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With a growing population of survivors following HCT, the importance of registry‐based studies focused on this patient population has become increasingly apparent. Research focused on long‐term HCT survivors is complex due to several factors, including the fact that certain HCT‐specific late effects may be rare, have long latencies, or occur more frequently in certain patient populations.

In order to overcome these research barriers, centralized and standardized data collection of large numbers of patients over extended periods of time is required. Recent studies evaluating risk factors for secondary neoplasms (SN), including central nervous system (CNS) tumor development in survivors of pediatric HCT and acute myeloid leukemia/myelodysplastic syndrome in survivors of autologous HCT, are examples of the critical importance of registry data to explore rare events that occur many years following the HCT process itself [13,'4]. The study focused on CNS tumor development found the cumulative incidence of tumor development to be 1.29% at 20 years following HCT. Though this equated to a total of 59 CNS tumors in 8720 patients, this was 33 times the expected rate in the general population and highlights the importance of surveilling for rare events in survivors [14].

Table 2.1 Transplant Registries Collecting Data on HCT Survivors

	Center for International Blood and Marrow Transplant Research (CIBMTR)	European Society for Blood and Marrow Transplantation (EBMT)	Asia‐Pacific Blood and Marrow Transplantation Group (APBMT)	Eastern Mediterranean Blood and Marrow Transplantation Registry (EMBMT)
Population/Region Included in Data	Over 320 centers. All US allograft centers (mandatory reporting for allogeneic transplants), various other regions in the world	Over 500 centers in Europe	Over 600 centers in 20 Asia‐Pacific countries/regions	38 centers in the Eastern Mediterranean Region
Year Data Collection Began	1968	1973	2007	2008
Number of Patients in Registry	>500,000	560,000	>200,000	>20,000
Transplant‐Related Data Collected	Transplant essential data (TED) for all patients includes Patient‐Disease‐Transplant related characteristics as well as several outcome data including disease status, aGVHD, cGVHD, cause of death and a number of infectious and noninfectious complications, such as second malignancies. Research level data on a subset (approximately 25%) of patients includes comprehensive disease history and late effects post‐transplant	Patient‐Disease‐Transplant related characteristics as well as several outcome data including disease status, aGVHD, cGVHD, cause of death and a number of infectious and noninfectious complications	Least Minimum Dataset (LMD) for all patients includes disease information, conditioning regimen (agents/radiation types), GVHD prophylaxis, best disease status after HCT, details of relapse or progression of disease, aGVHD, cGVHD, and cause of death. LMD also includes donor information	Limited database is obtained from all participating centers which includes: type of transplant, indication, type of conditioning, donor type and source of stem cells Few centers can provide outcome data on request.
Late Effects‐Specific Data Collected	Secondary malignancy and fertility outcomes for all patients. Research level patients: extensive organ‐specific late effects	Secondary malignancy; Details of relapsed or persisting disease including treatment; Pregnancy after HCT, Infectious and toxic complications are collected for the first 100‐day evaluation and subsequently for every yearly follow‐up on MED‐B forms (i.e. centers not reporting MED‐B forms do not report late effects except for secondary malignancy)	Secondary malignancy and the main cause of death	Few centers can provide outcome and late effect data on request. Late effects are addressed in few multicenter studies but not captured by the database.
Funding for Registry	Funding sources include federal, National Institute of Health grants and corporations	Through EBMT fundraising and locally paid data managers	Through APBMT funding only	Through EMBMT fundraising and partial support from the hosting institution
Regulations (voluntary/mandatory)	Mandatory TED level reporting for all allogeneic transplants in the US All other reporting is voluntary	Consent forms for patients, regulation for data collection; manuals for data management; regulation through JACIE accreditation which mandates data collection	In Japan, centers are obliged to make an effort to submit outcome data to the Japanese HCT registry. In all other countries or regions, reporting is voluntary.	Consent forms for patients, regulation for data collection. Few centers are JACIE accredited which mandates data collection
Research Opportunities	Open to the entire community through a system of peer‐review	Open for any EBMT member	Open for any APBMT member from a country/region participating and submitting data to outcome registry	Open for any EMBMT member. Few studies were done in partnership with CIBMTR and EBMT
Studies on late effects performed (Y/N)	Y	Y	N In some countries/regions with a long history of HCT, studies on late effects are performed by using their national registry data	Y One study at present

While pre‐HCT exposures, HCT‐related variables and demographic data have routinely been collected in most registries, the granularity and extent of late effects data collection has varied. SN development was the major long‐term complication captured by registries in their early stages of development. Though it differs depending on the specific registry, the late effects data obtained has now expanded to include long‐term outcomes such as fertility, cardiovascular disease, diabetes, cataracts, chronic GVHD (cGHVD), socioeconomic status and late disease relapse.