Table 2.

Comparison of hematologic malignancy incidence for patients transplanted using our regimens vs other conditioning regimens

ConditioningNHLBI HLA matchedNHLBI haploidenticalGene therapyFrench groupCIBMTR
Alemtuzumab
300 cGy TBI
Pentostatin/Cy
alemtuzumab
300 cGy TBI
(Chicago, IL, and Riyadh, Saudi Arabia)
alemtuzumab
300 cGy TBI
Alemtuzumab
400 cGy TBI
± PT-Cy
Pentostatin/Cy
alemtuzumab
400 cGy TBI
PT-Cy
BusulfanCy
± ATG busulfan
Cy
± ATG busulfan (mostly)
No. enrolled in study 57 24 64 21  19  47 234 (79 with mixed chimerism long-term) 908  
TRMN (MDS, AML), No. (%) 1 (1.8) 1 (4.2) 1 (1.6)  3 (14.3) 2 (4.3) 2 (0.22)  
Hematologic malignancies (including TRMN), No. (%) 3 (5.3) 2 (8.3) 1 (1.6)  3 (14.3) 2 (4.3) 1 (0.4) 3 (0.33)  
Median time to hematologic malignancy development, y 3.5 1.7 N/A 4.3 
No. deceased from hematologic malignancies 
Graft status 1 Graft failure, 2 mixed chimerism 1 Graft failure, 1 mixed chimerism 1 Graft failure 3 Graft failure N/A Group A 
Median follow-up, y 9.1 4.0 8.4 2.6 7.9 2.1-3.9 
Therapeutic goal Mixed chimerism Mixed chimerism Mixed chimerism Mixed chimerism Mixed chimerism Gene-corrected autologous HSPCs Full donor chimerism Full donor chimerism 
ConditioningNHLBI HLA matchedNHLBI haploidenticalGene therapyFrench groupCIBMTR
Alemtuzumab
300 cGy TBI
Pentostatin/Cy
alemtuzumab
300 cGy TBI
(Chicago, IL, and Riyadh, Saudi Arabia)
alemtuzumab
300 cGy TBI
Alemtuzumab
400 cGy TBI
± PT-Cy
Pentostatin/Cy
alemtuzumab
400 cGy TBI
PT-Cy
BusulfanCy
± ATG busulfan
Cy
± ATG busulfan (mostly)
No. enrolled in study 57 24 64 21  19  47 234 (79 with mixed chimerism long-term) 908  
TRMN (MDS, AML), No. (%) 1 (1.8) 1 (4.2) 1 (1.6)  3 (14.3) 2 (4.3) 2 (0.22)  
Hematologic malignancies (including TRMN), No. (%) 3 (5.3) 2 (8.3) 1 (1.6)  3 (14.3) 2 (4.3) 1 (0.4) 3 (0.33)  
Median time to hematologic malignancy development, y 3.5 1.7 N/A 4.3 
No. deceased from hematologic malignancies 
Graft status 1 Graft failure, 2 mixed chimerism 1 Graft failure, 1 mixed chimerism 1 Graft failure 3 Graft failure N/A Group A 
Median follow-up, y 9.1 4.0 8.4 2.6 7.9 2.1-3.9 
Therapeutic goal Mixed chimerism Mixed chimerism Mixed chimerism Mixed chimerism Mixed chimerism Gene-corrected autologous HSPCs Full donor chimerism Full donor chimerism 

AML, acute myeloid leukemia; ATG, antithymocyte globulin; CIBMTR, Center for International Blood and Marrow Transplant Research; Cy, cyclophosphamide; HSPC, hematopoietic stem and progenitor cells; MDS, myelodysplastic syndrome; N/A, not applicable; NHLBI, National Heart, Lung, and Blood Institute; PT-Cy, posttransplant cyclophosphamide; TRMN: therapy-related myeloid neoplasm.

One patient was transplanted on study for both protocols.

Two patients transplanted at the NHLBI and who developed therapy-related myeloid neoplasms were included with the NHLBI studies and are not included herein.

Incidence of hematologic malignancies and median duration of follow-up are reported from the time of article publication.

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