Patient characteristics and outcomes
Characteristic . | Incidence, n (%) . |
---|---|
Age,y | |
0 > age < 1 | 22 (12) |
1 ≥ age < 5 | 49 (27) |
5 ≥ age < 13 | 58 (32) |
13 ≥ age < 20 | 52 (29) |
Sex | |
Male | 104 (57) |
Female | 77 (43) |
Race* | |
Northern European | 130 (72) |
African/North African | 13 (7) |
Multiracial/other | 12 (7) |
Asian/Southeast Asian | 10 (5.5) |
Middle Eastern/Persian/Turkish | 10 (5.5) |
Eastern European/Russian | 6 (3) |
HCT years | |
2005-2008 | 22 (12) |
2009-2012 | 66 (37) |
2013-2016 | 93 (51) |
HCT indication | |
Malignancy | 90 (50) |
Primary immunodeficiency | 37 (20) |
Metabolic/inborn error of metabolism | 29 (16) |
Bone marrow failure syndrome | 21 (12) |
Autoimmune disease | 4 (2) |
Allograft donor† | |
Mismatched UCB | 72 (40) |
Matched UCB | 45 (25) |
Matched sibling donor | 37 (20) |
Matched unrelated donor | 27 (15) |
Conditioning‡ | |
Myeloablative | 172 (95) |
Reduced intensity | 9 (5) |
Serotherapy | |
ATG or alemtuzumab | 125 (69) |
BALF routine microbiology§ | |
Any abnormality | 90 (50) |
Virus detected on PCR | 54 (30) |
Bacterial culture growth | 31 (17) |
Fungal culture growth | 22 (12) |
GM positivity | 21 (13) |
Post-HCT lung injury | |
Yes | 39 (22) |
Infection | 13 (7) |
IPS or unknown | 16 (9) |
Bronchiolitis obliterans | 10 (6) |
No | 142 (78) |
Post-HCT mortality | |
NRM | 39 (21) |
Due to lung injury | 17 (9) |
Not due to lung injury | 22 (12) |
Relapse mortality | 12 (7) |
Overall survival | 130 (72) |
Characteristic . | Incidence, n (%) . |
---|---|
Age,y | |
0 > age < 1 | 22 (12) |
1 ≥ age < 5 | 49 (27) |
5 ≥ age < 13 | 58 (32) |
13 ≥ age < 20 | 52 (29) |
Sex | |
Male | 104 (57) |
Female | 77 (43) |
Race* | |
Northern European | 130 (72) |
African/North African | 13 (7) |
Multiracial/other | 12 (7) |
Asian/Southeast Asian | 10 (5.5) |
Middle Eastern/Persian/Turkish | 10 (5.5) |
Eastern European/Russian | 6 (3) |
HCT years | |
2005-2008 | 22 (12) |
2009-2012 | 66 (37) |
2013-2016 | 93 (51) |
HCT indication | |
Malignancy | 90 (50) |
Primary immunodeficiency | 37 (20) |
Metabolic/inborn error of metabolism | 29 (16) |
Bone marrow failure syndrome | 21 (12) |
Autoimmune disease | 4 (2) |
Allograft donor† | |
Mismatched UCB | 72 (40) |
Matched UCB | 45 (25) |
Matched sibling donor | 37 (20) |
Matched unrelated donor | 27 (15) |
Conditioning‡ | |
Myeloablative | 172 (95) |
Reduced intensity | 9 (5) |
Serotherapy | |
ATG or alemtuzumab | 125 (69) |
BALF routine microbiology§ | |
Any abnormality | 90 (50) |
Virus detected on PCR | 54 (30) |
Bacterial culture growth | 31 (17) |
Fungal culture growth | 22 (12) |
GM positivity | 21 (13) |
Post-HCT lung injury | |
Yes | 39 (22) |
Infection | 13 (7) |
IPS or unknown | 16 (9) |
Bronchiolitis obliterans | 10 (6) |
No | 142 (78) |
Post-HCT mortality | |
NRM | 39 (21) |
Due to lung injury | 17 (9) |
Not due to lung injury | 22 (12) |
Relapse mortality | 12 (7) |
Overall survival | 130 (72) |
ATG, antithymocyte globulin; IPS, idiopathic pneumonia syndrome; UCB, umbilical cord blood.
Includes 1 Hispanic patient and 1 patient from Suriname.
The majority of non–UCB grafts were obtained from bone marrow (n = 62); only 2 were from peripheral blood. Includes 1 8/10 mismatched sibling donor and 1 9/10 mismatched unrelated donor. The majority of cytomegalovirus serostatuses were unknown/assumed positive because of the use of UCB.
Most myeloablative conditioning was busulfan/fludarabine (n = 82), busulfan/fludarabine/clofarabine (n = 47), or total body irradiation/etoposide (n = 13). The majority of reduced-intensity conditioning was fludarabine based.
No organisms were detected with other diagnostic tests, such as cytology for Pneumocystis carinii pneumonia or PCR for Mycoplasma or Legionella. Some patients had >1 abnormality.