Myelodysplastic Syndromes: Determining Risk, Tailoring Therapy, Supporting Patients PDF
Preview Myelodysplastic Syndromes: Determining Risk, Tailoring Therapy, Supporting Patients
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A S T FAST FACTS F A with Fast Facts – the ultimate medical handbook series C T S Myelodysplastic M y Syndromes FAST FACTS e l o Myelodysplastic Syndromes d y s p 9 Epidemiology and etiology la s t i c 20 Pathogenesis and presentation S Peter Dyer and Martha L Arellano y n 31 Diagnosis and classification d r o m 51 Management e s 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: +44 (0)1235 523233 Book orders can be placed by telephone or email, or via the website. Please telephone +41 61 306 1440 or email [email protected] To order via the website, please go to karger.com Fast Facts is a trademark of S. Karger Publishers Ltd. All rights reserved. No part of this publication may be reproduced, stored in a retrieval system, or transmitted in any form or by any means, electronic, mechanical, photocopying, recording or otherwise, without the express permission of the publisher. The rights of Peter Dyer and Martha L Arellano to be identified as the authors of this work have been asserted in accordance with the Copyright, Designs & Patents Act 1988 Sections 77 and 78. The publisher and the authors have made every effort to ensure the accuracy of this book, but cannot accept responsibility for any errors or omissions. For all drugs, please consult the product labeling approved in your country for prescribing information. Registered names, trademarks, etc. used in this book, even when not marked as such, are not to be considered unprotected by law. 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 5 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. 7
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