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Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases. Blood Adv 2020 05 12;4(9):2084-2094

Date

05/13/2020

Pubmed ID

32396620

Pubmed Central ID

PMC7218429

DOI

10.1182/bloodadvances.2019000839

Scopus ID

2-s2.0-85086114432   1 Citation

Abstract

We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range, <1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was <1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.

Author List

Kahn JM, Brazauskas R, Tecca HR, Bo-Subait S, Buchbinder D, Battiwala M, Flowers MED, Savani BN, Phelan R, Broglie L, Abraham AA, Keating AK, Daly A, Wirk B, George B, Alter BP, Ustun C, Freytes CO, Beitinjaneh AM, Duncan C, Copelan E, Hildebrandt GC, Murthy HS, Lazarus HM, Auletta JJ, Myers KC, Williams KM, Page KM, Vrooman LM, Norkin M, Byrne M, Diaz MA, Kamani N, Bhatt NS, Rezvani A, Farhadfar N, Mehta PA, Hematti P, Shaw PJ, Kamble RT, Schears R, Olsson RF, Hayashi RJ, Gale RP, Mayo SJ, Chhabra S, Rotz SJ, Badawy SM, Ganguly S, Pavletic S, Nishihori T, Prestidge T, Agrawal V, Hogan WJ, Inamoto Y, Shaw BE, Satwani P

Authors

Ruta Brazauskas PhD Associate Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Larisa Broglie MD, MS Assistant Professor in the Pediatrics department at Medical College of Wisconsin
Saurabh Chhabra MD Associate Professor in the Medicine department at Medical College of Wisconsin
Kristin Page MD, MHS, MEd Associate Professor in the Pediatrics department at Medical College of Wisconsin
Rachel A. Phelan MD Assistant Professor in the Pediatrics 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

Adolescent
Anemia, Aplastic
Child
Hematopoietic Stem Cell Transplantation
Humans
Leukemia
Neoplasms
Transplantation, Homologous