1. Current Uses and Outcomes of Hematopoietic Stem Cell Transplantation
INTRODUCTION: The Summary Slides are an annual report on data submitted to the CIBMTR by centers worldwide and describes information related to practices and general survival outcomes after hematopoietic cell transplantation. The current edition includes transplants performed prior to 2015.
Slides 3 to 16 exhibit data on frequency of transplants according to age, donor and transplant type, graft source and disease, and causes of death. All frequencies represent first autologous and allogeneic transplants registered with the CIBMTR during the period. Slides 3, 4, 5 and 12 represent estimated frequencies of total number of transplants in the US
Slides 17 to 40 include overall survival outcomes according to disease, disease status, donor type, year of transplant and conditioning regimen intensity. Comparisons across survival curves are univariate and do not adjust for all potentially important factors; consequently, results should be interpreted cautiously.
Acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and chronic myelogenous leukemia (CML) are classified as early disease (first complete remission [CR1] or first chronic phase [CP1]), intermediate (second or subsequent CR or CP or accelerated phase [AP]), or advanced (primary induction failure, active disease, or blastic phase) disease. Myelodysplastic syndrome (MDS) is divided into early (refractory anemia [RA] or refractory anemia with ringed sideroblasts [RARS]), or advanced (refractory anemia with excess of blasts [RAEB] or chronic myelomonocytic leukemia [CMML]) disease. Lymphoma is classified according to sensitivity to prior chemotherapy (chemosensitive or chemoresistant).
The classification of conditioning regimen intensity is based on the agents, doses and schedules used. Several classification systems are available, and for this report we used a composite classification. Cases defined as reduced-intensity by the transplant center were classified as such. Cases without such information and with available data on chemotherapy agents, radiation and doses, were classified according to the CIBMTR operational definition of conditioning regimen intensity:
 Myeloablative conditioning regimen: regimens with total body irradiation doses of ≥500 cGY, single fractionated doses of ≥800 cGY, busulfan doses of >9mg/kg, or melphalan doses of >150 mg/m2 given as single agents or in combination with other drugs.
Reduced-intensity conditioning regimen: regimens with lower doses of total body irradiation, fractionated radiation therapy, busulfan, and melphalan than those used to define a myeloablative conditioning regimen (above).
Pasquini MC, Zhu X. Current use and outcome of hematopoietic stem cell transplantation: CIBMTR summary slides, Available at:

2. Location of Centers Participating in the CIBMTR 2015

3. Annual Number of Transplant Recipients in the US by Transplant Type
Slide 3: Estimated annual numbers of transplant recipients in the U.S. were compiled according to the number first transplants registered with CIBMTR. Estimates of how closely the numbers reported are representative of actual transplant activity vary according to the type of transplant and number of centers reporting data per year. Prior to 2007, all except unrelated donor allogeneic transplant facilitated by the NMDP were reported voluntarily. It was estimated that the CIBMTR captured 90% of all unrelated donor transplants performed in the US, 60-90% of related donor allogeneic transplants and 65 to 75% of autologous transplants. These estimates were extrapolated from other databases that capture transplant center activity, accreditation or hospital discharges. After 2007, the Stem Cell Transplant Outcomes Database (SCTOD) was initiated which changed reporting requirements and data capture to an electronic format. The SCTOD requires that all allogeneic transplants performed in the US be registered with CIBMTR. Data reporting of autologous transplants remains voluntary and the numbers in the CIBMTR database are estimated to be 80%. US numbers of allogeneic transplants in the CIBMTR are representative of the actual transplant activity.
The number of autologous transplants recipients in the U.S. continue to increase, most prominently since 2010 , mainly for treatment of plasma cell and lymphoproliferative disorders extending to older patients. The annual number of allogeneic transplants recipients is also increasing surpassing 8,000 a year in the US.

