TY - JOUR
T1 - Survival and Late Effects after Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancy at Less than Three Years of Age
AU - Vrooman, Lynda M.
AU - Millard, Heather R.
AU - Brazauskas, Ruta
AU - Majhail, Navneet S.
AU - Battiwalla, Minoo
AU - Flowers, Mary E.
AU - Savani, Bipin N.
AU - Akpek, Görgün
AU - Aljurf, Mahmoud
AU - Bajwa, Rajinder
AU - Baker, K. Scott
AU - Beitinjaneh, Amer
AU - Bitan, Menachem
AU - Buchbinder, David
AU - Chow, Eric
AU - Dandoy, Christopher
AU - Dietz, Andrew C.
AU - Diller, Lisa
AU - Gale, Robert Peter
AU - Hashmi, Shahrukh K.
AU - Hayashi, Robert J.
AU - Hematti, Peiman
AU - Kamble, Rammurti T.
AU - Kasow, Kimberly A.
AU - Kletzel, Morris
AU - Lazarus, Hillard M.
AU - Malone, Adriana K.
AU - Marks, David I.
AU - O'Brien, Tracey A.
AU - Olsson, Richard F.
AU - Ringden, Olle
AU - Seo, Sachiko
AU - Steinberg, Amir
AU - Yu, Lolie C.
AU - Warwick, Anne
AU - Shaw, Bronwen
AU - Duncan, Christine
N1 - Publisher Copyright:
© 2017 The American Society for Blood and Marrow Transplantation
PY - 2017/8
Y1 - 2017/8
N2 - Very young children undergoing hematopoietic cell transplantation (HCT) are a unique and vulnerable population. We analyzed outcomes of 717 patients from 117 centers who survived relapse free for ≥1 year after allogeneic myeloablative HCT for hematologic malignancy at <3 years of age, between 1987 and 2012. The median follow-up was 8.3 years (range, 1.0 to 26.4 years); median age at follow-up was 9 years (range, 2 to 29 years). Ten-year overall and relapse-free survival were 87% (95% confidence interval [CI], 85% to 90%) and 84% (95% CI, 81% to 87%). Ten-year cumulative incidence of relapse was 11% (95% CI, 9% to 13%). Of 84 deaths, relapse was the leading cause (43%). Chronic graft-versus-host-disease 1 year after HCT was associated with increased risk of mortality (hazard ratio [HR], 2.1; 95% CI, 1.3 to 3.3; P = .0018). Thirty percent of patients experienced ≥1 organ toxicity/late effect >1 year after HCT. The most frequent late effects included growth hormone deficiency/growth disturbance (10-year cumulative incidence, 23%; 95% CI, 19% to 28%), cataracts (18%; 95% CI, 15% to 22%), hypothyroidism (13%; 95% CI, 10% to 16%), gonadal dysfunction/infertility requiring hormone replacement (3%; 95% CI, 2% to 5%), and stroke/seizure (3%; 95% CI, 2% to 5%). Subsequent malignancy was reported in 3.6%. In multivariable analysis, total body irradiation (TBI) was predictive of increased risk of cataracts (HR, 17.2; 95% CI, 7.4 to 39.8; P < .001), growth deficiency (HR, 3.5; 95% CI, 2.2 to 5.5; P < .001), and hypothyroidism (HR, 5.3; 95% CI, 3.0 to 9.4; P < .001). In summary, those who survived relapse free ≥1 year after HCT for hematologic malignancy at <3 years of age had favorable overall survival. Chronic graft-versus-host-disease and TBI were associated with adverse outcomes. Future efforts should focus on reducing the risk of relapse and late effects after HCT at early age.
AB - Very young children undergoing hematopoietic cell transplantation (HCT) are a unique and vulnerable population. We analyzed outcomes of 717 patients from 117 centers who survived relapse free for ≥1 year after allogeneic myeloablative HCT for hematologic malignancy at <3 years of age, between 1987 and 2012. The median follow-up was 8.3 years (range, 1.0 to 26.4 years); median age at follow-up was 9 years (range, 2 to 29 years). Ten-year overall and relapse-free survival were 87% (95% confidence interval [CI], 85% to 90%) and 84% (95% CI, 81% to 87%). Ten-year cumulative incidence of relapse was 11% (95% CI, 9% to 13%). Of 84 deaths, relapse was the leading cause (43%). Chronic graft-versus-host-disease 1 year after HCT was associated with increased risk of mortality (hazard ratio [HR], 2.1; 95% CI, 1.3 to 3.3; P = .0018). Thirty percent of patients experienced ≥1 organ toxicity/late effect >1 year after HCT. The most frequent late effects included growth hormone deficiency/growth disturbance (10-year cumulative incidence, 23%; 95% CI, 19% to 28%), cataracts (18%; 95% CI, 15% to 22%), hypothyroidism (13%; 95% CI, 10% to 16%), gonadal dysfunction/infertility requiring hormone replacement (3%; 95% CI, 2% to 5%), and stroke/seizure (3%; 95% CI, 2% to 5%). Subsequent malignancy was reported in 3.6%. In multivariable analysis, total body irradiation (TBI) was predictive of increased risk of cataracts (HR, 17.2; 95% CI, 7.4 to 39.8; P < .001), growth deficiency (HR, 3.5; 95% CI, 2.2 to 5.5; P < .001), and hypothyroidism (HR, 5.3; 95% CI, 3.0 to 9.4; P < .001). In summary, those who survived relapse free ≥1 year after HCT for hematologic malignancy at <3 years of age had favorable overall survival. Chronic graft-versus-host-disease and TBI were associated with adverse outcomes. Future efforts should focus on reducing the risk of relapse and late effects after HCT at early age.
KW - Graft-versus-host disease
KW - Hematologic malignancy
KW - Hematopoietic cell transplantation (HCT)
KW - Infants
KW - Late effects
KW - Pediatric
KW - Relapse
KW - Survival
KW - Total body irradiation
UR - http://www.scopus.com/inward/record.url?scp=85021731451&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2017.04.017
DO - 10.1016/j.bbmt.2017.04.017
M3 - Article
C2 - 28461213
AN - SCOPUS:85021731451
SN - 1083-8791
VL - 23
SP - 1327
EP - 1334
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 8
ER -