Current Uses and Outcomes of Hematopoietic Stem Cell

Current Uses and Outcomes of Hematopoietic Stem Cell Transplantation 2010 CIBMTR Summary Slides SUM 10_1. ppt

Location of transplant centers participating in CIBMTR 2008 . . . . . . . . . Slide 2 SUM 10_2. ppt

Transplant activity worldwide 1980 -2009 35, 000 Autologous 30, 000 Allogeneic Transplants 25, 000 20, 000 15, 000 10, 000 5, 000 0 '80'81'82'83'84'85'86'87'88'89'90'91'92'93'94'95'96'97'98'99'00'01'02'03'04'05'06'07'08'09 Slide 3 SUM 10_3. ppt

Transplant activity in the U. S. 1980 -2009 18, 000 16, 000 Transplants 14, 000 Autologous Related Donor Unrelated Donor 12, 000 10, 000 8, 000 6, 000 4, 000 2, 000 0 '80'81'82'83'84'85'86'87'88'89'90'91'92'93'94'95'96'97'98'99'00'01'02'03'04'05'06'07'08'09 Slide 4 SUM 10_4. ppt

Allogeneic stem cell sources, by recipient age 1999 -2008 100 Bone Marrow (BM) Peripheral Blood (PB) Transplants, % 80 Cord Blood (CB) 60 40 20 0 1999 -2003 2004 -2008 Age £ 20 yrs 1999 -2003 2004 -2008 Age >20 yrs Slide 5 SUM 10_7. ppt

Autologous stem cell sources, by recipient age 1999 -2008 100 Bone Marrow (BM) Peripheral Blood (PB) BM + PB Transplants, % 80 60 40 20 0 1999 -2003 2004 -2008 Age £ 20 yrs 1999 -2003 2004 -2008 Age >20 yrs Slide 6 SUM 10_8. ppt

Trends in transplantation, by transplant type and recipient age* 1999 -2008 100 Transplants, % 80 £ 20 yrs 21 -40 yrs 41 -50 yrs 51 -60 yrs > 60 yrs 60 40 20 0 1999 -2003 2004 -2008 Allogeneic Transplants 1999 -2003 2004 -2008 Autologous Transplants * Transplants for AML, ALL, NHL, Hodgkin Disease, Multiple Myeloma Slide 7 SUM 10_9. ppt

Trends in transplantation, by transplant type and recipient age* 1999 -2008 100 < 50 years ³ 50 years < 60 years ³ 60 years Transplants, % 80 60 40 20 0 1988 -1994 1995 -2001 2002 -2008 Allogeneic Transplants Autologous Transplants * Transplants for AML, ALL, NHL, Hodgkin Disease, Multiple Myeloma Slide 8 SUM 10_29. ppt

Indications for hematopoietic stem cell transplant in North America 2008 5, 500 Allogeneic (Total N=6, 672) Number of Transplants 5, 000 Autologous (Total N=10, 302) 4, 500 4, 000 3, 500 3, 000 2, 500 2, 000 1, 500 1, 000 500 0 Multiple NHL Myeloma AML HD ALL MDS/MPD Aplastic CML Anemia Other Non. Leuk Cancer Malig Disease Slide 9 SUM 10_11. ppt

Indications for hematopoietic stem cell transplant for ages £ 20 years in North America 2008 800 Allogeneic (Total N=1, 496) Number of Transplants 700 Autologous (Total N=880) 600 500 400 300 200 100 0 Other Cancer ALL AML Aplastic Anemia HD NHL MDS/MPD CML Other Leuk Non. Malig Disease Slide 10 SUM 10_12. ppt

Indications for allogeneic hematopoietic stem cell transplantation in North America 2008 3, 000 Number of Transplants Related Donor (Total N=3, 282) Unrelated Donor (Total N=3, 389) 2, 500 2, 000 1, 500 1, 000 500 0 AML ALL MDS/MPD NHL Aplastic CML Multiple HD Anemia Myeloma Other Non. Leuk Cancer Malig Disease Slide 11 SUM 10_13. ppt

Allogeneic transplants in patients £ 20 years, by donor type and graft source, registered with CIBMTR, 1991 -2008 5, 000 Number of Transplants 4, 500 4, 000 Related, BM or PB Unrelated, CB 3, 500 3, 000 2, 500 2, 000 1, 500 1, 000 500 0 1991 -92 1993 -94 1995 -96 1997 -98 1999 -00 2001 -02 2003 -04 2005 -06 2007 -08* * Data incomplete Slide 12 SUM 10_14. ppt

