Faculty Collaboration Database

Survival and Late Effects after Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancy at Less than Three Years of Age. Biol Blood Marrow Transplant 2017 Aug;23(8):1327-1334

Date

05/04/2017

Pubmed ID

28461213

Pubmed Central ID

PMC5666571

DOI

10.1016/j.bbmt.2017.04.017

Scopus ID

2-s2.0-85021731451 (requires institutional sign-in at Scopus site)   37 Citations

Abstract

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.

Author List

Vrooman LM, Millard HR, Brazauskas R, Majhail NS, Battiwalla M, Flowers ME, Savani BN, Akpek G, Aljurf M, Bajwa R, Baker KS, Beitinjaneh A, Bitan M, Buchbinder D, Chow E, Dandoy C, Dietz AC, Diller L, Gale RP, Hashmi SK, Hayashi RJ, Hematti P, Kamble RT, Kasow KA, Kletzel M, Lazarus HM, Malone AK, Marks DI, O'Brien TA, Olsson RF, Ringden O, Seo S, Steinberg A, Yu LC, Warwick A, Shaw B, Duncan C

Authors

Ruta Brazauskas PhD Associate Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Peiman Hematti MD Professor in the Medicine department at Medical College of Wisconsin
Bronwen E. Shaw MBChB, PhD Center Director, Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Age Factors
Allografts
Child, Preschool
Chronic Disease
Disease-Free Survival
Female
Follow-Up Studies
Graft vs Host Disease
Hematologic Neoplasms
Hematopoietic Stem Cell Transplantation
Humans
Infant
Male
Survival Rate