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Survival and Late Effects after Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancy at Less than Three Years of Age
Dana Farber Boston Childrens Canc & Blood Disorde, Dept Pediat, 450 Brookline Ave, Boston, MA 02215 USA.
Med Coll Wisconsin, Dept Med, Ctr Int Blood & Marrow Transplant Res, Milwaukee, WI 53226 USA.
Med Coll Wisconsin, Dept Med, Ctr Int Blood & Marrow Transplant Res, Milwaukee, WI 53226 USA;Med Coll Wisconsin, Div Biostat, Inst Hlth & Soc, Milwaukee, WI 53226 USA.
Cleveland Clin, Taussig Canc Inst, Blood & Marrow Transplant Program, Cleveland, OH 44106 USA.
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2017 (English)In: Biology of blood and marrow transplantation, ISSN 1083-8791, E-ISSN 1523-6536, Vol. 23, no 8, p. 1327-1334Article in journal (Refereed) Published
Abstract [en]

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 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 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.

Place, publisher, year, edition, pages
2017. Vol. 23, no 8, p. 1327-1334
Keywords [en]
Hematopoietic cell transplantation (HCT), Survival, Infants, Pediatric, Late effects, Hematologic malignancy, Relapse, Total body irradiation, Graft-versus-host disease
National Category
Hematology
Identifiers
URN: urn:nbn:se:uu:diva-359267DOI: 10.1016/j.bbmt.2017.04.017ISI: 000406737000016PubMedID: 28461213OAI: oai:DiVA.org:uu-359267DiVA, id: diva2:1245791
Available from: 2018-09-06 Created: 2018-09-06 Last updated: 2018-09-06Bibliographically approved

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Olsson, Richard

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