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.
Registry Challenges
Though data collection through registries can address several of the issues surrounding late effects research, there remain many challenges. The correct methodology to conduct such research often requires intensive protocol development and complex statistical approaches. A recent report by the National Institutes of Health (NIH) Hematopoietic Cell Transplantation Late Effects Initiative highlights the importance of collecting the correct amount and type of data, thoughtfully approaching study design, and having the expertise to conduct the analysis [15]. In order to collect registry data and conduct appropriate studies, significant infrastructure and funding is required. When collecting and evaluating data internationally, cultural/language difference, regulation variances around data collection, variability in organizational structure, and resource barriers need to be considered. One of the most notable challenges, regardless of geographic location, is that of patient attrition as they get further from the acute post‐HCT period and may be less likely to be followed by the transplant center.
Future of Data Collection in Survivorship
Efforts are being made to improve data collection for long‐term survivors. Growth of dedicated late effects‐focused clinics in both the pediatric and adult setting has the potential to improve patient engagement and decrease the number of patients lost to follow‐up in the years following HCT [16–20]. The utilization of technology, such as mobile health (mhealth) platforms to track the health of survivors, electronic patient‐reported outcomes, and streamlined registry data ascertainment using electronic