Blood and Marrow Transplantation Long Term Management. Группа авторов

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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 health record systems, is being investigated as an alternative 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

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