4. Allogeneic Transplant Recipients in the US, by Donor Type
Slide 4: By further stratifying the number of allogeneic transplants recipients in the US by donor type, recipients of unrelated donor transplants represent the largest group. The number of unrelated donor transplant has surpassed the number of allogeneic transplants from related donors after 2006 and the gap between these two types of approaches continues to widen annually. Transplants performed with other alternative donors is also increasing. From 2003 to 2011, there were a steady increase in the numbers of transplants using umbilical cord blood as a result of several published studies demonstrating its benefit in both children and adults. From 2012 onward, there was an increase in the numbers of transplants from other relatives, which is likely due to rapid dissemination of using haploidentical donors with the post transplant cyclophosphamide strategy. In 2014, the numbers of transplants using other relatives surpassed the total numbers of umbilical cord transplant performed in the US, accounting for 11% of all allogeneic transplants performed in the US.

5. Cumulative Plot of Transplant Recipients in the US by Transplant Type
Slide 5: Cumulative number of transplant recipients in the US reached 340,000 in 2014.

6. HLA-Match Sibling Donor Allogeneic Transplants in Patients ≤20 Years
Slide 6: Annual numbers for HLA-match sibling transplants in pediatric population demonstrates a greater use of bone marrow grafts. Over time, the use of mobilized peripheral blood stem cells have decreased in the pediatric population to 20% in 2013.

7. Unrelated Donor Allogeneic Transplants in Patients ≤20 Years
Slide 7: Annual numbers for allogeneic transplants in pediatric population demonstrates increase in the overall utilization of alternative donors. Bone marrow remains the most common graft source for unrelated donors accounting for 49% in The use of umbilical cord blood is declining steadily after if peaked in 2009, from 46% to 32% of all unrelated donor transplants performed in this age group.

8. HLA-Match Sibling Donor Allogeneic Transplants in Patients Age >20 years
Slide 8: Among adult recipients of HLA-match sibling transplant, mobilized peripheral blood stem cells are the most common graft type. The proportion use of bone marrow grafts for sibling transplants ranged from 11% to 14% since 2006.

9. Unrelated Donor Allogeneic Transplants in Patients Age >20 years
Slide 9: Among adult recipients of unrelated donor transplant, there has also been an increase in overall number of transplants. Mobilized peripheral blood stem cells is the predominant graft utilized in this setting, accounting for 77% of unrelated donor transplants in This practice continues to increase despite clinical trial results demonstrating increase late complications such as chronic graft-versus-host disease with this graft source. The number of umbilical cord transplants in adults are steady since 2010, accounting for 10% of alternative donor grafts used in adults.

10. Trends in Autologous Transplants by Recipient Age*
Slides 10 & 11: The number of autologous and allogeneic transplants for treatment of malignant diseases in older patients continue to increase. Fourty-four percent of autologous transplant recipients and 22% of allogeneic transplant recipients in were older than 60.

11. Trends in Allogeneic Transplants by Recipient Age*
Slides 10 & 11: The number of autologous and allogeneic transplants for treatment of malignant diseases in older patients continue to increase. Fourty-four percent of autologous transplant recipients and 22% of allogeneic transplant recipients in were older than 60.

12. Indications for Hematopoietic Stem Cell Transplants in the US, 2013
Slide 12: The most common indications for HCT in the US in 2013 were multiple myeloma and lymphoma, accounting for 52% of all HCTs. AML and myelodysplasia are the most common indications for allogeneic transplants accounting for 53% of allogeneic HCTs.

13. Causes of Death after Autologous Transplants done in 2012-2013
Slides 13-15: After an autologous transplant, primary disease is the most commonly reported cause of death. Among allogeneic transplant recipients, unrelated donor transplants have fewer deaths related to the primary disease, however organ failure and infections are higher after unrelated donor transplants.

14. Causes of Death after HLA Match Sibling Transplants done in 2012-2013
Slides 13-15: After an autologous transplant, primary disease is the most commonly reported cause of death. Among allogeneic transplant recipients, unrelated donor transplants have fewer deaths related to the primary disease, however organ failure and infections are higher after unrelated donor transplants.

