Survival and Late Effects after Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancy at Less than Three Years of Age

Lynda M. Vrooman, Heather R. Millard, Ruta Brazauskas, Navneet S. Majhail, Minoo Battiwalla, Mary E. Flowers, Bipin N. Savani, Görgün Akpek, Mahmoud Aljurf, Rajinder Bajwa, K. Scott Baker, Amer Beitinjaneh, Menachem Bitan, David Buchbinder, Eric Chow, Christopher Dandoy, Andrew C. Dietz, Lisa Diller, Robert Peter Gale, Shahrukh K. HashmiRobert J. Hayashi, Peiman Hematti, Rammurti T. Kamble, Kimberly A. Kasow, Morris Kletzel, Hillard M. Lazarus, Adriana K. Malone, David I. Marks, Tracey A. O'Brien, Richard F. Olsson, Olle Ringden, Sachiko Seo, Amir Steinberg, Lolie C. Yu, Anne Warwick, Bronwen Shaw, Christine Duncan

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34 Scopus citations


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.

Original languageEnglish
Pages (from-to)1327-1334
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Issue number8
StatePublished - Aug 2017


  • Graft-versus-host disease
  • Hematologic malignancy
  • Hematopoietic cell transplantation (HCT)
  • Infants
  • Late effects
  • Pediatric
  • Relapse
  • Survival
  • Total body irradiation


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