Allogeneic transplant in patients >20 years, by donor type and graft source, registered with CIBMTR, 1991 -2008 Number of Transplants 13, 000 12, 000 11, 000 10, 000 9, 000 8, 000 7, 000 6, 000 5, 000 4, 000 3, 000 2, 000 1, 000 0 Related, BM or PB Unrelated, BM Unrelated, PB Unrelated, CB 1991 -92 1993 -94 1995 -96 1997 -98 1999 -00 2001 -02 2003 -04 2005 -06 2007 -08* * Data incomplete Slide 13 SUM 10_15. ppt

Unrelated donor stem cell sources by recipient age 1999 -2008 100 Bone Marrow (BM) Peripheral Blood (PB) Transplants, % 80 Cord Blood (CB) 60 40 20 0 1999 -2003 2004 -2008 Age £ 20 yrs 1999 -2003 2004 -2008 Age >20 yrs Slide 14 SUM 10_16. ppt

Unrelated cord blood transplantation registered with CIBMTR 1999 -2008 800 ³ 16 years Number of Transplants 700 < 16 years 600 500 400 300 200 100 0 1999 2000 2001 2002 2003 2004 2005 2006 2007* 2008* * Data incomplete Slide 15 SUM 10_17. ppt

One-year survival after myeloablative conditioning for acute leukemias in any remission phase, CML or MDS, age <50 years, by year of transplant and graft source, 1988 -2008 100 Sibling Donor One-Year Survival, % Unrelated Donor 80 60 40 20 0 1988 -90199119921993199419951996199719981999200020012002200320042005200620072008 Slide 16 SUM 10_18. ppt

100 -day mortality after autologous transplantation 2007 -2008 15 Early Disease Intermediate/Advance Disease 12 Sensitive Mortality, % Resistant Other 9 6 3 0 Acute Leukemia Non-Hodgkin Lymphoma Hodgkin Disease Multiple Myeloma Slide 17 SUM 10_19. ppt

100 -day mortality after HLA-identical sibling transplantation 2007 -2008 100 Early Disease Intermediate Disease 80 Advance Disease Mortality, % Chronic Phase Accelerated Phase 60 Blast Phase Other 40 20 0 AML ALL CML MDS/MPS Aplastic Anemia Immune Deficiency Slide 18 SUM 10_20. ppt

100 -day mortality after unrelated donor transplantation 2007 -2008 100 Early Disease Intermediate Disease 80 Advance Disease Mortality, % Chronic Phase Accelerated Phase 60 Blast Phase Other 40 20 0 AML ALL CML MDS/MPS Aplastic Anemia Immune Deficiency Slide 19 SUM 10_21. ppt

Autologous Causes of death after transplantations done in 2003 -2008 Primary Disease (73%) IPn*(1%) Infection (5%) Organ Failure (4%) Other Cause (17% HLA-identical sibling Primary Disease (43%) GVHD (10%) Unrelated donor IPn*(3%) Primary Disease (35%) Infection (14%) GVHD (12%) IPn*(5%) Organ Failure (8%) Other (22%) Infection (17%) Other Cause (19%) *IPn = Idiopathic pneumonia syndrome Organ Failure (12%) Slide 20 SUM 10_22. ppt

Allogeneic transplantations by conditioning regimen intensity and patient age, registered with CIBMTR 1999 -2008 11, 000 Number of Transplants 10, 000 9, 000 Reduced Intensity Conditioning, Age ³ 50 years Reduced Intensity Conditioning, Age < 50 years Standard Myeloablative Conditioning 8, 000 7, 000 6, 000 5, 000 4, 000 3, 000 2, 000 1, 000 0 1999 2000 2001 2002 2003 2004 2005 2006 2007* 2008* * Data incomplete Slide 21 SUM 10_23. ppt

Allogeneic transplantations using reducedintensity conditioning, by donor type, registered with CIBMTR 1998 -2008 3, 000 Number of Transplants Unrelated CB 2, 500 Unrelated PB/BM Related 2, 000 1, 500 1, 000 500 0 1999 2000 2001 2002 2003 2004 2005 2006 2007* 2008* * Data incomplete Slide 22 SUM 10_24. ppt

Number of allogeneic transplants, by disease, registered with CIBMTR 1998 -2008 3, 000 Transplants 2, 500 AML ALL CML AA LYM / MM / CLL 2, 000 1, 500 1, 000 500 0 1999 2000 2001 2002 2003 2004 2005 2006 2007* 2008* * Data incomplete Slide 23 SUM 10_25. ppt

Number of allogeneic transplants for AML, by disease status, registered with CIBMTR 1998 -2008 1, 400 Early Disease 1, 200 Intermediate Disease Advanced Disease Transplants 1, 000 800 600 400 200 0 1999 2000 2001 2002 2003 2004 2005 2006 2007* 2008* * Data incomplete Slide 24 SUM 10_26. ppt