15. Causes of Death after Unrelated Donor Transplants done in 2012-2013
Slides 13-15: After an autologous transplant, primary disease is the most commonly reported cause of death. Among allogeneic transplant recipients, unrelated donor transplants have fewer deaths related to the primary disease, however organ failure and infections are higher after unrelated donor transplants.

16. Allogeneic Transplants Registered with the CIBMTR
Slide 16: The overall number of transplants utilizing reduced intensity conditioning steadily increase from 2003 to 2012, peaking at 42% of all allogeneic transplants. In 2013, 40% of allogeneic transplants used reduce intensity conditioning.

17. Survival after HLA Match Sibling Donor Transplants for AML, 2003-2013
Slides 17 & 18: The CIBMTR has data for 30,955 patients receiving an HLA-matched sibling (n=13,016) or unrelated donor (n=17,939) transplant for AML between 2003 and Their disease status at the time of transplant and the donor type are the major predictors of post-transplant survival. The 3-year probabilities of survival after HLA-matched sibling transplant in this cohort was 59% ± 1%, 51% ± 1%, and 27% ± 1% for patients with early, intermediate, and advanced disease, respectively. The probabilities of survival after an unrelated donor transplant were 50% ± 1%, 47%± 1%, and 24% ± 1% for patients with early, intermediate, and advanced disease, respectively.

18. Survival after Unrelated Donor Transplants for AML, 2003-2013
Slides 17 & 18: The CIBMTR has data for 30,955 patients receiving an HLA-matched sibling (n=13,016) or unrelated donor (n=17,939) transplant for AML between 2003 and Their disease status at the time of transplant and the donor type are the major predictors of post-transplant survival. The 3-year probabilities of survival after HLA-matched sibling transplant in this cohort was 59% ± 1%, 51% ± 1%, and 27% ± 1% for patients with early, intermediate, and advanced disease, respectively. The probabilities of survival after an unrelated donor transplant were 50% ± 1%, 47%± 1%, and 24% ± 1% for patients with early, intermediate, and advanced disease, respectively.

19. Survival after HLA Match Sibling Donor Transplants for AML, Age <20 Years, 2003-2013
Slide 19: Among 1,724 patients younger than 20 with AML between 2003 and 2013, the 3-year probabilities of survival following transplant for early, intermediate, and advanced disease were 69% ± 1%, 58% ± 3%, and 34% ± 3%, respectively.

20. Survival after Allogeneic Transplants for Myelodysplastic Syndrome (MDS), 2003-2013
Slides 20 & 21: Allogeneic transplant is a potentially curative treatment for myelodysplastic syndrome (MDS). Outcomes differ according to disease status at the time of transplant. The CIBMTR has data on 6,461 patients receiving an allotransplants for early (n=2,586) and advanced (n=3,875) MDS. The 3-year probabilities of survival were 53% ± 2% and 44% ± 1% for recipients of sibling and unrelated donor transplants for early MDS, respectively. Among patients with advanced MDS, corresponding probabilities were 49% ± 1% and 39% ± 1%.

21. Survival after Allogeneic Transplants for Myelodysplastic Syndrome (MDS), 2003-2013
Slides 20 & 21: Allogeneic transplant is a potentially curative treatment for myelodysplastic syndrome (MDS). Outcomes differ according to disease status at the time of transplant. The CIBMTR has data on 6,461 patients receiving an allotransplants for early (n=2,586) and advanced (n=3,875) MDS. The 3-year probabilities of survival were 53% ± 2% and 44% ± 1% for recipients of sibling and unrelated donor transplants for early MDS, respectively. Among patients with advanced MDS, corresponding probabilities were 49% ± 1% and 39% ± 1%.

