Clinical Management of Acute Lymphoblastic Leukemia: From Bench to Bedside

439 Pages·2021·7.095 MB·English

by  Mark R. Litzow, Elizabeth A. Raetz

Clinical Management
of Acute Lymphoblastic
Leukemia
From Bench to Bedside
Mark R. Litzow
Elizabeth A. Raetz
Editors
123
Clinical Management of Acute Lymphoblastic
Leukemia
Mark R. Litzow • Elizabeth A. Raetz
Editors
Clinical Management
of Acute Lymphoblastic
Leukemia
From Bench to Bedside
Editors
Mark R. Litzow Elizabeth A. Raetz
Division of Hematology Department of Pediatrics
Mayo Clinic NYU Langone Health
Rochester, MN, USA New York, NY, USA
ISBN 978-3-030-85146-0 ISBN 978-3-030-85147-7 (eBook)
https://doi.org/10.1007/978-3-030-85147-7
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Preface
The genomic revolution has led to significant advances in understanding the genetic
mechanisms underlying the pathophysiology of acute lymphoblastic leukemia
(ALL). These studies in turn have identified new targets and treatment modalities
for this challenging form of acute leukemia. They have also resulted in advances in
our ability to detect minimal or measurable residual disease (MRD), which has
enhanced our ability to prognosticate and tailor therapy.
In this textbook, entitled Clinical Management of Acute Lymphoblastic Leukemia:
From Bench to Bedside, we have brought together noted experts in ALL to provide
the reader with comprehensive information on the basic and translational science
that underpins our new knowledge of the pathogenesis of ALL. Separate chapters on
the genetics of B-cell and T-cell ALL highlight these new developments. A chapter
on MRD assessment in ALL spotlights the translation of basic science discoveries
to refinements in this essential prognostic tool.
We follow these exciting developments with multiple chapters highlighting
advances in the treatment of B- and T-cell ALL across the age spectrum from infants
to elderly adults. We also highlight the significant progress that has been seen in the
treatment of children and adults with Philadelphia chromosome-positive ALL and
highlight the management of the new entity of Philadelphia-like ALL. This part
concludes with important chapters on the diagnosis and management of central ner-
vous system ALL and the progress and challenges in the management of late
sequelae of ALL therapy.
We conclude the book with an important part on new treatment modalities and
highlight the tremendous advances that have been seen with development of mono-
clonal antibody-based and chimeric antigen receptor T-cell therapies. While
advances in the treatment of T-cell ALL have lagged behind those in B-cell ALL,
there are new agents on the horizon for this challenging subtype of ALL and we
highlight these in a separate chapter. Finally, given all these new developments we
conclude with updated information which refines the role of hematopoietic cell
transplantation in both children and adults.
v
vi Preface
We hope readers will find this comprehensive textbook helpful as they confront
the new opportunities and challenges of treating children and adults with ALL.
Rochester, MN, USA Mark R. Litzow
New York, NY, USA Elizabeth A. Raetz
Contents
Part I Translational Science
1 Molecular Pathways and Targets in B-Cell
Progenitor Acute Lymphoblastic Leukemia . . . . . . . . . . . . . . . . . . . . . 3
Kathryn G. Roberts and Charles G. Mullighan
2 Genetic Mechanisms in T-Cell Acute Lymphoblastic Leukemia . . . . 33
Francesca Gianni and Adolfo Ferrando
3 Minimal Residual Disease in Acute Lymphoblastic
Leukemia: Techniques and Application . . . . . . . . . . . . . . . . . . . . . . . . 47
Xueyan Chen and Brent L. Wood
Part II Therapeutics
4 Treatment of Pediatric B- and T-Cell Acute Lymphoblastic
Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75
Laura E. Hogan, Luke D. Maese, Keith J. August,
and Jennifer L. McNeer
5 Treatment of Adult B- and T-Cell Acute Lymphoblastic
Leukemia: An Overview of Current Treatments
and Novel Advances . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105
Shimoli V. Barot and Anjali S. Advani
6 Acute Lymphoblastic Leukemia in Infants: A Distinctive,
High-Risk Subtype of Childhood Acute Lymphoblastic
Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 135
Erin H. Breese, Rishi S. Kotecha, and Erin M. Guest
7 Treatment of Elderly Patients with Acute Lymphoblastic
Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 149
Marc Schwartz and Matthew Wieduwilt
vii
viii Contents
8 Treatment of Childhood Philadelphia Chromosome-Positive
Acute Lymphoblastic Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171
Melissa A. Burns and Lewis B. Silverman
9 Treatment of Adult Philadelphia Chromosome-Positive
Acute Lymphoblastic Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 185
Oliver G. Ottmann
10 Treatment of Ph-Like Acute Lymphoblastic Leukemia . . . . . . . . . . . 209
Thai Hoa Tran and Sarah K Tasian
11 Prophylaxis and Treatment of Central Nervous