Number of allogeneic transplantations for ALL, by disease status, registered with CIBMTR 1998 -2008 1, 000 Early Disease Intermediate Disease Transplants 800 Advanced Disease 600 400 200 0 1999 2000 2001 2002 2003 2004 2005 2006 2007* 2008* * Data incomplete Slide 25 SUM 10_27. ppt

Probability of survival after HLA-matched sibling donor transplant for AML, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 Early (N=6, 898) 60 60 50 50 40 40 Intermediate (N=1, 805) 30 30 20 20 Advanced (N=3, 011) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 26 SUM 10_32. ppt

Probability of survival after unrelated donor transplant for AML, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 Early (N=3, 658) 60 60 50 50 40 40 Intermediate (N=2, 774) 30 30 20 20 Advanced (N=3, 242) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 27 SUM 10_33. ppt

Probability of survival after HLA-matched sibling donor transplant for AML, age <20 years, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 Early (N=1, 384) 60 60 50 50 Intermediate (N=285) 40 40 30 30 Advanced (N=309) 20 20 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 28 SUM 09_34. ppt

Probability of survival after HLA-matched sibling donor transplant for AML with reduced-intensity conditioning, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 60 60 50 50 Early (N=1, 129) 40 40 30 30 Intermediate (N=377) 20 20 10 P < 0. 0001 0 0 10 Advanced (N=571) 0 1 2 3 Years 4 5 6 Slide 29 SUM 10_35. ppt

Probability of survival after unrelated donor transplant for AML with reduced-intensity conditioning, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 60 60 50 50 Early (N=951) 40 40 30 30 Intermediate (N=585) 20 20 Advanced (N=728) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 30 SUM 10_36. ppt

Probability of survival after autologous transplant for AML, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 60 60 50 50 Early (N=2, 319) 40 40 30 30 Intermediate (N=769) 20 20 10 P < 0. 0001 0 0 10 Advanced (N=221) 0 1 2 3 Years 4 5 6 Slide 31 SUM 10_38. ppt

Probability of survival after allogeneic transplant for MDS, age <20 years, by disease status and donor type, 1998 -2008 100 Probability of Survival, % 90 90 Early, unrelated (N=145) 80 70 Early, HLA-matched sibling (N=63) 60 60 Advanced, HLA-matched sibling (N=114) 50 50 40 40 Advanced, unrelated (N=190) 30 30 20 20 10 10 P = 0. 002 0 0 0 1 2 3 Years 4 5 6 Slide 32 SUM 10_39. ppt

Probability of survival after allogeneic transplant for MDS, age ³ 20 years, by disease status and donor type, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 Early, unrelated (N=509) 60 60 Early, HLA-matched sibling (N=599) 50 50 40 40 30 30 Advanced, unrelated (N=1, 142) 20 20 Advanced, HLA-matched sibling (N=1, 237) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 33 SUM 10_40. ppt

Probability of survival after allogeneic transplant for MDS with reduced-intensity conditioning, by disease status and donor type, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 Advanced, HLA-matched sibling (N=366) 60 60 Early, HLA-matched sibling (N=217) 50 50 Early, unrelated (N=202) 40 40 30 30 20 20 Advanced, unrelated (N=383) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 34 SUM 10_41. ppt

Probability of survival after HLA-matched sibling donor transplant for ALL, age <20 years, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 Early (N=915) 60 60 50 50 Intermediate (N=1, 313) 40 40 30 30 20 20 Advanced (N=243) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 35 SUM 10_42. ppt

Probability of survival after unrelated donor transplant for ALL, age <20 years, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 60 60 Early (N=831) 50 50 40 40 Intermediate (N=2, 041) 30 20 Advanced (N=340) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 36 SUM 10_43. ppt

Probability of survival after HLA-matched sibling donor transplant for ALL, age ³ 20 years, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 60 60 50 50 Early (N=1, 993) 40 40 Intermediate (N=700) 30 30 20 20 Advanced (N=716) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 37 SUM 10_44. ppt

Probability of survival after unrelated donor transplant for ALL, age ³ 20 years, by disease status, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 60 60 50 50 Early (N=1, 363) 40 40 30 30 Intermediate (N=934) 20 10 Advanced (N=784) P < 0. 0001 0 20 0 1 2 3 Years 4 5 6 Slide 38 SUM 09_45. ppt

