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 purpose of the Registry is to provide a pool of data to EBMT members to perform studies, assess epidemiologic trends, and ultimately improve patient outcome.
The EBMT is a voluntary organization comprising more than 600 transplant centers mainly from Europe. Status as a member center requires submission of minimal essential data (MED‐A form) from all consecutive patients to a central registry in which patients may be identified by the diagnosis of underlying disease and type of transplantation.
Data collection
Completeness of data registration is checked at regular intervals in centers with accreditation by the Joint Accreditation Committee ISCT‐Europe (JACIE) & EBMT, Europe’s official accreditation body in the field of hematopoietic stem cell transplantation (HSCT) and cellular therapy, promoting high‐quality patient care and medical and laboratory practice through a profession‐led, voluntary accreditation scheme. Over 330 centers have been accredited at some point in time. Accreditation has been shown to be associated with improved outcome [32].
Informed consent for transplantation and data collection is obtained locally according to current regulations. Since 2003, all transplant centers are required to obtain written informed consent prior to data registration with the EBMT following the 1975 Helsinki Declaration.
Next to the MED‐A data with a restricted set of variables, MED‐B data, are collected by centers with the data management capacity to handle the burden of data management, a task with formidable challenges in a voluntary organization without remuneration for the data management effort. Data initiatives are sometimes used to increase data coverage in a particular disease or field