22. Survival after HLA Match Sibling Donor Transplants for Myeloproliferative Diseases, 2003-2013
Slides 22 & 23: Allogeneic transplants are a treatment choice for patients with myeloproliferative disease whom have high risk features. The CIBMTR has data for 2,824 patients receiving an HLA-matched sibling (n=1,284) or unrelated donor (n=1,540) transplant for myeloproliferative diseases between 2003 and The 3-year probabilities of survival were 56% ± 2% and 50% ± 1% for HLA-match sibling donor recipients with myelofibrosis and other myeloproliferative diseases. Among unrelated donor recipients with myeloproliferative diseases, corresponding probabilities were 51% ± 2% and 43% ± 2%.

23. Survival after Unrelated Donor Transplants for Myeloproliferative Diseases, 2003-2013
Slides 22 & 23: Allogeneic transplants are a treatment choice for patients with myeloproliferative disease whom have high risk features. The CIBMTR has data for 2,824 patients receiving an HLA-matched sibling (n=1,284) or unrelated donor (n=1,540) transplant for myeloproliferative diseases between 2003 and The 3-year probabilities of survival were 56% ± 2% and 50% ± 1% for HLA-match sibling donor recipients with myelofibrosis and other myeloproliferative diseases. Among unrelated donor recipients with myeloproliferative diseases, corresponding probabilities were 51% ± 2% and 43% ± 2%.

24. Survival after HLA Match Sibling Donor Transplants for ALL, Age <20 Years, 2003-2013
Slides 24 & 25: Among young patients with ALL, for whom chemotherapy has a high success rate, allogeneic transplantation is generally reserved for patients with high-risk disease (i.e. high leukocyte count at diagnosis and the presence of poor-risk cytogenetic markers), who fail to achieve remission or who relapse after chemotherapy. Among the 2,036 patients younger than 20 receiving an HLA-matched sibling transplant for ALL between 2003 to 2013, the 3-year probabilities of survival were 68% ± 2%, 56% ± 2 %, and 30% ± 4% for patients with early, intermediate, and advanced disease, respectively. The corresponding probabilities of survival among the 3,259 recipients of an unrelated donor transplant were 67% ± 2%, 52% ± 1%, and 36% ± 3%.

25. Survival after Unrelated Donor Transplants for ALL, Age <20 years, 2003-2013
Slides 24 & 25: Among young patients with ALL, for whom chemotherapy has a high success rate, allogeneic transplantation is generally reserved for patients with high-risk disease (i.e. high leukocyte count at diagnosis and the presence of poor-risk cytogenetic markers), who fail to achieve remission or who relapse after chemotherapy. Among the 2,036 patients younger than 20 receiving an HLA-matched sibling transplant for ALL between 2003 to 2013, the 3-year probabilities of survival were 68% ± 2%, 56% ± 2 %, and 30% ± 4% for patients with early, intermediate, and advanced disease, respectively. The corresponding probabilities of survival among the 3,259 recipients of an unrelated donor transplant were 67% ± 2%, 52% ± 1%, and 36% ± 3%.

26. Survival after HLA Match Sibling Donor Transplants for ALL, Age ≥20 Years, 2003-2013
Slides 26 & 27: Older age at disease onset is a high-risk feature in ALL. Consequently, a larger proportion of ALL patients 20 years of age or older undergo allogeneic HCT for early disease. Among 3,884 patients ≥20 years of age receiving HLA-matched sibling HCT for ALL between , the 3-year survival probabilities were 56% ± 1%, 36% ± 2%, and 27% ± 2% for patients with early, intermediate, and advanced disease, respectively. Corresponding probabilities among the 4,618 recipients of unrelated donor HCT were 55% ± 1%, 36% ± 2%, and 21% ± 2%.

27. Survival after Unrelated Donor Transplants for ALL, ≥20 Years, 2003-2013
Slides 26 & 27: Older age at disease onset is a high-risk feature in ALL. Consequently, a larger proportion of ALL patients 20 years of age or older undergo allogeneic HCT for early disease. Among 3,884 patients ≥20 years of age receiving HLA-matched sibling HCT for ALL between , the 3-year survival probabilities were 56% ± 1%, 36% ± 2%, and 27% ± 2% for patients with early, intermediate, and advanced disease, respectively. Corresponding probabilities among the 4,618 recipients of unrelated donor HCT were 55% ± 1%, 36% ± 2%, and 21% ± 2%.

