
indeterminate potential
MDS-MLD: MDS with multilineage
CI: confidence interval dysplasia
CMML: chronic myelomonocytic MDS-RS: MDS with ring sideroblasts
leukemia MDS-SLD: MDS with single-lineage
dysplasia
CR: complete remission
NGS: next-generation sequencing
DNA: deoxyribonucleic acid
OS: overall survival
ESA: erythropoiesis-stimulating agent
PNH: paroxysmal nocturnal
FDA: Food and Drug Administration
hemoglobinuria
FISH: fluorescence in situ
RBC: red blood cell
hybridization
RBC-TI: red blood cell-transfusion
GvHD: graft versus host disease
independence
HI: hematologic improvement
RIC allo: reduced-intensity
HIV: human immunodeficiency virus conditioning allogeneic HSCT
HLA-DR: human leukocyte RIPSS: Revised International
antigen-DR isotype Prognostic Scoring System
HMA: hypomethylating agent RNA: ribonucleic acid
RS: ring sideroblast
HR-MDS: high-risk MDS
SNP: single nucleotide polymorphism
HSC: hematopoietic stem cell
t-AML: treatment-related AML
HSCT: hematopoietic stem cell
transplantation t-MDS: therapy-related MDS
IPSS: International Prognostic WHO: World Health Organization
Scoring System
WPSS: WHO Classification-Based
IR-MDS: intermediate-risk MDS Prognostic Scoring System
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Introduction
Myelodysplastic syndromes (MDS) are a heterogeneous group of
closely related clonal hematopoietic myeloid disorders that affect
older adults. The name is derived from the Greek words ‘μυελός’ or
‘myelos’ meaning marrow, and ‘δυσπλασία’ or ‘dysplasia’ meaning
abnormal morphology. MDS are characterized by ineffective
hematopoiesis leading to cytopenias and their attendant
complications (infection, bleeding and symptomatic anemia), and
variable risk of transformation to acute myeloid leukemia (AML).
The heterogeneity of MDS relates not only to differences in the
morphological appearances of cells, but also to diversity in molecular
and cytogenetic abnormalities detectable in the blood and marrow of
patients with MDS.
Diagnosis of MDS must be distinguished from that of dysplasia
associated with marrow failure syndromes and other clonal disorders,
as well as non-clonal, non-malignant causes such as infections,
nutritional deficiencies and intoxications. Accurate diagnosis requires
excellent morphological evaluation, as well as cytogenetic and
molecular data, in the context of peripheral blood cytopenias. Clinical
correlation is also important to rule out familial syndromes and
non-neoplastic causes of dysplasia.
MDS can be indolent non-progressive disorders, but they can also
have an aggressive clinical course with (at times rapid) progression to
AML in approximately 15–30% of patients. Various prognostic scoring
systems may be used to predict clinical course and time to AML
progression, and thus tailor therapy.
The overall 5-year probability of survival for patients with MDS
remains poor at approximately 30%. Despite recent improvements in
therapies and support, and although several targeted therapies are
under investigation, current treatment options remain limited and
allogeneic hematopoietic stem cell transplantation is the only
potentially curative option for fit young patients with MDS.
This book is designed to provide hematology/oncology nurses,
physicians and trainees with concise up-to-date guidance for accurate
diagnosis, risk stratification and management of patients with MDS.
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