Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

Justine M. Kahn, Ruta Brazauskas, Heather R. Tecca, Stephanie Bo-Subait, David Buchbinder, Minoo Battiwala, Mary E.D. Flowers, Bipin N. Savani, Rachel Phelan, Larisa Broglie, Allistair A. Abraham, Amy K. Keating, Andrew Daly, Baldeep Wirk, Biju George, Blanche P. Alter, Celalettin Ustun, Cesar O. Freytes, Amer M. Beitinjaneh, Christine DuncanEdward Copelan, Gerhard C. Hildebrandt, Hemant S. Murthy, Hillard M. Lazarus, Jeffery J. Auletta, Kasiani C. Myers, Kirsten M. Williams, Kristin M. Page, Lynda M. Vrooman, Maxim Norkin, Michael Byrne, Miguel Angel Diaz, Naynesh Kamani, Neel S. Bhatt, Andrew Rezvani, Nosha Farhadfar, Parinda A. Mehta, Peiman Hematti, Peter J. Shaw, Rammurti T. Kamble, Raquel Schears, Richard F. Olsson, Robert J. Hayashi, Robert Peter Gale, Samantha J. Mayo, Saurabh Chhabra, Seth J. Rotz, Sherif M. Badawy, Siddhartha Ganguly, Steven Pavletic, Taiga Nishihori, Tim Prestidge, Vaibhav Agrawal, William J. Hogan, Yoshihiro Inamoto, Bronwen E. Shaw, Prakash Satwani

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16 Scopus citations


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.

Original languageEnglish
Pages (from-to)2084-2094
Number of pages11
JournalBlood advances
Issue number9
StatePublished - May 12 2020

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ASJC Scopus subject areas

  • Hematology


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