28. Survival after HLA Match Sibling Transplants for CML, 2003-2013
Slide 28: Allogeneic transplants for CML are currently reserved for patients who failed or poorly tolerate tyrosine kinase inhibitors. The CIBMTR has data for 2,346 HLA-matched sibling donor allotransplants for CML from 2003 to Three-year probabilities of survival were 68% ± 1%, 53% ± 3% and 24% ± 4% for patients in chronic phase (CP), in accelerated phase (AP) and blastic phase; respectively.

29. Survival after Allogeneic Transplants for Chronic Lymphocytic Leukemia (CLL), 2003-2013
Slide 29: Allogeneic transplants are the main transplant modality for treatment of chronic lymphocytic leukemia (CLL) patients who fail standard chemotherapy or have high-risk features (e.g. cytogenetic abnormalities, short remission intervals, purine analog resistant disease). Among the 2,979 patients who underwent transplantation for CLL, the 3-year probabilities of survival were 57% ± 1% and 48% ± 1% after transplants with HLA Match sibling and unrelated donors, respectively.

30. Survival after Allogeneic Transplants for Severe Aplastic Anemia, <20 Years, 2003-2013
Slides 30 & 31: Allogeneic HCT is the treatment of choice for young patients with severe aplastic anemia and available HLA-matched sibling donor. Among the 2,670 patients receiving HLA-matched sibling donor HCT for severe aplastic anemia between 2003 and 2013, the 3-year probabilities of survival were 89% ±1% for those younger than 20 years and 76% ± 1% for those 20 years of age or older. Among the 1515 recipients of unrelated donor HCT during the same period, the corresponding probabilities of survival were 75% ± 2% and 65% ± 2%.

31. Survival after Allogeneic Transplants for Severe Aplastic Anemia, ≥20 Years, 2003-2013
Slides 30 & 31: Allogeneic HCT is the treatment of choice for young patients with severe aplastic anemia and available HLA-matched sibling donor. Among the 2,670 patients receiving HLA-matched sibling donor HCT for severe aplastic anemia between 2003 and 2013, the 3-year probabilities of survival were 89% ±1% for those younger than 20 years and 76% ± 1% for those 20 years of age or older. Among the 1515 recipients of unrelated donor HCT during the same period, the corresponding probabilities of survival were 75% ± 2% and 65% ± 2%.

32. Survival after Autologous Transplants for Hodgkin Lymphoma, 2003-2013
Slide 32: Transplantation for Hodgkin Lymphoma (HL) is indicated in patients who have failed initial chemotherapy or radiation therapy. Survival after HCT for HL depends on disease response to previous salvage therapy. Among the 8,631 patients receiving autotransplants for HL between 2003 and 2013, the 3-year probabilities of survival were 81% ± 1% and 64% ± 2% for patients with chemosensitive and with chemoresistant disease, respectively.

33. Survival after Allogeneic Transplants for Hodgkin Lymphoma, 2003-2013
Slide 33: Allogeneic HCT for HL is generally performed in patients who experience disease relapse after receiving multiple lines of therapy including autotransplant or who have refractory disease and an available HLA-matched donor. Among 2,105 patients receiving allotransplants for HL between 2003 and 2013, the 3-year probabilities of survival were 54% ± 2% and 48% ± 2% after sibling and unrelated donor transplants, respectively.

34. Survival after Autologous Transplants for Follicular Lymphoma, 2003-2013
Slide 34: Transplantation for follicular lymphoma (FL) is generally reserved for patients with recurrent or aggressive disease. Autotransplantation is most commonly done in patients with chemosensitive disease. Among the 2,798 patients receiving an autotransplant for FL between 2003 and The 3-year probabilities of survival were 78% ± 1% and 62% ± 4% for patients with chemosensitive and chemoresistant disease, respectively.

