means of data collection that may not require patients to return to their specific transplant centers [21‐23]. Sharing research findings with patients and caregivers through various forums (i.e. online webinars, in‐person conferences) can demonstrate the importance of continued registry involvement and may result in improved patient follow‐up.
Registries are also adapting and expanding to include outcomes related to various novel cellular therapies, such as chimeric antigen receptor T‐cell (CART) therapy, in addition to HCT. Though only short‐term data are available for novel cellular therapies at this time, the importance of following these patients long‐term to evaluate for potential late effects has been acknowledged and is already being discussed. Facilitating the merging of other disease and treatment‐specific registries not centered on HCT alone (such as those focused on solid‐organ transplant, hematologic or solid‐tumor malignancy therapies, and pediatric or adult cancer survivors) is another area of interest [24]. Though merging data from diverse registries represents another opportunity to compare patient populations and use registry data to its full extent, challenges exist since data collection methodology is seldom standardized, and the collection of uniform, unique identifiers is inconsistent. Most importantly, continued and increased future collaborations between the major HCT registries presented herein will be critical to enhance research endeavors focused on the growing population of HCT survivors.
Center for International Blood and Marrow Transplant Research (CIBMTR)
Rachel Phelan and Bronwen E Shaw
Introduction to the registry
The CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin (MCW). This collaboration, agreed upon in 2004, brought together the strength of the two organizations and removed the need for centers to carry out duplicate reporting to both the NMDP and International Bone Marrow Transplant Registry (IBMTR) for patients who received stem cells from an unrelated donor. The mission of the CIBMTR is to promote collaborative research to understand and improve access to, and outcomes of, cellular therapies for the people served. Though CIBMTR initially collected data only for Hematopoietic Cell Transplant (HCT) recipients, this has recently been expanded to include recipients of non‐HCT cellular therapies. CIBMTR works closely with other international registries to harmonize data collection variables and facilitate joint research efforts.
Data collection
CIBMTR has been collecting longitudinal patient outcome data for over 45 years. The complexity and volume of data collected has increased with time, as the field has progressed. In the US, reporting of all allogeneic transplants is mandatory to meet the requirements of the Stem Cell Therapeutic Outcomes Database (SCTOD) contract (which CIBMTR holds) for the Health Resources and Services Administration (HRSA)‐sponsored C.W. Bill Young Cell Transplantation Program (CWBYCTP). This contract requires CIBMTR to produce an annual center‐specific survival analysis evaluating the one‐year survival rates among US centers. All patients participate at the “essential” level of data collection, which includes details related to patient, disease and transplant characteristics which impact transplant outcomes. A subset of patients who consent to research (approximately 25% of the registry) have more comprehensive data collected, which includes details of pretransplant disease course and therapy, as well as additional outcomes data such as infections and organ‐specific late effects. Data are collected for as long as the patient is followed clinically (lifelong if applicable).
Data quality is an essential aspect of the registry and multiple steps are in place to enhance this. These include: upfront data validations, automated cross‐form data validations, in‐system data queries, automated data quality checks performed by a dedicated data quality team and, finally, direct review by medical and scientific staff of individual data elements at the time of data analyses. In addition, CIBMTR has a routine system to ensure the completeness of form submission on a calendar basis, and an audit team who perform comprehensive on‐site data audits for participating sites every four years. There are ongoing challenges related to the burden of data entry at the transplant centers and laboratories, as well as challenges related to the need to regularly update the data variables collected on the forms to reflect changing transplant practice.
Late effects specific data collection
CIBMTR has made an effort to include collection of late‐effects data on patients for most of its history, however, the nature and completeness of these data has changed significantly over time. Data on subsequent neoplasms (SN) and fertility outcomes have been collected for many years on all patients. Centers are asked to provide pathology reports to CIBMTR to support the SN diagnosis and subsequent research studies in SNs, however, this can by logistically challenging, especially when SNs are not diagnosed at the original transplant center.
Data on other organ‐specific late effects, including renal, cardiac, metabolic, endocrine, psychiatric and others are collected for patients for whom research level data are collected. These data are complex to collect due to the broad spectrum of late effects and the inability to collect extensive detail on individual events due to the nature of registry reporting. Another serious challenge relates to the fact that these late effects most frequently occur at a time when the patient is no longer regularly (or at all) attending their transplant center and thus under‐ascertainment is highly likely.
In 2018, CIBMTR convened a group of experts to form a late effects task force to develop a strategy for the focused collection of late effects in a subset of patients reporting to CIBMTR. The task force made recommendations in three main areas: subsequent neoplasms, organ‐specific late effects and the inclusion of Patient‐Reported Outcomes (PRO) in routine registry data collection. Many of the recommendations focus on assessing the quality and completeness of late‐effect reporting to CIBMTR and include proposals to compare CIBMTR data to other disease or outcomes specific databases/registries. Enhancing the collection of risk factors, and correlative biologic material was also recommended in some circumstances. Finally, the value of adding assessments of quality of life by direct patient questioning (PROs) was strongly encouraged.
Most research on late effects in CIBMTR is performed in the Late Effects and Quality‐of‐Life (QOL) Working committee. Members of the community can propose studies to the committee which are then voted for through a peer‐review system at the annual meeting. The committee has performed studies addressing a diverse range of topics such as the incidence and risk factors for SN and other late effects after HCT, cardiovascular and metabolic complications, and return to work and QOL in HCT survivors [3,13,14,25‐31].
Future plans
CIBMTR is currently implementing the recommendations of the late effects task force. An IRB‐approved PRO protocol is in place (August 2019) to begin the collection of PROs. An important recommendation of the task force was to explore mechanisms to enhance patient engagement in later years following transplant, especially if they have left the care of the transplant center, to continue to understand the physical, emotional and social issues that they face. Such engagement, through direct patient contact, may also enhance the collection of late effects such as SN and fertility outcomes. Finally, CIBMTR is also transforming the way in which medical record/electronic data are collected to reduce the burden on transplant center staff and maximize the benefits of advances in IT systems and electronic data records.
The Registry of the European Society for Blood and Marrow Transplantation (EBMT)
Jakob R. Passweg and Helen Baldomero
Introduction to the registry
The EBMT Registry, established in 1974, is the backbone of the EBMT’s research and educational activities. More than half a million patients having received a hematopoietic stem cell transplantation (HSCT) procedure are included and cellular therapies now constitute a rapidly expanding field. The