Probability of survival after HLA-matched sibling donor transplant for CML, by disease status and transplant year, 1998 -2008 Probability of Survival, % 100 90 90 80 80 CP, 2001 -2008 (N=2, 412) 70 60 CP, 1998 -2000 (N=2, 302) 50 50 AP, 2001 -2008 (N=314) 40 40 AP, 1998 -2000 (N=301) 30 30 20 20 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 39 SUM 10_46. ppt

Probability of survival after autologous and HLA-matched sibling donor transplants for CLL, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 autologous transplant (N=399) 60 60 50 50 Myeloablative conditioning(N=458) 40 30 Reduced-intensity conditioning (N=679) 20 20 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 40 SUM 10_47. ppt

Probability of survival after allogeneic transplant for severe aplastic anemia, by donor type and age, 1998 -2008 100 Probability of Survival, % 90 90 HLA-matched sibling, £ 20 y (N=1, 388) 80 80 HLA-matched sibling, >20 y (N=1, 408) 70 70 60 Unrelated, £ 20 y (N=562) 60 50 Unrelated, >20 y (N=532) 50 40 40 30 30 20 20 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 41 SUM 10_48. ppt

Probability of survival after autologous transplant for Hodgkin disease, by disease status, 1998 -2008 100 Probability of Survival, % 90 90 Complete remission (N=2, 437) 80 80 70 70 60 60 Not in CR, sensitive (N=3, 237) 50 40 Not in CR, resistant (N=699) 30 30 20 20 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 42 SUM 10_49. ppt

Probability of survival after allogeneic transplant for Hodgkin disease, by donor type and conditioning regimen, 1998 -2008 Probability of Survival, % 100 90 90 80 80 Unrelated, reduced-intensity conditioning (N=110) 70 70 HLA-matched sibling, myeloablative (N=190) 60 60 HLA-matched sibling, reduced-intensity conditioning (N=153) 50 50 40 40 30 30 20 20 Unrelated, myeloablative (N=71) 10 10 P = 0. 021 0 0 0 1 2 3 Years 4 5 6 Slide 43 SUM 10_50. ppt

Probability of survival after autologous transplant for follicular lymphoma, by disease status, 2000 -2008 Probability of Survival, % 100 90 90 80 80 Chemosensitive (N=1, 995) 70 70 60 60 50 50 Chemoresistant (N=160) 40 40 30 30 20 20 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 44 SUM 10_51. ppt

Probability of survival after HLA-matched sibling donor allogeneic transplant for follicular lymphoma, by disease status and conditioning regimen, 1998 -2008 100 Probability of Survival, % 90 90 Chemosensitive, reduced–intensity conditioning (N=388) 80 80 Chemosensitive, myeloablative (N=351) 70 70 60 60 Chemoresistant, reduced–intensity conditioning (N=64) 50 50 40 40 30 30 Chemoresistant, myeloablative (N=85) 20 20 10 10 P = 0. 011 0 0 0 1 2 3 Years 4 5 6 Slide 45 SUM 10_52. ppt

Probability of survival after autologous transplant for diffuse large B-cell lymphoma, by disease status, 2000 -2008 Probability of Survival, % 100 90 90 80 80 70 70 Chemosensitive (N=6, 203) 60 60 50 50 40 40 30 30 Chemoresistant (N=447) 20 20 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 46 SUM 10_53. ppt

Probability of survival after HLA-matched sibling donor allogeneic transplant for diffuse large B-cell lymphoma, by disease status and conditioning regimen, 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 60 60 50 50 Chemosensitive, reduced-intensity conditioning (N=122) 40 40 30 30 Chemosensitive, myeloablative (N=315) 20 20 Chemoresistant, reduced-intensity conditioning (N=43) 10 P < 0. 0001 0 0 10 Chemoresistant, myeloablative (N=98) 0 1 2 3 Years 4 5 6 Slide 47 SUM 10_54. ppt

Probability of survival after transplant for mantle cell lymphoma, by donor type and conditioning regimen, 1998 -2008 100 Probability of Survival, % 90 90 HLA-matched sibling, reduced-intensity conditioning (N=229) 80 80 HLA-matched sibling, myeloablative (N=284) 70 70 autologous transplant (N=2, 390) 60 60 50 50 40 40 Unrelated, myeloablative (N=94) 30 30 Unrelated, reduced-intensity conditioning (N=184) 20 20 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 48 SUM 10_55. ppt

Probability of survival after transplant for multiple myeloma, by donor type 1998 -2008 Probability of Survival, % 100 90 90 80 80 70 70 autologous transplant (N=22, 254) 60 60 50 50 40 40 HLA-matched sibling, Allo (N=878) 30 20 Unrelated, Allo (N=143) 10 10 P < 0. 0001 0 0 0 1 2 3 Years 4 5 6 Slide 49 SUM 10_56. ppt