MD, PhD Hospital Clinic Hematopoietic Cell Transplantation Program Barcelona, Spain
Alicia Rovó MD Department of Hematology and Central Hematology Laboratory Inselspital, Bern University Hospital Bern, Switzerland
Nina Salooja FRCPath MD Department of Medicine Imperial College London Hammersmith Hospital London, UK
Insara Jaffer Sathick MD Renal Service, Division of Medicine Memorial Sloan Kettering Cancer Center New York, NY, USA
Bipin N. Savani MD Long Term Transplant Clinic (LTTC), Hematology, Stem Cell Transplantation and Cellular Therapy Section, Division of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center and Veterans Affairs Medical Center, Nashville, TN, USA
Angela Scherwath PhD Department and Outpatient Clinic of Medical Psychology University Medical Center Hamburg – Eppendorf Hamburg, Germany
Hélène Schoemans MD, PhD Department of Hematology University Hospitals Leuven and KU Leuven Leuven, Belgium
Dana Shanis MD, FACOG VHealth & Wellness Philadelphia, PA, USA
Bronwen E. Shaw MD, PhD Center of International Blood and Marrow Transplant Research; Department of Medicine Medical College of Wisconsin Milwaukee, WI, USA
Gérard Socié MD, PhD Hematology TransplantationAPHP Hospital Saint Louis and Université de Paris; Université de Paris & INSERM U976 Paris, France
Ayman O. Soubani MD Division of Pulmonary, Critical Care and Sleep Medicine Wayne State University School of Medicine Detroit, MI, USA
Katrina M. Stokes MSSW LCSW Tennessee Valley Healthcare System Nashville, TN, USA
Pamela Stratton MD Office of the Clinical Director National Institute for Neurological Disorders and Stroke Bethesda, MD, USA
Aurélien Sutra del Galy MD Hematology Transplantation APHP Hospital Saint Louis and Université de Paris; Université de Paris & INSERM U976 Paris; France
Sarah Thilges PhD Section of Psychosocial Oncology Loyola University Medical Center Maywood, IL, USA
André Tichelli MD Division of Hematology University Hospital Basel Basel, Switzerland
Mihkaila Wickline MD Seattle Cancer Care Alliance Fred Hutchinson Cancer Research Center Seattle, WA, USA
Lori Wiener, PhD, DCSW, LCSW‐C Psychosocial Support and Research Program Center for Cancer Research Pediatric Oncology Branch NIH Bethesda, MD, USA
John R. Wingard MD Division of Hematology & Oncology University of Florida College of Medicine Gainesville, FL, USA
CHAPTER 1 Introduction to long‐term survivorship after hematopoietic cell transplantation
Bipin N. Savani1 and André Tichelli2
1 Division of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN, USA
2 Division of Hematology, University Hospital Basel, Basel, Switzerland
Background
Hematopoietic cell transplantation (HCT) provides curative therapy for a variety of diseases. Over the past several decades, significant advances have been made in the field of HCT and now HCT has become an integral part of treatment modality for a variety of hematologic malignancies and some nonmalignant diseases. HCT remains an important treatment option for a wide variety of hematologic and nonhematologic disorders, despite recent advances in the field of immunologic therapies. Factors driving this growth include expanded disease indications, greater donor options (expanding unrelated donor registries and haploidentical HCT), and accommodation of older and less fit recipients [1,2‐4].
The development of less toxic pretransplant conditioning regimens, more effective prophylaxis of graft‐versus‐host disease (GVHD), improved infection control, and other advances in transplant technology have resulted in a rapidly growing number of transplant recipients surviving long‐term free of the disease for which they were transplanted. The changes over decades in the transplant recipient population and in the practice of HCT will have almost inevitably altered the composition of the long‐term survivor population over time. Apart from an increasingly older transplant recipient cohort, the pattern of transplant indications has shifted from the 1990s when chronic myeloid leukemia made up a significant proportion of allo‐HCT indications. Changes in cell source, donor types, conditioning regimens, GVHD prophylaxis, and supportive care have all occurred, with ongoing reductions in both relapse and non‐relapse mortality (NRM) have been demonstrated [5–10].
These patients have increased risks for a variety of late complications, which can cause morbidity and mortality (Figure 1.1). Most long‐term survivors return to the care of their local hematologists/oncologists or primary care physicians, who may not be familiar with specialized monitoring and management of long‐term complications after HCT for this patient population. As HCT survivorship increases, the focus of care has shifted to the identification and treatment of long‐term complications that may affect quality of life and long‐term morbidity and mortality [11–13].
Preventive care, as well as early detection and treatments, are important aspects to reducing morbidity and mortality in long‐term survivors after allo‐HCT [4,14‐17]. The second edition of the book Blood and Marrow Transplantation Long‐Term Management: Prevention and Complications updates our knowledge on diagnosis, screening, treatment, and long‐term surveillance of long‐term survivors after HCT and shall meet the long‐term follow‐up standard operative practice (SOP) requirement of Foundation for the Accreditation of Cellular Therapy (FACT)/ Joint Accreditation Committee of ISCT‐EBMT (JACIE) standards [17].
Rapidly rising numbers of long‐term transplant survivors
Since the first three cases of successful allo‐HCT in 1968, the number of HCTs performed annually has increased steadily over the past several decades and continues to do so each year [1–3]. Advances in HCT practice and supportive care have led to improved outcomes and an increasing number of long‐term HCT survivors.
Historically, the limitation of allo‐HCT has been transplant‐related mortality (TRM) and the availability of a compatible donor. In order to offer the curative allo‐HCT treatment option in most patients, safer regimens with acceptable GVHD‐associated morbidity and TRM are preferred, and donor availability have been expanded. In this era, a stem cell source can be found for virtually all patients who have an indication to receive allo‐SCT. All of these advances result in steadily increasing numbers of long‐term survivors after HCT, creating an expanding pool of children, and young and mature adults who are at risk of long‐term complications of HCT [18].