35. Survival after Allogeneic Transplants for Follicular Lymphoma, 2003-2013
Slide 35: Allogeneic transplants for FL are performed in patients who experience disease relapse after multiple lines of therapy or who have refractory disease and an available HLA match donor. Among 1,079 patients receiving HLA-matched sibling donor transplants for FL between 2003 and 2013, the 3-year probabilities of survival were 71% ± 2% and 58% ± 4% for patients with chemosensitive and chemoresistant disease, respectively. Corresponding probabilities among 901 patients receiving unrelated donor transplants in the same period were 64% ± 2% and 41% ± 4%.

36. Survival after Autologous Transplants for Diffuse Large B-cell Lymphoma (DLBCL), 2003-2013
Slides 36 & 37: Autotransplants are an accepted treatment indication for diffuse large B-cell lymphoma (DLBCL) and, similar to FL, most autotransplants are performed in patients with chemosensitive disease. Among the 11,356 patients who received an autotransplant for DLBCL between 2003 and 2013, the 3-year probabilities of survival were 64% ± 1% and 42% ± 2% for patients with chemosensitive and chemoresistant disease, respectively. Allogeneic HCT for treatment of DLBCL is performed less frequently than for FL and is generally used only in patients with aggressive disease that has been resistant to previous therapies, including autotransplants. Among the 1,040 patients who underwent an HLA-matched sibling HCT for DLBCL from 2003 to 2013, the 3-year probabilities of survival were 49% ± 2% and 25% ± 3% for patients with chemosensitive and chemoresistant disease, respectively.

37. Survival after HLA Match Sibling Transplants for Diffuse Large B-cell Lymphoma (DLBCL), 2003-2013
Slides 36 & 37: Autotransplants are an accepted treatment indication for diffuse large B-cell lymphoma (DLBCL) and, similar to FL, most autotransplants are performed in patients with chemosensitive disease. Among the 11,356 patients who received an autotransplant for DLBCL between 2003 and 2013, the 3-year probabilities of survival were 64% ± 1% and 42% ± 2% for patients with chemosensitive and chemoresistant disease, respectively. Allogeneic HCT for treatment of DLBCL is performed less frequently than for FL and is generally used only in patients with aggressive disease that has been resistant to previous therapies, including autotransplants. Among the 1,040 patients who underwent an HLA-matched sibling HCT for DLBCL from 2003 to 2013, the 3-year probabilities of survival were 49% ± 2% and 25% ± 3% for patients with chemosensitive and chemoresistant disease, respectively.

38. Survival after Transplants for Mantle Cell Lymphoma, 2003-2013
Slide 38: The optimal timing and type of HCT for mantle cell lymphoma (MCL) is not well defined. As with other mature B-cell lymphoproliferative disorders, autotransplantation is the most common transplant approach. Among the 4,360 patients who received an autotransplant for MCL between 2003 and 2013, the 3-year probability of survival was 78% ± 1%. Among 1,414 patients who underwent an allogeneic transplantation for MCL during the same period, the 3-year probabilities of survival was 52% ± 1% .

39. Survival after Transplants for Multiple Myeloma, 2003-2013
Slide 39: Multiple myeloma (MM) is the most common indication for autologous HCT. Among 37,285 patients who received an autotransplant for MM between 2003 and 2013, the 3-year probability of survival was 75% ± 1%. Allogeneic transplantation for MM is reserved for patients with high risk disease and the majority performed after an autologous HCT with reduced intensity or nonmyeloablative conditioning regimens. Among the 1,012patients who received an allogeneic HCT from 2003 to 2013, the 3-year probabilities of survival was 52% ± 2%.

40. Survival after Autologous Transplants for Multiple Myeloma, 2000-2013
Slide 40: Outcomes after autotransplants with the addition of novel agents and combinations for the treatment of multiple myeloma have significant impact survival in the last decade. The 3 year overall survival among autotransplant recipients for multiple myeloma were 68% ± 1%, 73% ± 1% and 77% ± 1% of the transplants were performed in 2000 to 2003, 2004 to 2007 and 2008 to 2011, respectively.