System (CNS) Acute Lymphoblastic Leukemia . . . . . . . . . . . . . . . . . . 235
Lauren D. Scherer and Eric S. Schafer
12 Late Effects of Therapy of Acute Lymphoblastic Leukemia . . . . . . . . 267
Liv Andrés-Jensen, Christoffer Johansen, and Kjeld Schmiegelow
Part III N ew and Existing Modalities of Therapy
13 Monoclonal Antibody-Based Treatment and Other
New Agents for B-Lineage Acute Lymphoblastic Leukemia . . . . . . . . 295
John C. Molina and Nirali N. Shah
14 New Agents for the Treatment of T-Cell Acute
Lymphoblastic Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 329
Sunil S. Raikar, David T. Teachey, and Nathan P. Gossai
15 The Development and Management of Treatment
with Chimeric Antigen Receptor T Cell (CAR T) . . . . . . . . . . . . . . . . 353
Colleen Annesley and Rebecca Gardner
16 Hematopoietic Stem Cell Transplantation in Adults with
Acute Lymphoblastic Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 373
Erik L. Kimble and Ryan D. Cassaday
17 Hematopoietic Stem Cell Transplantation in Pediatric
Acute Lymphoblastic Leukemia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 405
Divya Subburaj and Kirk R. Schultz
Index . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 431
Contributors
Anjali S. Advani Department of Leukemia, Taussig Cancer Institute, Cleveland
Clinic, Cleveland, OH, USA
Liv Andrés-Jensen Department of Pediatrics and Adolescent Medicine, University
Hospital Rigshospitalet, Copenhagen, Denmark
Institute of Clinical Medicine, Faculty of Health and Medical Sciences, University
of Copenhagen, Copenhagen, Denmark
Colleen  Annesley Department of Pediatrics, University of Washington,
Seattle, WA, USA
Cancer and Blood Disorders Center, Seattle Children’s Hospital, Seattle, WA, USA
Keith J. August Division of Pediatric Hematology/Oncology, Children’s Mercy
Hospital, Kansas City, MO, USA
Shimoli  V.  Barot Department of Internal Medicine, Cleveland Clinic,
Cleveland, OH, USA
Erin H. Breese Department of Pediatrics, University of Cincinnati College of
Medicine, Division of Oncology, Cancer and Blood Diseases Institute, Cincinnati
Children’s Hospital Medical Center, Cincinnati, OH, USA
Melissa  A.  Burns Department of Pediatric Oncology, Dana-Farber Cancer
Institute, Harvard Medical School, Boston, MA, USA
Ryan D. Cassaday Department of Medicine, University of Washington School of
Medicine, Seattle, WA, USA
Clinical Research Division, Fred Hutchinson Cancer Research Center,
Seattle, WA, USA
Xueyan Chen Department of Laboratory Medicine and Pathology, University of
Washington, Seattle, WA, USA
Adolfo  Ferrando Institute for Cancer Genetics, Columbia University, New
York, NY, USA
ix
x Contributors
Rebecca  Gardner Department of Pediatrics, University of Washington,
Seattle, WA, USA
Cancer and Blood Disorders Center, Seattle Children’s Hospital, Seattle, WA, USA
Francesca  Gianni Institute for Cancer Genetics, Columbia University, New
York, NY, USA
Nathan  P.  Gossai Department of Cancer and Blood Disorders, Children’s
Minnesota, Minneapolis, MN, USA
Erin  M.  Guest University of Missouri-Kansas City, Division of Pediatric
Hematology/Oncology/Blood and Marrow Transplantation, Children’s Mercy
Kansas City, Kansas City, MO, USA
Laura E. Hogan Stony Brook Children’s, Stony Brook, NY, USA
Christoffer  Johansen Institute of Clinical Medicine, Faculty of Health and
Medical Sciences, University of Copenhagen, Copenhagen, Denmark
Department of Oncology, University Hospital Rigshospitalet, Copenhagen, Denmark
Erik L. Kimble Department of Medicine, University of Washington School of
Medicine, Seattle, WA, USA
Clinical Research Division, Fred Hutchinson Cancer Research Center,
Seattle, WA, USA
Rishi S. Kotecha Department of Clinical Haematology, Oncology, Blood and
Marrow Transplantation, Perth Children’s Hospital, Perth, WA, Australia
Leukaemia Translational Research Laboratory, Telethon Kids Cancer Centre,
Telethon Kids Institute, University of Western Australia, Perth, WA, Australia
Curtin Medical School, Curtin University, Perth, WA, Australia
Luke  D.  Maese Department of Pediatrics and Huntsman Cancer Institute,
University of Utah, Salt Lake City, UT, USA
Jennifer L. McNeer Section of Pediatric Hematology/Oncology, The University
of Chicago Comer Children’s Hospital, Chicago, IL, USA
John C. Molina Pediatric Oncology Branch, Center for Cancer Research (CCR),
National Cancer Institute (NCI), National Institutes of Health (NIH),
Bethesda, MD, USA
Department of Pediatric Oncology, Johns Hopkins Hospital, Baltimore, MD, USA
Charles G. Mullighan Department of Pathology, Hematological Malignancies
Program, St. Jude Children’s Research Hospital, Memphis, TN, USA
Oliver G. Ottmann Division of Cancer and Genetics, School of Medicine, Cardiff
University, Cardiff, UK
Sunil S. Raikar Department of Pediatrics, Aflac Cancer and Blood Disorders
Center, Emory University and Children’s Healthcare of Atlanta, Atlanta, GA, USA